Data-Gouv-ML/pcri-informations-sur-les-projets-laureats huggingface dataset:
PanCare Studies in Fertility and Ototoxicity to Improve Quality of Life after Cancer during Childhood, Adolescence and Young Adulthood (PANCARELIFE)

Framework: FP7 — Cooperation
Programme: Health
Destination: Translating research for human health
Action type: Collaborative project
Countries involved: Germany,Ireland,Ireland,Netherlands,Germany,Netherlands,Germany,Germany,Switzerland,Italy,Czechia,France,Denmark,Czechia,Germany,Netherlands
Call:  (FP7-HEALTH-2013-INNOVATION-1, 2013)
Start date: 2013-11-01
End date: 2018-10-31
Duration (months): 60.0
Total project cost (EUR): 7803577.36
EU funding (EUR): 5998279.0
Participants: 20
Project status: CLOSED

Survival rates after childhood cancer now reach nearly 80% in developed European countries as a result of more effective therapies and better supportive care, leading to a steady increase in the number of survivors in the population. However, the treatments that have improved survival are harsh and cause serious side-effects that can greatly impact survivors’ quality of life in the long term. The goal of PanCareLIFE is that survivors of cancer diagnosed before age 25 should enjoy the same quality of life and opportunities as their peers who have not had cancer. Using observational studies and molecular genetic investigations PanCareLIFE will investigate late effects that impact fertility and hearing impairment (ototoxicity), and will assess health-related quality of life. Information from PanCareLIFE’s studies will be incorporated into new guidelines for fertility preservation. As the number of survivors with late effects in any one country is small, large cohorts are required for accurate estimation of risk. PanCareLIFE has assembled a team of prominent investigators from 8 European countries who will contribute in total over 12,000 well-characterised research subjects to identify risk factors, both genetic and non-genetic, linked to decrements in fertility and ototoxicity. Quality-of-life studies will evaluate the impact of fertility and ototoxicity. PanCareLIFE will advance the state-of-the-art in survivorship studies by evaluation of large cohorts with observational and genetic tools that will provide better knowledge of individual risk factors. Survivors can then be stratified into groups benefitting from personalized, evidence-based, care; future patients may expect effective therapies to have less severe side effects, and plans for a seamless transition to long-term follow-up care can be made. These approaches will result in better quality of life for survivors of cancer diagnosed at a young age.

Keywords: childhood/adolescent cancer,fertility,ototoxicity,quality of life,premature/primary ovarian failure,long-term side effects,genome-wide association studies
Targets and biomarkers for antiepileptogenesis (EPITARGET)

Framework: FP7 — Cooperation
Programme: Health
Destination: Translating research for human health
Action type: Collaborative project
Countries involved: Sweden,Germany,France,Finland,Italy,Germany,United Kingdom,Poland,Netherlands,Israel,Germany,France,Netherlands
Call:  (FP7-HEALTH-2013-INNOVATION-1, 2013)
Start date: 2013-11-01
End date: 2019-10-31
Duration (months): 72.0
Total project cost (EUR): 15776100.34
EU funding (EUR): 11999975.0
Participants: 20
Project status: CLOSED

Epilepsy is a devastating condition affecting over 50 million people worldwide. This multidisciplinary project is focused on the process leading to epilepsy, epileptogenesis, in adults. Our main hypothesis is that there are combinations of various causes, acting in parallel and/or in succession, that lead to epileptogenesis and development of seizures. Our central premise and vision is that a combinatorial approach is necessary to identify appropriate biomarkers and develop effective antiepileptogenic therapeutics. The project will focus on identifying novel biomarkers and their combinations for epileptogenesis after potentially epileptogenic brain insults in clinically relevant animal models, such as traumatic brain injury (TBI) and status epilepticus (SE); explore multiple basic mechanisms of epileptogenesis and their mutual interactions related to cell degeneration, circuit reorganization, inflammatory processes, free radical formation, altered neurogenesis, BBB dysfunction, genetic and epigenetic alterations; and translating these findings towards the clinic by validating biomarkers identified from animal models in human post TBI brain tissue and blood samples, post-mortem brain tissue in individuals that died soon after SE, and human brain and blood samples from chronic epilepsy cases. The project will identify novel combinatorial biomarkers and novel disease-modifying combinatorial treatment strategies for epileptogenesis, create an Epilepsy Preclinical Biobank, and validate translational potential of results from animal models in human tissue. To adequately address the proposed goals, the project will develop technological breakthroughs, such as completely novel chemogenetic approaches, novel MRI techniques, novel multimodal organic recording devices for simultaneous recordings of EEG / cellular unit activity and biochemical measurements, novel bioluminescence for in vivo promoter activity analysis, and novel systems biology approaches.

