Regenerative Hearing Drug Assessed in World-First Trial

UCL and UCLH have successfully completed a world-first trial of a regenerative hearing drug. The results, while not meeting the ambitious targets set by the research team, show promise for the future of hearing loss treatment.

Hearing loss is a significant unmet need, with the most common cause being the progressive loss of inner ear sensory hair cells.

The REGAIN trial, led by Professor Anne GM Schilder, tested the drug gamma secretase inhibitor LY3056480 on 59 patients with mild to moderate hearing loss. While the drug did not restore hearing across the group, deeper analysis of the data showed changes in various hearing tests in some patients, suggesting the drug has some activity in the inner ear.

“There are many important lessons from this study which will guide future studies of its kind.” - Professor Anne GM Schilder

The study highlights the need for better understanding of the mechanisms behind inner ear hearing loss and better hearing tests to identify its causes in patients. Big data and AI may speed up this process.

Participants received three injections of the drug into the ear, through the eardrum, and their hearing was tested with a range of tests before and after receiving the drug.

The UCLH BRC team, together with other BRCs, have set up the NIHR Hearing Health Informatics Collaboration (HIC) that will bring anonymised hearing data together from NHS hospitals across the UK for analysis with advanced computational techniques.

The huge demand among patients to take part in research was revealed by REGAIN, with over 5,000 patients with hearing loss worldwide requesting to take part.

The study paves the way for new hearing therapies, and the UCL Ear Institute researchers were among the first to understand the role of a protein found in inner ear hair cells, which paved the way for new hearing therapies such as the one trialled in REGAIN.

The study was able to go ahead thanks to the support of the NIHR UCLH BRC.