An 18-month-old British girl has become the first person in the world to take part in a groundbreaking clinical trial testing a new gene therapy. The little girl, born completely deaf, has regained her sense of hearing, reports DPA/PA Media on Thursday.
A revolutionary new gene therapy could restore hearing to people with impairments PHOTO Archive
Opal Sandy was born with no hearing at all due to a condition called auditory neuropathy. This is caused by disruption of the nerve impulses that travel from the inner ear to the brain. The little girl has become the test subject of an amazing new medical therapy.
Addenbrooke's Hospital in Cambridge was where the gene therapy took place, part of a clinical trial that could revolutionize the treatment of deafness. Professor Manohar Bance, the surgeon who led the study, said the results were “better than I hoped or expected”paving the way to curing this type of deafness.
Auditory neuropathy is often caused by a defect in the OTOF gene, which affects the production of a protein called otoferlin. Gene therapy provided by biotech company Regeneron inserts a copy of the gene into the ear, thereby correcting this defect.
After Opal received this gene in her right ear during surgery, her parents, Jo and James Sandy, noticed significant improvements within four weeks. The little girl reacted to a loud noise for the first time, an emotional experience for her family.
Further tests confirmed that Opal can also hear softer sounds, such as a whisper. Professor Bance explained that her hearing is now “almost normal”, offering hope that it will be fully back to normal in the next tests.
Opal is not the only beneficiary of this therapy; a second child also received the treatment and had positive results six weeks after the intervention.
The clinical trial, called 1/2 Chord, is being conducted on a sample of up to 18 children in the UK, Spain and the United States.
If the therapy is proven to be safe, more children would benefit from this treatment.
