FRIEDREICH’S ATAXIA: FUNDING GRANT HELPS BRIDGE INDUSTRY/RESEARCH DIVIDE ON RARE DISEASES

For further detail, read here
​
ERC awards PoC grant funding to “FAST” project headed by Prof. Roberto Testi
The European Research Council (ERC) has approved follow-up funding – in the form of a Proof of Concept (or PoC) grant – for the potential commercial development of therapeutic products devised by the “FAST” (or Friedreich’s Ataxia Seeks Therapy) project. The “FAST” project, led by Prof. Roberto Testi, director of the Laboratory of Signal Transduction at the Department of Biomedicine and Prevention of the University of Rome “Tor Vergata” and Full Professor of Immunology at the said University, was previously awarded an Advanced Grant by the ERC (under the Seventh Framework Programme [sic]) of around 1.5 million euro. Friedreich’s ataxia (FRDA) is a serious genetic disorder that affects the peripheral nervous system of children and adolescents, mainly between the ages of 5 and 15 years. Young people affected begin to present motor coordination disturbances that lead, more or less rapidly, to severe disability, often accompanied by cardiac insufficiency and reduced life expectancy. The genetic defect involves the gene tasked with the production of a protein called “frataxin”. In FRDA patients, the frataxin gene functions poorly and produces little protein, causing the death of nerve cells essential for motor coordination. Patients with FRDA make use of antioxidant drugs but as yet there is no effective specific treatment.
Testi’s research group has devised chemical molecules capable of increasing frataxin levels, or better still, of halting the degradation of frataxin. “Our approach”, explains Testi, “has been to actually think about how to make existing frataxin last longer. We have found that part of the frataxin produced is degraded even before being utilized, which is why we have focused on how to prevent this degradation and are working on transforming some of these molecules into drugs”.
The PoC grant was established to help bridge the gap between academia and industry, and to enable originators of a new treatment (as in this case) to receive funding for definitive proof of concept, so as to facilitate the protection of intellectual property and scouting for industry partners.