New hope for Fanconi Anemia

Two widely used compounds may help lower the chromosome instability associated with Fanconi anemia, a rare DNA repair disorder that affects about one in 350,000 people, according to researchers led by Beatriz Porto, PhD, of the Institute of Biomedical Sciences Abel Salazar in Porto, Portugal. The increased rate of DNA damage in Fanconi patients predisposes them to cancer, but, in a series of lab experiments, the researchers showed that alpha-lipoic acid and N-acetylcysteine markedly reduced DNA breaks in cells from Fanconi anemia patients. When they were used together, the rate of breakage fell by 58%, they reported in Orphanet Journal of Rare Diseases Alpha-lipoic acid is essential for metabolism, is widely sold as a nutritional supplement, and in some places is approved to treat polyneuropathies. N-acetylcysteine is used clinically to treat acetaminophen overdose. Because both substances are widely used and safe, the researchers argued, they could be tested immediately in phase III trials, writes Michael Smith from medpagetoday