Keywords: biomarkers for epileptogenesis,antiepileptogenic treatments,mechanisms of epileptogenesis
Personalisation of tREatment In Cardiovascular disease through next generation sequencing in Adverse Drug Reactions (PREDICTION-ADR)

Framework: FP7 — Cooperation
Programme: Health
Destination: Other Actions across the Health Theme
Action type: Collaborative project
Countries involved: United Kingdom,Netherlands,United Kingdom,Sweden,Estonia,United Kingdom
Call:  (FP7-HEALTH-2013-INNOVATION-1, 2013)
Start date: 2013-09-01
End date: 2017-02-28
Duration (months): 42.0
Total project cost (EUR): 3897656.39
EU funding (EUR): 2999952.0
Participants: 7
Project status: CLOSED

Prediction-ADR aims to discover the genetic factors predisposing patients to adverse drug reactions (ADRs) from cardiovascular disease (CVD) drugs. This will be achieved by assembling a consented blood bank and DNA for a population of ~500 cases of ACEi induced angioedema and statin induced myopathy defined using a standardised phenotypic criteria. An innovative next generation sequencing strategy will be used to find predisposing genetic changes to myopathy and angioedema. Our SME partner will use proprietary software to build a highly predictive genetic algorithm for subsequent performance evaluation in real world clinical datasets involving >50,000 patients. This will deliver a novel set of genomic biomarkers to improve effective prevention or treatment of CVD providing the EU SME competitive advantage in the emerging personalised medicine market. The number of CVD drug prescriptions globally means that the absolute number of adverse drug reactions (ADRs) is substantial resulting in harm to the patients and leading to discontinuation and increased risk of CVD progression. Genome wide association studies (GWAS) has recently accelerated the progress in determining genetic biomarkers for adverse responses to drugs and with the advent of affordable whole genome sequencing, genetic research is now positioned to accelerating it even further. This provides a new opportunity to discover improved genomic biomarkers for extreme phenotypes of severe ADRs. The ultimate goal is to have these markers accepted by EMA and FDA and to disseminate these findings not only to the scientific community but also to lay people and patient organisations. This genetic algorithm will be developed into a commercially available clinical test by Asper Biotech. As 7 Million individuals were treated with statins in the UK alone during 2012, the size of the potential market for this test is immense.

Keywords: cardiovascular disease population genetics pharmacogenomics
Personalized Medicine 2020 and beyond – Preparing Europe for leading the global way (PerMed) (PERMED)

Framework: FP7 — Cooperation
Programme: Health
Destination: Other Actions across the Health Theme
Action type: Coordination and support actions
Countries involved: Germany,Germany,Netherlands,France,United Kingdom,Israel,Finland,Spain,France,France,Italy,Canada,Netherlands,France,Luxembourg,Spain,Austria
Call:  (FP7-HEALTH-2013-INNOVATION-1, 2013)
Start date: 2013-09-01
End date: 2015-08-31
Duration (months): 24.0
Total project cost (EUR): 645115.0
EU funding (EUR): 497378.0
Participants: 18
Project status: CLOSED

