HEALTH

Relevance: WINTHER project focuses on the development and validation, in 24 months, of industry supported new tools and computational methods, to predict efficacy of drugs in cancer patients
Current medical needs: Selection of cancer targeted therapies based on molecular abnormalities (mutations /amplifications/ translocations) benefit today only to 10-40% of patients. For the remaining majority, therapeutic decision is still based according to decade-old protocols with limited efficacy. Drug development depends on efficiency of clinical trials, with a staggering 95% failure rate

Since biology is by and large a 3-dimensional phenomenon, 3D imaging has a significant impact on many challenges in life sciences. 3D matrix-assisted laser desorption/ionization (MALDI) imaging mass spectrometry (IMS) is an emerging label-free 3D imaging technology with high potential in proteomics and metabolomics. 3D MALDI-IMS is based on 2D MALDI-IMS, which in the last years has proven its value in metabolomics, glycomics, lipidomics, peptidomics, and proteomics.

The use of HTA has increased recently in Europe and more widely (e.g. Americas) to enable evidence-based coverage decisions and improve efficiency in resource allocation.
HTA has often resulted in different coverage decisions across settings despite the same evidence being used for this purpose. This may reflect in part societal preferences about value, priorities or risk perceptions, suggesting a significant need for methodological improvements and extensions.

The 40% protection against HIV-1 infection with vaginally-applied tenofovir gel demonstrated in the CAPRISA004 trial, has emphasized development of anti-retroviral (ARV)-based microbicides. Combining ARVs in a single product may provide increased protection. Development of a generally applicable formulation platform for combining ARVs (even if chemically incompatible) and understanding of the processes (drug uptake, efflux and metabolism) that determine tissue levels of microbicide are two important gaps in knowledge necessary for microbicide development.

The MDGs succeeded in generating consensus on and mobilising resources towards agreed goals. They were less successful at clarifying responsibilities for achieving them. The MDG target on sharing global health innovation is particularly ambiguous about the allocation of responsibilities.

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.

0 million diabetic EU citizens are using approved anti-diabetic agents to control their glycaemia. However, suboptimal glycemic control leads to 6 progressive diabetic complications, namely: nephropathy, retinopathy, cardiomyopathy, neuropathy and foot ulceration. In 2010, 11% of EU adult deaths (634,000) were caused by diabetic complications. These distinct disorders have few effective medicines and present challenging management issues for clinicians. Stromal Stem Cells (SSC) are a mixed population of plastic-adherent (PA) cells isolated from adult bone marrow.

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.

Neurodegenerative (ND) and neuromuscular (NM) disease is one of the most frequent classes of rare diseases, affecting life and mobility of 500,000 patients in Europe and millions of their caregivers, family members and employers. This NEUROMICS project brings together the leading research groups in Europe, five highly innovative SMEs and relevant oversea experts using the most sophisticated Omics technologies to revolutionize diagnostics and to develop pathomechanism-based treatment for ten major ND and NM diseases. Specifically we aim to: