Diflunisal shows efficacy in treating Amyloidosis

by Bruce Sylvester – Diflunisal, a generic anti-inflammatory drug, appears to reduce neurological decline and preserve quality-of-life in patients with familial transthyretin amyloidosis (ATTR).

Researchers reported the findings in the Dec. 25 issue of JAMA.

“Our results show that diflunisal represents an alternative to liver transplantation, the current standard of care for this devastating disease,” said John Berk, MD, associate professor of medicine at Boston University School of Medicine and clinical director of its Amyloidosis Center, who designed the trial and was its lead author.  “We hope that this study prompts the identification of other widely-used generic drugs for treatment of rare diseases.”

As background, the authors noted that amyloidosis is a rare inherited disorder in which mutated transthyretin protein aggregates in the blood and forms insoluble fibrils, causing tissue damage. Patients develop peripheral and autonomic nerve damage, heart disease and weight loss. Untreated, a patient will die 10-15 years after disease onset.

Investigators  in Sweden, Italy, Japan, England and the United States enrolled 130 patients between 2006 and 2010. The subjects were randomized to two years of treatment with diflunisal or placebo.

The investigators reported that diflunisal treatment dramatically inhibited the progression of neurologic disease and it also preserved quality of life, compared to placebo treatment.

The authors noted that amyloidosis patients have achieved this level of therapeutic benefit with no other drug.

“We are pleased and optimistic about the results of this study, and are encouraged about the potential for repurposing of generic medication to bring treatments to rare and more common diseases,” said Robin Conwit, MD, a Program Director with the (US) National Institute of Neurological Disorders and Stroke (NINDS).