Academia, Pre-clinical

Ben-Gurion University seeking partner to repurpose old lymphoma drug to treat ALS

Researchers at the Ben-Gurion University of the Negev are seeking collaborators who can help them teach an old lymphoma drug a new trick- to treat ALS.

Rachel Lichenstein, a researcher at Ben-Gurion in Israel had been studying the role of sugar molecules in cancer when a student drew her attention to a lab in a nearby hospital that happened to have 20 tissue samples from ALS patients. So Lichenstein figured she might as well check them out.

“I came by ALS by coincidence,” she explained to Endpoints News, “and Bingo! We found something.”

What she found was a unique sugar molecule in the samples that increases antibody’s ability to bind to immune cells by about 100 times. In the late stages of ALS, these antibodies guide immune cells to attack healthy motor neurons.

So Lichenstein set out to prevent the antibody from binding to the immune cells in the first place.

She started with Rituximab, a monoclonal antibody targeting CD20, that is already approved to treat some lymphomas. Splicing the molecule to remove the portion designed to kill cancer, leaving only the sugar that binds to the antibody, she created a new molecule she believes will block immune cells from binding to the antibody, and thus prevent the cells from attacking motor neurons.

So far, she says a single injection of the drug before the onset of the disease, extended life expectancy in a mouse model of ALS about three weeks. The mice also showed signs that their brain cells were better able to clear debris after the injection — a process that is disrupted in other neurodegenerative diseases as well. Of course, prolonging the life of a mouse a few weeks is a long way from meaningfully extending and improving the lives of humans suffering from ALS, or any other disease.

Still, the team is optimistic. “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,” said Lichenstein in a press release.

When asked if she had pursued a collaboration with the drug’s manufacturer, Lichenstein told me “It is in our plan to contact the manufacturer of Rituximab to ask for partnership.”

Like most neurodegenerative diseases, patient’s options for treatment are limited and underwhelming. There are so few options that when FDA regulators heard about a drug approved in Japan and Korea, edaravone, they reached out to the manufacturer, Mitsubishi Tanabe, and requested that the company file for approval in the United States. It was approved about a month ago. Before that, there was only one other drug for ALS, an antiglutamate called Riluzole, developed by Sanofi and approved in 1995. Both slow the progression of the disease slightly, giving patients a precious few extra months to a couple years of motor control. However, edavarone is administered intravenously, which has raised concerns. It also may come with an unaffordable price tag for many afflicted.

Lichenstein says the main obstacles are, of course, time and money.


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