Nonsteroidal anti-inflammatory agent slows rate of progression of neurodegenerative disease

December 24, 2013

Among patients with familial amyloid polyneuropathy, a lethal, genetic neurodegenerative disease, use of the nonsteroidal anti-inflammatory agent diflunisal compared with placebo for 2 years reduced the rate of progression in neurological impairment and preserved quality of life, according to a study appearing in the December 25 issue of JAMA.

Familial amyloid polyneuropathy is characterized by progressive neurologic deficits and disability which prove fatal if left untreated. Fewer than 10,000 people are estimated to be clinically affected worldwide, according to background information in the article. Diflunisal showed potential benefit in a phase 1 study.

John L. Berk, M.D., of the Boston University School of Medicine, and colleagues, pursuing the NIH mission to repurpose old drugs, randomized 130 patients from Sweden, Italy, Japan, England, and the United States to receive diflunisal (n=64) or placebo (n=66) twice daily for 2 years to determine the effect of diflunisal on polyneuropathy progression in patients with familial amyloid polyneuropathy.

Patients randomized to diflunisal exhibited less progression of polyneuropathy than those assigned to placebo. The inhibitory effect of diflunisal on neuropathy progression was detectable at 1 and 2 years, and at 2 years, 29.7 percent of the diflunisal group compared to 9.4 percent of the placebo group exhibited neurological stability. Physical quality of life stabilized from the beginning of the study to 2 years in those assigned to diflunisal treatment while decreasing in the placebo group.

The authors write that their trial is pivotal for several reasons: it is the first randomized trial involving a broad cross-section of the spectrum of disease; it provides invaluable natural history data on the rate of neurological disease progression; and it establishes that diflunisal, a low-cost treatment, is well tolerated by familial amyloid polyneuropathy patients with a spectrum of neuropathy.

"Although longer-term follow-up studies are needed, these findings suggest benefit of this treatment for familial amyloid polyneuropathy."
-end-
(doi:10.l001/jama.2013.283815; Available pre-embargo to the media at http://media.jamanetwork.com)

Editor's Note: Please see the article for additional information, including other authors, author contributions and affiliations, financial disclosures, funding and support, etc.

The JAMA Network Journals

Related Placebo Articles from Brightsurf:

Effect of fluvoxamine vs placebo on clinical deterioration in outpatients with symptomatic COVID-19
This randomized trial compares the effects of fluvoxamine, a selective serotonin reuptake inhibitor with immunomodulatory effects, versus placebo on a composite of dyspnea or pneumonia and oxygen desaturation among adult outpatients with polymerase chain reaction-confirmed mild COVID-19 illness.

Hydroxychloroquine no more effective than placebo in preventing COVID-19
Clinical trial with COVID-19 testing of participants shows health care workers in contact with coronavirus patients who took hydroxychloroquine each day did not reduce their rate of infection.

Compared to placebo, vitamin D has no benefit for severe asthma attacks
Contrary to earlier observational results, vitamin D supplements do not prevent severe asthma attacks in at-risk children, according to the first placebo-controlled clinical trial to test this relationship.

UMN trial shows hydroxychloroquine has no benefit over placebo in preventing COVID-19
Today, University of Minnesota Medical School researchers published the results from the first randomized clinical trial testing hydroxychloroquine for the post-exposure prevention of COVID-19.

The placebo effect and psychedelic drugs: tripping on nothing?
A new study from McGill suggests that, in the right context, some people may experience psychedelic-like effects from placebos alone.

Methotrexate reduces joint damage progression over placebo in erosive hand OA
According to new research findings presented at the 2019 ACR/ARP Annual Meeting, methotrexate did not demonstrate superior efficacy over placebo for pain relief and function evolution at three and 12 months in patients with erosive hand osteoarthritis, but did significantly reduce the progression of joint damage over placebo and seems to facilitate bone remodeling in these patients.

Botulinum toxin reduces chronic migraine attacks, compared to placebo
A growing body of evidence supports the effectiveness of botulinum toxin injections in reducing the frequency of chronic migraine headaches, concludes an updated review and analysis in the January issue of Plastic and Reconstructive Surgery, the official medical journal of the American Society of Plastic Surgeons (ASPS).

Opioids vs. placebo, nonopioid alternatives for chronic noncancer pain
An estimated 50 million adults in the United States were living with chronic noncancer pain in 2016 and many of them were prescribed opioid medications, even though a clinical benefit is uncertain.

Probiotic no better than placebo for acute gastroenteritis in children
While probiotics are often used to treat acute gastroenteritis (also known as infectious diarrhea) in children, the latest evidence shows no significant differences in outcomes, compared to a placebo.

Most common shoulder operation is no more beneficial than placebo surgery
In a landmark study published this week in the BMJ, Finnish researchers show that one of the most common surgical procedures in the Western world is probably unnecessary.

Read More: Placebo News and Placebo Current Events
Brightsurf.com is a participant in the Amazon Services LLC Associates Program, an affiliate advertising program designed to provide a means for sites to earn advertising fees by advertising and linking to Amazon.com.