UCLH and UCL successfully complete world-first trial of a regenerative hearing drug

UCLH and UCL researchers have successfully completed a world-first trial of a therapy designed to restore hearing loss.

Results from the REGAIN trial suggest the drug – which has the technical name gamma secretase inhibitor LY3056480 – did not restore hearing across the group of adults with mild to moderate hearing loss from the UK, Germany and Greece.

But deeper analysis of the data showed changes in various hearing tests in some patients, suggesting the drug has some activity in the inner ear. These signals call for further development of LY3056480 using the learnings from this trial.

The results are published in Nature Communications.

The REGAIN trial (Regeneration of inner ear hair cells with gamma secretase inhibitors) was led by Professor Anne GM Schilder (NIHR UCLH Biomedical Research Centre, UCL Ear Institute, UCLH Royal National ENT and Eastman Dental Hospitals). It was the first study of a regenerative hearing drug worldwide, delivered by an EU Consortium with clinical partners in Germany and Greece and the company Audion Therapeutics, and funded by an EU Horizon 2020 grant of €5.8million.

Prof Schilder said: “There are many important lessons from this study which will guide future studies of its kind. For example, the study will help how we best select the patients that may benefit from these new and highly targeted hearing treatments. This requires a 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.”

To progress this work, 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.

And locally, the team have opened a new patient registry called HEDGE, which offers people with hearing loss the opportunity  to participate in research to help uncover the genetic and environmental factors, molecular pathways and mechanisms that cause hearing loss.