The Health Directorate of the European Commission's DG Research and Innovation recently stated that Personalized Medicine is one of the most innovative areas in the future of health research with a high potential for patients, citizens and the economy. However, today the full potential can not be developed due to fragmented activities, insufficient communication and lack of generic solutions in the different areas of Personalized Medicine.The implementation of Personalized Medicine is, therefore, a major challenge in Europe and beyond. It calls for appropriate governance strategies at the European and global level as it challenges the way in which healthcare systems worldwide are set up. Thus, the EC itself, EuroBioForum, the European Health Forum Gastein (EHFG) and others have organized conferences to tackle these challenges. Furthermore, key European organizations and institutions have come up with reports, guidelines, roadmaps aiming to give guidance in this emerging and important health research field amongst others the European Science Foundation (ESF) Forward Look, the Manifesto of the European Alliance for Personalised Medicine (EAPM), the European Best Practice Guidelines of the Public Health Genomics European Network (PHGEN), the Roche report on Personalized Medicine or the report of the European Hospital and Healthcare Federation (HOPE). Based on these initiatives European and national decision-makers and funding bodies not only have expressed the urgent need for a CSA, but also initiated this specific CSA to step up coordination efforts between the European key stakeholders to allow synergies and avoid duplication or competition, to ensure maximum transparency and openness preparing Europe for leading the global way.

Keywords: personalized medicine,funding bodies,decision-makers,health and translational research,healthcare systems,health information and technologies,innovation policy,european competiveness
Advanced T-cell Engineered for Cancer Therapy (ATECT)

Framework: FP7 — Cooperation
Programme: Health
Destination: Translating research for human health
Action type: Collaborative project
Countries involved: United Kingdom,France,Italy,Netherlands,Switzerland
Call:  (FP7-HEALTH-2013-INNOVATION-1, 2013)
Start date: 2013-12-01
End date: 2018-11-30
Duration (months): 60.0
Total project cost (EUR): 7780342.22
EU funding (EUR): 5931151.0
Participants: 6
Project status: CLOSED

T-cell engineering strategies for Cancer therapy, either Chimeric Antigen Receptors (CARs) or TCR transfer holds promise to revolutionize cancer treatment. There are, however, considerable barriers to be overcome to take this form of therapy to a format that can benefit all EU citizens with a wide range of common cancers. The aim of this consortium is to exploit advances in T-cell engineering to allow the full potential of CAR therapy to be unleashed. At present, CAR therapy requires a bespoke autologous therapeutic product for each patient. This greatly limits practicality, scalability and commercialisation. The development of a strategy for creation of universal engineered T-cells is the first key aim of this consortium. There is an increased appreciation of the immunological hostilities (CAR) T-cells face in the tumour microenvironment, and prevention of this local immune suppressive effect will likely be critical in permitting effective tumour control. The second main aim of this proposal is therefore to engineer CAR T-cells to be resistant to the hostile microenvironment. CAR T-cells can only be effective if they can access the tumour site. Exploiting the fact that neo-angiogenesis is a hallmark of neoplastic progression, the third aim of the consortium is to utilise endothelial cues of neo-angiogenesis to direct CAR T-cell migration and activity. The central technological theme of this consortium is the application of TALEN-mediated gene editing strategies alongside genetic modification with integrating vectors. Using this approach, we will implement a clinical study of “universal” CAR T-cells in refractory lymphoma. Further, this work will be complemented with highly focused development of T-cells which are resistant to hostile microenvironments and which can home to sites of neovascularization. The legacy this consortium wishes is commercialization of universal CAR therapy for a broad swathe of human cancers.

Keywords: immunotherapy,cellular therapy,gene-therapy,chimeric antigen receptor,gene editing
Human pluripotent stem cell differentiation, safety and preparation for therapeutic transplantation in Huntington’s disease (REPAIR-HD)

Framework: FP7 — Cooperation
Programme: Health
Destination: Biotechnology, generic tools and medical technologies for human health
Action type: Collaborative project
Countries involved: United Kingdom,France,United Kingdom,United Kingdom,Germany,Italy
Call:  (FP7-HEALTH-2013-INNOVATION-1, 2013)
Start date: 2013-10-01
End date: 2018-03-31
Duration (months): 54.0
Total project cost (EUR): 7820103.95
EU funding (EUR): 6000000.0
Participants: 10
Project status: CLOSED

