Navigation Links
Widely-used anti-inflammatory drug shows success in treatment of amyloidosis
Date:12/24/2013

(Boston) A recent study led by researchers from the Amyloidosis Center at Boston University School of Medicine (BUSM) and Boston Medical Center (BMC) demonstrates 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.

This study, published in the Dec. 25 issue of JAMA, is one of the first examples of successful repurposing of a generic drug to treat a rare disease. The National Institutes of Health (NIH) has advocated this research strategy as a way to increase the availability of treatments for rare diseases such as amyloidosis.

John Berk, MD, associate professor of medicine at BUSM and clinical director of the Amyloidosis Center, designed the trial, led the international consortium of researchers, and served as the study's corresponding author. Jeffrey Kelly, PhD, a biochemist at The Scripps Research Institute, and Peter Dyck, MD, a neurologist at Mayo Clinic Rochester, were instrumental in development of the study.

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. Investigators 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.

"Our results show that diflunisal represents an alternative to liver transplantation, the current standard of care for this devastating disease," said Berk. "We hope that this study prompts the identification of other widely-used generic drugs for treatment of rare diseases."

"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 National Institute of Neurological Disorders and Stroke (NINDS).


'/>"/>

Contact: Jenny Eriksen Leary
jenny.eriksen@bmc.org
617-638-6841
Boston University Medical Center
Source:Eurekalert

Related biology news :

1. Oxygen levels increase and decrease the effectiveness of anti-inflammatory therapies
2. Researchers say tart cherries have the highest anti-inflammatory content of any food
3. Study shows Where Alzheimers starts and how it spreads
4. Wayne State cholesterol study shows algal extracts may counter effects of high fat diets
5. Telecoupling science shows Chinas forest sustainability packs global impact
6. H. pylori vaccine shows promise in mouse studies
7. Deepwater Horizon NRDA study shows possible oil impact on dolphins
8. Research shows how household dogs protect against asthma and infection
9. Harvard study shows sprawl threatens water quality, climate protection, and land conservation gains
10. Study shows first link between altitude and concussion
11. International gene therapy trial for bubble boy disease shows promising early results
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:5/16/2016)... , May 16, 2016   EyeLock LLC , ... announced the opening of an IoT Center of Excellence ... and expand the development of embedded iris biometric applications. ... level of convenience and security with unmatched biometric accuracy, ... identity aside from DNA. EyeLock,s platform uses video technology ...
(Date:4/28/2016)... SAN FRANCISCO and BANGALORE, India ... part of EdgeVerve Systems, a product subsidiary of Infosys (NYSE: ... service provider, today announced a global partnership that ... convenient way to use mobile banking and payment services. ... Mobility is a key innovation area for financial services, but ...
(Date:4/19/2016)... 20, 2016 The new GEZE ... compact web-based "all-in-one" system solution for all door components. ... or the door interface with integration authorization management system, ... systems. The minimal dimensions of the access control and ... building installations offer considerable freedom of design with regard ...
Breaking Biology News(10 mins):
(Date:6/27/2016)... ... June 27, 2016 , ... Parallel 6 , the ... today the Clinical Reach Virtual Patient Encounter CONSULT module which enables both ... physician and clinical trial team. , Using the CONSULT module, patients and physicians can ...
(Date:6/27/2016)... ... June 27, 2016 , ... ... will join the faculty of the University of North Carolina Kenan-Flagler Business ... strategy and entrepreneurship at UNC Kenan-Flagler, with a focus on the school’s international ...
(Date:6/27/2016)... --  Ginkgo Bioworks , a leading organism design ... awarded as one of the World Economic Forum,s ... innovative companies. Ginkgo Bioworks is engineering biology to ... in the nutrition, health and consumer goods sectors. ... including Fortune 500 companies to design microbes for ...
(Date:6/24/2016)... SAN DIEGO , June 24, 2016 /PRNewswire/ ... that more sensitively detects cancers susceptible to PARP ... individual circulating tumor cells (CTCs). The new test ... of HRD-targeted therapeutics in multiple cancer types. ... therapies targeting DNA damage response pathways, including PARP, ...
Breaking Biology Technology: