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First US Gene Therapy Offers Hope for Hearing Loss
18 Aug
Summary
- A toddler received a first-of-its-kind gene therapy for hearing loss.
- The treatment targets a gene mutation causing auditory neuropathy.
- The therapy can cost over $1 million but was free for this patient.
A toddler from Alabama recently underwent a pioneering gene therapy treatment in the United States for auditory neuropathy spectrum disorder (ANSD), a condition resulting from an OTOF gene mutation. This therapy, named Otarmeni and approved by the FDA in April, aims to restore hearing by introducing a functional OTOF gene to inner ear cells. The treatment's maker, Regeneron, provided it free of charge to U.S. patients, a significant benefit given its usual cost of over $1 million per dose.
Despite the free treatment, the family incurred travel expenses of approximately 1,200 miles to reach Boston Children's Hospital. The child, Everett, received the infusion directly into his cochlea, a procedure performed by Dr. A. Eliot Shearer. This method aims to restore a more natural hearing capability than cochlear implants. Early observations have shown improvements in Everett's awareness of his surroundings, fueling optimism for his future development.
Clinical trials leading to the FDA approval demonstrated promising results, with 80% of participants showing hearing improvements and 42% achieving normal hearing within a year. Experts are hopeful that this advancement will pave the way for treating numerous other patients with similar genetic hearing impairments, offering a new horizon for those affected by ANSD.
