• Canada
• European Commission close
• 2014 close

Rare diseases (RD) are diseases that affect not more than 5 per 10 000 persons (according to the EU definition). 7000 distinct rare diseases exist, affecting between 6% and 8% of the population (about 30 million EU citizens). The lack of specific health policies for rare diseases and the scarcity of the expertise, translate into delayed diagnosis, few medicinal products and difficult access to care. That is why rare diseases are a prime example of a research area that strongly profits from coordination on a European scale. At present only few European countries fund research on rare diseases through specific dedicated programmes. Therefore, the funding of transnational collaborative research is the most effective joint activity to enhance the cooperation between scientists working on rare diseases in Europe and beyond. The E-Rare consortium was built to link responsible funding bodies that combine the scarce resources and fund rare disease research via Joint Transnational Calls (JTCs). The current E-Rare-3 project proposal will extend and strengthen the transnational cooperation by building on the experience and results of the previous E-Rare-1&2 programmes. The consortium comprises 26 institutions from 17 European, Associated and non-European countries. Its international dimension will be directly translated into close collaboration with IRDiRC and other relevant European and international initiatives. IRDiRC guidelines and policies will be implemented in the four JTCs and representatives of the IRDiRC Scientific Committees will be invited to join the Advisory Board of E-Rare-3. Members of the EUCERD group will be involved in identifying rare disease research needs. Patients’ organizations from Europe (represented by EURORDIS) and beyond will be invited as a key partner towards collaborative efforts for research promotion and funding. The collaboration with European Research Infrastructures will be consolidated to enhance efficient and participative research.

IF-EBOLA has been strategically designed to efficiently respond to critical needs required to control Ebola outbreak from spreading and prepare a therapeutic approach to save lives. The work involves two of the main EVD outbreak sites, Sierra Leone and Liberia. MDs, public health authorities and virus experts working on site, under ethical regulatory rules, will extend their collaboration to companies and institutions to form a consortium of outstanding complementary partners, sharing their innovative technological approaches for a common goal. The main goal of our project is to contribute to provide innovative ultrasensitive diagnostics and therapeutic approaches for an early and accurate diagnostic for an early therapeutic treatment to control the epidemic and safe lives. We have been obligated to modify and extend for one year our initial two-year period IF-EBOLA action program due to both major foreseen and unforeseen changes produced during this “non-emergency” period: consisting mainly now in 3 main actions: (i) Preclinical validation of the therapeutic equine anti-Ebola antibodies; (ii) Clinical validation of the ultrasensitive detection (iii) homeostasis (“pathology”) profiling of patients and survivors. Originally (“emergency period”) IF-EBOLA included 2 phases: (I) a phase of preparation including, ethical authorizations, antibody production, technical and field organization as well as the beginning a follow-up of the homeostatic profile of contacts early-EBOV diagnosed and self-cured convalescent individuals in the absence of existing treatment, (with an ultrasensitive detection method of pernicious microorganisms, from the EC USDEP project qualified as a European success story “USDEP” project in 2010 by the EC-Project Officer) and (II) phase of a clinical study using a wide validated approach revisited with an innovative concept (strongly supported EC/EMA-WHO), we propose to carry out an experimental passive-immune therapy based on neutralizing capacity of horse anti-EBOV polyclonal F(ab’)2 on early-diagnosed patients to impact and reduce their pre-existing viremia, their mortality, the evolution of their homeostasis profile, during and after this treatment (once patients become convalescents). The homeostasis status evolution will help to generate high quality scientific data to understand the EVD, the effect of this therapy and cure parameters characterized at 3 different levels: immune (transcriptomes, NGS, metagenomics); infectious (other than EBOV, DNA arrays), and EBOV diversity (sequencing and metagenomics).