Data-Gouv-ML/pcri-informations-sur-les-projets-laureats huggingface dataset:
RD-CONNECT: An integrated platform connecting registries, biobanks and clinical bioinformatics for rare disease research (RD-CONNECT)

Framework: FP7 — Cooperation
Programme: Health
Destination: Translating research for human health
Action type: Collaborative project
Countries involved: United Kingdom,Spain,France,Italy,Sweden,Netherlands,Spain,Portugal,Sweden,Greece,France,France,France,Austria,France,Malta,Germany,Switzerland,Czechia,United States,Australia,Germany
Call:  (FP7-HEALTH-2012-INNOVATION-1, 2012)
Start date: 2012-11-01
End date: 2018-10-31
Duration (months): 72.0
Total project cost (EUR): 17938561.97
EU funding (EUR): 11997111.0
Participants: 32
Project status: CLOSED

Despite examples of excellent practice, rare disease (RD) research is still mainly fragmented by data and disease types. Individual efforts have little interoperability and almost no systematic connection between detailed clinical and genetic information, biomaterial availability or research/trial datasets. By developing robust mechanisms and standards for linking and exploiting these data, RD-Connect will develop a critical mass for harmonisation and provide a strong impetus for a global “trial-ready” infrastructure ready to support the IRDiRC goals for diagnostics and therapies for RD in close collaboration with the successful A/B projects. It will build on and transform the current state-of-the-art across databases, registries, biobanks, bioinformatics, and ethical considerations to develop a quality-assured and comprehensive integrated hub/platform in which complete clinical profiles are combined with -omics data and sample availability for RD research. The integrated, user-friendly RD-Connect platform, built on efficient informatics concepts already implemented in international research infrastructures for large-scale data management, will provide access to federated databases/registries, biobank catalogues, harmonised -omics profiles, and cutting-edge bioinformatics tools for data analysis. All patient data types will be linked via the generation of a unique identifier (“RD-ID”) developed jointly with the US NIH. The RD-Connect platform will be one of the primary enablers of progress in IRDiRC-funded research and will facilitate gene discovery, diagnosis and therapy development. RD-Connect has the RD field at its heart and brings together partners with a strong track record in RD research (gene discovery and development of innovative treatments), as well as committed IRDiRC funding partners and representatives of all major international RD initiatives (EU/US/AU/JP) spanning patient organisations, research and public health, to maximise impact to RD patients

Keywords: rare diseases,integrated it platform,databases,patient registries,biobanks,ethics,omics,irdirc,deep phenotyping,bioinformatics,hub,stakeholders,patients,therapy development.
Best EScalation Treatment in Multiple Sclerosis (MS) (BEST MS)

Framework: FP7 — Cooperation
Programme: Health
Destination: Biotechnology, generic tools and medical technologies for human health
Action type: Collaborative project
Countries involved: France,France,Spain,Spain,Germany
Call:  (FP7-HEALTH-2012-INNOVATION-1, 2012)
Start date: 2012-11-01
End date: 2016-10-31
Duration (months): 48.0
Total project cost (EUR): 6350712.13
EU funding (EUR): 4880575.0
Participants: 5
Project status: CLOSED

Multiple Sclerosis (MS) is a devastating disease of the central nervous system affecting 2.5 million worldwide. MS is a field of constant therapeutic innovation, a fact which brings hope to young adults since MS is one of the most frequent causes of severe handicap. A major unmet need is to rationalize treatment decisions. To date, there is no way of predicting which patients will best respond to one of the 15 drugs expected on the market by 2014 and which patients are at risk of severe adverse effects. Recent technical advances (the omics revolution) have brought the dream of personalized medicine (PM) closer to reality. Therefore the main objective of this project is to design a composite test (using genome based biomarkers associated with clinical and radiological information) in order to predict which patients are associated with the best benefit to risk ratio in MS treatment, using Natalizumab (NTZ) as the paradigm. For this purpose, we have already built up a unique cohort of 1500 Europeans MS patients with. We will address 5 secondary objectives: #1 determine a qualitative definition of response to NTZ with clinical and radiological parameters; #2 determine a quantitative biological response test based on an in vitro assay; #3 determine DNA-based biomarkers associated with NTZ response; #4 determine genetic susceptibility to progressive multifocal encephalopathy and NTZ-related severe adverse events; and #5 determine RNA-based biomarkers associated with NTZ response. When this work is completed, we will use the data generated to build our composite test with a multivariate approach. We postulate that our predictive test for choosing the best patients to treat with NTZ will be a paradigm for all MS treatment and, beyond MS, for biotherapies in general. This project should have a positive impact on patients’ quality of life and on the MS market, and will involve a network of 5 teams (4 academic and 1 SME) that will work in perfect synergy.

