A toddler can hear for the first time after becoming the world’s youngest patient to get pioneering gene therapy.
Opal Sandy 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.”
The Government hailed Opal's breakthrough treatment as an "historic milestone" Health Secretary Victoria Atkins said: “I am delighted and proud of what this government-backed trial has done for Opal, and what it could eventually do for children like her across the world.
"It marks an historic milestone as we develop a cure for this kind of deafness. I am determined that the UK continues to encourage innovation and improve lives across the world, which is why we are investing £1.2 billion a year through the National Institute for Health and Care Research to pursue groundbreaking research such as this.”
The Chord gene therapy trial was funded via the NIHR Cambridge’s Biomedical Research Centre, which receives some Government funding for research.