13 July 2012
We are pleased to announce that the clinical trial, led by Professor Richard Festenstein at Imperial College London, has now started. The team will test a promising type of potential treatment for Friedreich’s ataxia (FA) known as histone deacetylase inhibitors or HDACi. This type of drug is a protein that can switch-on the frataxin gene. In FA, the frataxin gene is switched off, resulting in limited production of the essential protein frataxin.
The first people with Friedreich’s ataxia have now been enrolled. Dr Paola Giunti, the neurologist at the Ataxia UK accredited Ataxia Centre in London, was responsible for the recruitment of patients and is collaborating with Prof Festenstein in this trial. At this early stage, the main aim of the trial is to find out whether the drug can increase their levels of frataxin. The team will also study how well the drug is tolerated and find out more about whether it is safe.
You can read about the Ataxia UK-funded research project in which the initial tests were carried out in this summary (a PDF file). And for more information on the current trial you can go to www.clinicaltrials.gov
Health warning: As with a number of potential treatments for the ataxias and other neurological conditions, the HDACi to be tested in this trial is already available for other usage. It is important to know that large doses of certain medications are known to be toxic and can have serious consequences. Clinical trials are designed specifically to enable testing in a controlled environment in which dosage and its effects can be managed and monitored with full medical support.