Potential ALS Therapy Based on Cancer Treatment Rituximab Shows Promise

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by Alice Melão |

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Researchers at Ben-Gurion University (BGU) believe that an existing FDA-approved anti-cancer drug, rituximab, could be modified and used to treat amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.

In lab studies with mice, the therapy restored resident immune cells of the central nervous system, which could indicate the potential to increase the life expectancy of ALS patients.

Scientists have not yet found the exact cause of ALS, and experts have focused on developing therapies to reduce symptoms and disease progression. The goal of these therapies is to extend post-onset life expectancy.

Only two drugs have been approved as ALS treatments. Rilutek (riluzole) was approved in 1995 and has helped extend life expectancy by only three to six months.

Twenty-two years later, the FDA approved Radicava (edaravone), which delays disease progression at early stages. Despite these therapeutic advances, more efficient treatments are needed.

Rachel Lichtenstein, PhD, and a team from BGU’s Avram and Stella Goldstein-Goren Department of Biotechnology Engineering have designed a new compound based on the structure of FDA-approved rituximab, whose brand name MabThera is used in treating chronic lymphocytic leukemia and non-Hodgkin’s lymphoma. More recently, its efficacy against autoimmune diseases such as rheumatoid arthritis was discovered.

Rituximab is a monoclonal antibody that was developed to deplete immune B cells in circulation. Taking advantage of its immunomodulatory effect, Lichtenstein hopes to reduce negative immune responses that lead to neuronal death in ALS. This new therapeutic approach could aid the nervous system by improving its tasks of surveillance and removal of dead cells and wastes that can be harmful.

“We found a way to thwart the glial cells from attacking and killing healthy brain cells,” Lichtenstein said in a news release. “Our experimental results on ALS transgenic mice showed a significant increase in life expectancy,” she added.

Because this investigational drug is based on an already approved drug with proven safety, the research team believes it will require fewer preclinical studies to reach the clinical development stage. This could significantly reduce the time it takes to develop the therapy. The researchers are now seeking a pharmaceutical company to partner with to proceed with further studies.

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