This project will tackle the huge complexity of taking stem cell therapies to clinical application for neurodegenerative disease by focusing on selective differentiation of a single neuronal phenotype (medium spiny striatal neuron: MSN) for a single well-defined disease (Huntington’s: HD). Our consortium contains expertise in all elements required to drive this technology to the point of clinical delivery, including expertise in stem cell differentiation and control of proliferation; in vitro genetic, molecular, cellular and functional characterisation; preclinical assessment in both rodents and primates models of HD; GMP knowledge, development and production; and clinical translation. Our clinical team includes world leaders in HD clinical trials, including fetal neural transplants and is well placed to design the translation process. We focus on human embryonic stem (hES) cells as our primary target for first-in-man proof-of-concept studies, as they are closest to clinical readiness. HD is the target disease as it provides both an excellent model relevant to a wide range of neurodegenerative conditions, and is a stringent test of the capacity of selectively differentiated stem cells to repair neural circuits. The starting point for the work is the existence within the consortium of three of the most advanced protocols to date for MSN differentiation, and a feature of our consortium is that the specificity of stem cell differentiation will be tested against primary fetal MSNs (current gold standard) at all stages of both in vitro and in vivo assessment. In order to maintain flexibility in an emerging ethical environment, we will develop induced pluripotent (hiPS) cells to the point of GMP validation as a second generation target to hESCs. This will build European infrastructure and capacity to deliver emergent stem cell therapies through the highest quality clinical trials into clinical practice in a broad range of human neurodegenerative diseases.

Keywords: human pluripotent stem cells,proliferation,tumourigenesis,differentiation,neuronal fate specification,gmp,neuronal transplantation,safety and efficacy,neurodegeneration,huntington's disease
Labeling of Enalapril from Neonates up to Adolescents (LENA)

Framework: FP7 — Cooperation
Programme: Health
Destination: Other Actions across the Health Theme
Action type: Collaborative project
Countries involved: Germany,Germany,Hungary,Netherlands,Serbia,Austria,Germany,United Kingdom,Belgium,Austria,Belgium
Call:  (FP7-HEALTH-2013-INNOVATION-1, 2013)
Start date: 2013-11-01
End date: 2019-04-30
Duration (months): 66.0
Total project cost (EUR): 7437966.34
EU funding (EUR): 5999991.0
Participants: 16
Project status: CLOSED

Children are often treated as small adults—not as the special patients they are. Regulations exist to encourage the development of drugs and formulation for children. However, off-patent drugs are often administered to children without appropriate formulations or systematic investigations. Therefore, the paediatric use marketing authorisation (PUMA) has been designed. The EMA Paediatric Working Party has prioritized the unmet need for the cardiovascular off-patent drug enalapril in the European paediatric population. Enalapril is an ACE inhibitor commonly used for cardiac failure in children under 1 year of age, although it is not labelled for any condition in patients <20 kg in European countries. Current problems with the use of enalapril in children include: no appropriate formulation and insufficient stability of the liquid soluble formulation for young paediatric patients; insufficient paediatric pharmacokinetic (PK) data for any formulation; insufficient safety data in neonates, infants, and young children; and limited pharmacodynamics (PD) and efficacy data for paediatric cardiological indications. The aim of the LENA (Labeling of Enalapril from Neonates up to Adolescents) project is to address these shortcomings and provide a basis for a future PUMA of enalapril by developing an age-appropriate solid oral formulation suitable for all paediatric subsets; generating PK and PD data; collecting data on the safety of enalapril in young paediatric patients; and providing dose recommendations based on PK/PD modelling and bridging from adult data. The international collaboration of the LENA investigators will provide the required expertise to develop and evaluate drug formulation, investigate dosing, and recruit paediatric patients for clinical trials while respecting current legislation, regulatory considerations, and ethical aspects, with the aim of applying for a PUMA.

