Gene therapy for a rare type of deafness shows lasting results

Health Gene therapy for a rare type of deafness shows lasting results April 22, 202611:00 AM ET Rob Stein Dr. Yilai Shu examines a young patient at the Eye & ENT Hospital of Fudan University in China. Partners HealthCare hide caption toggle caption Partners HealthCare An experimental gene therapy appears safe and highly effective for restoring hearing to people born with a rare form of deafness, researchers reported Wednesday. Shots - Health News Gene therapy shows promise for an inherited form of deafness The study, the largest and longest to date to evaluate a gene therapy for hearing loss, provides powerful new evidence that the approach may provide the first way to restore hearing to people who are deaf. "The results are really remarkable," Zheng-Yi Chen, an associate scientist at Mass Eye and Ear in Boston who led the study, published in the journal Nature. "This is really for the first time in the whole field a brand-new treatment option for genetic hearing loss. So that's very exciting." Sponsor Message The results, which confirm and extend a smaller study published two years ago, are consistent with those produced by several other research groups testing similar gene therapies for several forms of genetic deafness. In fact, a treatment developed by Regeneron Pharmaceuticals could soon become the first gene therapy for deafness to win approval from the Food and Drug Administration. Scientists are encouraged by treatment progress The flurry of advances is spurring increased interest in screening more babies for genetic deafness so they can be treated as young as possible. Early intervention appears to produce the best results. "Being able to restore natural hearing, I think, is a game changer for our field," said Dr. Lawrence Lustig, who chairs the Department of Otolaryngology-Head & Neck Surgery at Columbia University's medical school. While the form of deafness treated in the new study is very rare, affecting about 50 babies born each year in the U.S., the s...