Data-Gouv-ML/pcri-informations-sur-les-projets-laureats huggingface dataset:
New approach to treatment of the blinding disease Retinopathy of Prematurity (ROP) (PREVENTROP)

Framework: FP7 — Cooperation
Programme: Health
Destination: Translating research for human health
Action type: Collaborative project
Countries involved: Sweden,Norway,United States,Sweden,Sweden,Italy,Netherlands,United Kingdom,Germany
Call:  (FP7-HEALTH-2012-INNOVATION-1, 2012)
Start date: 2012-10-01
End date: 2018-09-30
Duration (months): 72.0
Total project cost (EUR): 7590759.85
EU funding (EUR): 5990236.0
Participants: 10
Project status: CLOSED

The overall objective is to develop a novel preventative intervention for the blinding disease retinopathy of prematurity (ROP) and other complications of prematurity. The PREVENTROP consortium proposes to conduct preclinical studies (pharmacological, pharmacodynamics, pharmacokinetics and toxicological) in models and/or clinical studies (including phase III clinical trial) of an EU designated orphan medicinal product. This orphan medicinal product has been granted the EU orphan designation. We have completed both a Phase I study and the first section of the Phase II study administering the growth factor complex IGF-I/IGFBP-3 (Premiplex®) successfully to preterm infants in order to prevent ROP– a discovery that we have taken from bench to bedside. Due to improved neonatal care, the survival of preterm babies has increased dramatically during the last decades. The downside of this improvement in survival rates is higher morbidity affecting these vulnerable infants. It should be emphasised that in a preterm infant any lasting morbidity will have a negative impact on the quality of life for a whole life span for both the individual and their families. One of the most severe morbidities affecting these infants is retinopathy causing severe visual impairment and blindness. Our research findings have rendered several editorials pointing at the hope of prevention strategies which if successful will change the paradigm for ROP and other morbidities in preterm infants. There are three major impacts of the present proposal; (1) clinical availability of a new orphan designated drug product, Premiplex®, and (2) improved care of preterm infants and (3) a significant contribution towards the goal of the International Rare Disease Research Consortium (IRDiRC) by delivering one new therapy for a rare disease.

Keywords: orphan drug,prevention,rare disease,retinopathy of prematurity,blindness,insulin-like growth factor-1
From Health Research to Market – Advanced Services and Training Actions for the IPR Management and Business Exploitation of the EU-funded Research Results in Health/life sciences (HEALTH-2-MARKET)

Framework: FP7 — Cooperation
Programme: Health
Destination: Other Actions across the Health Theme
Action type: Coordination and support actions
Countries involved: Germany,Spain,Sweden,France,Italy,Greece,Hungary,France,Belgium
Call:  (FP7-HEALTH-2012-INNOVATION-1, 2012)
Start date: 2012-09-01
End date: 2015-08-31
Duration (months): 36.0
Total project cost (EUR): 2394509.5
EU funding (EUR): 1999785.0
Participants: 10
Project status: CLOSED

Health-2-Market is an ambitious project aiming to boost the economic exploitation of EU-funded research results in the area of Health/life science. The project focuses on the actual Health/life science knowledge generators, namely the researchers, involved in EU-funded research in Health with a view to cultivate to them an enhanced innovation and entrepreneurial culture. More specifically, Health-2-Market will aim at providing Health researchers with the necessary knowledge and skills for more viable IPR management as well as market exploitation of their results through the setting up of new business ventures in health/life science sectors or in a more broad sense through their more active involvement in interdisciplinary cooperation actions. More specifically, Health-2-Market is expected to offer a set of high-level services including advanced training courses,ad-hoc and on demand counselling as well as specific coaching (both through web as well as face-2-face) addressing innovation issues such as: IPR and asset management, formulation of business and exploitation plans, preparation for the launch of start-ups in health/life science as well as on demand knowledge transfers. Health-2-Market services will be offered by a pool of experienced internal specialists as well as external ones coming from leading academic and industrial organisations. The provided services will be escalating to address the needs of the potential target groups: Web (e-learning) courses for broader audience, two-day seminars on specific topics, advanced training courses (intensive training academies), hands-on support and ad-hoc counselling services either through face-to-face meetings, or distance ones, etc. The project will cover all EU MS and AC through its broad partnership as well as through a network of regional experts that will be set up. The project with take into account existing networks and services already deployed as the EEN, the Fit for Health Network as well as the NCP network.

