The gene therapy-based research project to tackle Friedreich's ataxia launched in November in labs at the Institute for Research in Biomedicine (IRB) –based in the Parc Científic de Barcelona (PCB)– and the "Centro de Biología Molecular Severo Ochoa" (CBMSO), in Madrid, has received 100,000 US$ per year for two years from the Friedreich's Ataxia Research Alliance (FARA). FARA is one of the main patients' organisations in the United States and since 1998 it has provided a rigorous and solid funding programme for research projects all over the world that aim to tackle Friedreich's ataxia, a rare and serious hereditary neurodegenerative disease for which only palliative treatments are currently available.

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The peculiarity is that the idea of the project came from those affected by the disease, patients and relatives, who, in their endeavours to find a cure got in touch with basic research groups in order to start a long-term project. The GENEFA Platform, in close collaboration with FEDAES and the BabelFAmily, started fund-raising efforts in May 2013, and in November signed an agreement with IRB and CMBSO, headed by Joan Guinovart, director of the IRB, and Margarita Salas, president of the “Fundación Severo Ochoa”.

The project involves the research groups headed by Javier Díaz-Nido at the CBMSO, an international expert in gene therapy and Friedreich’s ataxia, and Ernest Giralt at IRB, an international authority on the design of transporters, such as nanoparticles, that can carry drugs into the brain and thus overcome the blood-brain barrier. Patients with this ataxia inherit a mutated version of the frataxin gene, which causes neurodegeneration. The project seeks to rescue this defect in cells of the central nervous system.

The support provided by FARA will account for 50% of the project budget of 300,000 euros. Of note, FARA’s funds come from the support of patient families and communities raising funds at a grassroots level and from other International advocates such as FARA – Ireland.

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