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gen_e987771e87df5ff696b4debead2c39a1
Understanding how clonal hematopoiesis feeds lymphoma
Worldwide Cancer Research
Institute of Oncology Research
HRCS22_15962
As the fifth most frequently occurring cancer type in humans, lymphoma still requires to be mechanistically better understood in order to achieve a satisfactory relative survival. A promising approach to close this knowledge gap concerning lymphoma consists in investigating the microenvironment and potentially occurring inflammatory processes, which may have a supportive effect on the emerging and maintenance of tumor progression. Clonal hematopoiesis (CH) is the accumulation of mutations in the myeloid-monocyte lineage in healthy individuals. CH has been correlated with inflammation and aging of immune response. One of the most-common mutations in CH is loss-of-function alleles in TET2. TET2 encodes an enzyme involved in epigenetic regulation of hematopoietic cells. Our working hypothesis is that TET2 mutated CH creates an inflamed niche of myeloid cells that support the tumor and/or impair the anti-tumor immune response. Our working hypothesis will be addressed through distinct tasks: 1.) Provide experimental proof that TET2 mutated myeloid cells are enriched in the intra-tumoral microenvironment of lymphoma patients with CH, and that they have a pro-inflammatory/tumor supportive phenotype; 2.) Validating that Tet2–deficient hemopoiesis promotes lymphoma development and 3.) Proofing that TET2 mutated CH is an unfavorable prognostic biomarker in lymphoma. The project leverage state-of-the-art and novel single cell technologies, novel strains of genetically modified mouse models of lymphoma, and clinical trial cohorts. The project will impact on our understanding of lymphoma biology and will create a new platform for therapeutic advances in this tumor. In terms of knowledge, we will discover a completely new and actionable mechanism of lymphoma promotion that may be termed as “monoclonal immune microenvironment” that will advance the understanding of the interplay between immune-aging and tumor development. In terms of treatment, we will generate both reagents and candidate targets from pre-clinical studies aiming at re-educating or eradicating the “monoclonal immune microenvironment”.
2.1 Biological and endogenous factors
6project_grants_public
gen_de1a6783eba71dee9b3619f6076e1feb
Early exposure to radio-frequency electromagnetic fields and cancer: complete evaluation of the Ramazzini Institute long-term carcinogenicity bioassays on Sprague-Dawley rats
Children with Cancer UK
Istituto Ramazzini
HRCS22_15966
Humans in modern society are exposed to an ever-increasing number of radiofrequency electromagnetic fields (RFR). Key sources of RFR include mobile phones, wireless networks and base station antenna. Concern is expressed about possible health effects, particularly children appears to be more sensitive to RFR than adults due to their developing system. In 2011, the International Agency for Research on Cancer (IARC) classified RFR as “possibly carcinogenic” to humans (group 2B) based on epidemiological evidence of a positive association between exposure to RFR and nervous system tumours. In the meantime, both the National Toxicology Program (NTP) and the Ramazzini Institute (RI) had already started a program to perform experimental studies on the health effects of RFR on rodents. The RI partial findings on far field exposure to RFR are consistent with and reinforce the results of the NTP study on near field exposure, as both reported an increase in the incidence of tumours of the brain and heart in RFR-exposed rats starting from prenatal life. Another carcinogenicity bioassay had been also performed by the RI to test the potential syn-carcinogenic effects of RFR in combination with gamma radiation. Up to now, the in vivo phase of both the RI ‘studies has been concluded and processing of tissues is still ongoing. In the present project, we aim to complete the histopathological evaluation of all the other tissues in order to provide sufficient evidence in experimental animals to call for the re-evaluation of IARC conclusions regarding the carcinogenic potential of RFR in humans.
No Research Activity assigned
6project_grants_public
gen_32649fa9a27b236ed545112a763deac1
Biomarkers Part 2
Duchenne UK
Duke University
HRCS22_15982
This project will be looking at biomarkers to be used in clinical trials.
4.1 Discovery and preclinical testing of markers and technologies
6project_grants_public
gen_216a3d785370cc502f5b1dd798bb4c0f
Exploring the functional role of EZH2-dependent autocrine signaling in T-cell leukemia
Worldwide Cancer Research
Casa Sollievo della Sofferenza
HRCS22_15987
BACKGROUND: T-cell acute lymphoblastic leukemia/lymphoma (T-ALL) is a malignancy with refractory/relapsed cases, presumably due to the ineffective targeting of leukemia initiating cells (LICs). DNA mutations, leading to the hyperactivation of Notch1 signaling, are found in >50% of human T-ALL. NOTCH1 binding induces the eviction of PRC2, which promotes the repressive H3K27me3 chromatin modification. Moreover, we observed that in NOTCH1-induced mouse T-cell leukemias, the shRNA-mediated knockdown of EZH2 (a functional component of PRC2) stimulates cell growth/survival by inducing an autocrine IGF-like signaling, critical for LIC activity. OBJECTIVE/HYPOTHESIS: This proposal intends to characterize the functional role of EZH2 in the NOTCH1-driven malignant transformation of human cells and to define the EZH2-dependent autocrine signaling involved in T-ALL pathogenesis. We hypothesize that the silencing of EZH2 modulates functional pathways of T-cell transformation and LIC activity underlying the different outcomes of relapsed/recurrent leukemias. SPECIFIC AIMS: Determine whether the loss of EZH2 promotes the malignant transformation of hematopoietic stem/progenitors(HSPCs) from human cord blood; Characterize the EZH2-dependent autocrine signaling in human NOTCH1-induced HSPCs; Define the functional role of EZH2 in the LIC activity of human T-ALL. STUDY DESIGN: We propose to use novel models of human T-ALL in which HSPCs from cord blood are transformed into clonal T-ALL-like malignant cells, after transduction with lentiviral vectors encoding NOTCH1-ΔE (a constitutively active NOTCH1 variant) alone or in combination with three known T-ALL oncogenes, and subsequent injection into immunocompromised mice. We will perform gain/loss-of-function assays to assess the EZH2 contribution to leukemogenesis and LIC-related phenotypes. We will employ ELISA/Luminex analyses, combined with RNA-Seq, ChIP-Seq and TaqMan gene expression assays for characterizing the EZH2-dependent autocrine signaling. Conventional EZH2 inhibitors and patient-derived xenograft(PDX) leukemias will be also included. SIGNIFICANCE: This study will outline relevant pathways of leukemia maintenance, development and chemoresistance and provide rationale for drug design by identifying novel molecular targets.
2.1 Biological and endogenous factors
6project_grants_public
gen_f54ec7d406a1b5bf92bb518f137478a7
Targeting GH signalling: a novel radiosensitising strategy
Worldwide Cancer Research
University of Auckland
HRCS22_15993
No abstract available for this analysis.
No Research Activity assigned
6project_grants_public
gen_efac2d3960b02e7f52d7cfe480d2672c
Role of PI3K-C2G in pancreatic cancer metabolism
Worldwide Cancer Research
University of Turin
HRCS22_15995
Pancreatic cancer (PC) is the most lethal cancer across the world, with incidence equaling mortality. Recently, PC emerged as a metabolic outlier characterized by several metabolic dependencies, representing an unmet opportunity for the development of novel therapeutic approaches. A wealth of studies report that enhanced activation of PI3K/mTOR pathway has a critical role in the pathogenesis of PC and it is required for sustaining malignant growth and metabolism rewiring. The mTOR Complex 1 (mTORC1) is a key regulator of metabolic adaptation to nutrient availability, promoting anabolic pathways. Recently, it has been reported that PI(3,4)P2, a class II PI3K signaling product, can act as a local repressor of the mTORC1 signaling. Differently from other class II members which are ubiquitous, In particular, the PI3K-C2γ isoform is mainly expressed in the pancreatic tissue where it plays a pivotal role in cell metabolism. In human PDAC, the PIK3C2G gene is homozygous-deleted in about 7% of patients. Therefore, we decided to model PI3K-C2γ loss (PIK3C2G gene KO) in a mouse model of PC (KPC). We found that lack of PI3K-Cϒ increases tumor development and progression, strongly reducing mice mean survival rate and driving rapid progression to cancer. Biochemical analyses of PIK3C2G-deficient tumors revealed a specific hyperactivation of mTORC1 pathway, increasing metabolic alterations. To consolidate these findings, we aim at: i) assessing the oncogenic potential of PI3K-C2ϒ ii) dissecting PI3K-C2ϒ signaling pathway iii) exploiting metabolic dependencies of PI3K-C2ϒ loss iv) defining the prognostic and predictive value of PI3K-C2ϒ in PDAC. With this project, we expect to validate PI3K-C2ϒ as a novel tumor suppressor in PC and to establish a new mechanism by which PI3K-C2ϒ controls metabolic alterations induced by mTORC1 hyperactivation. Eventually, data obtained from this study will validate selective inhibition of critical metabolic pathways in PI3K-C2ϒ-deficient tumors to maximize therapeutic success.
2.1 Biological and endogenous factors
6project_grants_public
gen_717cf461daf5404dbd6e59f3c727f716
NUMB splicing modulation with AONs as a novel lung cancer treatment
Worldwide Cancer Research
Centre for Genomic Regulation
HRCS22_15997
No abstract available for this analysis.
No Research Activity assigned
6project_grants_public
gen_29b67c9117ecbf97c79e003bcb62a1f8
Identifying key amino acid metabolic steps as potential drug targets for Chagas disease treatment
Wellcome Trust
University of Sao Paulo
HRCS22_16008
Trypanosoma cruzi, the causal agent of Chagas disease, has a complex digenetic life cycle. This organism is well adapted to handle environmental changes, presenting high metabolic flexibility. In this context, amino acids and their metabolism play crucial roles in cell differentiation, maintenance of cell volume and response to different types of stress. This proposal aims to identify the key steps of the amino acid metabolic network which are crucial for T. cruzi to complete its life cycle. With this purpose, we will use the bar-seq CRISPR-Cas9 genome editing strategy to generate mutant knockout cell lines for the coding sequences of 40 enzymes putatively involved in the metabolism of amino acids. Each cell line will be identified by a unique sequence barcode and they will be pooled to allow parallel phenotyping during cell differentiation and infection, both in vitro and in vivo. Relative fitness will be assessed by next-generation DNA sequencing. Identification of the enzymes that are essential for parasite development and infectivity will provide insights on the metabolic strategies T. cruzi evolved to adapt to diverse environments as well as novel targets for target-specific drug design and ligand structure-based screenings in the future.
2.1 Biological and endogenous factors
6project_grants_public
gen_0c93fb3636de6edfdd63ec36359fbcd6
Research Enrichment Funding for the Public engagement programme of CIDRI-Africa
Wellcome Trust
University of Cape Town
HRCS22_16027
The Wellcome Centre for Infectious Diseases Research in Africa (CIDRI-Africa) at the University of Cape Town leads research programmes on high-burden infectious diseases, particularly HIV and tuberculosis. Through this enrichment grant, we will develop our researcher community as public engagement champions, improve our research through inolvement of our local communities and stakeholders, and raise awareness of our research to maximise health outcomes. To develop our researcher community’s involvement in PE, we will create and implement a PE communication toolkit, which will become an embedded component of study conduct at CIDRI-Africa, and enable our researchers to confidently engage the wider public. We will continue to engage the community with whom we conduct research through health research awareness activities, and establish a community advocacy programme to extend the work of CIDRI-Africa’s Community Advisory Group. Members of the advocacy programme will bridge research and non-research communities, providing input to both and effectively advocating tuberculosis and HIV research. We will engage young people through our school’s engagement project, drawing on methods from a previous pilot. This will follow a workshop format, and improve understanding amongst school learners and the people they connect with of tuberculosis and HIV as well as research into these conditions.
7.1 Individual care needs
6project_grants_public
gen_60ff6246b8b55ab2fd56a82a1c02d80f
Decoding the role of lipids in the metabolic control of CD8+ T cells by the gut microbiota to improve outcomes in colorectal cancer.
Worldwide Cancer Research
European Institute of Oncology
HRCS22_16034
Over a third of resected early-stage colorectal cancer (CRC) relapse and patients die from metastatic disease. Infiltrating lymphocytes are independent positive predictor of survival in CRC, however only a small fraction of patients respond to immune checkpoint inhibitors (ICI). This project originates from two milestone discoveries. First, the gut microbiome modulates response to ICI, but the overlap across different cohorts is mostly functional rather than compositional. Second, accumulation of specific lipids in the tumor microenvironment (TME) impact the anti-tumor activity of CD8+ T cells. We have conducted comprehensive metagenomic, metabolomic and immune characterization on CRC patients, observing parallel changes in mucosa and tissues. Thus, we hypothesize that therapeutic manipulation of the metabolic output of mucosal bacteria can reshape the TME and improve the response of CRC to ICI. This prospective study is designed to: . DECODE the interplay between metabolic activities in the intestinal mucosa and immune cell functions on CRC patients; . VALIDATE the effect of lipids derived or induced by bacteria on immune cell functions; . TRANSLATE new knowledge into innovative strategies to improve ICI efficacy. Main objectives are: . Quantification of lipids and reconstruction of bacteria-host metabolic pathways, in tumours and surrounding tissues; . Characterization of the immunomodulatory impact of the microbiota on CD8+ T cells; . Mechanistic validation and improvement of ICI’s response on a novel patient-derived organ-on-chip device. This study will enable multi-scale profiling and predictive modelling on a fully annotated cohort while simultaneously spawning novel actionable targets for therapy. Tumour sensitivity to ICI will be tested, providing initial experimental validation on a patient-relevant and cost-effective system. Data generated through this project may also reconcile the limited association between the overall composition of the microbiome and the response to immunotherapy across different cohorts of patients, a crucial step toward new stratification strategies and therapeutic approaches.
