Foto by José Cano
The Carlos III Health Institute (ISCIII in Spanish) will fund, in the framework of the “International Rare Disease Research Consortium (IRDiRC), three collaboration research projects in the whole of Spain, one of which is “DRUGS4RARE: drug repositioning for rare diseases”. This project is lead by Jordi Quintana, in charge of the Drug Discovery Platform at the Barcelona Science Park (PCB in Spanish), with the participation of Jordi Mestres, the coordinator of the Chemogenomics research group of the IMIM (Hospital del Mar Research Institute) and Pompeu Fabra University (UPF), together with Mabel Loza, from the University of Santiago de Compostela (USC).
IRDiRC is an international initiative launched in 2011 upon a proposal made by the European Commission and the US National Institutes of Health (NIH) to study and make progress on gaining further knowledge on minority diseases, with the aim of developing 200 new therapy and diagnosis strategies for rare diseases and to promote global registers based on national registers. Spain was one of the first European countries to sign for adhesion to this consortium.
According to Jordi Mestres, “academic centres have achieved a level of maturity and expertise in designing, synthesizing and testing molecules with a therapeutic interest which is one of the keys during the first stages of developing new drugs for rare diseases when pharmaceutical companies may be more reticent to prioritize this, both from the point of view of investment risks and a limited potential market”.
For this project, the IMIM will be in charge of designing and selecting small molecules focusing on a list of priority rare diseases, which will then be synthesized or purchased by the PBC, and then be sent to the USC for in vitro testing against biological targets predicted by the IMIM. The duration of the project is two years with an investment of €346,000 which will be allocated to covering personnel and purchasing costs, synthesizing and testing small molecules which could be potentially bioactive in relevant target molecules for rare diseases.
The other two projects funded by the ISCIII are one to set up a national register of rare diseases, based on the existing regional registers, led by Manuel Posada, the director of the Research Institute for Rare Diseases (IEER in Spanish), and a trans-national experimental and therapeutic investigation on the Charcot-Marie-Tooth disease, lead by Francisco Palau, the scientific director of the Network Biomedical Research Centre for Rare Disease (CIBERER in Spanish).