Data-Gouv-ML/pcri-informations-sur-les-projets-laureats huggingface dataset:
Single-Port Insulin Infusion for Improved Diabetes Management (SPIDIMAN)

Framework: FP7 — Cooperation
Programme: Health
Destination: Translating research for human health
Action type: Collaborative project
Countries involved: Austria,France,France,United Kingdom,Germany,Luxembourg,Germany
Call:  (FP7-HEALTH-2012-INNOVATION-1, 2012)
Start date: 2012-11-01
End date: 2017-10-31
Duration (months): 60.0
Total project cost (EUR): 6372167.28
EU funding (EUR): 4941738.75
Participants: 9
Project status: CLOSED

Existing therapeutic devices for diabetic patients suffer from bulky size, inaccurate measurements and the difficulties of handling two body interfaces. Suboptimal control of blood glucose levels in type 1 or type 2 diabetes mellitus patients results in periods of hypo- and hyperglycaemia leading to severe and life-threatening complications. Exploiting a novel glucose sensor technology, SPIDIMAN aims to improve glycaemic management for better quality of life and healthier aging. The consortium will develop a new coating technology to apply a glucose-sensitive fluorescent dye onto a standard insulin catheter and incorporate this integrated glucose sensor into a single-port artificial pancreas system. Advanced optical continuous glucose monitoring technology (smart tattoos) with improved sensor accuracy, faster response times, wider dynamic range and higher signal stability will advance diabetes management by reducing hypo- and hyperglycaemic episodes. Within SPIDIMAN, research-intensive European SMEs will develop an innovative artificial pancreas approach, and experienced participants will perform clinical validation in a European network of specialised diabetes centres. SPIDIMAN will thus pave the way for a single-port device that integrates improved glucose measurement and more accurate insulin delivery to provide better glycaemic management in patients with insulin-dependent diabetes. The new device is expected to be particularly suitable for patients in childhood and adolescence, who will form a special focus of the project.

Keywords: single-port artificial pancreas,optical glucose sensor,glycaemic control,t1dm,smart tattoo,paediatric diabetes,continuous glucose measurement. control algorithm,transcutaneous cgm
A phase I/IIa clinical trial in Duchenne muscular dystrophy using systemically delivered morpholino antisense oligomer to skip exon 53 (SKIP-NMD)

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,Italy,France,Belgium,United Kingdom,United States
Call:  (FP7-HEALTH-2012-INNOVATION-1, 2012)
Start date: 2012-11-01
End date: 2016-04-30
Duration (months): 42.0
Total project cost (EUR): 7527838.99
EU funding (EUR): 5512424.07
Participants: 10
Project status: CLOSED

Duchenne muscular dystrophy (DMD) is a progressive, lethal muscle degenerative condition arising from the absence of dystrophin in skeletal and cardiac muscles. 65% of DMD boys have out-of-frame deletions. Modulation of pre-mRNA splicing by exon skipping is the most promising molecular intervention in DMD. 2 Phase Ib and 2 Phase IIa clinical trials (MDEX Consortium in collaboration with Sarepta Therapeutics; a Dutch Consortium) demonstrated that delivery of antisense oligonucleotides (AOs) to mediate exon skipping of exon 51 were able to return specific DMD mutations in-frame (~13% of all mutations) leading to new dystrophin protein expression after intramuscular and systemic delivery. The Dutch study of repeated 2-O-methylated phosphorothioate (2OMe) AO administration suggested limited efficacy after 5 weeks of treatment. Our MDEX Consortium study using a morpholino (PMO) AO demonstrated a clear dose response, robust dystrophin restoration and reduction of muscle inflammation after 12 weeks at doses up to 20mg/Kg, with no drug related adverse events. This, and preclinical studies focused on level of protein expression, clearly indicate that PMO have a superior therapeutic index compared to 2OMe. New PMOs are needed to target other DMD mutations. We will develop a PMO to skip exon 53 and perform a clinical trial in DMD boys using a world leading pan-European consortium. This will allow us to advance this class of PMO therapy in DMD by i. assessing the safety and efficacy of targeting another exon; ii. exploring the use of non-invasive techniques to monitor dystrophin restoration. This new PMO will be administered over 12 weeks in 3 groups, each of 4 DMD boys, receiving between 4 to 30mg/kg or placebo. If well tolerated, all boys will be treated for another 24 weeks at a dose of 30mg/kg. Safety and dystrophin restoration in a muscle biopsy at the end of this period will be the study endpoints. MRI, MRS and serum miRNA will be used to monitor muscle pathology non-invasively.

