New drug cocktail from Harvard Medical School could potentially treat hearing loss

Researchers at Harvard Medical School have developed a new drug cocktail that could help regenerate hair cells in the inner ear and can cure hearing loss, according to a paper published in Proceedings of the National Academy of Sciences. 

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The team reprogrammed genetic pathways within the inner ear of mice to regenerate hair cells using a cocktail of molecules, and are now exploring future clinical applications for humans suffering from hearing loss. The research is expected to pave the way for gene therapy clinical trials that could one day be used to treat patients with hearing loss. The researchers hope that by combining a surgical procedure with a refined gene therapy delivery method, they can bring this treatment to patients as soon as possible.

Hair cells in the inner ear act like biological microphones, responsible for passing sound signals from the inner ear to the brain. Humans do not have the ability to regenerate hair cells, unlike certain species of fish, birds, and reptiles. Hearing loss affects about 48 million Americans and 430 million people worldwide, with more than 90% of individuals affected having sensorineural hearing loss caused by damage to the inner ear and the destruction of hair cells responsible for relaying sounds to the brain.

In a previous study, the team was able to make other types of cells divide and develop hair cell characteristics in mice, and this latest research builds on that success by activating the same pathways with the use of newly developed chemical compounds. Using molecules called “small interfering RNAs” (siRNAs), the team was able to remove genes that suppressed the activation of a genetic pathway that allows for the growth of hair cells in the inner ear.

“These findings are extremely exciting because throughout the history of the hearing loss field, the ability to regenerate hair cells in an inner ear has been the holy grail,” said Zheng-Yi Chen, an HMS associate professor of otolaryngology and associate scientist in the Eaton-Peabody Laboratories at Mass Eye and Ear. “We now have a drug-like cocktail that shows the feasibility of an approach that we can explore for future clinical applications.”

The researchers plan to test the treatment on larger animals before moving on to human clinical trials, but if successful, the research could have a profound impact on individuals who have lost part of their hearing. With hearing loss becoming more prevalent as populations age, this research has the potential to improve the quality of life for millions of people worldwide.

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