2.1 Biological and endogenous factors
6project_grants_public
gen_71360dbc15ca1d51bf69d731fa5a8b61
Dissecting the molecular mechanisms that sustain frequent oncogenic BAP1 mutations
Worldwide Cancer Research
European Institute of Oncology
HRCS22_16035
No abstract available for this analysis.
No Research Activity assigned
6project_grants_public
gen_2cacb7896490467096a363147d172390
Investigating the roles of NF-κB subunits as master regulators of CD8+ T-cell function in cancer immunity and immunotherapy.
Worldwide Cancer Research
Cancer Center of Lyon
HRCS22_16036
Cancer progression is greatly influenced by a delicate balance between tolerance to and immune response against tumors. The recent success of immunotherapies such as checkpoint-blockade therapies, demonstrates that this balance can be shifted effectively towards enhanced anti-tumor immunity, to delay or even stop cancer progression, particularly by enhancing the function of CD8+ T cells. However, only a fraction a patients respond to these regimens, and the molecular mechanisms that drive the response and resistance to immunotherapies are largely unknown. The transcription factor NF-kappaB (NF-kB) has been implicated in the regulation of T-cell function. However NF-kB is in fact a family of transcription factors composed of 5 distinct subunits; due to the lack of suitable tools, the contribution of individual subunits of NF-kB to CD8+ T-cell biology has never been addressed. In this application, we propose to investigate the exact roles of NF-kB subunits that control CD8+ T-cell function in cancer immunity. We will use unique mouse models carrying ablation of NF-kB subunits specifically in mature CD8+ T cells, CRISPR-based gene edition in primary human cells and high-throughput sequencing analyses. Based on our previous publications and preliminary data, we hypothesize that the activation of selective NF-kB subunits orchestrates CD8+ T-cell biology and underlies the clinical response to checkpoint-blockade therapies. Importantly, these studies may reveal specific NF-kB subunit activities that can be targeted by novel therapeutics to enhance T-cell function alone or in concert with existing immunotherapies.
2.1 Biological and endogenous factors
6project_grants_public
gen_431da97927d45c6519c56d8db25052f7
Identifying properties of tumour suppressive pericytes for cancer therapy (PERISUPPRESS)
Worldwide Cancer Research
CERCA Institution
HRCS22_16064
Non-transformed co-habitant cells, defined as stroma, play key roles in the development and progression of tumours. Thus, understanding their function is instrumental for the efficacious eradication of the disease. Taking advantage of computational analyses, a unique collection of prostate cancer (PCa) mouse models, and our know-how expertise in pericyte biology, we have identified that pericytes inform about PCa recurrence and metastasis. Here, we postulate that the study of molecular cues governing the pericyte-cancer cell relationship provides an innovative perspective of the aggressive properties of cancer cells. This may serve to define therapeutic strategies that would be missed in a classical tumour cell-centred discovery approach. PERISUPRESS aims to identify the molecular and cellular mechanisms which promote tumour suppressive properties in pericytes by: (1) a discovery approach to identify pericyte-related molecular cues associated with PCa progression; (2) the study of the impact of pericyte-specific molecular programs on PCa progression; (3) the identification of pericyte-related vulnerabilities associated to PCa progression. This will be done through awell-balanced experimental plan which includes mouse models and patient-derived samples from PCa at different disease grades, computational biology and multiple in vitro and in vivo functional and mechanistic approaches. In summary, the study of pericyte biology in PCa has the potential to open a new window in the comprehension and treatment of aggressive PCa that may also be applied to other types of cancer.
2.1 Biological and endogenous factors
6project_grants_public
gen_00c124ae962579836cb4cb0bb2f9d026
‘We are the heroin capital of Europe’: Marginal Communities, Health, Identity, and the Opioid Epidemic in Twentieth-Century Ireland
Wellcome Trust
University College Dublin
HRCS22_16079
Keywords: Heroin, drugs, Ireland. By 1990 heroin had devastated working-class communities across Dublin and, in some families, wiped out entire generations. But how did things reach that stage when three decades previously heroin was only known in Ireland as a symbol of distant, undesirable, foreignness? This project will explore the history of heroin in Ireland, from its first public discussion (1918) to the end of the first heroin epidemic (1990). It will approach the history from two directions. First, analysing the public discourse about the drug. This will reveal, inter alia, different ways that heroin was used as a symbol of dissolute otherness, and how this shaped health policy and provision. Secondly the project will collect 100 oral histories to investigate how heroin affected everyday life in the communities at the centre of the heroin epidemic of the 1970s and 1980s. Finally, the project will use reception theory to integrate these approaches and achieve three key goals: • To understand how the public discourse shaped community members’ interaction with each other, the wider world, and healthcare providers. • To give marginalised communities a voice in the production of their own history. • To produce the first history of heroin-use in Ireland.
8.1 Organisation and delivery of services
6project_grants_public
gen_66f98f411b2deb166b213db3216ce699
Unravelling the requirement of Rad18 in glioblastoma proliferation
Worldwide Cancer Research
Cancer Research Center of Toulouse
HRCS22_16094
Glioblastoma is the most frequent and aggressive primary brain tumor characterized by a strong resistance to therapy involving treatment with ionizing radiations and alkylating agents. The molecular grounds of this acquired resistance are currently unknown. The identification of novel therapeutic targets constitutes therefore a priority. We and others have recently reported an implication of the Rad18 (E3) ubiquitin ligase in the resistance of glioblastoma to cisplatin and ionizing radiations. Rad18, a critical component of DNA damage bypass, activates translesion synthesis by catalyzing the transfer of an ubiquitin monomer on PCNA, a well-known proliferation-associated antigen. This results in recruitment of translesion synthesis DNA polymerases to allow DNA synthesis through DNA lesions thus facilitating escape of apoptosis and contributing in resistance to therapy. In addition, Rad18 is also implicated in repair of single and double DNA strand breaks as those generated by the radiotherapy. Unexpectedly, by analyzing the phenotypes associated with Rad18 downregulation in glioblastoma, we have observed that Rad18, which is non-essential for life, is essential for the proliferation of glioblastoma cells, and in particular of glioblastoma cancer stem cells (GCSCs), those cells currently strongly implicated in the resistance to the therapy. The aim of this work is to unveil the specific function of Rad18 in the proliferation of GCSCs and to propose Rad18 as novel therapeutic target in the treatment of glioblastoma. This will be achieved thought the identification of the molecular pathway(s) in which Rad18 is implicated through genome-wide analysis of genes and proteins affected by Rad18 expression, as well as by the identification of Rad18 substrates in GCSCs. We will also generate a proof-of-concept for a role of Rad18 in sustaining glioblastoma proiliferation in preclinical models using both wild-type and Rad18 knock-out mice.
2.1 Biological and endogenous factors
6project_grants_public
gen_db4c505020bebfa2fb6acba203da3e18
Impact of a Typhoid Conjugate Vaccine Campaign on Antimicrobial Use in Harare, Zimbabwe
Wellcome Trust
Biomedical Research and Training Institute
HRCS22_16112
Typhoid fever is a common cause of non-specific febrile illness in low-resource settings. Empirical treatment for typhoid may be a major driver of AMR in typhoid-endemic communities, where estimates suggest that up to 25 additional cases receive antimicrobial treatment for each one confirmed. Harare in Zimbabwe has experienced a major increase in typhoid since 2016, with recent emergence of ciprofloxacin-resistance. In February 2019 a mass typhoid vaccine campaign was performed, targeting infants and children living in 9 affected suburbs with the new typhoid conjugate vaccine (TCV). In work being performed at primary health clinics in these densely-populated suburbs as part of the FIEBRE study, which aims to assess the causes of febrile illness in low-resource settings, we have already identified a significant reduction in typhoid in vaccinated children. In this project our key goals are to investigate the impact of the 2019 mass TCV campaign on antimicrobial resistance in S. Typhi and on antimicrobial prescribing and related practices in community clinics. Defining these effects will have major policy implications for TCV use, both locally, in determining the benefits of further routine/repeat vaccination, and internationally to inform estimates of the direct and indirect impacts on AMR infection and antimicrobial use.
3.4 Vaccines
6project_grants_public
gen_156df0077ff39bd9ec6e643d49f29902
Defining NRF2-independent ROS management as bottleneck to tumor initiation
Worldwide Cancer Research
New York University
HRCS22_16120
Cancer is generally viewed as a genetic disease, which develops when cells acquire mutations in proto-oncogenes and tumor suppressors. These mutations are thought to progressively derail tissue homeostasis, allowing for an expansion of tumorigenic clones with unlimited proliferative potential that fuels long-term tumor growth. Surprisingly, recent mutational analyses of physiologically normal skins unveiled a mutational spectrum and burden that is similar to what had been reported for squamous cell carcinomas (SCCs). Additionally, intravital imaging revealed that skin epithelial progenitor cells (EPCs) with oncogenic mutations persist only transiently and are primarily outcompeted by normal EPCs. These observations, along with stereotypic transcriptional and metabolic changes, which distinguish tumor propagating SCC cells (TPCs) from normal EPCs, suggest that epigenetic adaptation is critical for tumor initiation and persistent expansion. Still, the mechanisms facilitating this epigenetic and metabolic rewiring are largely unknown. Here, we will test the hypothesis that de novo Paired-like homeodomain transcription factor 1 (PITX1) expression facilitates the epigenetic and metabolic reprogramming of KrasG12Dmutant EPC into TPCs. PITX1 dependent changes in chromatin accessibility will enhance self-renewal and increase cell survival by reducing Ras generated reactive oxygen species (ROS). Specifically, we will (1.) determine how PITX1 cooperates with KrasG12Das they reprogram the transposase accessible open chromatin of homeostatic EPCs into the TPC specific epigenetic landscape; (2.) measure how these changes in chromatin accessibility influence ROS management; and (3.) determine if PITX1 dependent antioxidant production is a rate limiting step for tumor initiation. The results of our proposed research are expected to illuminate how oncogenic stress co-operates with sequence specific transcription factors to epigenetically rewire a homeostatic state into a tumorigenic epigenome, which allows TPCs to meet the metabolic demands required for tumor expansion. Once identified, these mechanisms could improve the early diagnosis and prevention of this exceedingly common and deadly human cancer.
1.1 Normal biological development and functioning
6project_grants_public
gen_ec4962254d6cab4c2bef16eeb33d31fb
Preclinical development of antisense oligonucleotide therapy for RIPOR2-associated adultonset hearing loss
Royal National Institute for Deaf People
Radboud University Nijmegen Medical Centre
HRCS22_16125
We recently identified a 12-nucleotide deletion in RIPOR2 (rs760676508) as a frequent cause of dominantly inherited adult-onset sensorineural hearing loss (DFNA21). Based on the allele frequencies in gnomAD, it is estimated that ~30,000 individuals in Northwest Europe are at risk to develop hearing loss due to this RIPOR2 variant. The mutated protein exhibits an aberrant localization in stereocilia. The proposed project builds on these findings and aims to put a genetic treatment on the horizon of DFNA21 patients. The age of onset and progressive nature of the hearing loss provide a window of opportunity for therapeutic intervention. This, together with the high prevalence, makes DFNA21 an important target for therapy development. As the 12-nucleotide deletion in RIPOR2 is indicated to have a dominant negative or toxic gain of function effect, we hypothesize that suppression of mutant RIPOR2 synthesis will prevent or delay the onset and/or progression of the hearing loss. To test this therapeutic strategy, the aims of the project are: 1)_x0001_To design and test antisense oligonucleotides (AONs) for effective and specific targeting of the mutant RIPOR2 (pre-)mRNA for degradation in HEK293T and patient-derived cells. 2)_x0001_To provide insight into the feasibility of AON-mediated therapy for DFNA21 in Δ12-nt Ripor2 mutant mice . Allele-specific quantitative PCR will be employed to demonstrate AON-effectivity and specificity in HEK293T cells transfected with plasmids encoding mutant and wildtype RIPOR2 and in patient-derived fibroblasts and otic progenitor cells. Audiometric testing and (immuno)histology will be the readouts for therapeutic efficacy in the DFNA21 mouse model.
5.2 Cellular and gene therapies
6project_grants_public
gen_67a2749fc72a81a51cca8712f9289f03
Characterising the role of RET in breast cancer brain metastasis as a facilitator of colonisation and a novel targeted therapy
Breast Cancer Now
Royal College of Surgeons in Ireland
HRCS22_16127
Brain metastasis occurs in 10-30% of breast cancer patients and is fast becoming the foremost clinical challenge in the management of metastatic disease. Limited therapeutic options exist for patients with brain metastasis and the current treatment paradigm consists of surgical resection, stereotactic radiosurgery and whole-brain radiation. Clearly, there is an urgent need to better understand the mechanisms of breast cancer metastasis to the brain and to define novel therapeutic targets. Recent comprehensive sequencing studies from our group and collaborators have revealed a number of brain metastasis enriched oncogenic alterations with potential clinical significance. Aims: We aim to determine whether altered aberrant RET signalling in metastatic breast cancer is a key mediator of tumour colonisation of the brain. We also aim to determine the efficacy of RET inhibition to abolish cell growth and survival of breast cancer brain metastatic cells. Technique and Methodology: To address the suitability of RET as a target we will use both genetic and pharmacological inhibition of RET in comprehensive in vitro, ex vivo and in vivo pre-clinical models of breast cancer brain metastasis representing all three clinical subtypes. In vitro experiments will be accompanied by functional interrogation while in vivo experiments will be complemented by next-generation sequencing of the brain invading tumours. Impact on breast cancer research: The outcomes of this project have the potential to significantly enhance our understanding of the oncogenic significance of recurrent, acquired aberrations in the RET pathway in breast cancer brain metastasis. This project has the potential to uncover the clinical utility of brain metastasis-acquired clinically actionable alterations that could lay the foundations for a clinical trial utilising RET inhibition for patients with breast cancer brain metastasis.
