Ben-Gurion University of the Negev (BGU) researchers are developing a new therapy for Amyotrophic Lateral Sclerosis (ALS), using part of an existing FDA-approved drug that restores the central nervous system’s immune defenses and increases life expectancy.
ALS, also known as Lou Gehrig’s disease, is a lethal, progressive neurodegenerative disorder that affects the motor nerve cells in both the brain and the spinal cord, which leads to atrophy, paralysis and eventually death due to failure of the respiratory muscles.
Because the exact cause is unknown, current research has focused on extending the post-onset life expectancy, now between two and five years for most patients.
Part of the disease’s progression is linked to 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 environment. Dr. Rachel Lichtenstein of the Department of Biotechnology Engineering at BGU has focused on reducing this negative immune response.
“We found a way to thwart the glial cells from attacking and killing healthy brain cells,” she says. “Our experimental results on ALS transgenic mice showed a significant increase in life expectancy.”
She redesigned a portion of an already FDA-approved drug, which means they will only need limited preclinical testing to reach the clinical phase.
Dr. Ora Horovitz, senior vice president of business development at BGN Technologies, BGU’s technology transfer and commercialization company, said, “This could also have major implications on the life expectancy of other neurodegenerative disease patients with Alzheimer’s and Parkinson’s. Our new drug candidate may prove effective in boosting the self-cleansing mechanism of the human brain, thereby improving the lives of millions of people.”
The researchers are now seeking a pharmaceutical company partner.
Currently, two drugs are available for ALS patients, one which extends survival by only three to six months, and another new drug, which shows relatively modest success.