4BPA Promise for Cystic Fibrosis?

A drug which is already on the market for the treatment of urea cycledisorders may also have potential as a therapy for cystic fibrosis, according to researchers at the Johns Hopkins Children's Center in the USA who published their findings in the Journal of Clinical Investigation (November 15 issue).

CF sufferers have mutations in the CF transmembrane conductance regulator gene, which codes for a chloride-transporting protein. Around 70% of patients have the deltaF508 mutation in the CFTR gene, which means that instead of transporting chloride to the cell membrane, the proteins become stuck and are degraded. The subsequent chloride imbalance causes a thick mucus to build up, resulting in airway and intestinal obstructions.

Ucyclyd Pharma's orphan drug, Buphenyl (sodium phenylbutyrate; 4BPA), which was approved by the US Food and Drug Administration last year after nearly two years of discussions, allows patients to excrete waste nitrogen which would otherwise accumulate as ammonia (Marketletter September 30, 1996). Scientists collected cells from the lungs and nasal linings of CF patients and exposed them to 4BPA for up to seven days, Reuters reports.