4.1 Discovery and preclinical testing of markers and technologies
6project_grants_public
gen_184fb2511af737ecc47b0160f942738d
Improved surveillance of zoonotic Mycobacteria through rapid direct detection and genotyping in livestock, wildlife, and their environment from low-resource areas in South Africa.
Wellcome Trust
Stellenbosch University
HRCS22_16129
Recent studies in Africa and India (countries with high TB- and HIV-burdens) reported increased frequency of zoonotic tuberculosis (zTB) in humans caused by other members of the Mycobacterium tuberculosis complex (MTBC) like M. orygis, indicating that M. bovis infection may be an inadequate proxy for zTB. Similarly, nontuberculous mycobacteria (NTM) lung disease in people is more frequently reported in Africa because of environmental NTM like M. avium complex. NTM are naturally resistant to various drugs commonly used to treat MTBC infection and are often indistinguishable from MTBC when conventional MTBC “specific” immunological tests are used in humans and animals. Consequently, the management and treatment of infected patients are substantially different and complicated by the absence of appropriate culture-independent tests for the combined detection and differentiation of MTBC and NTM. We hypothesize that zoonotic pathogens like M. orygis and M. avium complex are more abundant in livestock, their environments and nearby wildlife than previously thought, especially in areas with immunocompromised people, leading to potentially high transmission risk interfaces. The goal is to develop culture-independent detection and genotyping tests to enhance zoonotic Mycobacteria spp. detection and differentiation directly from livestock specimens collected from low-resource areas, their environment and near-by wildlife reservoirs.
2.2 Factors relating to physical environment
6project_grants_public
gen_689ecadafb4996278c5e4c8b07f6e97a
PPS - RTI
Duchenne UK
RTI International
HRCS22_16136
This is a collaboration funded by Duchenne UK. It involves RTI International, Parent Project Muscular Dystrphy and Newcastle University, who are all recepients of funding based on their contribution. This collaborative project will assess and explore clinician, patient, and caregiver attitudes, priorities, and risk tolerance regarding gene transfer technologies. These technologies offer great promise to be disease modifying but come with risks and burden. The study will employ a research and community engagement approach previously developed by Holly Peay at RTI and the collaborating Parent Project Muscular Dystrophy (PPMD) team. Instruments from the prior PPMD gene therapy preference study will be revised to meet current state of the science and will be reused in the proposed study. Data will be collected in the United States and the United Kingdom. Data on attitudes, priorities, and risk tolerance will be compared across the countries.
7.1 Individual care needs
6project_grants_public
gen_3806d039adf3dd495e3f5a04aff46c97
Determining the neuroprotective and neurorestorative efficacy of AAV-hGDF5 in the AAV-α-synuclein rat model of Parkinson’s.
Cure Parkinson's
University College Cork
HRCS22_16139
In Europe, 1.2 million people have Parkinson’s disease (PD), with a total socioeconomic cost of €13.9 billion per annum, which will double by 2030. No current therapy stops the symptoms of PD from worsening. This project proposes that a neurotrophic factor called growth differentiation factor 5 (GDF5) can be applied as a therapeutic to slow or prevent disease deterioration. GDF5 has known neurotrophic effects in preclinical models on midbrain dopaminergic neurons, the cells that degenerate in this disorder. To date, clinical trials of neurotrophic factors have used intracerebral delivery of either glial cell line-derived neurotrophic factor (GDNF) or neurturin (NRTN), but disappointingly, none of the trials have reached their primary end-point and thus are deemed to have failed. GDF5 is distinct from these two neurotrophic factors, in that it acts through a different signalling mechanism. Here the neuroprotective efficacy of an adeno-associated viral vector carrying the GDF5 transgene (AAV-GDF5) will be assessed in the rat α-synuclein model of PD. They will compare the effects of intrastriatal delivery of AAV-GDF5 with those of AAV-GDNF, in the AAV-a-synuclein rat model of PD. They will determine if this molecular therapy can protect dopamine neurons and their axons against a-synuclein-induced degeneration, to maintain brain dopamine levels and improve motor function. In parallel, they will study the protective effects of GDF5 treatment on cultured dopaminergic neurons which have a-synuclein mutations. Specifically, we will evaluate the effects of GDF5 on dopaminergic neuron survival, neurite growth and arborisation, in embryonic rat midbrain primary cultures and in induced pluripotent stem cell (iPSC)-derived dopaminergic neurons carrying SNCA triplication or the A53T mutation, both of which are common mutations in familial cases of PD. Overall, this research will lay the foundation for the translation of GDF5 viral vector therapy to clinic trials for PD.
5.2 Cellular and gene therapies
6project_grants_public
gen_567c42aca34551493d87189788a1bf64
Notch3 as a regulator of cancer-associated fibroblast reprogramming and functions in intestinal cancer
Worldwide Cancer Research
Alexander Fleming Biomedical Sciences Research Center
HRCS22_16166
The Notch signaling pathway plays an important role in development, homeostasis, and disease, such as cancer, where its therapeutic potential is under consideration. However, the role of individual receptors, their cell-specific functions, and their implication in cancer therapy is not yet fully understood. Notch3 is of particular interest, as it is overexpressed in colorectal cancer (CRC) and associated with worst prognosis. Our preliminary data suggest that in patients and animal models of CRC, Notch3 is predominantly expressed in cancer-associated fibroblasts (CAFs) and more specifically in intratumoral perivascular fibroblasts. However, the mechanisms driving Notch3 activation in fibroblasts and its role as a regulator of CAF reprogramming and downstream functions are not known. In this proposal, we bring forward the hypothesis that endothelial-specific Notch ligands can activate Notch3 signaling in adjacent perivascular fibroblasts, leading to their activation and downstream functions associated with either or both angiogenesis and cancer cell proliferation, ultimately leading to tumor growth. Notch3 inhibition could thus have a therapeutic effect either alone or in combination with standard therapy. To address this hypothesis, we will combine in vitro and in vivo genetic targeting, 2D and 3D cell co-culture techniques, high-throughput single-cell, and bulk RNA sequencing, and mouse models of disease. We expect that this study could lead to a better understanding of the role of fibroblast-specific Notch3 in CRC and the potential of its inhibition in the clinic. In addition, it will provide further insight into the mechanisms driving CAF reprogramming and functions and thus help identify novel targets for therapeutic approaches, as well as diagnosis and patient stratification.
2.1 Biological and endogenous factors
6project_grants_public
gen_cc1c24b75904911738d57444b0766583
Pre-clinical evaluation of lonafarnib as a treatment for Parkinson's disease
Cure Parkinson's
Northwestern University
HRCS22_16195
Genetic and pathological evidence strongly suggests that alpha-synuclein (a-syn) accumulation and lysosomal dysfunction are major contributors to Parkinson’s disease pathogenesis. Dr Mazzulli's group recently discovered a novel mechanism for a-syn-induced toxicity and lysosomal dysfunction using patient-derived iPSC-midbrain neurons. They found that a-syn accumulation impedes the trafficking of lysosomal hydrolases by disrupting a SNARE protein called ykt6. Ykt6 preferentially enhances the trafficking of lysosomal proteins, including beta-glucocerebrosidase (GCase) between the endoplasmic reticulum (ER) and the Golgi. However when a-syn accumulates, it aberrantly interacts with ykt6 and impedes its ability to traffic hydrolases. This results in lysosomal dysfunction, further perpetuating and augmenting a-syn accumulation. The team have discovered a novel way to rescue lysosomes by activating ykt6 through small molecule farnesyltransferase inhibitors (FTIs). Ykt6 is a unique SNARE in that it is inhibited by farnesylation, which keeps it in a locked, inactive conformation. Non-farnesylated ykt6 promotes an open, active conformation allowing it to bind to its cognate binding partners syntaxin 5 and bet1 at the cis-Golgi and promote trafficking. Blocking ykt6 farnesylation, either genetically or pharmacologically, was found to dramatically rescue lysosomal hydrolase trafficking and function including GCase activity. Using a previously established farnesyltransferase inhibitor (FTI) called LNK-754, 1-to-10 nanomolar concentrations could activate ykt6 and enhance lysosomal GCase by 2 fold in patient cultures and mouse brain, which was sufficient to reduce pathological a-syn. Dr Mazzulli's group's new, unpublished data shows that ykt6 is present in red blood cells, and that FTI treatment actives ykt6 in blood and brain to a similar extent. This provides a unique opportunity to track active ykt6 in RBCs as a marker of mechanistic target engagement for future clinical trials. While LNK-754 is a promising FTI, it requires extensive long-term safety assessment prior to treating PD patients. However, another FTI called Lonafarnib, has already passed long-term clinical safety tests and is FDA approved for the treatment of Progeria. To determine if FTIs hold promise as a disease altering PD therapy, we will determine if Lonafarnib can activate ykt6 and lysosomes in patient cultures and brains of a-syn transgenic mice. They will measure RBC ykt6 from lonafarnib-treated mice, to assess its potential as a biomarker for future clinical trials. At the end of the funding period, the project aims to determin if Lonafarnib can activate the ykt6-lysosomal pathway and reduce a-syn pathology in the brain, after oral administration. Since the mechanism for how FTIs enhance lysosomal function was uncovered here, they are uniquely positioned to assess the utility of RBC ykt6 as a blood biomarker to track mechanistic target engagement. If successful, they hope to translate these findings into PD patients as a novel disease modifying therapy within 2 years.
2.1 Biological and endogenous factors
6project_grants_public
gen_65734e05463318635939a86c5d427b54
Uncovering the effects of DNA replication stress on cell fate determination in cancer and stem cells.
Worldwide Cancer Research
University of Milan
HRCS22_16200
Several studies have indicated that most cancer cells are associated with stress caused by the perturbation of DNA replication inducing replication stress (RS). RS and in general genome instability are major causes of genetic and epigenetic heterogeneity in human tumors. However, despite this heterogeneity, tumor mass and metastasis display stereotypical behaviors that allow them to invade surrounding tissues and escape immune surveillance. These features are remarkably similar to the ones displayed by trophoblast cells supporting placenta development. Using mouse embryonic stem cells (ESCs) we have recently found that RS-mediated activation of ATR and CHK1 promotes the expression of thousands of genes many of which involved in trophoblast differentiation, suggesting that RS can impact on cell fate determination in stem cells. The activation of several of these genes is coordinated by DUX, the master regulator of the two-cell stage in mouse ESCs, which we showed to be required for multinucleated trophoblast giant cells differentiation in response to RS. DUX and its human ortholog DUX4 are suppressed in normal adult cells. However, they are specifically reactivated in tumors where they promote the emergence of trophoblast-like features. Here we will study the molecular mechanisms linking RS to trophoblast activation in ESCs, identify pathways shared between placenta and cancer cells and uncover the mechanisms that suppress placenta activating mechanisms in adult somatic cells. Our studies will set the stage for targeting these pathways in order to develop new and effective therapeutic strategies to eradicate tumors.
2.1 Biological and endogenous factors
6project_grants_public
gen_ce72dcebc272d150014e00ac915d6e36
Targeting TFEB transcription factor as a novel therapeutic approach for the treatment of Birt-Hogg-Dube’- associated kidney cancer
Worldwide Cancer Research
Telethon Institute Of Genetics And Medicine
HRCS22_16209
Birt-Hogg-Dube’ is a hereditary cancer syndrome, due to loss of function mutations in the gene encoding Folliculin and characterized by benign skin tumors, lung and kidney cysts and renal cell carcinoma. Renal tumors represent the most severe manifestation of the disease and to date surgery is the only available therapeutic intervention. The design of effective targeted therapies depend on a better understanding of the primary cellular pathways in which folliculin serves as tumor suppressor. We recently found that depletion of TFEB in a kidney-specific mouse model of BHD syndrome fully rescued cystogenesis suggesting a key role of TFEB in Folliculin dependent pathologies. Encouraged by these findings, in this grant application we propose to understand the contribute of TFEB to the development of kidney tumors in Flcn heterozygous mice, which faithfully recapitulate kidney pathologies in BHD disease. In addition, we aimed at dissecting the TFEB downstream pathways responsible for tumor growth in BHD patients and functionally characterize a novel mechanism of regulation of TFEB localization, which could represent a novel therapeutic strategy for the treatment of BHD renal tumors.
2.1 Biological and endogenous factors
6project_grants_public
gen_303c4bb32b2cfd131161892c09827889
Pre-clinical evaluation of combination treatments for invasive lobular breast cancer
Worldwide Cancer Research
Royal College of Surgeons in Ireland
HRCS22_16210
Invasive lobular carcinoma (ILC) is the second most common type of breast cancer (approximately 10-15% of all breast tumors). Women with ILC are usually older, used hormone replacement therapy and are more likely to have ER+ disease. Compared to other ER+ breast cancers, ILCs are more likely to metastasize to the peritoneum, gastrointestinal tract, and ovaries and are more frequently bilateral. Like other ER+ breast cancers, anti-estrogen resistance has emerged as a significant problem in the management of ILC, however, ILC is considered to be chemo-resistant and patients receive no additional benefit from chemotherapy. As such, there is a pressing need to develop tailored therapeutic options for endocrine-resistant ILC patients. As part of the FP7 RATHER project, we discovered that the BET protein Brd3 is a marker of poor prognosis in ILC but not in ER+ breast cancers as a whole. We subsequently validated this in an independent cohort from the METABRIC study. We also found that ILC cell lines that do not respond to anti-endocrine therapy are sensitive to BET inhibition (iBET), either as a single agent, or in combination with an FGFR inhibitor. Here we aim to progress our in vitro observations to anchor the therapeutic potential of iBET for endocrine-resistant ILC. To achieve this, we will capitalize on the recent discovery that ILC xenograft models can be readily established by mammary intraductal implantation. The efficacy of iBET either as a single agent or in combination with a FGFR inhibitor will be determined. One of our ILC cell models displays de novo resistance to iBET. ChIP-Seq, RNA-Seq and label-free quantitative mass spectrometry-based comparison of this model with our iBET sensitive ILC model will also allow us to dissect the transcriptional plasticity that underlies resistance to iBET and determine how best to overcome these in ILC patients.
