Opal Sandy, a little British girl born deaf, has regained her hearing thanks to a new gene therapy trial in the United Kingdom. She is the first patient treated as part of a global gene therapy trial, the NHS, Britain’s public health service, said in a press release published this Thursday, May 9.
An innovative treatment
Opal, who suffers from auditory neuropathy, has been deaf since birth. This pathology leads to the degeneration of the nerves responsible for transmitting information to the nervous system. In other words, in patients with this disease, sounds enter the inner ear normally, but their transmission to the brain is altered.
Around 20,000 people in the UK, Germany, France, Spain and Italy are deaf due to a defect in a gene that produces otopherin, a protein needed by hair cells in the inner ear to communicate with the auditory nerve.
During the clinical trial which took place in September 2023 at Addenbrooke’s Hospital in Cambridge, Opal received an infusion containing the active gene into her right ear. The surgical operation lasted only 16 minutes, indicates Sky News.
A new era for gene therapies
Four weeks after the operation, Opal’s parents, Jo and James Sandy, noticed initial improvements. After 24 weeks, the little one’s hearing was almost normal for soft sounds, like whispers.
“When Opal could hear us clap without help, it was incredible,” her mother testified.
“The results with Opal are very dramatic and very close to normal restoration of hearing, so we are hopeful that this could be a potential cure,” said Professor Manohar Bance of Cambridge University Hospital and principal investigator of the trial. CHORD.
“We hope this is the beginning of a new era for gene therapies for the inner ear and many types of hearing loss,” he said.
According to Sky News, the treatment Opal received was designed specifically for children with OTOF mutations. Other similar studies are underway or expected to launch soon in the United States, Europe and China.
Earlier this year, Children’s Hospital of Philadelphia announced that an 11-year-old boy “who was born profoundly deaf” was hearing “for the first time in his life” after gene therapy and four months after surgery. The child now has only mild to moderate hearing. hearing loss.
A study published earlier this year in the medical journal The Lancet also revealed that a similar treatment administered in China to six deaf children allowed five of them to regain their hearing.
Source: BFM TV
