Opal, who was born completely deaf, can hear for the first time after a ground-breaking gene cure involving a harmless virus was given to her at just 11 months old
A toddler can hear for the first time after becoming the world’s youngest patient to get pioneering gene therapy.
Opal was born completely deaf because of a rare genetic condition that disrupts nerve impulses travelling from the inner ear to the brain. When she was 11 months old she took part in a medical trial in which a harmless virus containing a working copy of her faulty gene was injected into in the cochlear of her right ear. For comparative purposes, a cochlear implant – an electronic device that can provide a sense of sound – was fitted in her left. Opal is the first British patient to receive this type of treatment.
Her teacher mum Jo, 33, said: “I was testing [loud sounds] and hadn’t realised that her implant had actually come off, and she turned to pretty loud clapping.
“When she first turned, I couldn’t believe it. I thought it was a fluke or something that had caught her eye, but I repeated it a few times. I texted my husband James and said, ‘I think it’s working’. I was absolutely gobsmacked.”
Scientists behind the technique at Addenbrooke’s Hospital in Cambridge said her progress was “mind-blowing”. The trial’s chief investigator Prof Manohar Bance said: “These results are spectacular and better than I expected. This is hopefully the start of a new era for gene therapies for the inner ear.”
Prof Stephen Powis, NHS England’s national medical director, said: “This is another example of the NHS being a global leader in developing gene therapy for patients.” Jo and James, 33, said Opal now enjoys playing with her toy drums, a piano and wooden blocks. Jo added: “She loves slamming cutlery on the table asking us where dinner is.”
