Deaf baby hears for first time after gene therapy

LONDON — A baby girl born with profound genetic deafness can now hear unaided after receiving a “groundbreaking” gene therapy trial, Britain’s National Health Service said Thursday.

Opal Sandy, an 18-month-old from Oxfordshire, England, is the first patient treated in a global gene therapy trial that is showing “mind-blowing” results, Addenbrooke’s Hospital in Cambridge said in a statement. Opal is “the first British patient in the world and the youngest child to receive this type of treatment,” the hospital said.

The treatment is already having practical results, the hospital said: Opal can now respond to her parents’ voices and can communicate words such as “Dada” and “bye-bye.”

“When Opal could first hear us clapping unaided it was mind-blowing,” her mother, Jo Sandy, said in a statement. “We were so happy when the clinical team confirmed at 24 weeks that her hearing was also picking up softer sounds and speech.”

The results are another example of the potential of gene therapies, which use the insertion of genetic material to treat diseases.

Opal, like her elder sister, was born deaf because of a rare genetic condition known as auditory neuropathy, which is caused by the disruption of nerve impulses traveling from the inner ear to the brain, the hospital said. However, within four weeks of having the gene therapy infusion to her right ear, Opal “responded to sound,” it added. Now, at 24 weeks after treatment, clinicians have been able to confirm “close to normal hearing levels for soft sounds,” even including “whispering, in her treated ear.”

Manohar Bance, chief investigator of the trial and an ear surgeon at Cambridge University Hospitals NHS Foundation Trust, said the results were “spectacular and better than I expected.”

“This is the start of a new era for gene therapies for the inner ear and many types of hearing loss,” he added.