Researchers to accelerate search for ALS therapies

LOS ANGELES, Oct. 23, 2024 /PRNewswire/ -- The ALS Network, formerly ALS Golden West, has awarded a $125,000 grant through its global research program to AUTTX, LLC. to help develop novel therapeutics for people living with ALS.

Led by Isabelle Draper, Ph.D. and Alan S. Kopin, M.D., the project will study the abnormal processing of RNA molecules which provide instruction to neurons - a hallmark of amyotrophic lateral sclerosis (ALS).

"Our work is critical in helping expedite the development of targeted drug therapies that can reverse abnormal molecule processing," Dr. Kopin said. "We are grateful to the funders and supporting community that include the ALS Network, Dr. Richard Smith, Dr. Clotilde Lagier-Tourenne, and a number of academic institutions and foundations."

The ALS Network's grant will allow AUTTX to build on research that has identified a mechanism that can reverse abnormal RNA processing. AUTTX already discovered ASOs that stimulate this repair mechanism. To expedite the development of drugs targeting this pathway, AUTTX will screen a selected library of small molecules. Such compounds will shortcut the development process leading to novel therapeutics for people living with ALS. 

The ALS Network's multifaceted research program and partnering scientists seek to yield significant results in a disease category historically void of positive outcomes. The ALS Network's Scientific Advisory Committee, composed of world-renowned researchers, scientists, and healthcare industry leaders, reviews and selects the most promising projects and moonshot efforts for funding. 

"The ALS Network's research-funding model is an ambitious program driven by urgency and innovation," said Sheri Strahl, MPH, MBA, president and CEO of the ALS Network. "We seek to propel promising science forward quickly with a relentless focus on improving and saving lives. AUTTX's project will move us closer to the discovery of new therapies for ALS and other motor neuron diseases."

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's Disease, is a fatal, neurodegenerative illness that affects nerve cells in the brain and the spinal cord. People with ALS lose the ability to move, speak, swallow, and, eventually, to breathe. People who have served in any branch of the military are diagnosed with ALS nearly twice as often as the general population. Currently, there are no known cures for ALS.

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SOURCE ALS Network