Opal Sandy, a three-year-old girl from the United Kingdom, was recently treated with gene therapy to restore her hearing, marking a historic first. Thanks to this innovative treatment, she’s regained her sense of hearing due to mutations in Opal’s OTOF gene. Her path to soundness took a major twist when she was one of the first recipients of revolutionary therapy through a clinical trial for DB-OTO gene therapy.
The clinical trial, which lasted from 2016 through 2018, involved participants from the U.K., the United States, Spain, and Germany. It was designed to determine how successful this new intervention is at treating the inherited form of hearing loss. Before the surgery, Opal Sandy was completely deaf. This complication impacted not just her but her understanding, older sister, Nora. She was born deaf, courtesy of the very same genetic freak luck.
At the time, only 11 months old, Opal received the gene therapy procedure, which was conducted in the United Kingdom. The entire treatment was done in just 15 minutes and under general anaesthesia. Once open, surgeons provided an in-service of sorts, inserting a functional copy of the OTOF gene directly into Opal’s right cochlea. Meanwhile, they implanted a cochlear implant in her left ear.
Incredibly, two years after the surgery, Opal Sandy can finally hear. This is an important and encouraging development. That’s because she’s able to hear sounds with her cochlear implant off, an indication that the gene therapy is working. Her mother, Jo Sandy, told the Gazette of her surprise over the change, which she said has been “mind-blowing.”
Opal’s successful treatment is an inspiring example of how gene therapy provides hope to dramatically improve and extend people’s lives. It is a historic breakthrough in the science of audiology and for those who suffer from genetic hearing loss. These treatments need not only benefit the treating patient. Further, they unlock new possibilities for research and therapies that can help millions of others with similar conditions.
