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Dr Julie Greenfield, Research Manager at Ataxia UKNews kindly supplied by Goran Ilic

3 January 2012 - Researchers at the San Luigi Hospital in Torino, Italy, have been given the go-ahead to run a Phase I clinical trial of a new drug designed specifically to treat Friedreich’s ataxia. Regulatory approval was granted for this trial in which up to 20 patients with the condition will be tested with the drug, known as RG2833.

Scientists will aim to find out whether the drug is safe, and to learn more about its effects on the body. In particular, they will measure any changes in levels of frataxin, a key protein that is reduced in people with Friedreich’s ataxia.

RG2833 is a type of drug called a ‘histone deacetylase inhibitor’ that was developed by the pharmaceutical company Repligen, working with US scientists. Histone deacetylase inhibitors may provide a way to increase levels of frataxin by activating the gene responsible for frataxin production, which is switched off in people with Friedreich's ataxia. European research initiative GoFAR have given their support to the study.

Dr Julie Greenfield, Research Manager here at Ataxia UK said “There has been much interest recently in histone deacetylase inhibitor drugs as a potential therapy for Friedreich’s ataxia, and Ataxia UK has played a role in funding such research. This is an exciting step towards getting this important work underway.”

Source: Ataxia UK