Anti-Inflammatory Drug Treats Rare Disease

(Ivanhoe Newswire) –Researchers have discovered that a generic anti-inflammatory drug, diflunisal, successfully reduced neurological decline and preserved the quality of life in patients with familial transthyretin amyloidosis (ATTR).

This study is one of the first examples of successful repurposing of a generic drug to treat a rare disease. Patients with hereditary ATTR amyloidosis develop debilitating peripheral and autonomic nerve damage, weight loss, and heart disease. Patients who aren’t treated die ten to fifteen years after disease onset.

The investigators at the Amyloidosis Center at Boston University School of Medicine (BUSM) and Boston Medical Center (BMC) found that diflunisal dramatically altered the progression of neurologic disease while preserving quality of life when compared to placebo treatment. "Our results show that diflunisal represents an alternative to liver transplantation, the current standard of care for this devastating disease. We hope that this study prompts the identification of other widely-used generic drugs for treatment of rare diseases,”

For more information, go to:http://www.bu.edu/amyloid/files/2013/12/DiflunisalPressRelease.pdf

SOURCE: JAMA, December 2013