In an incredible medical miracle, three-year-old Opal Sandy has been able to hear for the first time in her life. She recently received the first-ever gene therapy treatment for profound congenital hearing loss. The therapy demonstrates extraordinary clinical trial success using one-off DB-OTO gene therapy to treat monogenic deafness. Because of this breakthrough, Opal is able to hear normally even with her cochlear implant shut off. This groundbreaking treatment took place in the UK. It forms a new and critical milestone in the treatment of inherited deafness.
Opal Sandy was born deaf due to mutations in the OTOF gene. This rare inherited condition affected her big sister, Nora. Prior to the treatment, neither girl was able to hear at all. Their mother, Jo Sandy, said their situation was critical.
“Both girls had no hearing whatsoever,” – Jo Sandy
At just 11 months old, Opal became the youngest patient globally to receive a gene therapy injection aimed at restoring her hearing. Her treatment took only 15 minutes. In the meantime, physicians had injected a functioning copy of the OTOF gene directly into her right cochlea and implanted a cochlear prosthetic device into her left ear.
The diverse group of participants in the clinical trial came from the U.K., the United States, Spain, and Germany. Since undergoing the procedure, Opal’s change has been nothing short of “mind blowing” according to her mom with an additional 13 genes to come. She’s done incredible work the past two years, learning to hear and showing what’s called “normal hearing” through her cochlear implants.
“So we’re talking like big, massive drums and cymbals being banged behind their heads. There was never a response, even at the very highest levels that they can test,” – Jo Sandy
This pioneering accomplishment demonstrates the promise of gene therapy as a cure for inherited genetic forms of hearing loss. Opal’s successful treatment represents hope for families worldwide stricken by conditions like hers. Beyond this immediate application, it serves as a launching point for exciting future research and development into auditory restoration therapies.
