World-first trial of regenerative hearing drug is successfully completed

Researchers at UCL and UCLH have successfully completed the first trial of a therapy designed to restore hearing loss. The REGAIN trial, the results of which were published in Nature Communications , was the first study of a treatment aimed at restoring lost hearing, focusing on a drug with the technical name gamma secretase inhibitor LY3056480. The researchers found that while the therapy did not restore hearing across the group of adults with mild to moderate hearing loss, a 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 so-called efficacy signals call for further development of LY3056480 - using the learnings from this trial. Trial participants were aged between 18 and 80, hailed from the UK, Germany and Greece, and had mild to moderate hearing loss. 15 patients took part in the phase 1 trial, which showed the treatment was safe and well tolerated, and 44 patients took part in the phase 2a trial designed to establish if the drug worked. Participants received three injections of the drug into the ear, through the eardrum.