It’s not a cure, but a new therapy for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is being hailed as a breakthrough that can extend the life expectancy of those suffering from ALS.

Developed by researchers at Ben-Gurion University of the Negev (BGU), the therapy may come to market much faster than typical treatments because it is based on an existing FDA-approved drug called MabThera, which is used to treat certain autoimmune diseases and cancers.

Dr. Rachel Lichtenstein of the Avram and Stella Goldstein-Goren Department of Biotechnology Engineering at BGU redesigned a portion of MabThera into a new molecule to treat ALS.

“Since the drug is already approved, we believe that we will need only limited preclinical testing to reach the clinical phase,” Lichtenstein said. The drug has been tested so far with mice, which “showed a significant increase in life expectancy.”

Part of the progression of ALS results from increased activity of glial cells, a type of immune cell that damages and kills the body’s motor neuron cells and decreases their ability to cleanse the central nervous system (CNS). MabThera restores the immune defenses of the central nervous system.

Lichtenstein realized that MabThera could work its magic with ALS, as well. “We found a way to thwart the glial cells from attacking and killing healthy brain cells,” she explained.

Lichtenstein added that the new molecule she designed “meets the conditions for patenting for the treatment of ALS” – a potential commercial boon for BGU. The researchers are seeking a pharmaceutical company partner and recently participated in the 16th National Life Sciences & Technology Week in Tel Aviv.

ALS, a lethal, progressive neurodegenerative disorder that affects the motor nerve cells in the brain and spinal cord, leads to atrophy, paralysis and eventually death due to failure of the respiratory muscles. The only drugs available currently are Reluzole, which helps extend life by only three to six months, and Edaravone, which has demonstrated relatively modest success.

The new Israeli drug candidate could have benefits beyond ALS, said Dr. Ora Horovitz, senior vice president of business development at BGN Technologies, BGU’s technology transfer and commercialization company.

“This could also have major implications on the life expectancy of other neurodegenerative disease patients with Alzheimer’s and Parkinson’s.”

(via Israel21c)

[Photo: John Voo / Flickr ]