Deaf Toddler Hears After Gene Therapy Treatment

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Facts

• Eighteen-month-old Opal Sandy from Oxfordshire, England — who was born deaf due to defective OTOF genes — can now hear 'almost normal for her age,' including 'soft sounds' such as whispers, thanks to a new gene therapy called DB-OTO.¹
• Due to a faulty gene, Opal was born with auditory neuropathy, which disrupts nerve impulses that travel from inside the ear to the brain. As part of the DB-OTO trial, which lasted 16 minutes, she was infused with a working copy of the gene.²
• Opal's sister Nora, 5, has the same condition but uses cochlear implants, which bypass the damaged sound-sensing hair cells and directly stimulate the auditory nerve. Opal received the therapy in her right ear and has a cochlear implant in her left.³
• Opal is part of a trial being run by Cambridge University Hospitals NHS Foundation Trust. She began in September and her positive hearing results came roughly 24 weeks later in February.⁴
• Opal's trial consists of three children like her who will receive a low dose of the gene in one ear, with another three set to receive one high dose. Up to 18 children are being sought to enter the trial, with a follow-up to be had five years later.⁴
• According to a surgeon at the NHS Foundations Trust, an estimated 20K people across the UK, US, France, Germany, Italy, and Spain have auditory neuropathy caused by OTOF defects.¹

Sources: ¹The Telegraph, ²Guardian, ³BBC News and ⁴Independent.

Narratives

• Narrative A, as provided by embopress.org. Recent gene therapy breakthroughs are not only exciting because they're helping children hear, but because these studies have resulted in no adverse effects. As scientists continue to cure deafness for these rare genetic disorders, these results will hopefully help further research for hearing-impaired people around the world.
• Narrative B, as provided by New York Times. These treatments, unfortunately, seem to be fully effective just for those born recently. Whether for deafness, muscular atrophy, or any disorder, current gene therapies for older people can only slightly mitigate the symptoms that have set in over long periods. More research should be conducted into fixing these disorders at all stages.

Predictions