Keywords: puma,pip,heart failure,enalapril,mini-tablets,pharmacokinetics/pharmacodynamics,physiology based modeling,neonates,infants,bridging
Systems biology for the functional validation of genetic determinants of skeletal diseases (SYBIL)

Framework: FP7 — Cooperation
Programme: Health
Destination: Translating research for human health
Action type: Collaborative project
Countries involved: United Kingdom,Italy,United Kingdom,Germany,Italy,Switzerland,Italy,France,United Kingdom,Germany,Germany,Austria,Germany,Germany,Belgium,France
Call:  (FP7-HEALTH-2013-INNOVATION-1, 2013)
Start date: 2013-10-01
End date: 2018-09-30
Duration (months): 60.0
Total project cost (EUR): 15778074.05
EU funding (EUR): 11968569.0
Participants: 18
Project status: CLOSED

The aim of SYBIL is to carry out extensive functional validation of the genetic determinants of rare and common skeletal diseases and the age related factors contributing to these painful conditions. To achieve this goal SYBIL will gather complementary translational and transnational scientists, systems biologists, disease modellers, leading SMEs and industrialists that will perform in-depth characterisation (complete molecular phenotyping) of pre-clinical models (cellular and animal) for a variety of common and rare skeletal diseases. SYBIL will establish a systematic pipeline of in vitro, ex vivo and in vivo models of increasing complexity and will also make use of novel technologies such as iPS cells and exclusive ‘Virtual Patient’ software to identify potential therapeutic targets for further validation through simultaneous modelling of fundamental and complex physiological pathways. SYBIL will rely on i) an ‘Omics Knowledge Factory’ for systematically generating new knowledge on skeletal disease pathophysiology and to generate the relevant Omics profiles necessary to detect and validate new disease determinants, biomarkers and therapeutic targets for future clinical developments, and ii) a “Systems Biology Hub” to integrate the high-throughput and data-dense information, to gain a global understanding of pathophysiological commonalities between different skeletal diseases and recognize predominant shared pathways and mechanisms that may represent new targeted routes to treatment. SYBIL will also identify potential modifier genes and study the epigenome that will ultimately influence the timing and efficacy of new personalised treatments. Overall SYBIL achievements will tremendously boost the efficient pre-clinical assessment and development of therapeutics against skeletal diseases and thus indirectly reduce their social and healthcare burden.
New approaches in the development of Hypoallergenic implant material in Orthopaedics: steps to personalised medicine (HYPORTH)

Framework: FP7 — Cooperation
Programme: Health
Destination: Biotechnology, generic tools and medical technologies for human health
Action type: Collaborative project
Countries involved: Germany,Estonia,Poland,Hungary,Denmark,Spain,Switzerland,Germany
Call:  (FP7-HEALTH-2013-INNOVATION-1, 2013)
Start date: 2013-08-01
End date: 2018-07-31
Duration (months): 60.0
Total project cost (EUR): 7330776.2
EU funding (EUR): 5282600.0
Participants: 10
Project status: CLOSED

Europe is confronted by a demographic challenge as a decreasing work force has to support an increasing elderly population. The economic risk implied by this burden could be addressed by efforts to achieve an increase in Healthy Life Years. One key element would be to ensure unrestricted mobility for especially the elderly, allowing them to stay at work for longer. Irreversible joint deterioration often requires a joint replacement. Implantation of artificial joints is one of the most successful orthopaedic interventions. However, an increasing number of patients receive revision surgery with these 10 % of these contract an infection and 50 % develop an adverse immune reaction (AIR) to conventional implant material. At the moment the reasons for the development of AIR are inadequately understood. Our proposal contains innovative solutions concerning this problem. A predictive approach using biomarkers will identify patients with risk to develop AIR. These patients will receive hypoallergenic endoprostheses, avoiding AIR to conventional material. Novel hypoallergenic material combinations will be developed in the frame of this consortium by material scientists and implant manufacturer. Via immunological, microbiological and biocompatibility testing the development and production process will be improved constantly. The matching of implant material with the allergenic background of patients will avoid complicated and cost-intensive reverse reactions and is a step towards personalised medicine. A further approach is to achieve a better understanding of mechanisms of AIR, and its faster and easier diagnosis using sensitive diagnostic biomarkers for an accurate differentiation from low-grade infection. Additionally, mathematical modelling of results from different methods will show us the gene regulatory network that leads to an amplification of the adverse immune response triggered by prosthetic implants and will develop predictive models of AIR process.

Keywords: orthopaedics,implant,endoprosthesis,adverse immune reaction,allergy,infection,bioceramic,healthy aging,personalised,transcriptomics,exomics,reverse engineering,arthroplasty,biomaterial