Keywords: health,ipr management,exploitation of research results,training,exploitation/business plans,start-ups,knowledge transfer
Rapid development and distribution of statistical tools for high-throughput sequencing data (RADIANT)

Framework: FP7 — Cooperation
Programme: Health
Destination: Translating research for human health
Action type: Collaborative project
Countries involved: United Kingdom,Germany,Italy,Italy,France,Switzerland,United Kingdom,United Kingdom,Germany
Call:  (FP7-HEALTH-2012-INNOVATION-1, 2012)
Start date: 2012-12-01
End date: 2015-11-30
Duration (months): 36.0
Total project cost (EUR): 4675174.0
EU funding (EUR): 3672249.4
Participants: 10
Project status: CLOSED

High-throughput sequencing (HTS) is a powerful and rapidly evolving family of technologies with a multitude of applications. They include genetics of rare and common diseases, understanding of disease mechanism and progression through transcriptome and epigenome profiling, cancer stratification, personalised medicine and molecular systems biology of gene regulation. The genome, epigenome, transcriptome and interactome are all intricately connected, and modern HTS technology can probe all of these -omic levels. Statistical analysis is a crucial component of many experiments and studies, and the quality and efficiency of the analysis often determines the success of a project. In this collaborative project we will develop a range of new statistical analysis tools to solve open problems in HTS data analysis, ranging from low-level processing of sequence reads up to systems-level modelling of disease associated and cellular processes. We will provide to a wide audience an integrated computational framework for HTS data analysis and interpretation that is robust, efficient and user-friendly. We will establish improved procedures for the publishing of statistical software as an integral part of the scientific publication process, within the framework of the Bioconductor project. We will provide tools to benchmark experimental protocols and statistical methods, and we will provide training materials and a extensive training programme to rapidly disseminate these new tools to the broader biomedical community. SME partners will integrate these new tools within their analysis pipelines with associated user-friendly commercial software providing access to their additional proprietary tools. SMEs will benefit from basic methodology development done in a public, pre-competitive arena and will be able to use these technologies to enhance their products and services.

Keywords: high-throughput sequencing,short read sequencing,next generation sequencing,transcriptome,epigenome,bioconductor,statistical software,genomics,epigenetics,gene expression,systems biology
Orphan Drug for Acanthamoeba Keratitis (ODAK)

Framework: FP7 — Cooperation
Programme: Health
Destination: Translating research for human health
Action type: Collaborative project
Countries involved: France,United Kingdom,Italy,Italy,Netherlands
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): 5779906.8
EU funding (EUR): 4050255.0
Participants: 6
Project status: CLOSED

This project will undertake preclinical and clinical research of the Orphan Drug Polihexanide (PHMB). The main objective is to provide a safe and effective drug for the treatment of the rare ocular disease Acanthamoeba keratitis (AK) tested according to international regulatory standards. This debilitating infectious disease is caused by a free living protozoan which, in the absence of treatment, can have catastrophic consequences such as severe pain, visual loss and eye enucleation. There are no approved drugs to treat this disease. After Orphan Drug Designation Protocol Assistance was requested from the European Medicines Agency on our drug development research plan. The proposed protocol incorporates the EMA advice and will include a non-clinical phase, a double-blind placebo controlled Phase I trial and a randomised double-blind, active controlled, parallel groups Phase 3 study (efficacy and safety therapeutic confirmatory study). The primary deliverables will be: 1) experimental scientific evidence on the quality, safety and efficacy of PHMB to provide the basis for a Marketing Authorisation within 5 years; 2) recommendations aiming to improve clinical practices in the management of AK based on the efficacy and safety evidence. ODAK is an industry led project mobilising the critical mass of industrial, pharmaceutical and academic expertise. needed to develop and optimise therapeutic approaches to alleviate the severe negative impacts of AK on the health and quality of life of patients. In particular, through identifying optimal PHMB formulations and recommending the best dose-benefit treatment regimes. ODAK directly contributes to the International Rare Diseases Research Consortium goal towards 200 new therapies. An estimated 95% of the total estimated EU contribution to the project will go to industrial partners (of this 32% goes to SMEs). The industrial strength assures a rapid translation of research to market application.

