Diflunisal, an anti-inflammatory drug, has shown success in treatment of patients with familial transthyretin amyloidosis


A study led by researchers from the Amyloidosis Center at Boston University School of Medicine ( BUSM ) and Boston Medical Center ( BMC ) has demonstrated that Diflunisal, a generic anti-inflammatory drug, successfully reduced neurological decline and preserved the quality of life in patients with familial transthyretin amyloidosis ( ATTR ).
Diflunisal is an inexpensive and safe medication marketed over the past 40 years for arthritis and pain.

The study, published in the JAMA, is one of the first examples of successful repurposing of a generic drug to treat a rare disease.

Familial amyloidosis is a rare inherited disorder in which mutated transthyretin protein aggregates in the blood and forms insoluble fibrils that cause tissue damage. Patients with hereditary ATTR amyloidosis develop debilitating peripheral and autonomic nerve damage, heart disease and weight loss. Untreated, patients die 10-15 years after disease onset.

Researchers at Centers in Sweden, Italy, Japan, England and the United States enrolled 130 patients between 2006 and 2010. Patients were randomized to receive Diflunisal or placebo treatment for two years.

The investigators found that Diflunisal dramatically inhibited the progression of neurologic disease while preserving quality of life when compared to placebo treatment. Known to physicians as Dolobid, generic Diflunisal is inexpensive and readily available.

To date, no other drug treatment has achieved this level of benefit for this rare disorder.

The results have shown that Diflunisal represents an alternative to liver transplantation, the current standard of care for this devastating disease. ( Xagena )

Source: Boston University Medical Center, 2013

XagenaMedicine_2013



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