FDA launches 5-year initiative for rare neurodegenerative diseases 

FDA has unveiled its Action Plan for Rare Neurodegenerative Diseases, including amyotrophic lateral sclerosis (ALS).

The plan will involve the use of public-private partnerships and incorporate feedback from patients. The initiative will encompass regulatory science initiatives, changes to existing programs and new policy initiatives.

Milestones of the initiative include the creation of an FDA rare neurodegenerative diseases task force and public-private partnerships for rare neurodegenerative diseases in the fiscal year 2022. In addition, from fiscal years 2022 to 2026, the plan aims to develop disease-specific science strategies.

The initiative is an outgrowth of a Public Law 117-79, the “Accelerating Access to Critical Therapies for ALS Act” (ACT for ALS) President Biden signed into law on December 23, 2021.

“We recognize the urgent need for new treatments that can both improve and extend the lives of people diagnosed with these diseases,” said…

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Scientists find new potential drug targets for neurodegenerative diseases

Maria Clara “Maca” Franco, center, with Kyle Nguyen, left, and Lydia Bastian. Franco’s research investigates neurodegenerative diseases. Image courtesy of Oregon State University.

Researchers at Oregon State University (OSU) have discovered a new class of potential drug targets for diseases like Alzheimer’s, Parkinson’s and amyotrophic lateral sclerosis (ALS).

The scientists are working to identify the best method to attack the targets — oxidized proteins. The most potent oxidant of the bunch is peroxynitrite, which is produced in conditions involving inflammation. Oxidized proteins and free radicals can damage DNA, lipids and proteins implicated in neurodegenerative diseases and other conditions.

Peroxynitrite is produced thanks to the diffusion-limited reaction of nitric oxide and superoxide.

Peroxynitrite appears to be especially pernicious when it oxidizes heat shock prote…

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What is needed to develop disease-modifying therapies for Parkinson’s and Alzheimer’s 

Photo by Edward Jenner from Pexels

Developing disease-modifying therapies for neurodegenerative diseases remains a pressing need.

The incidence of neurodegenerative disease is ramping up in the U.S. and elsewhere as much of the global population ages. One out of three seniors dies with Alzheimer’s disease or another form of dementia, according to the Alzheimer’s Association.

Parkinson’s disease is also becoming more widespread. Between 2015 and 2040, the number of people with Parkinson’s could nearly triple, rising from 6.3 to 17.5 million.

But developing drugs that can slow or stop the progression of such diseases poses a significant challenge for drug developers. Eli Lilly’s donanemab, for instance, showed promise earlier this year in treating Alzheimer’s in a Phase 2 study summarized in NEJM.

Another Alheimer’s candidate, aducanumab from Biogen, has also shown promise, although late l…

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