Keywords: duchenne muscular dystrophy,exon skipping,antisense oligomers,morpholino backbone,pmo,exon 53,therapeutic trial
Healthy Aging Through Internet Counselling in the Elderly (HATICE)

Framework: FP7 — Cooperation
Programme: Health
Destination: Translating research for human health
Action type: Collaborative project
Countries involved: Netherlands,Sweden,Finland,France,United Kingdom,Netherlands
Call:  (FP7-HEALTH-2012-INNOVATION-1, 2012)
Start date: 2013-01-01
End date: 2018-06-30
Duration (months): 66.0
Total project cost (EUR): 7423520.0
EU funding (EUR): 5824039.0
Participants: 7
Project status: CLOSED

Diabetes mellitus, hypertension, obesity, hypercholesterolemia, smoking and physical inactivity are common in elderly persons and are all associated with an increased risk of myocardial infarction, stroke and dementia. Efficacious treatments of these cardiovascular diseases are available, but in elderly patients with multiple cardiovascular diseases and risk factors however, treatment is currently insufficient. Optimisation of management of these diseases in order to decrease the burden of cardiovascular disease and its long-term complications is urgently needed. In this project we will develop and evaluate an innovative intervention strategy to improve pharmacological and non-pharmacological treatment for cardiovascular disease in elderly patients. First, data from three large ongoing clinical trials on multi-component vascular interventions in the elderly will be used to explore efficacy and feasibility of various treatment regimens. Second, an innovative and interactive internet platform for self-management of vascular diseases in the elderly will be developed. Interactive support by practice nurses and patient’s own physician and monitoring of adverse events will be integrated. Third, the efficacy of access to this new internet platform will be evaluated in a randomised controlled trial in 2600 elderly with multiple cardiovascular diseases. Patients randomised to the control condition will receive regular care. Primary outcome is a composite score including three vascular risk factors: systolic blood pressure, LDL cholesterol and BMI. Secondary outcomes are new cardiovascular events, handicap, cognition and survival. This flexible internet-based intervention strategy can be easily translated and adapted for use in different health-care systems in the EU. It will allow for tailor-made interventions specifically suited to the needs of older people and meticulous monitoring of side effects. The expected decrease in new cardiovascular events and dementia will result in lowering of healthcare costs substantially.

Keywords: aging,elderly,cardiovascular disease,internet,health,adverse events,self-management
Better Experimental Screening and Treatment for Primary Ciliary Dyskinesia (BESTCILIA)

Framework: FP7 — Cooperation
Programme: Health
Destination: Translating research for human health
Action type: Collaborative project
Countries involved: Germany,Switzerland,Cyprus,United States,Denmark,Netherlands,United Kingdom,Poland,Greece,United Kingdom
Call:  (FP7-HEALTH-2012-INNOVATION-1, 2012)
Start date: 2012-12-01
End date: 2016-05-31
Duration (months): 42.0
Total project cost (EUR): 3850875.6
EU funding (EUR): 2993674.6
Participants: 12
Project status: CLOSED

Primary Ciliary Dyskinesia (PCD) is a rare genetically heterogeneous disorder which results from dysfunction of motile hair-like organelles (cilia) that results in severe, chronic airways disease. Due to other cilia-related disease mechanisms several other organ systems like the heart can be affected. The complexity of the disease phenotype, late diagnosis, as well as lack of evidence based management guidelines contribute to a high burden of disease and cause high health care costs. Therefore, there is a great need for observational trials as well as well-designed randomised controlled trials to put evidence-based diagnostic and treatment approaches into effect. The main objective of our project is to improve diagnosis and treatment of PCD patients. To accomplish this, we propose to: 1) Establish widespread, early diagnosis by introduction of nasal Nitric Oxide measurement as screening tool, and by introduction of high-speed videomicroscopy as diagnostic tool; 2) Develop new outcome criteria, especially a PCD-specific quality of life questionnaire, as a prerequisite for controlled PCD trials; 3) Establish a PCD registry for both cross-sectional analysis of current disease status and longitudinal observational analysis of disease progression under different regimens; 4) Generate evidence-based treatment guidelines by conducting two prospective randomized trials on the inhalation of hypertonic saline and long term azithromycin therapy. To achieve these goals members of the European Respiratory Society’s PCD task force will join forces with members of the NIH-funded US-PCD-network. In our multi-national project, we will for the first time establish evidence-based guidelines for diagnosis, clinical management and therapy. We expect that in a high proportion of children the diagnosis will be established before irreversible lung damage has occurred. In later diagnosed individuals the disease burden will be reduced and chronic respiratory failure retarded.

Keywords: primary ciliary dyskinesia,nasal no,videomicroscopy,questionnaire,registry,hypertonic saline,azithromycin
Biomarkers of renal graft injuries in kidney allograft recipients (BIOMARGIN)

Framework: FP7 — Cooperation
Programme: Health
Destination: Biotechnology, generic tools and medical technologies for human health
Action type: Collaborative project
Countries involved: France,Belgium,Belgium,Germany,France,France,Sweden
Call:  (FP7-HEALTH-2012-INNOVATION-1, 2012)
Start date: 2013-03-01
End date: 2018-02-28
Duration (months): 60.0
Total project cost (EUR): 7836208.0
EU funding (EUR): 6000000.0
Participants: 13
Project status: CLOSED

In renal allograft recipients, 10-year graft survival has not improved over the past decades. Histological examination of graft biopsies has long been the gold standard to confirm graft injuries, but biopsies are invasive and histological grading is not very robust. There is thus a need for robust, non-invasive methods to predict and diagnose acute and chronic graft lesions, to improve patient treatment, quality of life and long-term graft survival. Also, there is an unmet need for better understanding of the immune and non-immune mechanisms of interstitial fibrosis /tubular atrophy and graft loss. Combining all the skills required to build upon previous findings, BIOMARGIN will offer such opportunities in renal transplantation by integrating several omics approaches (mRNA, miRNA, peptides, proteins, lipids and metabolites) in blood, graft tissue and urine, in a well thought out, multistage discovery-to-validation translational programme, following the highest European ethics and regulatory requirements, as well as quality controls and quality assessments at all clinical and analytical steps. It is probably one of the first programmes to pursue such an integrated and systematic research approach. BIOMARGIN aims to: (i) discover, select and validate blood and/or urine biomarkers of renal allograft lesions in adult and pediatric kidney transplant recipients; (ii) provide renal transplant physicians with non-invasive, robust diagnostic tests and interpretation algorithms enabling closer, more accurate, more predictive and/or less invasive monitoring of transplanted patients; (iii) help to avoid or diminish the use of biopsies and improve patient treatment, quality of life and long-term graft survival; (iv) help understand the mechanisms involved in the allograft injury processes which, combined with mass spectrometry imaging should offer pathologists new molecular targets and tools for renal graft biopsy analysis.

Keywords: biomarkers kidney transplantation graft humoral rejection interstitial fibrosis mrna mirna proteins peptide lipid metabolite spectrometry imaging data integration algorithms disease progression models
Heart OMics in AGEing (HOMAGE)

Framework: FP7 — Cooperation
Programme: Health
Destination: Translating research for human health
Action type: Collaborative project
Countries involved: France,France,Austria,United Kingdom,Spain,Ireland,United Kingdom,Netherlands,Italy,Germany,Belgium,United States,United Kingdom,United Kingdom
Call:  (FP7-HEALTH-2012-INNOVATION-1, 2012)
Start date: 2013-02-01
End date: 2019-01-31
Duration (months): 72.0
Total project cost (EUR): 16366890.17
EU funding (EUR): 12000000.0
Participants: 22
Project status: CLOSED

As more people survive into old age, the prevalence of heart failure (HF), one of the most common and debilitating diseases in older people, will rise still further. Delaying or preventing HF will have great benefit to those at personal risk, their families, society and the economy. HOMAGE aims to provide a biomarker (BM) approach that will a) help identify i. patients at high risk of developing HF before the onset of symptoms and ii. subsets of patients who are more likely to respond to specifically targeted therapies (personalized medicine). In available cohorts, we will identify the most promising ‘omics-based BM profiles for the pre-symptomatic diagnosis and future prediction of HF in patients at risk. The predictive value of the BMs for other co-morbidities commonly associated with HF and ageing will also be investigated. Furthermore, in a prospective trial, we will investigate the potential for targeting preventive therapy at patients with the greatest likelihood of response and the lowest risk of adverse effects. Our selection of innovative ‘omics-based BMs is based on knowledge of biological pathways of the disease, which may facilitate identification of ‘Biotargets’ for future therapies. On the economic side, HOMAGE will act as an economic catalyst for European SMEs in the field of cardiovascular and ageing BMs, estimated to peak annual turnovers of up to 800 M€.

Keywords: omics,biomarkers,heart failure,elderly,inflammation,extracellular matrix remodelling,fibrolisis,myocyte injury
Personalized diagnostics and treatment of high risk coronary artery disease patients. (RISKYCAD)

Framework: FP7 — Cooperation
Programme: Health
Destination: Biotechnology, generic tools and medical technologies for human health
Action type: Collaborative project
Countries involved: Greece,Finland,Germany,United Kingdom,Finland,Switzerland,Italy,Germany,France
Call:  (FP7-HEALTH-2012-INNOVATION-1, 2012)
Start date: 2012-11-01
End date: 2017-10-31
Duration (months): 60.0
Total project cost (EUR): 7901914.51
EU funding (EUR): 5992101.0
Participants: 12
Project status: CLOSED

Although the accurate diagnosis and prevention of coronary artery disease (CAD), acute myocardial infarction (AMI) and death is a major public health issue, risk stratification for CAD with current diagnostic tools is not properly supporting clinical decision making. RiskyCAD’s overall goal is to identify novel biomarkers for asymptomatic patients in high risk of major coronary events, and develop new diagnostic tools and ‘personalized’ therapeutic strategies for this selected group of patients. Taking advantage of the latest technologies, we will look into new biomarkers for asymptomatic patients in high risk of CAD using some of the finest cohorts (WP1), identify new molecules and develop new diagnostic kits that will be further validated in additional cohorts (WP6). We will generate reprogrammed iPS cell based human models for the study of metabolic aberrations in selected individual vulnerable CAD patients (WP2). We will identify defects, e.g. in liver lipid metabolism, in high risk CAD patients (WP3) and test targeted treatments in traditional and new preclinical animal models to provide proof-of-concept level evidence (WP4, WP5). As our approach is based on well characterized patients and phenotypes, we will generate clinically applicable stratification methods that will allow targeted treatment and patient enrichment for clinical trials (WP1). Unique predictive modelling and link identification platforms including Drug Repositioning will be used to select drugs or drug combinations that will best match the patient’s biomarker profile (WP7). The final (translational) outcomes of this project will be: (i) a set of distinct biomarker test(s) for asymptomatic patients in high risk of CAD that can be ordered both by primary care physicians and specialized cardiologists from routine clinical laboratories; (ii) new CAD risk estimation models; (iii) a set of repositioned drugs ready to be exploited further for the optimal treatment of patients in high risk of CA

Keywords: personalized medicine,vulnerable patient,coronary artery disease,ips cells,mirna,lipidomics
Nanomedicine for target-specific imaging and treatment of atherosclerosis: development and initial clinical feasibility (NANOATHERO)

Framework: FP7 — Cooperation
Programme: Nanosciences, Nanotechnologies, Materials and new Production Technologies - NMP
Destination: Nanosciences and Nanotechnologies
Action type: Collaborative project
Countries involved: France,Netherlands,Austria,Denmark,Germany,Netherlands,Germany,Switzerland,Germany,Hungary,Italy,Israel,United Kingdom
Call:  (FP7-NMP-2012-LARGE-6, 2012)
Start date: 2013-02-01
End date: 2018-07-31
Duration (months): 66.0
Total project cost (EUR): 12993053.5
EU funding (EUR): 9833348.0
Participants: 16
Project status: CLOSED

