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



Indietro

Altri articoli

Farxiga ( Dapagliflozin ), a sodium-glucose cotransporter 2 ( SGLT2 ) inhibitor, has been approved in the US to reduce...


Mineralocorticoid receptor antagonists ( MRAs ) have been demonstrated to improve outcomes in reduced ejection fraction heart failure ( HFrEF...


The European Commission ( EC ) has approved Kymriah ( Tisagenlecleucel, formerly CTL019 ). The approved indications are for the...


Results from the phase 2b ixCELL-DCM clinical study of Ixmyelocel-T in patients with advanced heart failure due to ischemic dilated...


A study has examined the correlation between pre-operative coronary artery disease ( CAD ) and post-operative cardiovascular events in lung...


The European Commission ( EC ) has approved Revolade ( Eltrombopag ) for the treatment of adult patients with severe...


Revolade ( Eltrombopag ) is used in adults for the treatment of: • long-term immune thrombocytopenic purpura ( ITP ), a...


Clinical trials in heart failure have focused on the improvement in symptoms or decreases in the risk of death and...


The BENEFIT trial has shown that a 40 to 80 day treatment with the antiparasitic medication Benznidazole ( Rochagan,...


The 2015 ESC/ERS Guidelines for the diagnosis and treatment of pulmonary hypertension are published in the European Heart Journal and...