5.1 Pharmaceuticals
6project_grants_public
gen_462388c127e3278955c13cf742959e02
Understanding spontaneous loss of heterozygosity
Worldwide Cancer Research
Institute Curie
HRCS22_16214
Using a novel model established in my team of spontaneous stem cell genome instability in fruit flies, our proposed work will address several important currently open questions about a major pathway of tumor suppressor inactivation: loss of heterozygosity via mitotic recombination. How do genomic features contribute to initiation of mitotic recombination? What tissue-intrinsic and extrinsic factors contribute to LOH initiation and neoplastic growth? We will explore these questions using whole-genome sequencing approaches combined with sophisticated Drosophila genetics. Our findings will bring to light mechanisms that are at work in vivo in adult stem cells and have numerous implications both for basic science as well as potential future preventative measures for patients.
2.1 Biological and endogenous factors
6project_grants_public
gen_9ab61cd6bc4a972e1cc2e12f9a8e12ff
Novel BBB penetrating peptide for the treatment of Parkinson’s disease
Cure Parkinson's
University of Helsinki
HRCS22_16255
The prevalence of neurodegenerative diseases, such as Parkinson’s disease (PD), is growing rapidly due to an aging population. Current treatments for PD only relieve symptoms and cannot stop the progression of the disease, thus there is an urgent need for new therapies. Neurotrophic factors (NTFs) are secretory proteins that regulate the growth, survival, regeneration and plasticity of neurons. They have been explored as novel drugs for the treatment of Alzheimer’s disease (AD), amyotrophic lateral sclerosis (ALS) and PD but their efficacy in clinical trials has been variable. Cerebral dopamine neurotrophic factor (CDNF), discovered in our lab, is an ER located protein that works completely differently from known NTFs, but protects and restores the function of dopamine (DA) neurons in rodent and rhesus monkey toxin models of PD more effectively than other NTFs. CDNF is currently in phase 1/2 clinical trials on PD patients in three medical centers. Despite the promising results in animal models of PD, NTF and CDNF-based treatments share a fundamental drawback: they require direct delivery to the brain through invasive surgery, since they cannot pass through the blood-brain barrier (BBB) and do not treat non-motor symptoms. Prof Saarma's group's recent discovery, however, may overcome this difficulty: they showed that a novel CDNF variant, the 61 amino acids (aa) long C-CDNF, enters DA neurons efficiently in culture. Furthermore, these data show that C-CDNF can cross through the BBB as measured by four different methods and has a neurorestorative effect in both 6-OHDA and MPTP toxin models of PD when administered subcutaneously. Although C-CDNF data are promising, they believe it is not yet the optimal CDNF-derived molecule for clinical trials. C-CDNF is 61 amino acids (aa) long and harbors three alpha-helical regions. The preliminary data suggest that deletion of helix 1 or helix 3 and the sequence after the helix 3 from C-CDNF does not affect the biological activity in vitro. Systemic administration of the shortest 33 aa long C-CDNF peptide (CDNF33) is even more effective in rat 6-OHDA model of PD and passes through the BBB more readily than the longer 61 aa long C-CDNF peptide (not shown). Thus, the goal of this project is to generate the smallest active C-CDNF polypeptides, using deletion and site-directed mutagenesis, and to optimize BBB penetration properties, stability, and activity to design the optimal C-CDNF polypeptide. Structural analysis will be followed by in vitro and in vivo testing in 6-OHDA and in alfa-synuclein (aSyn) aggregation models of PD in order to identify the best drug candidate for the disease. The innovative aspect of this proposal is a new groundbreaking concept of peripheral delivery of BBB-penetrating CDNF-derived polypeptides with trophic factor properties and their potential to simultaneously treat both the non-motor and motor symptoms of PD. The effect of C-CDNF variant on non-motor symptoms will also be tested and its mode of action studied.
5.1 Pharmaceuticals
6project_grants_public
gen_c09925a5fe2e427501ebed470e67ec22
Combinatorial Engineering of Proteolytically Resistant APPI Variants that Selectively Inhibit Human Mesotrypsin for Cancer Therapy
Worldwide Cancer Research
Ben-Gurion University of the Negev
HRCS22_16274
Mesotrypsin is a serine protease that is upregulated with tumor progression and associated with poor prognosis in many human cancers. In cancer models, mesotrypsin promotes tumor growth, invasion, and metastasis, making it an attractive target for therapeutic intervention. To date, no selective inhibitors against mesotrypsin, either natural or synthetic, have been reported. Developing selective inhibitors for mesotrypsin presents special challenges, as mesotrypsin shares high sequence homology and structural similarity with other serine proteases, and is resistant to inhibition by many polypeptide inhibitors. The human amyloid β-protein precursor Kunitz protease inhibitor domain (APPI) offers an attractive scaffold for engineering mesotrypsin inhibitors, but has the inherent disadvantage of rapid cleavage by the enzyme. In preliminary studies, we have used directed evolution to generate a novel prototype mesotrypsin inhibitor, based on the APPI scaffold, possessing picomolar affinity and improved proteolytic resistance to mesotrypsin. Herein, we propose to identify highly selective novel mesotrypsin inhibitors based on this prototype for clinical translation as imaging and therapeutic agents. We will use a yeast surface display platform and novel competitive screening strategy to identify selective mesotrypsin antagonists from inhibitor libraries. We will evaluate candidate inhibitors for mesotrypsin selectivity, proteolytic stability, and anticancer efficacy in cell culture models. Finally, we will begin preclinical evaluation of the best candidate as a targeting agent for tumor imaging and as a therapeutic. The proposed strategy is likely to produce mesotrypsin inhibitors of low toxicity and immunogenicity, with substantial translational potential as imaging agents and therapeutics. We further envision that our approach introduces a versatile platform that can be extended to target other serine proteases as well as other important target proteins, for the creation of new-generation therapeutics for cancer and other diseases.
5.1 Pharmaceuticals
6project_grants_public
gen_7f843d36245720da689a8b3c93783a15
Climate adaptation and sustainable rural health outcomes in Southern Africa
Wellcome Trust
Human Sciences Research Council
HRCS22_16275
The proposed project seeks to assess the complex health impacts of some of the major climate adaptation actions in rural Southern Africa, using case studies of Zimbabwean communities located in the mid-Zambezi Valley area, in the north of the country, along the borders with Zambia and Mozambique. The specific focus will be on investigating nutritional and psychosocial health impacts of two of the main adaptation actions in the case study area. The project is expected to run for 30 months and will build on a previous longitudinal (2010-2016) academic research work on livelihoods and climate change adaptation in the area undertaken by the lead applicant. A blended methodology combining qualitative and quantitative data collection and analytical techniques will be used. A Theory of Change, setting out the links between processes, activities, outcomes and context and the changes that occur in the short, medium and long term, will anchor the impact evaluation. The project ultimately aims to generate an evidence-based framework for tracking and assessing the health impacts of climate adaptation actions in marginal rural Southern African communities, and to proffer means and ways of harnessing the positive impacts and addressing the negative outcomes for rural household and community resilience
No Research Activity assigned
6project_grants_public
gen_0fbae4f6d98a397d5fc3677fd553d520
Identification of novel drug targets in the Notch pathway that are relevant to tumor formation
Worldwide Cancer Research
University of Milan
HRCS22_16294
Recent work in Drosophila melanogaster has revealed a central role of the endo-lysosomal compartment in Notch signaling activation. However, endo-lysosomal Notch signaling is ill-understood in mammals and strategies to counteract it have not emerged yet, limiting our ability to act on tumors arising from Notch signaling alterations. We postulate that endo-lysosomal Notch signaling might play a yet unrecognized important role in Notch-dependent tumorigenesis that awaits experimental exploration. In Task 1, we aim to study 30+ new drug targets that we have identified in a recent screen for trafficking regulators of Notch in human cells. For this, we have established a pipeline for quantitative assessment Notch trafficking and activation. New genes that are found to act in the endo-lysosomal activation process will be modulated in specific Notch-dependent cancer cell lines, upon depletion or by pharmacologic treatment. In Task 2, the Drosophila homologs of the most promising ones will be analyzed to determine their effect on Notch-dependent tumorigenesis using genetic assays in vivo. For such effort, we will generate mutants by CRISPR/Cas9 gene editing and will study them using Notch and endocytic assays that recapitulate features of tumorigenesis in vivo. In task 3, based on the indications of task 2, we will use Drosophila as a host for tumor transplantation and drug testing. In summary, we will identify new regulators of endo-lysosomal Notch signaling, understand their role in tumorigenesis, and characterize their potential pharmacologic modulators. At a time in which clinical therapies based on targeting Notch signaling are still an unfulfilled promise, detailed understanding of Notch events associated to the endo-lysosomal system will be key to devise more effective and less toxic pharmacologic options.
2.1 Biological and endogenous factors
6project_grants_public
gen_b15f8f0b58c1a4333cb2c1c3bbd8f905
Adoptive immunotherapy of acute Leukemia with CD1c retargeted iNKT cells
Worldwide Cancer Research
Fondazione Centro San Raffaele
HRCS22_16304
We have shown that a group of human T lymphocytes kills acute leukemia blasts by recognizing the leukemia-associated lipid antigen methyl-lysophosphatidic acid (mLPA) presented by the CD1c molecule, which is identical in all individuals, suggesting a donor-unrestricted adoptive immunotherapy approach. Indeed, we efficiently engineered total T lymphocytes from any donor with a selected mLPA-specific TCR to kill CD1c-expressing leukemia cell in vitro and in immunodeficient mouse xenografts. In this project, we aim at assessing whether the transfer of the mLPA-specific TCR into invariant Natural Killer T (iNKT) cells, a subset of lipid-specific lymphocytes expressing an invariant TCR restricted for CD1d molecules, may further improve efficacy and safety of this adoptive immunotherapy strategy. The advantages of using iNKT instead of T cells resides in their ability to: i. migrate into the bone marrow; ii. control graft versus host disease (GVHD) while promoting graft versus leukemia (GVL); iii. modulate/eliminate immunosuppressive cells in the tumor microenvironment (TME). We posit that iNKT cells may be harnessed to target, at the same time, leukemia cells with the mLPA-specific TCR, and therapeutically reprogram the TME with the endogenous one. The final aim of the project is to generate iNKT cells armed with a universal mLPA-specific TCR for off-the-shelf adoptive immunotherapy of human acute leukemia.
No Research Activity assigned
6project_grants_public
gen_8fce168ff591389867faeccc09dbf0af
Translational Control of the Immune Response in the Tumor Microenvironment
Worldwide Cancer Research
University of Milan
HRCS22_16313
No abstract available for this analysis.
No Research Activity assigned
6project_grants_public
gen_821844c9c3413023019c2fbde1cd63a9
Role of the interferon system in DNA replication fork stability and chemo-resistance
Worldwide Cancer Research
University of Zurich
HRCS22_16321
No abstract available for this analysis.
HRCS Research Uncodeable
6project_grants_public
gen_6354f90a7d1525287b7f259fc5ee5f92
Chromatin organization and transcription factor targeting in the establishment of oncogene-induced senescence and cancer progression.
Worldwide Cancer Research
Fondazione Centro San Raffaele
HRCS22_16334
Background. Oncogene-induced senescence (OIS) acts as a powerful tumor suppressive mechanism by inducing quasi-irreversible cell-cycle arrest when cells experience oncogenic stress. Senescent cells, however, can still overcome OIS leading to cancer onset. Senescence is caused by persistent DNA damage and it is characterized by massive chromatin reorganization and altered gene expression profiles, including the establishment of SASP, an inflammatory transcriptional program. Senescence can propagate to bystander cells, due to SASP-dependent paracrine and SASP-independent juxtacrine mechanisms. Key transcription factors (TFs) such as p53 and NF-kB mediate these processes but how chromatin impacts their activation and binding dynamics in OIS, in bystander senescence (BySen) and upon senescence overcome (SenOv) has been only marginally explored. Objective. We will test an uncharted hypothesis on early tumorigenesis: the massive chromatin reorganization observed upon OIS induction shape the search mechanism and the activity of the key TFs implicated in OIS, BySen and SenOv. To this end, we will integrate genomic technologies with unique microscopy approaches that we developed to probe TF activity at the single-cell and single-molecule level. Experimental Plan. We will model OIS – by oncogene induction - and SenOv – by histone demethylases overexpression - in human fibroblasts and melanocytes. We will analyze chromatin reorganization, TFs binding, and target gene expression in single cells using super-resolution and single-molecule microscopy to answer these questions: Does chromatin reorganization in OIS control the capability of TFs to find, bind and activate their targets? How does the further chromatin remodeling in BySen and SenOv affect TFs activity? How do paracrine signaling in modulate chromatin organization and TFs action? Significance. We will picture with single-cell resolution the interplay between chromatin architecture, TFs activity and paracrine signaling in a tumorigenesis model, leading to fundamental insights about early cancer progression that could be translated into the identification of novel druggable targets.
2.1 Biological and endogenous factors
6project_grants_public
gen_db1f6fe56b4301cd9b75b3350b31dc2d
Role of the RNA helicase DDX3X in Myc-induced lymphomagenesis
Worldwide Cancer Research
European Institute of Oncology
HRCS22_16355
No abstract available for this analysis.
