"Hearing loss is a chronic non-communicable disease disabling over 328 million adults, and 32 million children worldwide. Untreated hearing loss currently costs Europe €213 billion each year. Hearing loss in children and youngsters hinders language learning and cognitive development, acquired hearing loss in adults impairs social integration and participation, and in the elderly, (progressive) hearing loss accelerates the progression of cognitive impairment (every 20 dB of hearing loss is associated with a loss of 7 cognitive years). The treatment of hearing loss is currently limited to the use of hearing aids or cochlear implants. These devices often perform poorly in noisy environments and can be very costly.
Damage to the hair cells in the cochlea ""sensorineural hearing loss"" (SNHL) is a major cause of acquired hearing loss. Since hair cells do not regenerate spontaneously, the dogma has been that SNHL is irreversible. No pharmacological treatment for SNHL currently exists. Studies in animal models have, however, shown that new hair cells can be generated through local treatment with a drug product and that this leads to an improvement in hearing capacity. The REGAIN (REgeneration of inner ear hair cells with GAmma-secretase INhibitors) study aims to develop a locally delivered small-molecule drug to treat hearing loss caused by the loss of sensory hair cells in the inner ear. The REGAIN Consortium is in the unique position to take the next crucial step in translation of these findings to the clinic, by providing clinical validation of a highly potent gamma-secretase inhibitor (GSI) as a treatment of SNHL.
The objective of the REGAIN project is to establish the efficacy of a locally administered GSI in terms of improving hearing through regeneration of inner ear hair cells. The project involves 1) upscaling of GMP production of the clinical GSI candidate, 2) generation of preclinical data on GSI dosing and local safety, 3) ethics approval for the clinical trial l and 4) demonstration of proof of concept for the GSI for the treatment of patients with adult onset SNHL. Small molecule drugs targeting the underlying biological causes of hearing loss in a safe way are expected to meet an urgent clinical need for millions of patients, who currently rely on the limited benefits provided by hearing aids and cochlear implants.
The REGAIN Consortium is uniquely skilled and placed to advance this proof of concept to and through clinical studies, and brings together eight dedicated partners, coordinated by Audion Therapeutics BV and including University College London Ear Institute, University College London Hospitals, University of Tübingen, The National and Kapodistrian University of Athens, Eli Lilly, Nordic Biosciences and ttopstart BV). These Consortium partners represent the current state of the art in regenerative hearing loss research in the EU and bring together expertise in hearing loss biology and GSI’s with a deep understanding of preclinical and clinical drug development. The REGAIN project is funded by the European Commission in the Horizon 2020 - research and innovation framework programme."