Keywords: acanthamoeba keratitis,orphan drug development,polyhexamethylene biguanide,phmb,rare diseases,biguanide,ophthalmology
Platform for sharing best practices for management of rare diseases (RARE-BESTPRACTICES)

Framework: FP7 — Cooperation
Programme: Health
Destination: Translating research for human health
Action type: Coordination and support actions
Countries involved: Italy,Sweden,United Kingdom,United Kingdom,France,Germany,Netherlands,United Kingdom,Spain,Bulgaria,Spain,Belgium
Call:  (FP7-HEALTH-2012-INNOVATION-1, 2012)
Start date: 2013-01-01
End date: 2016-12-31
Duration (months): 48.0
Total project cost (EUR): 2293654.86
EU funding (EUR): 2000000.0
Participants: 17
Project status: CLOSED

RARE-Bestpractices will develop a sustainable networking platform, supporting the collection of standardized and validated data and efficient exchange of knowledge and reliable information on rare diseases (RD). RD are characterized by low prevalence (EU – 5:10000 persons). There are more than 5000, overall affecting about 30 million citizens of all ages in the EU. RD are often life-threatening and chronically debilitating, and healthcare is impaired by limited knowledge. Collaborative efforts are needed to tackle RD to prevent significant morbidity, perinatal or early mortality, to reduce socio-economic burdens and to improve an individual’s quality of life. RARE-Bestpractices aims to improve clinical management of RD patients, narrowing the existing gap among EU MS and other countries, also considering the application of patients’ rights in cross-border healthcare (EU Directive 2011/24). The platform deals with RD as a global health issue, exploiting and integrating contributions from all EU MS and other world areas (Caucasus, Europe, America, Oceania, PAHO/WHO) and will identify additional research needs to further improve clinical practice. Fostering synergistic collaboration among experts, patients representatives, policy makers, institutions, agencies, and other organizations experienced in systematic reviews and guidelines production, RARE-Bestpractices will focus on: a) collection, evaluation and dissemination of existing best practices; b) an agreed methodology suitable to develop and update best practice guidelines; c) training activities targeted at key stakeholders to spread expertise and knowledge; and d) a forum for exchanging information, sharing lessons learnt, and facilitating collaborations. The platform is conceived for health care providers, experts, patients, policy makers and best practice guideline developers with outcomes that support closure of healthcare gaps among countries and improved clinical management of RD patients globally.

Keywords: rare diseases,community network,practice guidelines,information systems,healthcare quality assurance,clinical management,business process modeling
Personal Health Systems Foresight (PHS FORESIGHT)

Framework: FP7 — Cooperation
Programme: Health
Destination: Other Actions across the Health Theme
Action type: Coordination and support actions
Countries involved: Austria,United Kingdom,Spain
Call:  (FP7-HEALTH-2012-INNOVATION-1, 2012)
Start date: 2012-09-01
End date: 2014-08-31
Duration (months): 24.0
Total project cost (EUR): 525458.4
EU funding (EUR): 485339.0
Participants: 3
Project status: CLOSED