No Research Activity assigned
6project_grants_public
gen_c82a4c643c1b2c1333b40f525b2c9534
Effects of intra-uterine HIV exposure on antibody functionality and B cell development
Wellcome Trust
University of Cape Town
HRCS22_16390
Approximately 30% of pregnant women in South Africa are HIV-infected resulting in over 300 000 infants exposed to HIV in utero every year. Rates of infectious mortality are reportedly higher among HIV-exposed uninfected infants (iHEU) compared to HIV-unexposed uninfected infants (iHUU). Studies evaluating the effects of maternal HIV infection on vaccine responses in their respective infants show that iHEU have similar antibody titres as those elicited in iHUU. These results imply efficacious vaccination in iHEU which contradicts the observed higher morbidity and mortality rates in iHEU. Therefore, I hypothesise that exposure to HIV in utero may impact the maturation of B cells leading to production of antibodies with poor Fc functions. Our preliminary analysis revealed altered B cell phenotypes across age in iHEU compared to iHUU. Therefore, in this study I aim to characterise B cell properties such as reduced somatic hypermutation for affinity maturation among iHEU using RNA-sequencing. Furthermore, I will analyse IgG Fc glycosylation that is associated with Fc-FcR affinity and Fc effector functions. Subsequently, I will develop in vitro models to measure antibody dependent cellular phagocytosis (ADCP) and cytotoxicity (ADCC) specific to early childhood vaccines.
2.1 Biological and endogenous factors
6project_grants_public
gen_ad32ffeefe662f7e18d7cfd0d7cbc2fd
Ultra-Sensitive, Switchable MRI Reporters for Lung Metastases
Worldwide Cancer Research
German Cancer Research Center
HRCS22_16398
Early cancer detection and visualisation of disease progression are highly relevant for succesful therapy. Imaging reporters for minimally invasive methods such as MRI are thus an indispensable component and visualising information of the biological complexity of tumour tissue beecomes increasingly important for choosing the right therapy. This is particularly relevant for metastatses detection which is a central aspect of diagnostic imaging. MRI provides excellent soft tissue contrast but suffers from low sensitivity, particularly in the lung which is low in tissue water density but is the most common organ targeted by metastases. MRI reporters with molecular spcificity for metastaes are therefore highly needed but cannot be implemented with conventional detection. Here we address this problem with an imaging method that includes a pre-targeting approach for LHRH receptor-carrying metastases and subsequent detection through-spin hyperpolarised xenon. The enhanced magnetization of this noble gas together with a saturation transfer technique from the molecular reporter onto the gas will enable ca. 10 million-fold enhanced sensitivity. We will design "smart" nanoparticles that selectively highlight the signal of xenon that has been in contact with these reporters that carry luteinizing hormone-releasing hormone (LHRH) for specific binding to cancer cells. This approach shall visualize pulmonary micrometastates from breast cancer cells. The method serves as an example for a molecular target from which a platform can be developped for different tumour markers. The combination of novel techniques from MRI physics and bioengineering will provide "switchable" MRI agents with a specific MR response frequency for xenon through the metastases-bound reporters and will bring diagnostic lung imaging to an uprecedented sensitivity level. A versatile design respects the need for easy adaptation to implement personalized diagnostics to interrogate the presence of various molecular targets.
4.1 Discovery and preclinical testing of markers and technologies
6project_grants_public
gen_d4f0807b2be3a578a274a80b945fe521
Determinants of EML4-ALK Driven Lung Cancer Growth and Therapeutic Responses
Worldwide Cancer Research
German Cancer Research Center
HRCS22_16421
Lung adenocarcinomas (LUAD) in non-smokers often harbour specific genetic aberrations, such as gene fusions involving the Anaplastic Lymphoma Kinase (ALK) and the echinoderm microtubule-associated protein-like 4 (EML4). This oncogenic fusion has been reported to have 15 different variants, which harbour the same ALK sequence but differ in the EML4 breakpoint. Variant 1 and variant 3 differ greatly in the proportion of EML4 and are two most frequent oncogenic inversion in ALK-positive patients. Despite the development of second-and third-generation ALK inhibitors, patient responses are limited by drug resistance. In half of relapsed cases, resistance arises from secondary mutations in the TK domain; however, the mechanisms of resistance in patients lacking secondary mutations remain poorly characterized. Besides, the impact of concurrent genomic alterations in EML4-ALK driven tumours remains largely unknown. TP53 is mutated in 30% of ALK-rearranged LUAD and appears to be synergistic with variant 3, giving rise to more aggressive human disease. Despite the direct relevance to patient treatment, how these co-incident genomic alterations influence the robustness of therapeutic responses has never been investigated. Here we propose to employ state-of-the-art technologies and methodologies to model Eml4-Alk variants in vivo and study the tumour evolution and dynamics of the most aggressive variants (Aim1). In Aim 2, we will determine the impact of co-incident genomic alterations on EML4-ALK-driven lung cancer development, by inactivating putative tumour suppressor genes in a variant-specific manner. Finally, in Aim 3, we will determine the impact of different Eml4-Alk variants and concomitant genomic alterations on response to ALK inhibition. By combining multiplex genome editing with drug treatment, we will perform the equivalent of hundreds of genotype-directed clinical trials in parallel and therefore this project will have a direct impact on personalised treatment for patients with EML4-ALK lung tumours.
2.1 Biological and endogenous factors
6project_grants_public
gen_a2b783270e9eebd6f811c01fd8d6514f
Oncogene discovery in esophageal cancer through interrogation of 3D regulatory landscapes
Worldwide Cancer Research
Max Planck Institute for Molecular Genetics
HRCS22_16429
Esophageal cancer is one of the most common and lethal cancers worldwide, yet currently no targeted therapeutics exists for its treatment. A critical roadblock in the development of targeted therapeutics for esophageal cancer is the lack of knowledge of the key oncogenic drivers in this cancer and the regulatory landscape that controls the expression of those genes. For example, genome-sequencing efforts of esophageal adenocarcinoma (EAC), the most frequent subtype of esophageal cancer, have revealed few frequently mutated genes to date, and those mutations have limited prognostic benefit, which suggests that mutations in non-coding DNA may play critical roles in EAC development. Gene expression programs are implemented in the three dimensional context of 3D chromosome structures, and our previous work suggests that the boundary elements of such 3D chromosome neighborhoods are frequently mutated in EAC. In this work, we propose to generate maps of the 3D regulatory landscape for multiple esophageal adenocarcinoma cells for the discovery of new oncogenic drivers. We will combine the 3D regulatory maps with whole genome sequence information to create a compressive catalog of candidate EAC driver mutations that occur in non-coding DNA elements, including chromosome neighborhood boundaries, and will use the 3D regulatory landscapes to identify oncogenes dysregulated around the mutated neighborhoods. We will functionally validate the candidate driver mutations and the new candidate oncogenes in cell line- and organoid models and using clinical information. This work will generate a functionally validated set of EAC oncogenes whose genomic sequence is otherwise intact. Results of this work will provide insights into the clinical utility of mutations disrupting chromosome neighborhoods in EAC, and facilitate the development and application of new therapeutic approaches for this common and deadly cancer.
2.1 Biological and endogenous factors
6project_grants_public
gen_f03e5353734b98356503042328a9f123
Mechanisms of resistance to anti-TGF-beta therapies in metastatic colorectal cancer
Worldwide Cancer Research
Institute for Research in Biomedicine
HRCS22_16434
Most colorectal cancer (CRC) patients die as a result of metastasis. Neither conventional chemotherapy nor current targeted therapies offer significant benefits once the disease has spread to distant organs. Immune checkpoint inhibitors have shown efficacy in only a small subset of patients bearing hypermutated CRCs. Arguably, these failures result from the incomplete understanding of metastatic CRC, which is in part due to lack of mouse models that recapitulate the advanced stages of the disease. We permuted conditional alleles of the WNT, EGFR, TGF-beta and p53 pathways in intestinal stem cells of mice. Quadruple mutant mice developed metastatic human-like intestinal adenocarcinomas. These tumors display key hallmarks of aggressive CRCs including T cell exclusion and a TGF-beta activated stroma. From these genetic mouse models we derived mouse tumor organoids (MTOs), which upon transplantation in immunocompetent recipients recreated both the primary and metastatic disease. Analysis of metastasis generated by MTOs revealed that tumor cells build an immunoprivileged microenvironment during liver colonization, by promoting T-cell exclusion and blocking the acquisition of a Th1 effector phenotype. Inhibition of TGF-beta signaling reverts this process and unleashes a potent cytotoxic T-cell response against tumor cells, which is long lasting and protects mice from metastatic disease. In mice with progressed liver metastatic disease, blockade of TGF-beta signaling rendered tumors susceptible to anti-PD-1/PD-L1checkpoint therapy. As poor prognosis CRCs are characterized by high levels of TGF-beta and immune evasion, our findings suggest that TGF-beta signaling inhibitors may have broad applications to treat patients with advanced CRC. However, we have discovered that a subset of mouse CRCs develop resistance to TGF-beta-based therapies during metastatic outgrowth. Here we propose to investigate this process and identify the mechanism co-opted by resistant CRCs to evade the action of the adaptive immune system triggered by treatment with TGF-beta inhibitors.
2.1 Biological and endogenous factors
6project_grants_public
gen_885d690711506c6d78ca8beb6599649b
International integrated analysis to identify markers of poor survival in high-risk neuroblastoma
Solving Kids' Cancer
Vall d'Hebron Hospital Universitari
HRCS22_16441
The outcome for patients with high-risk neuroblastoma is currently inadequate despite intensive multimodal therapy and despite continued treatment intensification (such as the addition of immunotherapy or tandem high-dose chemotherapy). To date, most patients with high-risk are treated in a broadly similar manner. Many researchers have looked at biomarkers (biological characteristics) that could identify those patients with the poorest outcome right from the time of diagnosis, so that these patients could be offered novel therapies very early in their disease course. So far, despite hundreds of studies evaluating factors routinely used in the clinic (such as age or number of metastasis) or more advanced technologies including complex genomic analyses, none of these biomarkers has been validated nor is available for clinical-decision making. The novelty of our approach is that we aim to integrate all (or as many as possible) biomarkers that have been described to date, pull together all the data, and analyse and compare all biomarkers head to head in the same dataset. We will do this in three phases: First, we will look at all published literature about neuroblastoma biomarkers from 1995 onwards and conduct a thorough analysis (this is called a systematic review and meta-analysis) to select the best biomarkers reported to date. Second, we will gain access to patient databases for each of the biomarkers (at the moment, each biomarker has been evaluated in a separate study/dataset) and create an enlarged INRG database including as many datasets, variables and biomarkers as possible; to then conduct an analysis integrating all the data together, which is called multivariate analysis. Thirdly, in order to confirm which biomarker(s) found on the first two phases are definitely the best one(s), we will analyse the best performing biomarkers in a homogeneous cohort of patients treated within a contemporaneous clinical trial, i.e. repeating all biomarkers in the same patients (which has never been done), to confirm which one(s) perform better and is taken forward to make treatment decisions in real clinical practice. With this ambitious approach, our proposal aims to identify patients with high-risk neuroblastoma with the poorest outcome at the time of diagnosis, with the final aim of giving these patients access to early therapies upfront, without waiting for them to relapse or become refractory to treatment.
4.1 Discovery and preclinical testing of markers and technologies
6project_grants_public
gen_62f9927e81b95dd91c2b860fce8bff0e
Targeting CPEB-mediated translational balance in Hepatocellular Carcinoma
Worldwide Cancer Research
Institute for Research in Biomedicine
HRCS22_16442
No abstract available for this analysis.
HRCS Research Uncodeable
6project_grants_public
gen_d46680739a45054cad449254bbccacbe
Resolving the tumour suppressor function of the E3 ubiquitin ligase Parkin
Worldwide Cancer Research
Walter and Eliza Hall Institute of Medical Research
HRCS22_16445
The E3 ubiquitin ligase Parkin is critical for maintaining mitochondrial quality control as it is the key effector in damage-induced mitophagy. As well as a have key role in early onset Parkinson’s disease, Parkin is emerging as an important tumour suppressor, with mounting evidence indicating that the gene encoding Parkin (PRKN/PARK2) is one of the most frequently deleted or mutated genes in a wide variety of cancers including glioblastoma, colorectal, breast cancer. We have also identified links between Parkin loss and neuroblastoma, a common childhood cancer with poor prognosis and limited treatment options. How Parkin limits the growth of such diverse tumours is unclear. We hypothesise that the ability of Parkin to limit inflammation promoted by damaged mitochondria underpins its tumour suppressor function. We will use complementary cell and new mouse models coupled with innovative proteomics analyses to resolve how the E3 ubiquitin ligase Parkin controls inflammation as well as critical processes including apoptosis, cell cycle regulation and metabolism to define how Parkin activity limits the development of neuroblastoma. This proposal will identify Parkin or its specific substrates as new targets to treat neuroblastoma and potentially other cancers.
2.1 Biological and endogenous factors
6project_grants_public
gen_8f7aec31911494f31e59c727e2fc5756
Harnessing immune circadian rhythms to advance anti-tumour immunotherapeutic strategies
Worldwide Cancer Research
Pompeu Fabra University
HRCS22_16446
No abstract available for this analysis.
