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Gene Therapy Gives Deaf Child Natural Hearing
16 Aug
Summary
- A new gene therapy allows a toddler to hear naturally without aids.
- The treatment delivers a working copy of a defective gene to the inner ear.
- This groundbreaking therapy was recently approved and is the first of its kind.
A 16-month-old boy named Everett Tynes, who has been deaf since birth, has become one of the first patients to receive a new gene therapy called Otarmeni. This innovative treatment targets a rare genetic variant that prevents sound signals from reaching his brain.
The therapy works by delivering a working copy of Everett's defective OTOF gene directly into his inner ear. A modified virus carries the gene, instructing cells to produce a missing protein essential for transmitting sound. This approach aims to restore the natural hearing process, unlike cochlear implants.
Boston Children's Hospital administered the treatment, which was approved in April and is the first of its kind in the US. Clinical trials showed significant hearing improvements in most participants, with some achieving normal hearing.
Regeneron, the therapy's maker, is offering Otarmeni free to US patients under a federal agreement, though other care costs remain. The family faced challenges, including travel and accommodation expenses, to access the treatment located 1,200 miles from their Alabama home.
While it is still early to determine the full success of Everett's treatment, his parents have observed increased awareness and engagement. The medical community is optimistic about the potential of gene therapy for hearing loss, with ongoing research for other genetic causes of deafness.
