New hope for Tay-Sachs disease at UMass Medical School


A new Voices of UMassMed podcast highlights the progress being made at UMass Medical School in developing gene therapy for Tay-Sachs disease. Tay-Sachs is a rare, genetic degenerative disease that destroys nerve cells in the brain and spinal cord. Few children with Tay-Sachs will reach their fifth birthday. There is no cure.

Miguel Sena-Esteves, PhD, associate professor of neurology, has been a leading force behind more than a decade of collaborative research toward better understanding Tay-Sachs disease with an eye toward finding a treatment using gene therapy.

“Now, I think there is a good chance that what we’re doing today can have an impact. Can it be therapeutic? We don’t know,” said Dr. Esteves, member of the Horae Gene Therapy Center, and the prinicipal investigator of the Tay-Sachs gene therapy program.

Heather Gray-Edwards, PhD, DVM, assistant professor of radiology, is collaborating with Sena-Esteves, and shared an update on her research.

“I agree with what Miguel said 100 percent,” Dr. Gray-Edwards said. “I don’t think we’ll stop doing it until there is a cure.”

The podcast details the early results of a trial of the new gene therapy on young patients under an “expanded access” protocol approved by the U.S. Food and Drug Administration.

Listen to Voices of UMassMed at: You can also subscribe and listen on SoundCloud and iTunes.

The Tay-Sachs research is featured in the new edition of the @umassmed magazine, which can be read online here.