Repurposing Diflunisal for Familial Amyloid Polyneuropathy

in Uncategorized
December 24th, 2013

December 25, 2013

An international consortium of investigators led by Amyloidosis Center Clinical Director Dr. John Berk have completed a randomized, placebo-controlled, phase III clinical trial of the non-steroidal anti-inflammatory drug diflunisal for patients with ATTR polyneuropathy, published today in the Journal of the American Medical Association. 130 participating patients received either diflunisal (250 mg twice a day) or placebo for up to two years. Diflunisal met its primary endpoint, showing a significant reduction in progression of the Neuropathy Impairment Score + 7, and its secondary endpoint of quality of life, measured by SF-36 questionnaire. Details can be found on the JAMA website. This ground-breaking research  derived from biophysical studies of Dr. Jeffery Kelly and colleagues of the Scripps Institute in La Jolla, California, and used techniques for measuring neuropathy developed by Dr. Peter Dyck of the Mayo Clinic. The diflunisal clinical trial was supported by the  National Institute of Neurological Diseases and Stroke at the NIH (R01 NS051306), the Food and Drug Administration Office of Orphan Products Development (FD-R 002532), the Young Family Amyloid Research Fund, the BU Clinical and Translational Science Institute, and by private donors; Merck Sharp and Dohme donated the diflunisal.

Further information and Dr. Berk’s comments can be found in the Boston University School of Medicine and Boston Medical Center press release: Diflunisal Press Release

This story was featured in Deborah Kotz’s “Daily Dose” blog on Boston.com: Daily Dose, 12/26/2013