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 (CNS) 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. The
progressive degeneration of motor neurons leads to atrophy, paralysis and
eventually death due to failure of the respiratory muscles.
Since the exact cause of
ALS is unknown, current research has focused on extending the post-onset life
expectancy, which is currently between two to 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 CNS environment.
Dr. Rachel Lichtenstein (pictured above center with students in her lab) of the Avram and Stella Goldstein-Goren 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,” says Dr. Lichtenstein.
She successfully
redesigned a portion of MabThera, an FDA-approved drug used to treat certain
autoimmune diseases and types of cancer, into a new molecule to treat ALS.
“Our experimental
results on ALS transgenic mice showed a significant increase in life
expectancy,” says Dr. Lichtenstein. “Since the drug is already approved, we
believe that we will only need limited preclinical testing to reach the
clinical phase earlier than other initiatives.”
"This could also
have major implications on the life expectancy of other neurodegenerative
disease patients with Alzheimer’s and Parkinson’s,” says Dr. Ora Horovitz,
senior vice president of business development at BGN Technologies, BGU’s technology transfer and
commercialization company. “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, there are
only two drugs available for ALS patients: Reluzole (Rilutek), which helps
extend patient survival by only three to six months; and the recently FDA-
approved Edaravone (Radicava), which has demonstrated relatively modest
success.
