Vitamin B3 treatment for ataxia shows promise in first human trial

A form of vitamin B3 has shown early promise against Friedreich's ataxia, a debilitating degenerative disease with no treatment or cure, in the first human trial of the treatment involving UCL researchers. Friedriech's ataxia is thought to be caused by a lack of frataxin, a protein important in regulating iron levels within cells. Frataxin deficiency causes iron overload in mitochondria, the 'batteries' of cells, and problems regulating free radicals within cells. These damaging effects lead to cellular death. As the damage builds up, people with the condition suffer from progressively worsening symptoms including problems walking, talking, swallowing, seeing and hearing. Many patients are wheelchair-bound and unable to live independently a decade or two after symptoms appear and often die of heart disease within 35 years. In a collaborative effort, researchers from UCL and Imperial College London gave patients a form of vitamin B3 called nicotinamide in much higher doses than found in vitamin supplements.