Keywords: multiple sclerosis pharmacogenetics pharmacogenomics,natalizumab fingolimod,predictive test
Co-morbidity in relation to Aids (COBRA)

Framework: FP7 — Cooperation
Programme: Health
Destination: Translating research for human health
Action type: Collaborative project
Countries involved: Netherlands,United Kingdom,Sweden,Netherlands,Italy,Netherlands,Belgium,Germany
Call:  (FP7-HEALTH-2012-INNOVATION-1, 2012)
Start date: 2013-03-01
End date: 2017-02-28
Duration (months): 48.0
Total project cost (EUR): 7800514.21
EU funding (EUR): 5998758.1
Participants: 12
Project status: CLOSED

Persons with HIV on combination antiretroviral therapy (cART) are at increased risk of the premature development of age-associated non-communicable comorbidities (AANCC), including cardiovascular, chronic kidney, liver and pulmonary disease, diabetes mellitus, osteoporosis, non-AIDS associated malignancies, and neurocognitive impairment. It has therefore been hypothesised that such individuals, despite effective cART, may be prone to accelerated ageing. The underlying pathogenesis is likely to be multifactorial and include sustained immune activation, both systemically and within the central nervous system. By building on an established infrastructure for conducting longitudinal HIV cohort studies in Amsterdam and London, we will provide a detailed, prospective evaluation of AANCC among HIV-infected patients suppressed on cART and appropriately chosen and comparable non-infected controls. In this way, we will provide a robust estimate of the effect of treated HIV infection on the prevalence, incidence and age of onset of AANCC, thus clearly establishing a link between HIV and AANCC. Through the “Human Immune System” (HIS) mouse model, experimental studies will permit us to differentiate the effects of HIV and cART on metabolic outcomes when applied under controlled conditions, thereby further elucidating the causative nature of the link between HIV and AANCC. To further clarify potential pathogenic mechanisms underlying this causative link, including the possible induction of an inflammation-associated accelerated ageing phenotype, biomarkers which reflect each of these mechanisms will be investigated in biomaterial obtained from HIS mice and humans, and subsequently validated in patients with HIV on cART. The successful execution of the experimental and clinical research outlined in this proposal will be ensured through a strong interdisciplinary collaboration between clinical, basic and translational scientists bridging the fields of HIV, AANCC and ageing.

Keywords: hiv infection and age-associated non-communicable comorbidity,accelerated ageing,humanised immune system mouse-model,prospective cohort study,predictive biomarkers
Systems medicine of chronic inflammatory bowel disease (SYSMEDIBD)

Framework: FP7 — Cooperation
Programme: Health
Destination: Translating research for human health
Action type: Collaborative project
Countries involved: United Kingdom,Germany,Germany,United Kingdom,Germany,Netherlands,Israel,Germany,Germany,New Zealand,Germany
Call:  (FP7-HEALTH-2012-INNOVATION-1, 2012)
Start date: 2012-12-01
End date: 2017-11-30
Duration (months): 60.0
Total project cost (EUR): 15784306.68
EU funding (EUR): 11999998.0
Participants: 13
Project status: CLOSED

