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Long-awaited new ALS drug shows promise at UT Health San Antonio

Experimental pill promises extended life for ALS patients

SAN ANTONIO – With so many troubling health concerns during the coronavirus pandemic, UT Health San Antonio has some promising news for those with ALS.

For the first time in years, a new drug appears to have a positive effect on those with amyotrophic lateral sclerosis, for which there is no cure.

Researchers have released the results of a small clinical trial for a drug combination that looks to extend the life of a patient, and perhaps just as important, help them retain motor function longer.

Dr. Carlayne Jackson is an ALS specialist and professor of neurology at UT Health San Antonio. She co-authored the Centaur study, which examined the effect of phenylbutyrate-taurursodiol. It’s a combination of two drugs, which seemed to magnify the efficacy that each held in earlier testing when used singularly.

Many ALS patients die within two to three years and suffer from a rapid decline in motor function, and currently there are only two other drugs approved by the FDA to treat it.

Dr. Jackson explained of the new drug trial, “That difference is like the difference between being able to walk with a walker and not walk at all or being able to feed yourself versus having a feeding tube.”

Her study published in the New England Journal of Medicine last week shows the Centaur study pill slowed the progression of the disease in a measurable fashion on the motor function scale used for the disease. Five of the patients used in the phase two clinical trial were from UT Health San Antonio.

“It did show that it actually improves survival as well as slow the decline in function. So it did both,” said Jackson. It’s no small feat considering there’s been little progress in this area for years.

Jackson notes if you were part of the ALS Ice Bucket Challenge, you too can help take some credit for this new one. “I do want to shout out to everyone who participated in the Ice Bucket Challenge because that money helped fund this trial, so everyone who did that should feel proud.”

The phenylbutyrate-taurursodiol combo still must advance now to a larger phase three clinical trial before the FDA will consider it for approval.

A full report on the experimental drug treatment can be found at here.

One other positive finding according to Jackson is that during Covid-19′s use of telemedicine, the most vulnerable ALS patients managed to remain in better contact with their doctors.

She says even hospice patients were able to maintain their connection to their doctors, something that will be utilized going forward.

RELATED: Higher rates of ALS found in war veterans


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