This support action will coordinate strategic stakeholder engagements in the realm of Personal Health Systems (PHS) within and beyond European Union up to 2030. PHS assist in the provision of continuous, quality controlled and personalised health services to empowered individuals regardless of location. PHS are not limited to dealing with aging and eldercare, though PHS communities contribute to the European Innovation Partnership (EIP) on Active and Healthy Aging. PHS provides a horizontal development area across variety of patients, clinical specialties, technology fields and health services. Hence, the development of PHS requires and can mobilize the emergence of novel cross-disciplinary and -sectoral innovation partnerships. With this proposal we want to achieve a deeper understanding of mismatches between the potential of PHS and current policy and innovation initiatives and framework conditions; to support more mobilized and networked innovation communities, promoting PHS around jointly formulated issues which support pooling resources and streamlining diverse innovation initiatives; to tackle future opportunities and alternative trajectories, aligning actor perspectives for the development of a joint strategic action plan, including recommendations for a possible new EIP; and finally, to achieve a transparent, open and inclusive engagement of stakeholders, and targeted dissemination of results in society. Sufficient expertise will be provided by the consortium, the stakeholders involved, and by the scientific advisory board.

Keywords: personal health systems,innovation,sme,competitiveness,ehealth,aal,empowerment,innovation partnership,foresight,stakeholder participation
Improving the lives of Parkinson’s Disease patients while reducing side-effects through tailored deep brain stimulation (IMPACT)

Framework: FP7 — Cooperation
Programme: Health
Destination: Biotechnology, generic tools and medical technologies for human health
Action type: Collaborative project
Countries involved: Netherlands,Netherlands,Germany,Sweden,France,Germany,Sweden,Belgium
Call:  (FP7-HEALTH-2012-INNOVATION-1, 2012)
Start date: 2012-09-01
End date: 2016-08-31
Duration (months): 48.0
Total project cost (EUR): 7536580.0
EU funding (EUR): 4980907.0
Participants: 9
Project status: CLOSED

The IMPACT project is about improving the lives of brain diseased patients through a novel approach that leaps beyond currently available Deep Brain Stimulation (DBS) devices and procedures. The initial project focus is on Parkinson’s Disease (PD), but further brain-disease indications will be included in the later phase of the project. The personalized approach that IMPACT brings is essential in delivering full therapeutic benefits to DBS patients while preventing the stimulation-induced side-effects that occur with today's DBS implants. PD is well known for its characteristic symptoms: shaking, rigidity, slowness of movement and postural instability. Millions are suffering from PD including famous people like Michael J. Fox. Drugs are used as first treatment, but as the disease progresses they become ineffective and increasingly higher doses are needed. This leads to many side-effects, while symptoms still persist. DBS is a ‘pacemaker for the brain’, analogous to the function of pacemakers for the heart: mild electrical stimuli are delivered to brain tissue to suppress unwanted activity and restore desired neuronal functions. When stimulation is optimal, the impact of DBS is spectacular: shaking and rigidity are strongly improved, and medication doses may be lowered significantly. Despite its successes, DBS is still in its infancy. Programming for optimal therapy is complicated since physicians lack the appropriate tools to support them. Around 15 – 30% of DBS patients suffer from stimulation-induced side-effects resulting from stimulation leaking outside intended target regions. IMPACT addresses these barriers to adoption exploiting the directivity provided by next generation high-resolution implants. IMPACT delivers a physician tool for tuning the high-resolution implant based on a personalized patient brain stimulation model that takes into account imaging data (MRI, X-ray) as well as pre-operative data (local field potentials).

Keywords: parkinson disease (pd),deep brain stimulation (dbs),brain atlas,side effects,adverse events,micro electrode recording (mer),steering brain stimulation (sbs).
Does vascular endothelial growth factor gene therapy safely improve outcome in severe early-onset fetal growth restriction? (EVERREST)

Framework: FP7 — Cooperation
Programme: Health
Destination: Biotechnology, generic tools and medical technologies for human health
Action type: Collaborative project
Countries involved: United Kingdom,Finland,Germany,Spain,Sweden,United Kingdom
Call:  (FP7-HEALTH-2012-INNOVATION-1, 2012)
Start date: 2013-01-01
End date: 2018-12-31
Duration (months): 72.0
Total project cost (EUR): 7754709.33
EU funding (EUR): 5998984.0
Participants: 11
Project status: CLOSED