Inflammatory bowel disease (IBD) is a major health problem with severe co-morbidities, requiring life-long treatment. Oscillating processes, like biological clocks are well studied and modeled in a number of systems. Circadian rhythms are extremely important for optimal treatments of patients. Recently, the NfkB pathway has been shown to be oscillating. In this project, we will model NfkB oscillation in chronic inflammatory bowel diseases in animal models and patient cohorts with immunosuppressive treatments and controls. The aim is to build an experimentally validated model the NfkB oscillation in 4D within the gut tissue. Dynamic, experimental validation will be done for various types of cells in the gut by a combination of methods, including single-cell based transcriptomics, multi-photon microscopy and time-dependent, multi-component profiling. The validated model framework will enable searching for critical components of the NfB oscillation and to assess their relevance for the disease in patients. Interfering with the oscillation of biological pathways may provide new possibilities to influence biological processes like inflammation. Hence, we will search (assisted by the models and databases developed) for small molecules interfering with the NfkB oscillation in chemical databases and validate selected candidates in experimental systems. To this end, we will use cell lines with the correct indicator constructs using high content microscopy. To better translate the findings in animal models to patients, we will use a mouse model with transplanted human tissue so that we can verify the mathematical model in human tissue and verify functionality of small molecules in vivo. Owing to its systems, highly focused approach, the project will generate substantial insights into key mechanisms underlying IBD and will provide ways to modulate the oscillatory behavior of the NfB in IBD and IBD-dependent co-morbidities.
High-Dimensional Phenomena and Convexity (DIMENSION)

Framework: FP7 — Ideas
Programme: European Research Council
Theme: European research council
Destination: Starting grants
Action type: Support for frontier research (ERC)
Panel: Mathematical foundations
Countries involved: Israel
Call:  (ERC-2012-StG_20111012, 2012)
Start date: 2013-01-01
End date: 2018-12-31
Duration (months): 72.0
Total project cost (EUR): 998000.0
EU funding (EUR): 998000.0
Participants: 2
Project status: CLOSED

High-dimensional problems with a geometric flavor appear in quite a few branches of mathematics, mathematical physics and theoretical computer science. A priori, one would think that the diversity and the rapid increase of the number of configurations would make it impossible to formulate general, interesting theorems that apply to large classes of high-dimensional geometric objects. The underlying theme of the proposed project is that the contrary is often true. Mathematical developments of the last decades indicate that high dimensionality, when viewed correctly, may create remarkable order and simplicity, rather than complication. For example, Dvoretzky's theorem demonstrates that any high-dimensional convex body has nearly-Euclidean sections of a high dimension. Another example is the central limit theorem for convex bodies due to the PI, according to which any high-dimensional convex body has approximately Gaussian marginals. There are a number of strong motifs in high-dimensional geometry, such as the concentration of measure, which seem to compensate for the vast amount of different possibilities. Convexity is one of the ways in which to harness these motifs and thereby formulate clean, non-trivial theorems. The scientific goals of the project are to develop new methods for the study of convexity in high dimensions beyond the concentration of measure, to explore emerging connections with other fields of mathematics, and to solve the outstanding problems related to the distribution of volume in high-dimensional convex sets.

Keywords: high dimension,convex geometry,central limit theorem
Prevention of hepatitis C virus (HCV) and HIV-1 co-infections through induction of potent T cell responses using prime-boost viral vector vaccine regimens (PEACHI)

Framework: FP7 — Cooperation
Programme: Health
Destination: Translating research for human health
Action type: Collaborative project
Countries involved: United Kingdom,Switzerland,Belgium,Italy,Ireland
Call:  (FP7-HEALTH-2012-INNOVATION-1, 2012)
Start date: 2013-02-01
End date: 2017-10-31
Duration (months): 57.0
Total project cost (EUR): 6062182.86
EU funding (EUR): 4388813.0
Participants: 5
Project status: CLOSED

