Biotech, Cures For Hearing Loss, Health Care, Hearing Loss, Journal Reports, Restore Hearing Loss, Reverse Hearing Loss, Akouos, Decibel Therapeutics

Biotech, Cures For Hearing Loss

Cures for Hearing Loss May Be Found in New Drugs

Researchers are trying to counter the biological causes of hearing loss—rather than just treating the effects

16.9.2019

Researchers are trying to counter the biological causes of hearing loss —rather than just treating the effects

Researchers are trying to counter the biological causes of hearing loss —rather than just treating the effects.

“The potential of drug treatments [is] phenomenal,” says Frank Lin, professor of otolaryngology, medicine, mental health and epidemiology at Johns Hopkins University.

One way biotech companies seek to cure hearing loss in children is by using a healthy gene to compensate for a defective gene that causes deafness. Researchers have identified some 150 genes that when mutated lead to hearing loss, says Manny Simons, chief executive of Boston-based Akouos Inc.

Lawrence Lustig, who is the Howard W. Smith professor and chair of the department of otolaryngology, head and neck surgery at Columbia University Medical Center and New York Presbyterian Hospital, says he sees potential in gene therapies such as the one Akouos is working on to offset the effects of defective genes.

The fluid the virus is delivered into comes into contact with sensory hair cells in the inner ear that are required for hearing. The virus infects the hair cells, allowing the healthy gene to enter their nuclei.

Sensory hair cells, equipped with the normal otoferlin gene, can now perform their job of transmitting sound vibrations that they convert into electrical signals to the brain.

A synthetic virus carrying a healthy copy of the otoferlin gene is injected into an ear structure known as the round window.

Sensory hair cells, equipped with the normal otoferlin gene, can now perform their job of transmitting sound vibrations that they convert into electrical signals to the brain.

The fluid the virus is delivered into comes into contact with sensory hair cells in the inner ear that are required for hearing.

Boston-based Decibel Therapeutics Inc. is testing a medicine in clinical trials that may prevent cisplatin-induced hearing loss without disrupting cisplatin’s ability to fight cancer. The company’s drug, for injection into the middle ear before cisplatin treatment begins, is designed to prevent cisplatin from killing vital cells in the inner ear. Decibel, which previously tested the drug in healthy volunteers, is launching its first study of the treatment in adults being treated with cisplatin.

Kay Chang, a professor of otolaryngology-head and neck surgery at Stanford University School of Medicine, says he thinks that the mechanistic approach of Decibel’s drug is sound. But, Dr. Chang adds, delivering the therapy through an injection into the middle ear of children would be challenging to implement because most would be unable to sit still through the procedure. Children would typically need general anesthesia to get the drug before each cycle of cisplatin therapy, which would be impractical, he says.

Kathleen C.M. Campbell, an audiologist and research professor at Southern Illinois University School of Medicine who developed the drug version of d-methionine, says she is preparing the manuscript of the clinical trial for publication and can’t discuss the results right now.

He says it is possible that an effective treatment that regenerates hair cells in humans could be developed, though perhaps not quickly. The science will need to progress faster for that to occur, he says.

Frequency Therapeutics Inc. seeks to spark the creation of new hair cells through a treatment designed to activate progenitors in the inner ear. In April, the Woburn, Mass.-based biotech company said it saw improvements in hearing functions in multiple patients treated in an early-stage clinical trial of its drug. Frequency said in late August that it was preparing to launch midstage clinical research of the treatment, but it declined to comment for this article.

“If these treatments are effective they’re going to radically change the way we provide hearing care,” Dr. Schilder says.

Mr. Gormley is a reporter for The Wall Street Journal in Boston. He can be reached at brian.gormley@wsj.com.

Read more: The Wall Street Journal

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