UK Toddler Who Was Born Deaf Can Now Hear After World's First Groundbreaking Gene-Therapy Trial
Opal with mum and dad Jo and James Sandy. (Image credit: UK NHS)
A toddler in the United Kingdom has had her hearing restored, becoming the first person worldwide to benefit from a groundbreaking gene-therapy trial.
Opal Sandy was born deaf and unable to hear due to auditory neuropathy, a condition caused by a faulty gene that disrupts nerve impulses from the inner ear to the brain.
However, after undergoing surgery that lasted just 16 minutes and receiving an infusion containing a functional copy of the gene, the 18-month-old can now hear almost perfectly and delights in playing with toy drums.
Treated shortly before her first birthday, Opal can now hear sounds as soft as a whisper and is beginning to speak, uttering words such as “Mama,” “Dada,” and “uh-oh.”
Her parents were astonished when they witnessed her hearing for the first time after the treatment. “I couldn’t really believe it,” Opal’s mother, Jo Sandy, remarked. “It was… unbelievable.”
Opal, from Oxfordshire, is the first patient treated in a global gene therapy trial that has shown “mind-blowing” results. She is not only the first British patient to undergo this treatment but also the youngest child to receive it worldwide.
The toddler received treatment at Addenbrooke’s Hospital, part of Cambridge University Hospitals NHS Foundation Trust, which is conducting the Chord trial.
Professor Manohar Bance, an ear surgeon at the trust and the chief investigator for the trial, expressed his surprise at the initial results, stating they were “better than I hoped or expected” and could potentially cure patients with this type of deafness, as per The Guardian report.
“We have results from [Opal] which are very spectacular – so close to normal hearing restoration. So we do hope it could be a potential cure,” Bance stated.
He further added: “There’s been so much work, decades of work … to finally see something that actually worked in humans …. It was quite spectacular and a bit awe-inspiring really. It felt very special.”
As per the report, Opal is part of a trial recruiting patients in the UK, US, and Spain. Doctors in other countries are also exploring similar treatments for the Otof gene mutation that Opal has.
The gene therapy, known as DB-OTO, is designed specifically for children with OTOF mutations, utilising a harmless virus to deliver the functional gene into the patient’s cells.