A Big Step Forward for ALS Treatment Research

Published Feb-13-17

A gene therapy approach increased the lifespan of mouse models with a terminal neurodegenerative disease.

Prize4Life, United States

The Story:

A Big Step Forward for ALS Treatment Research Amyotrophic Lateral Sclerosis (ALS) is a neurodegenerative disease that causes the death of nerve cells in the brain and spinal cord. Affected people gradually lose the ability to speak, eat, move and breathe, and approximately 80 percent of sufferers die within five years of diagnosis. It also known as Lou Gehrig’s disease, after the baseball legend was diagnosed with the condition in the 1930s.

Prize4Life, a non-profit organization dedicated to the discovery of treatments and a cure for ALS, launched the ALS Treatment Prize to accelerate research in this area. It was the third open innovation challenge of its original triptych of Prize4Life Challenges aimed at identifying new drug targets, biomarkers and therapies, and computational methods for improving ALS clinical trial design.

Global Pool of Scientists

The open innovation ALS Treatment Prize challenge galvanized scientists around the world who were tasked with finding a treatment candidate that increased lifespan of SOD1 ALS mouse models by at least 25 percent, much more than had ever been done before. SOD1 was the first ALS-causing gene to be identified and it is muted in a small percentage of case.

In early 2017, Prize4Life announced the $1 Million ALS Treatment Prize Winners were Drs. Martine Barkats and Maria-Grazia Biferi, team leaders from the Institute of Myology in Paris, France. They developed a gene therapy approach that shuts down the expression of SOD1. This results in not only in increasing mouse survival by more than 50%, but also improved the motor function of mice.

"We launched this prize with the goal of creating momentum around testing of new treatments for ALS, and are enthusiastic about the potential of the winning team's solution," said Dr. Nicole Szlezak, Chair of Prize4Life's Board of Directors.

"It far exceeds the survival threshold we laid out, and is beyond anything that's been done before in this mouse model. We are hopeful these results will ultimately translate to humans."

Moving Forward

The previous winners of the ALS open innovation challenge were:

In 2011, a $1M ALS Biomarker Prize was awarded to Dr. Seward Rutkove of Beth Israel Deaconess Medical Center in Boston for a technology that measures disease progression of ALS patients and can help reduce Phase II clinical trial results.

In 2012, the DREAM-Phil Bowen ALS Prediction Prize went to two teams for their algorithms to predict disease progression and improve clinical design. A market research company that employed one of the winning teams spin off the company Origent Data Sciences that is now working with partners on clinical trial design.

Commenting on all the prize winners, Prize4Life Founder and Board of Director Avi Kremer, who was diagnosed with ALS in 2004, said:

"It’s exciting to see the progress being made by our prize participants, including winners Drs. Barkats and Biferi, as well as the broader industry, when it comes to finding therapies that can quickly advance to human trials."

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