HRCS Research Uncodeable
6project_grants_public
gen_79d56275bdbb9eeaa8903942708b6eea
PRECISION - supplementary award
Cancer Research UK
Netherlands Cancer Institute
HRCS22_16455
Background Ductal carcinoma in situ (DCIS) now represents 20-25% of all breast neoplasia. This is due to large-scale detection by widely adopted population-based breast cancer screening programs . As a result, thousands of women are confronted with DCIS each year: more than 8,000 in the UK, 2,300 in the Netherlands, and over 50,000 in the US. Conventional management includes surgery, supplemented by radiotherapy and/or endocrine therapy, but overtreats the majority of DCIS as ~1% recur annually and breast cancer mortality is ~3% at 20 years. Uncertainty as to which DCIS lesions will progress to invasive cancer or, after excision, which will return with recurrent DCIS or invasive breast cancer drives this overtreatment. Distinguishing DCIS that may progress to lethal disease from the majority of harmless DCIS is therefore an urgent need to save thousands of women with low risk DCIS the burden of radical treatment without any survival benefit. Aim We aim to reduce the burden of overtreatment of DCIS (surgery, radiation therapy, hormonal therapies) through the development of novel tests that promote informed and shared decision-making between patients and clinicians, without compromising the excellent outcomes for DCIS management presently achieved. Methods First, three large retrospective DCIS cohorts (and supplementary sources) will be collected enabling subsequent in depth molecular studies. Second, extensive genomic characterisation, immune profiling and imaging analysis will be performed. In vivo and in vitro modelling will be performed to study the biology of DCIS in detail. Finally, all clinical, immune, and molecular data will be incorporated into a clinical risk prediction model. This risk prediction model can be validated in three prospective DCIS trials. Specifically, in the current project we will a.) collect tissue and blood samples from these trials and b.) start validation of the biomarkers detected in the retrospective series. How the results of this research will be used The discoveries from our laboratory studies, including a risk stratification model, will be cross-validated in prospective trials of DCIS active surveillance versus conventional treatment (the LORIS, LORD and COMET trials). As such, the main result of this study will be that we can identify a group of women for which active surveillance for DCIS could be a safer alternative to intensive treatment. Ultimately, this may also contribute to a more reassuring perception of risk regarding non-life threatening precancerous lesions in general, reducing anxiety and preserving quality of life.
2.6 Resources and infrastructure (aetiology)
6project_grants_public
gen_adcb7da91c067a15c348736677fb974a
How can we harness cancer mechanobiology to prevent tumour progression?
Worldwide Cancer Research
University of South Australia
HRCS22_16463
Metastatic breast cancer is currently incurable and new methods to halt invasion and metastasis are an important unmet clinical need. Rho-ROCK signalling in tumour cells is a key arbiter of the paracrine program by which cancers shape their microenvironments. We have established using an in vivo model of mammary cancer, that two key proteins, Creld2 and Ptx3 secreted by progressive breast cancers of all subtypes, mediate the recruitment and polarisation of cancer-associated fibroblasts (CAFs) and tumour-associated macrophages (TAMs). Crucially, these genetically normal cells are brought into the tumour's influence to promote tumour progression. We hypothesise that specific effector pathways downstream of ROCK synergise with tumour mechanobiology to selectively mediate the production and secretion of these paracrine factors. Identifying crucial signalling nodes within these pathways may prove a fertile source of novel approaches to target cancer progression. These studies complement ongoing research to identify the receptors in CAFs and TAMs that mediate the functions of Creld2 and Ptx3. Here we plan to: 1. Employ an in vivo model of microenvironment education and multiplex immunolabelling approaches to characterise the molecular mechanisms underlying the production and secretion of Ptx3 2. Adapt ex vivo primary cell culture approaches and in vivo functional assays to assess the contributions of ECM stiffness and the tumour secretome to the production of tumour-promoting CAFs and TAMs 3. Employ an alginate-based ex vivo 3D culture system to establish the biological relevance of CRELD2 and PTX3 to the tumour microenvironment of human patient samples. We believe that this highly innovative approach to understanding the interplay between signalling and mechanobiology in the tumour microenvironment will allow us to identify novel vulnerabilities that permit the targeting of breast and other cancers.
2.1 Biological and endogenous factors
6project_grants_public
gen_83e3dbf83ec6033207321f7963d0941e
Motorized Traffic and Risk of Childhood Leukaemia
Children with Cancer UK
University of Modena and Reggio Emilia
HRCS22_16494
This research project aims to assess the association between exposure to chemicals emitted by motorized traffic and industry emissions and childhood leukaemia onset. Specifically, we plan to carry out measurements of motorized traffic contaminant exposure through laboratory analyses of these pollutants and their metabolites in blood, urine, and hair samples of children newly-diagnosed with leukaemia at the Paediatric Haematology/Oncology Clinics of the Parma, Modena and Catania University Hospitals (n=100) along with sex- and age-matched controls (n=100) in 2019-21. The overall metabolomics pattern potentially induced by these air pollutants will also be assessed. We will focus on contaminants that are established or suspected carcinogens, including benzene, 1,3-butadiene, and polycyclic aromatic hydrocarbons (PAHs) together with heavy metals and metalloids, to clarify the role of these substances, and their mixtures, in the aetiology of childhood leukaemia. We will eventually seek to confirm exposure to motorized traffic by direct measurements through the installation of benzene and PM sensoring systems within the vicinity of the subjects residential houses. Moreover, we will assess potential lifestyle risk factors (including diet, smoking habits and occupation) through the administration of questionnaires to the children and their parents.
2.2 Factors relating to physical environment
6project_grants_public
gen_fc40e29d45bbda9de7c3a1ffc5772033
Characterisation of gene-lifestyle interactions associated with obesity-related traits in African populations
Wellcome Trust
University of the Witwatersrand
HRCS22_16509
Obesity is increasing in Africa and elsewhere, but little is known of how genetic and environmental factors interact.The proposed research has the following components: (i) discovery phase: heterogeneity of variance analysis (HEVA) to determine candidate gene-environment interacting (GEI) variants in the Human Hereditary and Health in Africa (H3Africa) cardiovascular (CVD) working group to replicate the top-ranking signals. Additional variants will be selected from the GWAS trans-ethnic meta-analysis of East Asians, Europeans and Africans based on exhibiting heterogenous effects across the ethnic groups. Gradient Boosted LD adjusted (GraBLD) machine learning algorithms will be used to compute highly predictive genetic risk scores; (ii) cross-sectional and longitudinal analysis phase: single candidate GEI variants and genetic risk scores (of candidate GEI variants and from GraBLD analysis) will be combined with lifestyle factors in generalised linear models (cross-sectional analysis) and linear mixed models (repeated measures analysis) to determine plausible variants responsible for gene-lifestyle interactions in obesity-related traits. Assessments of sensitivity and specificity will be used to determine the predictiveness of these gene-lifestyle interactions and genetic risk scores. The transferability of the identified GEIs across African and European populations will be evaluated. The proposed research may help elucidate relevant risk factors and/or identify obesity intervention targets.
2.1 Biological and endogenous factors
6project_grants_public
gen_3e9bfd4a7fefbdfc12d1e75ad06bfc1f
Causes and consequences of mental health challenges in autistic individuals during the transition to adulthood
MQ Mental Health Research
Karolinska Institutet Innovations (Sweden)
HRCS22_16513
Research Questions Autistic individuals1 face profound challenges across their lives, including high rates of mental health problems. Many of these mental health problems emerge during the transition to adulthood, as illustrated in Figure 1, highlighting the critical need to understand mental health problems faced by autistic individuals during this period. The purpose of this project is to investigate causes and consequences of educational, occupational, and health challenges mental health problems in autistic individuals during the transition to adulthood. As shown in Figure 2, I will: 1._x0001_Identify risk factors for mental health problems in transitional-aged autistic adults: a) genetics; b) comorbidity; c) trajectories of childhood autistic traits. 2._x0001_Assess sex-specific mental health problems 3._x0001_Investigate long-term impacts of mental health problems during the transition to adulthood. Methods Cohorts: I will leverage three richly phenotyped cohorts, with follow-up spanning childhood and adulthood. Their coverage is shown in Figure 2. Swedish registers record healthcare, education, and welfare for the entire Swedish population. I have identified 26,538 autistic individuals from these registers, born 1973-1998, followed-up to ages 18-43. The Child and Adolescent Twin Study in Sweden (CATSS) includes 18,250 participants (10,848 genotyped), followed-up from ages 9-24. The Twins Early Development Study (TEDS) includes 7,124 participants, followed from birth to age 21. Risk factors, mental health problems, and outcomes are all assessed using questionnaires and registry data. All data are collected and have ethical approval. Study Designs: I will define genetic factors linked with autism using polygenic scores, and test their association with mental health problems from ages 18-24 in CATSS. The utility of genetic factors in predicting these outcomes will be compared with more conventional indicators, such as symptoms (aim 1a). I will test whether the association between autism and mental health problems is mediated by comorbidity in CATSS and Swedish registers (aim 1b). I will compare mental health problems at ages 16-21 across trajectories of autistic traits in TEDS, identified using latent growth curve models (aim 1c). Sex-specific mental health problems at ages 18-24 in CATSS and Swedish registers will be assessed using a stratified cohort design (aim 2). I will compare long-term outcomes from ages 25-43 between autistic individuals showing different numbers of mental health problems during the transition to adulthood, utilizing the extended follow-up of Swedish registers (aim 3). Regression models will be the chief analytic approach in all aims, with models varying by outcome (linear, logistic, or time-to-event). Expected Outcomes This truly interdisciplinary project unites contemporary methods and concepts from epidemiology, behavioural genetics, and lifespan psychology to address three novel aims of crucial priority in autism research. This project will move the field beyond establishing that autistic adults face challenges, towards assessing why this is so. This will subsequently inform strategies to identify and support particularly vulnerable autistic adults in navigating adult life, with the goal of improving their mental health and wellbeing by lifting barriers to their participation in society. 1'Autistic individuals' is the preferred terminology of many stakeholders when referring to autism
2.1 Biological and endogenous factors
6project_grants_public
gen_6d17cd26acca2b5ae4e11085d917d909
Making vital connections; exploring how desmoplasia gets on pancreatic cancer's nerves
Worldwide Cancer Research
Temple University Health System
HRCS22_16520
Pancreatic ductal adenocarcinoma (PDAC) will soon become the second USA deadliest neoplasia. PDAC features a fibrous stroma, desmoplasia, with low blood/nutritional supply and extensive innervation (neoneurogenesis/axonogenesis) associated with poor prognosis, neuropathy and perineural invasion. The roles of cancer-associated fibroblasts (CAFs) and their self-derived desmoplastic extracellular matrix (D-ECM), in aiding nutritional-deprived tumor survival and neoneurogenesis, which affect PDAC’s onset/progression, are understudied. Using mRNA from patient’s D-ECM producing CAFs vs. matching normal stellate cells, we discovered a specific glutamatergic presynaptic protein. A simultaneous multiplex immunofluorescent approach validated its overexpression and that of its postsynaptic partner. Increased expression of these proteins is typical in neurological syndromes allied to increased cancer risk. Preliminary data indicated that: D-ECM and CAFs increase both neuron and tumor cell longevity; functional synapses between CAF and other cells are formed; and, CAF-secreted vesicles expressing presynaptic protein engage with PDAC cells, expressing the postsynaptic protein to promote tumor growth. Hypothesis: PDAC is locally enabled by D-ECM, CAFs, neurons and CAF secreted vesicles in a synapse-like mechanism. Aim1: Investigate if CAFs support neoneurogenesis and PDAC cells in a synapse-like manner. We will test the functional need for CAF and neural synaptic proteins, as well as downstream enzymes and other proteins, in maintaining high extracellular glutamate levels, posited to sustain neoneurogenesis and PDAC survival under nutritional stress. Our D-ECM system together with gain and loss of function approaches will be used to investigate the synaptic protein roles in these processes. Aim2: Assess requirement for and level of “pre- and post-synaptic” proteins linked to human PDAC. We will use our multiplex immunofluorescent approach in a well annotated human PDAC cohort to question neoneurogenesis levels associated with quantities and locations of the synaptic pair and question their association with patient outcomes. Vesicles, positive for the pre-synaptic protein, will be probed in matching liquid biopsies.
2.1 Biological and endogenous factors
6project_grants_public
gen_400d5632ef2530d52502b27892b96f78
Enhancing CAR-T cell therapies for gliomas
The Brain Tumour Charity
Harvard University
HRCS22_16527
No abstract available for this analysis.
No Research Activity assigned
6project_grants_public
gen_fe07735477a82674108f8aa1b67877e9
Understanding the immune cell dysfunction in pediatric lymphoma
Worldwide Cancer Research
Karolinska Institutet Innovations (Sweden)
HRCS22_16528
Lymphoma is the third most common type of cancer among children and accounts for 10-15% of all childhood cancers. Lymphoma in children and adults are divided into three main groups; Hodgkin lymphoma, B- and T-cell lymphoma. Lymphoma stems from faulty activation of B cells and, less frequently, T cells. Upon an infection, B cells undergo affinity maturation in germinal centers. B cells in germinal centers mutate their antibody-coding genes in a process that induce DNA damage during rapid B cell proliferation. B cell transformation into lymphoma may occur because of faulty DNA repair, defects in cell division, and impaired migration and interaction with other cells during the process of mutating and selecting the best fit B cells for antibody production. Patients with inborn errors in the immune system, primary immunodeficiency diseases, are prone to develop pediatric and adolescent lymphoma of very poor prognosis. An unmet need is to understand the molecular and cellular pathophysiology to define better treatment for patients with pediatric lymphoma, both in the primary and relapse setting, and for poor prognosis lymphoma of primary immunodeficiency patients. The goal of this project is two-fold: 1) To gain molecular insight into pediatric lymphoma by analysis of single cells from lymphoma specimens and 2) To develop and use methods where specific alteration in gene expression can be examined functionally and for defining new personalized and generalized therapeutic approaches.
2.1 Biological and endogenous factors
6project_grants_public
gen_eeb08da993b2c367d522457ad46a5e84
A computational approach to identifying trans-diagnostic predictors of antidepressant response
MQ Mental Health Research
Trinity College Dublin
HRCS22_16535
We need to develop tools that can improve the precision with which we allocate treatments in psychiatry. Current psychiatric disease classifications (DSM-5, ICD-10) ensure reliable diagnoses across clinicians, but their diagnostic categories do not allow for individual treatment predictions – for example, most patients with major depression do not recover after their first antidepressant treatment. This project aims to remedy this by using machine learning to develop an algorithm that can quantify how likely an individual is to respond to a range of antidepressant medications. Large samples are required to train a robust predictive model. To achieve this, all data collection will be web-based and subjects will be recruited worldwide. Predictors will include cognitive tests, which are linked to specific brain circuits and neurotransmitter systems that relate to psychiatric disease and treatment action, clinical symptoms and treatment variables, and demographics. Once the algorithm has been trained and validated on an independent test set, we will test its generalizability in three local clinics. The long-term goal of this work is to develop a tool that can be used by clinicians to achieve individual treatment predictions, something that will ultimately be tested in a randomized controlled trial.
