French scientists win $1 m. for results of treatment on mice with ALS

The successful results, in which the rodents’ lives were extended by more than 50%, surprised even the researchers.

By JUDY SIEGEL-ITZKOVICH
Updated: FEBRUARY 2, 2017 23:17
A mouse [illustrative]. (photo credit: INGIMAGE)
A mouse [illustrative].
(photo credit: INGIMAGE)
A genetic treatment developed by French scientists for amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease, slows the advancement of the incurable neurological disease and has been called a “breakthrough” by Prize4Life, an organization founded in 2006 by a group of Harvard Business School students when one of them – Israeli Avi Kremer – was diagnosed with ALS.
Kremer, who was 29 years old when he was diagnosed with the disease, is still active in the organization, which is dedicated to accelerating research into the disease.
An ALS Treatment Prize of $1 million, named in honor of Kremer, will be awarded in April to the team of Dr.
Martine Barkats and Dr. Maria-Grazia Biferi of the Myologie Institute in Paris for their development of a gene therapy approach targeted at one of the most common known causes of inherited ALS.
This approach, tested pre-clinically on lab mice, significantly extends survival and slows disease progression beyond any results reported to date.
The treatment involves the “silencing” of a gene named SOD1. The experiment was repeated and proved successful when carried out at the ALS Therapy Development Institute in Cambridge, Massachusetts, according to Prize4Life.
The successful results, in which the rodents’ lives were extended by more than 50%, surprised even the researchers.
The treatment also significantly improved the motor functioning of the mice even after their ALS symptoms appeared.
The prize recipients said they were excited and satisfied by the promising results of their technique. “We are proud that we have helped speed up the way to developing a treatment using our new technique for dealing with the SOD1 gene,” they said, adding that they “look forward to its use in clinical trials.”