A team of researchers has discovered a gene therapy capable of restoring hearing in some cases of childhood deafness. The therapy has just been approved by the FDA.
Last month, the FDA approved the My ottera gene therapy for severe to profound hearing loss caused by mutations in a gene called OTOF.
In a clinical trial, 80% of treated patients regained measurable hearing and 42% reached the level necessary to hear whispers. Two and a half years after treatment, 90% of patients.
According to , the advancement in the field of genetic therapies, such as Otarmeni, which introduce a functional copy of a defective gene directly into the patient’s cells, seems like a miracle.
Em 1999the field of gene therapy was thrown into crisis when a young man named Jesse Gelsingerdied four days after receiving an injection of an experimental gene therapy in University of Pennsylvaniathe first publicly identified death in a clinical trial of this type of therapy.
It took several years and changes in the way gene therapies are administered for the field to recover. Now, 27 years after the young man’s death, there is a gene therapy capable of effectively reversing some types of .
The first change was a change in administration method. Gene therapies use modified viruses to introduce repair genes into the patient’s cells.
In Gelsinger’s case, the therapy used was carried by an adenovirus, which is highly immunogenic, meaning the human immune system recognizes it and reacts violently. It was this immunological reaction that killed the young man.
Thus, the field began to increasingly resort to adeno-associated viruses (AAV), which are smaller, more tolerable and capable of introducing the payload into the right cells without triggering an immunological reaction of great magnitude.
The second thing was the CRISPRwhich was adapted in 2012 to become a precision gene editing tool. CRISPR was able to do something that AAV could not, find a specific point in the patient’s own DNA and rewrite the letters in that location, correcting the damaged gene at that location.
CRISPR also gave gene therapy a moment of cultural prominence not seen since before Gelsinger.
Finally, while cost may not be an issue for families who would qualify for Otarmeni, it is not the only concern. You cochlear implantsthe standard treatment for OTOF patients for decades, have been contested in the deaf community since the 1980s, with many arguing that deafness should be viewed as an identity rather than a disability.
Gene therapy applied to babies makes this issue even more delicate, since children treated with gene therapy cannot give their consent to the change, and not everyone would make that choice.