4.1 Discovery and preclinical testing of markers and technologies
6project_grants_public
gen_cc56e0228e76e5c2e55b1e705687218e
Understanding oncogenic transcriptional processes in synovial sarcoma
Worldwide Cancer Research
Trinity College Dublin
HRCS22_16545
Synovial sarcoma is an often aggressive malignancy primarily effecting children and young adults. Unfortunately, these tumours often do not respond well to conventional therapeutic interventions and overall survival rates remain poor. The disease is caused by a recurrent chromosomal translocation generating a characteristic fusion protein, SS18-SSX. In synovial sarcoma cells the fusion protein assembles into SWI/SNF complexes, which are essential chromatin regulators that alter chromatin structure to regulate gene expression. Incorporation of SS18-SSX into these complexes alters their function, resulting in significant oncogenic gene expression changes. Targeting the oncogenic function of SS18-SSX presents and attractive therapeutic option for the development of mechanistically anchored cancer treatments; however the fusion itself has proven intractable for direct targeting. Here, we are pursuing a research program aimed at better understanding the function of SS18-SSX containing complexes and exploring new opportunities to therapeutically target complexes containing the fusion protein. Using CRISPR/Cas9 based approaches in functional genomics screening we’ve identified the acetyltransferase enzyme p300 as functionally essential in synovial sarcoma cells. By combining these data with in depth proteomic analyses of SS18-SSX interacting proteins we further identified that p300 physically associates with SS18-SSX. In Aims 1 and 2 of this research program we are combining biochemical and genomics approaches to characterise the composition and function of SS18-SSX/p300 containing complexes in synovial sarcoma cells. In Aim 3 we are taking an ambitious approach combining CRISPR/Cas9 screening with single-cell genomics analysis platforms to characterise the molecular function of SS18-SSX containing complexes in cancer cells. Together, these aims will provide a mechanistic understanding and potential therapeutic rationale for targeting p300 function as a novel therapy in patients. In addition to providing unprecedented molecular understanding of disease biology, which will likely highlight more opportunities to explore and develop future approaches to more effectively treat synovial sarcoma patients.
2.1 Biological and endogenous factors
6project_grants_public
gen_32f69f1719f5ab612e881cf48aeaacb6
Hysteresis conveys the EMT spread to the neighborhood tumor cells and metastasis
Worldwide Cancer Research
CERCA Institution
HRCS22_16556
Cancer metastasis is a complex process with multiple challenges that disseminated tumour cells need to overcome to form overt metastasis, yet many biological aspects of metastasis are poorly understood. One of the most studied tumour cell phenotypic changes during metastasis is the epithelial-to-mesenchymal transition (EMT). This is a reversible transdifferentiation into a mesenchymal phenotype that favours cancer cell dissemination and cell plasticity to adapt to new conditions. We have recently described the dynamics of EMT at single cell level, by a molecular feedback loop that governs a bistable switch among the epithelial and mesenchymal state, also called hysteresis. However, how the dynamics affects group-decision processes at the cell population level has not been yet well studied. Remarkably, our recent data in 2D cell cultures show that EMT-like cells can spatially spread the EMT induction to the neighbourhood when the recipient cells are wired to undergo EMT by hysteresis. In this proposal, we aim to study the collective effects of hysteretic-EMT that resonates into spreading the EMT across cancer cell populations and its epigenetic mediators. For this purpose, we will use unique models of hysteresis and non-hysteresis cancer cells that we have previously generated, and inducible EMT systems with precise space and time control in 3D cultures in vitro, as well as in vivo breast cancer models. Advanced microscopy imaging, flow-cytometry analysis, ATAC-sequencing, and genome-wide DNA methylation will be used to model and unveil the mechanisms involved in the EMT spreading effect. Overall, this proposal will characterize the ability of cancer cells to amplify the EMT signal received by tumour cells, and the epigenetic mechanisms underlying it. This can lead to specific treatments to disrupt the spreading effect in order to halt cancer metastasis.
2.1 Biological and endogenous factors
6project_grants_public
gen_2f1105a4f7a7f4b610ef9810946661a3
Chlomipramine derivatives to target the ubiquitin ligase NEDD4 in lung cancer
Worldwide Cancer Research
FIRC Institute of Molecular Oncology
HRCS22_16567
No abstract available for this analysis.
No Research Activity assigned
6project_grants_public
gen_fb30e657d54aff27c027d377fabd4f11
Antimicrobials and antimicrobial resistance (AAMR)
Wellcome Trust
University of Nottingham
HRCS22_20539
No abstract available for this analysis.
Missing/Incomplete
6project_grants_public
gen_77d92e9a7628adb155b41e092c9daebc
Mechanisms of inflammatory disease.
Wellcome Trust
University of Birmingham
HRCS22_20629
No abstract available for this analysis.
Missing/Incomplete
6project_grants_public
gen_5422e382c95be38e247a37f5a624a910
Tissue Repair
Wellcome Trust
University of Edinburgh
HRCS22_20633
No abstract available for this analysis.
Missing/Incomplete
6project_grants_public
gen_cd8822c3d8350c939513b9fdec9ed6c8
Molecular and Cellular Basis of Infection
Wellcome Trust
Imperial College London
HRCS22_20654
No abstract available for this analysis.
Missing/Incomplete
6project_grants_public
gen_06bcffcfaec74710d0ae9796d1f1c6ee
Cell therapies and regenerative medicine.
Wellcome Trust
King's College London
HRCS22_20670
No abstract available for this analysis.
Missing/Incomplete
6project_grants_public
gen_bdb880e2e8f6a642f82387c97eace49d
BeSecAT: Blockchain-enabled Secure and Resilient Supply Networks for Advanced Therapies Medicinal Products
Innovate UK
Cardiff University
HRCS22_21294
Abstracts are not currently available in GtR for all funded research. This is normally because the abstract was not required at the time of proposal submission, but may be because it included sensitive information such as personal details.
Missing/Incomplete
6project_grants_public
gen_9ffa039131d68a566654ae5564d8294d
RespiTrain - A novel medical technology to reduce mechanical ventilation duration by 30%
Innovate UK
University of Oxford
HRCS22_21632
no public description
Missing/Incomplete
6project_grants_public
gen_18b3e15d13421cd3f95b56d528b19be1
Songbirds Music UK: Music Interaction Telepresence Robot Research Trial
Innovate UK
Songbirds Music UK Cic
HRCS22_21895
Songbirds Music UK: Music Interaction Telepresence Robot Research Trial Songbirds Music UK (Northwest England) run an award-winning hospital musical interaction programme with children with complex healthcare needs and disabilities. SMUK will research, develop and evaluate the use of a telepresence robot as an interface for delivering enhanced remote live musical interaction sessions with hospitalised children in paediatric settings. The research will use music as a vehicle to innovate new knowledge for professionals across health and communication in using computerised devices to interact with patients at increased risk of isolation due to debilitating illness, limited communication, immuno-suppression and COVID-19 restrictions.
Missing/Incomplete
6project_grants_public
gen_f2a39981cd68f004ddf9d995d9b06b0e
University of the West of Scotland and Kibble Education and Care Centre
Innovate UK
University of the West of Scotland
HRCS22_22045
To develop an advanced remote health monitoring and behavioural analysis solution utilising machine vision and artificial intelligence to improve operational efficiency, enhance care provision and generate new revenue streams through commercialisation of resulting IP.
Missing/Incomplete
6project_grants_public
gen_b4952a1054c2399518befea36878c8d5
Advancing Nuclear Science via Theory and Experiment
Science and Technology Facilities Council
University of Surrey
HRCS22_22487
For a hundred years, atomic nuclei have been probed more or less exclusively by studying collisions between stable beams and stable targets. This restricted the nuclei that could be studied to just a just a small fraction of those that are thought to exist. Most of the nuclei important to making all of the elements (in various stellar processes) have for example been inaccessible to experiment. The major thrust in nuclear physics worldwide, and a key priority in the UK's programme, is to reach out and study these exotic nuclei by using beams produced from short-lived radioactive isotopes. This in turn reveals that nuclear structure is not always like it seems to be for the stable nuclei, and nuclei are found to have surprising trends in stability and to have different shapes that will affect reaction rates inside stars and supernovae. At Surrey we take the UK priorities and the new opportunities very much to heart, and we seek out and lead programmes at the world's best facilities for making radioactive beams. To make the beams is difficult and the facilities - as well as the research effort - are international in scale. Surrey builds and runs innovative experimental equipment at these facilities. The present grant request is focused on the exploitation of the best capabilities at the best laboratories. Experimental progress is intimately linked with theory, and the development of novel and better theoretical approaches are a hallmark of the Surrey group. An outstanding feature of the group as a whole, which is key to our research plans and acknowledged as a rare and valuable strength, is our powerful blend of theoretical and experimental capability. Our science goals are aligned with current STFC strategy for nuclear physics, as expressed in detail through the Nuclear Physics Advisory Panel's road map. We wish to understand the boundaries of nuclear existence, i.e. the limiting conditions that enable neutrons and protons to bind together to form nuclei. Under such conditions, the nuclear system is in a delicate state and shows unusual phenomena. It is very sensitive to the properties of the nuclear force. It is unknown whether, and to what extent, the neutrons and protons can show different collective behaviour or even how many neutrons can bind to a given number of protons. It is features such as these that determine how stars explode. To tackle these problems, we need a more sophisticated understanding of the nuclear force, we need more powerful theories that can build this understanding into the calculations, and we need experimental information about nuclei with unusual numbers of neutrons relative to protons so that we can test our theoretical ideas. Therefore, theory and experiment go hand-in-hand as we push forward towards the nuclear limits. An overview of nuclear binding reveals that about one half of predicted nuclei have never been observed, and the vast majority of this unknown territory involves nuclei with an excess of neutrons. Much of our activity addresses this "neutron rich" territory, exploiting the new capabilities made possible with radioactive beams and exploiting advances in computational power and analytical theories to bring superior new theoretical tools to bear on the latest observations. Our principal motivation is the basic science and the STFC "big questions", and we contribute strongly to the world sum of knowledge. The radiation-detector advances that our work drives can be incorporated in medical diagnosis and in environmental management. We engage strongly with the National Physical Laboratory on these topics. Our work also relates to national nuclear security and we have strong links in this area with AWE. We provide excellent training for our research students and staff, many of whom go on to work in the nuclear power industry, helping to fill the current skills gap. Furthermore, we are enthusiastic about sharing our research, and actively pursue a public engagement agenda
Missing/Incomplete
6project_grants_public
gen_68a1d7bb73ee3a06da3b4e970fc4bc16
Astronomy at the Open University 2017-2020
Science and Technology Facilities Council
The Open University
HRCS22_22489
Our research programme, Astronomy at the Open University, covers the breadth of cosmic evolution, from dark energy to the birth of planets. We do this research by observation, laboratory experiments, simulations and modelling. We use purpose-designed laboratories and instruments, and instruments on telescopes and spacecraft to make our observations and measurements. Our group is based in the Department of Physical Sciences at the OU. So what are we trying to find out? We have 8 separate projects, from exoplanets and stars to distant galaxies. We already know a lot about how the Solar System came about. The Sun and planets formed from a cloud of dust and gas about 4570 million years ago. The cloud collapsed to a spinning disk and dust and gas spiralled inwards. The core of the disk became hot, forming the Sun, while the leftover dust and gas formed the planets. Boulders gravitated together to make planets, but no-one knows how the dust grains became boulders. We are experimenting with colliding centimetre-sized particles in zero-gravity conditions to see if they stick together, to find the missing link in how planets form. We also look at processes that cause stars to change as they age. Only recently has it been recognised that so many stars are binary systems, where two or more stars are in close association and affect each others' motion. Such systems affect the way mass and energy is lost from a star, and how they are transferred into the interstellar medium. We will study how 'binarity' affects the behaviour of massive stars (>20 times the mass of the Sun) and low mass stars (< the mass of the Sun), and how star populations change as they age. Studying these effects is vital, because the environment of a star influences any planets that surround it. Many hundreds of planets have been discovered around other stars (exoplanets) and we are working to describe the range of properties of these planets, especially when they are located close to their central star. A star can even completely destroy a close-in exoplanet, which could be an important new source of dust in the nearby universe and even in distant galaxies in the early Universe. Also in the early Universe, we can use the way that galaxies warp space and time to learn about the dark matter that surrounds them, and the dark energy that drives them apart. What else do we do? We build and test instruments for ground-based telescopes and for space missions, striving to make them smaller and lighter, and explore how they can be used on Earth for medical or security purposes. One of the most important benefits of our research is that it helps to train and inspire students: the next generation of scientists and engineers. We also enjoy telling as many people as possible about our work, and what we have learned from it about our origins.
