New trial raises hope for first drug to restore hearing

Image of anatomy of ear.There are currently no drugs available that treat hearing loss. At present, patients have to opt for hearing aids or cochlear implants, which don’t address the root cause of hearing loss.

Damage to the sensory hair cells in the cochlea – known as sensorineural hearing loss – is a major cause of hearing loss acquired later in life: 90% of cases of hearing loss are sensorineural. Overall, 1 in 6 people in the UK – and around half a billion people worldwide and over 360 million people worldwide – have hearing loss.

Hair cell loss has long been thought to be irreversible, but various earlier studies in animals indicate that functioning inner ear sensory hair cells may be regenerated through the use of a small molecule substance called a gamma-secretase inhibitor.

And now, researchers at the UCL Ear Institute and UCLH’s Royal National Throat Nose and Ear Hospital (RNTNEH) and are leading a trial – being undertaken by the REGAIN (Regeneration of inner ear hair cells with gamma-secretase inhibitors) consortium made up of partners in the Netherlands, UK, Greece, Germany and Denmark – to test a drug in patients with hearing loss based on these studies.

Professor Anne Schilder, Director of evidENT at the UCL Ear Institute and NIHR Research Professor, who is supported by the National Institute for Health Research UCLH Biomedical Research Centre and is leading the design and delivery of the REGAIN clinical trial, said: “We are proud to be part of the REGAIN consortium and to be leading on translating this scientific breakthrough into a treatment that may improve people’s hearing and lives.

For ‘phase 1’ of the REGAIN trial, which took place throughout 2018, the clinical research team at RNTNEH gave the drug via injections to the ear to 15 patients with mild to moderate sensorineural hearing loss, to test the safety and tolerability of the drug.

Researchers at UCL and UCLH – along with sites in Germany and Greece – are now moving on to the next stage of the REGAIN clinical trial: a ‘Phase 2 study’ which will test the efficacy of the drug in 40 adults with mild to moderate adult-onset sensorineural hearing loss.

Professor Shakeel Saeed, Professor of Otology and Clinical Director at RNTNEH and lead clinician on the REGAIN clinical trial, said: “We are very excited by this research and are thankful to all the participants in the study without whom we could not have delivered on this trial.”

Hearing loss can prevent and hinder participation in social situations, and the overall health and quality of life of people with hearing loss is poorer than that of the general population. Hearing loss has been identified as an important risk factor for dementia with mild hearing loss doubling the risk, and severe hearing loss increasing the risk of dementia five-fold.

Current treatments – hearing aids and cochlear implants – work by amplifying the sound waves entering a person’s ear, but have no impact on the underlying physical problems in the ear responsible for hearing loss, and don’t work well in noisy environments.

Patients interested in participating in phase 2 of the REGAIN clinical trial at UCLH and UCL can visit


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