NIH announces new Transformative Research Award program for ALS

New initiative encourages innovative research on devastating neurological disease.

The National Institutes of Health plans to invest $25 million over five years in a new program to spur innovative research on amyotrophic lateral sclerosis (ALS), a progressive and fatal neurological disease that weakens and eventually paralyzes voluntary muscles. Accelerating Leading-edge Science in ALS (ALS2) aims to answer critical questions about this disease.

“Over the past few decades, there has been significant progress in our understanding of ALS, but we still do not have any breakthrough treatments for this terrible disease,” said NIH Director Francis S. Collins, M.D., Ph.D. “We hope this program will inject new ideas to the field to push us rapidly toward effective therapies.”

Sometimes known as Lou Gehrig’s disease, ALS is a neurodegenerative disease that affects motor neurons in the spinal cord that control voluntary muscles. For reasons that are currently unknown, the motor neurons die off, resulting in progressive muscle weakness that usually leads to respiratory failure. ALS is virtually always fatal, and many people die within three to five years of developing symptoms.

There is no cure for ALS. The U.S. Food and Drug Administration-approved drugs riluzole and edaravone can prolong life by a few months but do not improve symptoms. Ongoing clinical trials are looking at a range of therapeutic strategies including new drug candidates, devices to improve quality of life, and stem cell therapies. Genetic research has recently provided clues to understanding the disease, but it will take a focused investment in research from a range of scientific disciplines in order to capitalize on these insights.

ALS2 will be part of the NIH Common Fund’s High-Risk, High-Reward (HRHR) research program. The four initiatives of the HRHR program are a time-tested, powerful approach to sparking innovation. They support exceptionally creative research, which is inherently riskier, but has the potential to transform biomedical science. NIH will apply this potential to ALS2 through the HRHR program’s Transformative Research Award initiative. The Transformative Research Award is particularly well-suited to interdisciplinary teams of scientists looking to combine their expertise and pursue new ideas with the potential to transform ALS research.

ALS2 will take a three-pronged approach to improve understanding of ALS:

Adapt emerging tools and technologies from neuroscience and cell biology to identify what causes ALS at the molecular level, and how the disease progresses, leading to new therapeutic strategies.
Attract new talent from a range of scientific disciplines, including neuroscience, cell biology, bioengineering, genomics, chemistry, biophysics, environmental health sciences, and computational science.
Examine biological similarities between ALS and motor neuron disease in other neurodegenerative disorders, including frontotemporal dementia, chronic traumatic encephalopathy, Kennedy’s disease, spinal muscular atrophy, and primary lateral sclerosis.

Information on a Notice of Special Interest to accompany the Transformative Research Award funding opportunity will be posted here: https://commonfund.nih.gov/tra/grants.

This initiative is managed collaboratively by the NIH Common Fund, the National Institute of Neurological Disorders and Stroke, the National Institute on Aging, the National Institute of Environmental Health Sciences, and the National Institute of General Medical Sciences.

About the National Institutes of Health (NIH): NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit www.nih.gov.

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