The past few months have brought electrifying news that, for the first time, a gene treatment has provided some hearing to children born with deafness.
Eli Lilly announced this week, for example, that a profoundly deaf boy from Morocco given its treatment as part of a clinical trial in Philadelphia can now hear. And five children in China treated similarly at younger ages gained hearing with some able to verbally communicate without their cochlear implants(人工耳蜗). Their hearing recovery, first covered by the press in October 2023, is described in de tail this week in The Lancet.
"It's an enormous achievement," says geneticist Karen Avraham of Tel Aviv University. Otolaryngologist(耳鼻喉科专家) and gene therapist Lawrence Lustig of Columbia University, whose lab was among the first to test the same approach in mice, agrees. "Other than cochlear implants, we haven't really had any successful treatment s to treat deafness," he notes.
The various efforts from companies and academic centers each use a virus to insert the same gene, OTOF, into the children's inner ear so the so-called hair cells there can sense sound and transmit it to the brain.
The new deafness treatments add to a string of recent successes for the gene treatment field, but also raise questions. The ear's hair cells don't divide, so the new copies of OTOF they contain should persist and continue to instruct the cells to make OTOF. Gene expression could drop off over time or the ear could mount an immune response that shuts it off.
But Lustig is optimistic that the various challenges will be overcome. "Now that we've got one success story, there's going to be more money coming in to fund some of these other projects," he says.