Missing/Incomplete
6project_grants_public
gen_c19c07c83850e499fd34db38ef8a56e6
Demonstration of a base metal Low-temperature Catalytic converter for deNOx (LowCat Demo)
Science and Technology Facilities Council
University of Leeds
HRCS22_22491
Poor air quality results in around 400,000 excess deaths per year in Europe. In 2017, 39 % of NOx emissions were from road transport, with a further 45 % from sectors which include combustion or chemical processing. 86 % of observations which exceed EU air quality legislation regarding ambient NO2 concentrations were made at roadside stations, with 98 % of these in urban or suburban areas. Diesel powered vehicles and machines are one of the major sources of NOx pollutions. Though the sales of pure petrol and diesel vehicles will be banned in 2030 according to the Prime Minister's ten point plan, the policy only applies to light duty vehicles. For heavy duty vehicles and machines, diesel engines will still be the main powertrains in the foreseeable future. There are various technologies for NOx emissions control from diesel engines. SCR (Selective Catalytic Reduction) as one of the major deNOx technologies has been used in HDVs for more than a decade. However, the NOx conversion efficiency of SCR at low temperature (<200 oC) is poor (less than 50%). In addition, the high cost of SCR catalyst has hindered its wide application in small to medium engines. This project has developed an iron silicate material, originally intended as an analogue for meteoric material in Venus' atmosphere, which we have shown to be effective in catalysing the selective reduction of exhaust NOx over N2. The material shows a remarkable ability to catalyse the reduction of NO2 at low (including ambient) temperatures, so we refer to it as LowCat. The aim of this project is to scale up production of this low-cost novel catalyst, improve and consolidate its advantages on low temperature NOx conversion and demonstrate its conversion efficiency and cost effectiveness in real world applications. The ultimate goal of the project is to contribute to better urban air quality and improve human health and well-being.
Missing/Incomplete
6project_grants_public
gen_3bf75dcbde9f25e56df6f9bc41b2dc39
Depleted Monolithic CMOS and Ultra-fast silicon detectors for particle physics and other applications
Science and Technology Facilities Council
University of Oxford
HRCS22_22492
Semiconductor detectors are used widely in particle physics and other applications. This thesis concentrates on the development of two types of novel detectors. LGADs Both ATLAS and CMS are planning precision timing layers outside their trackers for the HL-LHC upgrades. These will be instrumented with a type of ultra-fast silicon detector called Low Gain Avalanche Detectors (LGADs) that achieve a timing resolution of ~30 ps. LGADs can be used to connect charged tracks to the correct production vertices and therefore reduce the effect of pile up at the High Luminosity LHC. Many other experiments nuclear and medical physics would also benefit from precision timing information. LGADs use charge multiplication due to the avalanche initiated by a charge moving in large electrical fields to reach the picosecond regime. Unfortunately, the performance of these devices is limited by radiation damage when exposed to particle fluences higher than about 1014 particles/cm2. Simulation of the doping profiles to improve LGAD radiation damage performance and prompt characterization of structures fabricated by Teledyne-e2V before and after radiation exposure will be conducted during this thesis. A study of the performance degradation (loss of signal, loss of gain, inhomogeneous spatial response, etc.) as a function of radiation fluence will also be performed. These studies will require careful laboratory measurements and test beam campaigns. The measurements will validate the TCAD simulations, improve our understanding of these devices, and lead to new optimised rad-hard designs. Depleted Monolithic Active Pixels Sensors (DMAPS) DMAPS are position-sensitive detectors fabricates using standard CMOS processes. These sensors are extremely attractive for experiments in particle physics because they integrate the sensing and the readout electronics in a single layer of silicon. This removes the need for interconnection between the sensor and the readout-chip with the solder bump technology which is complex and expensive. DMAPS will become the sensor technology of choice for the next generation of experiments in particle physics. This thesis will focus on the optimization of the DMPAS technology for two different regimes: radiation-hard detector for further upgrades of the LHC pixel detectors and high-precision low-mass detector for future e+e- colliders. The R&D programme on DMPAS requires simulation with TCAD and sensor characterization, including radiation hardness studies for the HL-LHC application. This programme will be conducted in collaboration with TowerJazz using its 180 nm and 65 nm technologies.
Missing/Incomplete
6project_grants_public
gen_fbf32aaaaf707998feb13afac4da4eb4
Gates Foundatoin
Praedicare Inc
INV-004622
to evaluate novel TB drug combinations in the in vitro hollow fiber system for TB to inform our understanding of efficacy and dose selection
Family Planning|Global Health and Development Public Awareness and Analysis / Global Health
6project_grants_public
gen_56f607fe7c4bfa6d3e7207ff0e998b0d
Gates Foundatoin
International Centre for Diarrhoeal Disease Research, Bangladesh
INV-016370
to develop shelf-stable, locally-sourced, microbiome-directed, ready-to-use therapeutic foods (MD-RUTFs) for malnourished children.
Global Education / Gender Equality
6project_grants_public
gen_06509a572837eaed14375c75d22622f5
Gates Foundatoin
University of Pretoria
INV-022216
to support novel imaging and metabolic biomarker discovery in birth asphyxia, and the use of this information for planning for future clinical trials of neuroprotective agents
Global Education / Gender Equality
6project_grants_public
gen_fbef573745455f975921ccc06e3f8fb1
Gates Foundatoin
Armauer Hansen Research Institute
INV-023730
to introduce a novel rapid molecular method - digital droplet PCR (ddPCR) - to detect hrp2/3 gene deletions in Plasmodium falciparum in Ethiopia
Global Health and Development Public Awareness and Analysis / Global Health
6project_grants_public
gen_1ebf149db621514a1313b311e72d4a9e
Gates Foundatoin
CDC Foundation
INV-024558
to evaluate the impact of the pneumococcal conjugate vaccine introduction on pneumococcal nasopharyngeal carriage in children in Indonesia
Research and Learning Opportunities / Global Health
6project_grants_public
gen_4e3ad04e214d9c4c01af4da08d599f31
Gates Foundatoin
Brigham and Women's Hospital, Inc.
INV-025023
to assess whether remdesivir administered in a new simplified manner can produce effective antiviral protection in people
Global Health and Development Public Awareness and Analysis / Global Health
6project_grants_public
gen_bc061b28dd6d4637c40d0d9efa16bda6
Gates Foundatoin
Vector Borne and NTD Division
INV-026449
to increase MDA coverage through the use of &#34;infotainment&#34; , an effort to modernize delivery and retention of NTD messages with a focus on reaching young populations.
Development of Solutions to Improve Global Health|Malaria / Global Health
6project_grants_public
gen_3423ae516988709745a703082b2f62d8
Gates Foundatoin
FIOCRUZ
INV-027961
to fund 12 Ki GCE Brazil-based data science projects that advance and strengthen maternal and child health, women’s health and nutrition efforts, by using administrative data to develop better evidence and insights for policymakers
Tuberculosis / Global Health
6project_grants_public
gen_223d7c23388cbdd213afbfe1d0264155
Gates Foundatoin
Faecal Sludge Management Alliance
INV-033458
to build capacity of professionals from low and middle income countries in non-sewered sanitation
Discovery and Translational Sciences / Global Growth & Opportunity
6project_grants_public
gen_5ebd078d3783804bb61d37a120280ac6
Gates Foundatoin
Mastory
INV-028468
to support development of novel resources to accelerate student mastery of algebra
Research and Learning Opportunities / U.S. Program
6project_grants_public
gen_d9593cf830c177e08996364bc99ed695
Gates Foundatoin
Johns Hopkins University
INV-028582
to guide future programs seeking to improve the nutrition of young women prior to pregnancy in South Asia and elsewhere
Pneumonia & Pandemic Preparedness / Gender Equality
6project_grants_public
gen_a9fb47f67dca8e94a54df3bf846fa2fc
Gates Foundatoin
Calibr-Skaggs Institute for Innovative Medicines
INV-028691
to deliver a drug candidate to treat coronavirus diseases
Maternal, Newborn, Child Nutrition and Health / Global Health
6project_grants_public
gen_69eea08e19d42556981d74cdd6392c34
Gates Foundatoin
The Pirbright Institute
INV-028944
to support foot-and-mouth disease control in India
Global Health and Development Public Awareness and Analysis / Global Growth & Opportunity
6project_grants_public
gen_82eb347c241583344d90f11ff8f1e2fa
Gates Foundatoin
The Center for Disease Dynamics, Economics & Policy, Inc.
INV-029062
to contribute to improved understanding of the epidemiology of COVID-19 in India
Community Engagement Grantmaking / Global Development
6project_grants_public
gen_2bee1c228d3e58d4b9c9e81fd0a8b297
Gates Foundatoin
WeRobotics
INV-029257
to determine whether cargo drones can enable more rapid and reliable diagnosis of children who may have polio
AFRICA|ASIA / Global Development
6project_grants_public
gen_eed79f8ee66de21e81de5efa943f1217
Gates Foundatoin
CARE
INV-029674
to support rigorous costing and additional analysis of a multisectoral model to support married adolescent girls to delay their first birth and achieve their desired fertility intentions in Niger and Bangladesh
Vaccine Development / Gender Equality
6project_grants_public
gen_57038fff9103244ad48085a830fc19dc
SHared automation Operating models for Worldwide adoption
European Commission
Gruppo Torinese Trasporti S.P.A.; KAPSCH TrafficCom AG; IRIZAR E-MOBILITY SL
CORDIS-875530
SHOW aims to support the migration path towards affective and persuasive sustainable urban transport, through technical solutions, business models and priority scenarios for impact assessment, by deploying shared, connected, cooperative, electrified fleets of autonomous vehicles in coordinated Public Transport (PT), Demand Responsive Transport (DRT), Mobility as a Service (MaaS) and Logistics as a Service (LaaS) operational chains in real-life urban demonstrations in 5 Mega, 6 Satellite and 3 Follower Pilots taking place in 20 cities across Europe. By deploying a fleet of 74 L4/L5 AVs of all types (buses, shuttles, pods, robo-taxis, automated cars connected with MaaS and cargo vehicles) and for all transport operators (passengers, cargo and mixed transport) in both dedicated lanes and mixed traffic, connected to a wide range of supporting infrastructure (5G, G5, IoT, etc.) and operating under traffic speeds ranging from 18 to over 50km/h, it aims to satisfy 7 UCs families and 22 single UCs; that together cover all urban automated mobility needs and wants of the stakeholders (i.e. as reported within SPACE initiative and in ERTRAC roadmap). Project pilots will last for 24 months, with real service seamless operation in each pilot site lasting at least 12 months and will transport with AV fleets over 1,500,000 passengers and 350,000 units of goods. Being the bigger and more holistic ever real life CCAV urban demonstration initiative, it is user led (by UITP) and realised by a Consortium of 77 Partners, 13 third parties, [terminating 9 partners and 6 third parties] and with the additional support of 60 stakeholders (connected through LoS, including major stakeholder Associations) and twinning actions with 11 organisations the US, S. Korea, Australia, China, Taiwan and Singapore.
H2020-EU.3.4. / 3.4 Societal Challenges - Transport
6project_grants_public
gen_fed90038b75edec8fc10e21613e8f2e0
Gates Foundatoin
PATH
INV-030072
to better understand the risks and external costs of the COVID-19 vaccine rollout
Development of Solutions to Improve Global Health / Global Development
6project_grants_public
gen_e7d47b7daa37603eb8ac80755102f210
Gates Foundatoin
University of California, Los Angeles
INV-028840
to support development of novel resources to accelerate student mastery of algebra
Washington State Education and Human Service Needs / U.S. Program
6project_grants_public
gen_a2c75bd607eccc213f9b48577cf6e8f1
Gates Foundatoin
University of Notre Dame
INV-005898
to introduce a novel rapid molecular method - digital droplet PCR (ddPCR) - to detect hrp2/3 gene deletions in Plasmodium falciparum in Ethiopia
Postsecondary Education / Global Health
6project_grants_public
gen_44e73cb6b9c2082dae5f6e27c6f918ad
Gates Foundatoin
Penn State
INV-016091
to develop novel approaches and methodologies to integrate multiple data sources to evaluate and communicate measles risk and inform programmatic decision-making at the national and sub-national level
Vaccine Delivery / Global Development
6project_grants_public
gen_1652d9f30ed2ddbce2aa3db90917ae92
Gates Foundatoin
Indian Institute of Science
INV-024649
to develop subunit-based immunogens to the newly emerged Coronavirus as candidate vaccines
Global Health and Development Public Awareness and Analysis / Global Development
6project_grants_public
gen_02a213796cdfd133278016cf39d6ee3d
Gates Foundatoin
Addis Ababa University - Center for Innovative Drug Development and Therapeutic Trials for Africa
INV-024950
to train clinical trial coordinators to manage trials and support the operations at sites in Africa
Tuberculosis / Global Health
6project_grants_public
gen_ddf3bfcc255f568767acdfa84ac22b89
Gates Foundatoin
Lgenia Inc.
INV-025337
to contribute to the generation of new tuberculosis drug candidates
Global Health and Development Public Awareness and Analysis / Global Health
6project_grants_public
gen_4a400b3a378f85fa5f7d9e7cc93d956a
Gates Foundatoin
Shenzhen Institute of Advanced Technology, Chinese Academy of Sciences
INV-025841
to design and engineering of a synthetic enzyme pathway from AA to Artemisinin
Vaccine Development / Global Health
6project_grants_public
gen_f98323c56980293f20721163dee43659
Gates Foundatoin
Wuhan University
INV-026361
to provide scientific evidence to policy makers to lower the price and improve multi-financing policies of TB drugs that lead to improved accessibilities of high quality TB services and reduced financial burden for TB patients
K-12 Education / Global Policy and Advocacy
6project_grants_public
gen_45f54edc4de882bb6a91f7ecdd400b96
Gates Foundatoin
Indian Institute of Technology Delhi
INV-027027
to accelerate COVID19 vaccine distribution in India by Ministry of Health and Family Welfare
Polio / Global Development
6project_grants_public
gen_40d5a80a167c5beb7bef30cac65fa5e4
Gates Foundatoin
Shanghai Jiao Tong University
INV-027291
To improve global market need for antimalaria treatment supply of artemisinin.
K-12 Education / Global Health
6project_grants_public
gen_24dfc889761445b43ffc0512be94ffab
Gates Foundatoin
The School Board of Broward County, Florida
INV-028815
to support development of novel resources to accelerate student mastery of algebra
Inclusive Financial Systems / U.S. Program
6project_grants_public