Fetal growth restriction (FGR) globally occurs in 8% of pregnancies, is severe and early onset in 1:500 cases, affecting 11,000 babies annually in the EU. In most cases, reduced uterine blood flow restricts substrate delivery to the fetus causing growth to slow or cease. There is no treatment. Currently the fetus is delivered very preterm before fetal death or irreversible organ damage occurs. Affected neonates suffer intracranial haemorrhage, chronic lung disease, cerebral palsy, with heart disease and diabetes as adults; mortality is high. Recent improvements in the care of premature growth restricted neonates, means that more of them survive delivery, but at great cost. Small increases in fetal growth and gestation at birth are associated with major improvements in survival and morbidity. Improving uterine blood flow is key and Vascular Endothelial Growth Factor (VEGF) is important to achieve this. In preclinical animal models we showed that local VEGF gene transfer to the uteroplacental circulation using adenovirus vectors increases uterine blood flow, attenuates constriction of uterine arteries and increases angiogenesis; these changes result in improved growth of severely growth restricted fetuses. This is the first clinically-applicable evidence based therapy that could improve perinatal outcome in severe early onset FGR in man. In collaboration with an SME experienced in gene therapy trials, our aim is to complete an agreed toxicology programme, identify specific ethical issues in stakeholders, and to perform a Phase I/II study in women with severe early onset FGR at four EU centres of excellence, using interventional radiology to deliver an adenovirus vector containing the mature processed form of human VEGF-D into the uterine artery. Data on safety, tolerability and efficacy will be analysed, and used, if successful, to inform phase II and III trials of this innovative therapy, leading to the first treatment for this intractable obstetric condition.

Keywords: targeted nucleic acid delivery,severe fetal growth restriction,uteroplacental insufficiency,vascular endothelial growth factor,gene therapy,adenovirus,phase i trial,pregnancy,neonatal outcome
Identifying correlates of protection to accelerate vaccine trials: systems evaluation of two models of experimentally-induced immunity to malaria (SYSMALVAC)

Framework: FP7 — Cooperation
Programme: Health
Destination: Translating research for human health
Action type: Collaborative project
Countries involved: Spain,Spain,Switzerland,Germany,Netherlands,Netherlands
Call:  (FP7-HEALTH-2012-INNOVATION-1, 2012)
Start date: 2013-01-01
End date: 2014-12-31
Duration (months): 24.0
Total project cost (EUR): 3635594.36
EU funding (EUR): 2803651.93
Participants: 7
Project status: CLOSED

Vaccine development is an empirical process (trial and error) and involves a long, expensive clinical development pipeline to license an efficacious vaccine candidate. Better tools for vaccine evaluation are needed to adapt to a rising number of candidate vaccines entering clinical trials for many diseases. Surrogate biomarkers of immunity offer the possibility of expediting the clinical development by eliminating non-viable candidates earlier in the pipeline, shortening vaccine trial timeframes by giving a proxy measurement for efficacy and by guiding future vaccine design. In the case of malaria and other complex diseases, a surrogate biomarker of immunity has been difficult to achieve with classical immunological assays. We propose using a systems biology analytical approach in two efficacious malaria vaccination models to identify combinatorial biomarkers of protection. First, newly generated cellular transcriptome profiles and previously generated immunological read-outs common to both trials will be integrated into a database for this analysis. An already developed artificial intelligence-based analytical tool that generates biological network maps, transforms experimental data to the map and discriminates transcriptional gene signatures to physiological states (protection or susceptibility) will be applied in both vaccination models. The aim is to determine malaria signatures of protection that will then be refined and validated in an experimentally induced immunity non-human primate model. The optimized model will be further validated on additional samples from the two protective human trials. The identified biomarkers of protection will be used to produce a customised Immunome Chip, which together with traditional immunological read-outs will be used to evaluate vaccine efficacy, shortening times and costs of clinical trials. This strategy may also prove useful for other diseases and support the systems medicine approach.

Keywords: clinical trials,systems biology,biomarkers,vaccines,malaria,transcriptomics,computational models,protection,vaccine efficacy