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ALS drug breakthrough shows promise in animal studies

ALS Drug
Posted at 5:17 AM, May 31, 2022
and last updated 2022-05-31 08:05:41-04

EVANSTON, IL — Inside the state-of-the-art Richard and Barbara Silverman lab at Northwestern University, scientists have invented a new experimental drug to treat amyotrophic lateral sclerosis — commonly known as Lou Gehrig’s disease.

“All of a sudden, you feel weakness in your arms, in your legs. You can’t swallow, you can't speak and you can't move, and your breathing ability is also reduced," said Hande Ozdinler, an associate professor of neurology at Northwestern University. "After a while, you get stuck in your body.”

The fatal nerve disease has few treatments, with most people only surviving about two to five years after a diagnosis, on average.

“The patient actually knows what's happening to their body every day and sees that it’s getting worse by the day, and there's nothing they can do about it,” said Dr. Richard Silverman, the Patrick G. Ryan AON professor in the department of chemistry, molecular biosciences and pharmacology at Northwestern University.

Silverman, a renowned scientist, came to worldwide prominence after inventing the anti-seizure and pain medication Lyrica, which generated billions of dollars for Pfizer.

“I think NU-9 is his next baby, and I think NU9 is going to be the second Lyrica for neurodegenerative diseases,” Ozdinler said.

Ozdinler said NU-9, now called AKV9, could be more effective than anything else on the market currently used to treat ALS.

“We have, for the first time, a molecule that actually has the ability to improve the health of the diseased motor neurons in many different aspects,” she said.

Ozdinler said while there’s no translation from extending the lifespan of a mouse to that of a human patient, they can find that translation at the cellular level. This is why they focused on improving the health of the upper and lower neurons.

“Once their health is improved, that's going to improve their circuitry, that's going to help their ability to move, and that's how we will eradicate the disease,” she said.

Deteriorating axons contribute to the rapid and fatal paralysis of ALS patients. Scientists say in animal studies that this compound stops degeneration to the point where diseased neurons become similar to healthy control neurons after 60 days of treatment.

“We're hoping that means that it not only maintains it but corrects the degeneration as well,” Silverman said.

AKV9 is now moving toward clinical trials. Silverman’s own company carries out animal safety studies to determine dosage levels and potential toxic effects.

They hope to receive FDA approval as an investigational drug and move into human trials early next year.

“It could be another year after that before patients actually will be able to take it,” Silverman said.

It would provide a new glimmer of hope for those living with this fatal disease, if it gets approved.