Akouos, Inc announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) issued a positive opinion on the company’s application for orphan drug designation for AK-OTOF, a gene therapy intended for the treatment of otoferlin gene-mediated hearing loss. The positive opinion was subsequently adopted by the European Commission.
This study suggests that not properly treating infections or other conditions that chronically affect the middle ear may lead to neural deficits and increased difficulties hearing in noisy environments.
In the future, scientists may be able to use the data to steer stem cells toward the hair cell lineage, helping to produce the specialized cells they need to test cell replacement approaches for reversing some forms of hearing loss.
Researchers examined 120 inner ears collected at autopsy. They used multivariable statistical regression to compare data on the survival of hair cells, nerve fibers, and the stria vascularis with the patients' audiograms to uncover the main predictor of the hearing loss in this aging population. They found that the degree and location of hair cell death predicted the severity and pattern of the hearing loss, while stria vascularis damage did not.
Researchers looked at mice engineered to have progressive hearing loss, and found that their neurotransmitter receptors—responsible for communication between brain cells—exhibited changes in sensory processing regions related to memory.