New trials on gene therapy to restore hearing
Wired.com reported that two companies have launched clinical trials to see if they can restore hearing to children who have a rare type of genetic deafness.
The companies are Akous and Decibel Therapeutics and both are based in Boston.
The article said the trials focus on a gene called OTOF. Those who have alterations in this gene lose a key protein needed for hearing called otoferlin.
A top official with Decibel Therapeutics, Vassili Valayannopoulos, explained that there are thousands of sensory hair cells in the inner ear. When sound waves hit these cells, they vibrate and release a chemical messenger called a neurotransmitter that initiates an electrical signal in the auditory nerve that is sent to the part of the brain that interprets sound. The protein otoferlin is key to allowing the neurotransmitter to work. Those who have a mutation in the OTOF gene are unable to hear because the otoferlin protein isn’t working properly. Scientists are going to try to deliver working copies of the OTOF gene to hair cells through a small incision behind the ear and infusing it into the inner ear.
There are about 20,000 people in the U.S., U.K., Germany, France, Spain, and Italy who have genetic deafness due to issues with the OTOF gene.
Decibel Therapeutics plans to enroll up to 22 children in the U.S., U.K., and Spain for the trials and follow them for five years. Akouos plans to have 14 participating in the U.S. and Taiwan.
Wired.com said this gene therapy has worked in experiments with deaf mice. There was a previous trial in 2014 by the company Novartis that tried to regrow damaged hair cells but it didn’t work out.
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Wired.com did interview two deaf people to get their insights on gene therapy for deaf people. One of them, Jaipreet Virdi, said this is an extension of the debate that began in the 1990s over cochlear implants. She said it is problematic because deaf children, as individuals, may be denied a chance to access Deaf culture. The second deaf person is Wyatte Hall, who said “As long as deaf people have been around on the earth, people have always been trying to fix us.” Hall said he’s not opposed to cochlear implants or gene therapy but does not agree with parents limiting access to sign language in favor of a medical-only approach. He encourages parents, if they want to take a medical approach, to use both technology and sign language.
https://www.wired.com/story/new-trials-aim-to-restore-hearing-in-deaf-children-with-gene-therapy/