LONDON (dpa-AFX) - A British toddler got her hearing restored after taking part in a breakthrough Chord trial, making her the first child in the U.K. and the youngest in the world to receive the new treatment.
Opal Sandy, aged 18 months, was born deaf due to a rare genetic disorder called auditory neuropathy, a condition caused by the disruption of nerve impulses traveling from the inner ear to the brain.
'When Opal could first hear us clapping unaided it was mind-blowing - we were so happy when the clinical team confirmed at 24 weeks that her hearing was also picking up softer sounds and speech,' Opal's mother Jo Sandy said. 'The phrase 'near normal' hearing was used and everyone was so excited such amazing results had been achieved.'
The Oxfordshire-based toddler had undergone the gene therapy - DB-OTO, from the biotechnology firm Regeneron, at Addenbrooke's hospital.
The trial results, presented during the annual meeting of the American Society of Gene and Cell Therapy in Baltimore, noted that auditory neuropathy arises from a single variation in a gene called OTOF, which is responsible for producing otoferlin. The protein is required by the tiny hairs of the inner ear to transmit nerve signals responsible for hearing.
Speaking about the result, chief investigator of the new trial Dr. Manohar Bance said, 'Gene therapy has been the future in otology and audiology for many years and I'm so excited that it is now finally here. This is hopefully the start of a new era for gene therapies for the inner ear and many types of hearing loss.'
The researcher noted that the gene disorder often goes undetected in newborn babies until the age of 2 or 3.
'We have a short time frame to intervene because of the rapid pace of brain development at this age,' Bance commented. 'Delays in the diagnosis can also confuse families as the many reasons for delayed speech and late intervention can impact a child's development.'
During the therapy, Opal received a working copy of the OTOF gene in her right ear's cochlea with the help of a harmless virus called AAV1. She also got fitted with a cochlear implant in her left ear.
The ongoing global trial is recruiting more deaf children from the UK, Spain, and the U.S., who will be followed up for the next five years.
'Many families will welcome these developments, and we look forward to learning about the long-term outcomes for the children treated,' Martin McLean, senior policy advisor at the National Deaf Children's Society in Britain, commented. 'This trial will teach us more about the effectiveness of gene therapy in those cases where deafness has a specific genetic cause.'
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