The goal of the PEACHI project is to develop simple, affordable and effective vaccine strategies that can be given alone or in combination to prevent hepatitis C virus (HCV), human immunodeficiency virus type 1 (HIV-1) and co-infection. The vaccines are based on novel and powerful viral vectors for in vivo delivery of antigens.The PEACHI Consortium members have employed replication-defective simian adenovirus (ChAd) and modified vaccinia virus Ankara (MVA) vector technology to develop the most immunogenic HCV and HIV-1 vaccines to date. We will assess the safety and immunogenicity of ChAd prime / MVA boost HCV vaccines in a key target group - HIV-positive individuals receiving antiretroviral therapy. These data are essential to support future efficacy studies aiming to assess protection of HIV-infected people from HCV infection. In addition, we will conduct the first phase I clinical studies using two distinct ChAd vectors simultaneously, one hosting an HCV immunogen spanning the entire NS region of HCV and the other, highly conserved HIV-1 sequences. This strategy aims to prime responses against both HCV and HIV-1 antigenic targets concurrently. Similarly, responses will be boosted simultaneously, using MVA vectors that host the respective HIV-1 and HCV immunogens. Finally, recent work by Consortium members has shown that the immunogenicity of ChAd and MVA vectors is markedly improved when the encoded HCV immunogen is fused to mouse or human MHC class II invariant chain. This may be critical to the effectiveness of HCV vaccines in HIV-infected people and will be applicable to vaccine development for other major infectious diseases. Therefore, a large component of this project will be the first assessment of this novel technology in humans. Clinical studies will be complemented by comprehensive laboratory analyses to assess the strength and quality of vaccine-induced T cell responses using state-of-art assays, which will facilitate the discovery of surrogate markers of protective immunity.

Keywords: hepatitis c virus,hiv,coinfection,vaccine,immunogen,vector,adenovirus,modified vaccinia virus ankara,t cell,immune response,phase i trial,prevention
Orphans Unite: chILD better together – European Management Platform for Childhood Interstitial Lung Diseases (CHILD-EU)

Framework: FP7 — Cooperation
Programme: Health
Destination: Translating research for human health
Action type: Collaborative project
Countries involved: Germany,United Kingdom,United Kingdom,France,Türkiye,Germany,Germany,Italy,Germany,Germany
Call:  (FP7-HEALTH-2012-INNOVATION-1, 2012)
Start date: 2012-12-01
End date: 2016-11-30
Duration (months): 48.0
Total project cost (EUR): 3854000.37
EU funding (EUR): 3000000.0
Participants: 10
Project status: CLOSED

Children with diffuse lung disease, also called childhood interstitial lung diseases (chILD), may have one of more than 200 entities, the biggest group of respiratory orphan lung diseases. Frequently undiagnosed because of lack of awareness or complex differential diagnosis, they lead to much morbidity, mortality (about 15%) and psychosocial stress for the families. Current lack of evidence based guidelines reflects the absence of any real scientific evidence for management. All current therapeutic options are off label. We propose that leading European clinical scientists and paediatric pulmonologists collaborate to assemble cohorts in which children with well defined disease entities, verified by international panels of clinicians, radiologists, geneticists and pathologists are followed in a pan-European database and biobank compatible with others worldwide to allow common projects. Outcomes and treatment schemes will be rigorously defined and their value systematically assessed. We will put defined treatment protocols systematically into practice to allow their evaluation and perform a randomised controlled trial in line with the EU recommendations, to put prescribing for children on an evidence based footing. This will give evidence to use medicines available based on their objectively determined effects and side effects. The project will lead to accepted evidence-based and consensus-agreed diagnostic and management clinical guidelines, to a better care of patients afflicted by rare chILD and lead to improved quality of life for children with these incurable diseases.

Keywords: children,rare respiratory diseases,diffuse lung disease,interstitial lung disease,no orphan drug,off-label treatment,outcome measures,child-patient organisations,evidence-based guidelines
A Portable bihormonal Closed Loop for Diabetes (PCDIAB)

Framework: FP7 — Cooperation
Programme: Health
Destination: Translating research for human health
Action type: Collaborative project
Countries involved: Netherlands,Austria,Denmark,Germany,Netherlands,Türkiye
Call:  (FP7-HEALTH-2012-INNOVATION-1, 2012)
Start date: 2012-09-01
End date: 2016-05-31
Duration (months): 45.0
Total project cost (EUR): 5465086.2
EU funding (EUR): 4335284.0
Participants: 7
Project status: CLOSED

