As ALS Community Waits for Cure, Scientists Turn to Repurposed Cancer Drug

Scientists redesigned a portion of rituximab into a new molecule to treat amyotrophic lateral sclerosis.

Bringing a new drug to market is expensive and time consuming. The entire process can take approximately 10 years and costs billions of dollars. As such, it is no surprise that many scientists are repurposing drugs already approved by the FDA to treat other conditions.

The latest team to enter the drug repurposing space are scientists from Ben-Gurion University (BGU), who believe the FDA-approved cancer drug rituximab (MabThera) could be modified to treat amyotrophic lateral sclerosis (ALS).

ALS is a neurodegenerative disease that affects the nerve cells in the brain and spinal cord. As the motor neurons die off, the muscles begin to atrophy, resulting in the inability to walk, speak, swallow, and breathe. Currently, there is no cure and the average life expectancy is 2 to 5 years.

Part of the progression of the disease is linked to increased activity of glial cells, which damage and kill motor neurons.

“We found a way to thwart the glial cells from attacking and killing healthy brain cells,” said scientist Rachel Lichtenstein, in a BGU press release.

Increased activity of glial cells has been linked to part of the disease’s progression, and the investigators found a way to thwart the glial cells from attacking and killing brain cells.

Rituximab is indicated to treat chronic lymphocytic leukemia and non-Hodgkin’s lymphoma, as well as rheumatoid arthritis.

The scientists successfully redesigned a portion of rituximab into a new molecule to treat ALS.

In lab studies using mice, the investigators found that the therapy restored the immune cells of the central nervous system, which could potentially help extend survival in ALS patients

“Our experimental results on ALS transgenic mice showed a significant increase in life expectancy,” Dr Lichtenstein said in the release. “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.”

To date, there are only 2 drugs approved by the FDA to treat ALS: riluzole (Rilutek) in 1995, which extends life expectancy by only 3 to 6 months; and edaravone (Radicava), which was approved earlier this month and delays disease progression at early stages.

“This could also have major implications on the life expectancy of other neurodegenerative disease patients with Alzheimer’s and Parkinson’s,” Dr Ora Horovitz, senior vice president of business development at BGN Technologies, said in a release. “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.”