NanoAthero aims to have demonstration of initial clinical feasibility of nanosystems for targeted imaging and treatment of advanced atherosclerotic disease in humans. The nanosystems are assemblies of following components: nanocarrier, targeting, imaging agent/drug. They have proven safety records, and strong preliminary in vitro and in vivo proofs of efficacy are available. Partners have patented and provided evidence of efficacy of carriers and ligands. Over 5 years, NanoAthero will integrate GMP production, the initiation of clinical investigations in high-risk patients, including the preparation of regulatory dossiers, risk and ethical assessments, and the evaluation of the performance of optimized diagnostic and therapeutic compounds. NanoAthero offers a unique opportunity for combining in-depth knowledge of nanocarrier bioengineering and production with state-of the art expertise in imaging and treatment of cardiovascular patients providing a full bench-to-bedside framework within one collaborative consortium of 16 partners from academia, a European association, SMEs and a large pharmaceutical company. NanoAthero gathers together leading chemists, engineers, pharmacists, biologists, toxicologists, clinicians, analysts, ethicists and key-opinion leaders in the field of cardiovascular medicine and early drug development. In NanoAthero, the nanocarriers carrying compounds to visualize thrombus or vulnerable plaques, or to deliver therapeutic agents should be suitable for proof-of-concept in patients. Phase I clinical trials targeting pivotal pathways in atherothrombosis will be performed with nanosystems for diagnosis and treatment of carotid atheroma. NanoAthero aims to propose nanosystems for thrombus imaging, stroke treatment and plaque stabilization in high-risk patients. Molecular imaging and therapeutic treatments in NanoAthero are based on feasible approaches.

Keywords: atherosclerosis,stroke,nanocarriers,molecular imaging,therapeutic nanosystems,target-specific imaging,initial clinical feasibility,mri,spect,pet,gmp production,injectable medicinal product
Innate sensing of HIV and immune responses (HIVINNATE)

Framework: FP7 — Ideas
Programme: European Research Council
Theme: European research council
Destination: Starting grants
Action type: Support for frontier research (ERC)
Panel: Immunity and infection
Countries involved: France
Call:  (ERC-2012-StG_20111109, 2012)
Start date: 2013-01-01
End date: 2017-12-31
Duration (months): 60.0
Total project cost (EUR): 1680000.0
EU funding (EUR): 1680000.0
Participants: 1
Project status: CLOSED

Since its isolation in 1983 as a causative agent of AIDS, HIV-1 has remained an exceptional challenge. The human immune system usually ultimately fails at controlling infection by the virus. In contrast, most individuals infected with the related and poorly studied lentivirus HIV-2 do not develop AIDS. The immune response is clearly implicated in this protection, but the mechanisms are not well understood. DCs are a unique type of immune cell that sense pathogens and couple this sensing to the activation of innate and adaptive immunity. We have recently discovered that unlike HIV-1, HIV-2 is sensed in dendritic cells (DCs), a finding that is strikingly parallel to the in vivo situation. We found that sensing of the virus in DCs in vitro induces an antiviral innate response and activation of CD4+ and CD8+ T cells. Whether this adaptive response contributes to the lack of AIDS pathology in HIV-2 infected patients is unknown. Therefore, while the vast majorities of studies have focused on HIV-1, we believe that the HIV-2 represents a unique opportunity to discover how HIV-sensing in DCs translates into protective immune responses. To address this question, we propose an approach at the interface of immunology and virology. We propose to: 1) Unravel the type of CD4+ and CD8+ T cell responses induced by DCs that sense HIV-2. We will examine how naïve T cells respond to HIV-2 in vitro and how this compares with the response of memory T cells isolated from HIV-2 infected patients who control their virus. 2) Determine what is sensed by DCs in HIV-2. 3) Identify cellular regulators of HIV sensing in DCs. Altogether, this study will integrate molecular sensing of the virus and adaptive immunity to provide an understanding of how the immune response against HIV-2 operates. We believe that this may lead to novel approaches based on the manipulation of innate immunity against HIV-1 and HIV-2 infections and in other types of infection.

Keywords: hiv-2,hiv-1,dendritic cells,innate sensing,innate immunity,adaptive immunity,capsid,cyclophilin