The aim of PCDIAB is to build and evaluate a bihormonal (insulin and glucagon) artificial pancreas (AP) with automated closed loop glycaemic control for insulin treated patients with diabetes. This will be a breakthrough in diabetes management. We will miniaturize our current prototype consisting of well-established continuous glucose monitors, an insulin pump and a glucagonpump. The housing will be redesigned with dedicated miniature motors and the software will be embedded. The algorithm will be improved and a continuous glucose sensor (CGM) per-formance alert will be developed. In parallel, glucagon pharmacology will be investigated and a stable liquid glu-cagon analogue will be developed. Furthermore, administration of insulin and glucagon together with continuous glucose monitoring at the same subcutaneous site will be investigated, to enable even further miniaturization in the future. Deliverables include description of system integration of the bihormonal AP system and of an online detection of continuous glucose monitor performance. In a multinational controlled trial the bihormonal AP will be compared with standard intensive insulin therapy in daily life. Impact of the project includes simplified diabetes care, improved quality of life for patients with diabetes, dimin-ished occurrence of diabetes related complications and diminished health costs in the long run. Also, the project will strengthen competitiveness of European industry across a complete value chain involving large, mid-sized and small companies, enabling Europe to lead progress in AP systems. Finally, the project will put European research and clinical organizations in leading positions with an increased number of high-skilled jobs in the medical device industry. Dissemination and exploitation comprises a website, a conference, patents and scientific publications. The bihormonal closed loop system and the glucagon analogue can be developed into a product and brought to the market.

Keywords: portable artificial pancreas,insulin delivery,glucagon delivery,continuous glucose monitor
Community-based scheduled screening and treatment of malaria in pregnancy for improved maternal and infant health: a cluster-randomized trial (COSMIC)

Framework: FP7 — Cooperation
Programme: Health
Destination: Translating research for human health
Action type: Collaborative project
Countries involved: Netherlands,Belgium,Burkina Faso,United Kingdom,United Kingdom,Switzerland,Benin
Call:  (FP7-HEALTH-2012-INNOVATION-1, 2012)
Start date: 2012-09-01
End date: 2017-05-31
Duration (months): 57.0
Total project cost (EUR): 3944701.4
EU funding (EUR): 2997218.0
Participants: 8
Project status: CLOSED

Pregnant women are very susceptible to malaria infection and Malaria in Pregnancy (MiP) is a major cause of maternal anaemia and low birth weight (LBW) that leads to infant mortality, poor growth and development. In low transmission areas, malaria can become severe, resulting in maternal and foetal death. In sub-Saharan Africa (SSA) MiP is responsible for 8–14% of LBW, 3–8% of infant deaths, higher risk of post-partum haemorrhage and >10,000 maternal deaths/year. Prevention like, bed nets and intermittent preventive treatment with sulphadoxine-pyrimethamine (IPTp/SP), is cheap and cost-effective, but coverage achieved by these interventions is low. Therefore, we propose Community Health Workers (CHW) to implement scheduled intermittent screening at community level with RDT of pregnant women and if positive treat with anti-malarials (SST). In addition CHWs will encourage pregnant women to attend antenatal clinics (ANC) for other pregnancy-targeted interventions and IPTp/SP, thereby improving its coverage. This approach combines existing IPTp/SP with SST at village level as an extension of Home based management of malaria (HMM). This low cost (based on existing practice) and simple (diagnosis by RDTs) intervention improves maternal and newborn health and capitalise on an already existing intervention (HMM). The aim of this proposal is to determine the added value (as compared to IPTp/SP alone implemented in health facilities) of community SST of pregnant women implemented through the CHW involved in HMM. Objectives are: 1) Identify bottlenecks for implementation by CHW involved in HMM of SST; 2) determine impact of introducing SST in pregnancy on quality of HMM; 3) determine the impact of SST on ANC attendance and IPTp/SP coverage; 4) determine the impact of SST on LBW, anaemia and placenta malaria; 5) estimate cost-effectiveness of SST as compared to IPTp/SP alone and 6) formulate recommendations for possible implementation of the intervention.

Keywords: malaria,pregnancy,diagnosis,community health workers,rapid diagnostic tests