Potential treatment of cystic fibrosis and Duchenne

PubMed, the Internet portal of biomedical and life sciences literature, indexed an interesting article, entitled “Drug evaluation: PTC-124-a potential treatment of cystic fibrosis and Duchenne muscular dystrophy.” (IDrugs. 2006 Nov;9(11):783-9). Author is Hamed SA, from the Saudi German Hospital – Aseer, Khamis Mushayt 61961, Saudi Arabia. PTC-124, a 1,2,4-oxadiazole compound, is in development as an orally active small molecule that can override nonsense stop translation signals to produce full-length proteins. PTC-124 is currently being evaluated in phase II clinical trials against cystic fibrosis (CF) and Duchenne muscular dystrophy (DMD). The functional properties of PTC-124 are similar to the aminoglycoside antibiotic gentamicin, but the two compounds are chemically distinct and PTC-124 does not exhibit any antibiotic characteristics. Phase I clinical trials reported that PTC-124 was well tolerated in healthy patients. The author concludes that the encouraging results observed to date make PTC-124 an attractive option for further well-designed, long-term human studies on larger sample populations. To access the abstract of the article, click here.