Scientists treat deafness in 11-year-old boy using gene therapy
In a major scientific breakthrough, researchers may have just managed to use gene therapy to help a young deaf boy hear.
The boy in question is 11-year-old Aissam Dam from Morocco. He had congenital deafness because of a mutation to the otoferlin gene in his body, according to the New York Times. This gene normally manufactures a protein that plays a key role in transferring sound from the ear to the brain, so if it's not quite right then that process is impeded.
Dam was reportedly treated at the Children’s Hospital of Philadelphia, as part of a clinical study supported by pharmaceutical company Eli Lilly and Akouos.
Advert
There are apparently around 200,000 people with a similar condition to Dam around the globe.
The researchers effectively made a benign virus containing new otoferlin genes, and then embedded that in a liquid. This liquid was squirted into Dam's cochlea, in the inner ear, where it set to work.
Just a few days later, Dam was already able to hear sounds that he never would have picked before, and two months later his hearing was apparently close to full strength.
Dam seems euphoric, and told the New York Times with the help of interpreters: "There’s no sound I don’t like. They’re all good."
He was born in Morocco but now lives in Spain, and was referred to the trial by a hearing expert there, and there are now two more kids lined up for the same treatment.
The fact that the process only took a few days is perhaps the most remarkable part of it.
Dr. Margaret A. Kenna, an investigator in the Lilly trial, said: "It’s been a long time coming. For decades people have been saying, 'When is this going to work?' I didn’t think gene therapy would begin in my practice lifetime. But here it is."
And it seems like this type of treatment is picking up speed - with more studies being carried out all ove the world, from Europe to China.
That said, this type of disorder only accounts for a few percent of the worldwide population of those with congenital deafness. But the template that the gene therapy uses might later be adapted to suit at least some of these other congenital issues.
However, not everyone is so keen on the idea of 'treating' deafness. Dr. Robert C. Nutt, a developmental and behavioral pediatrician in Wilmington, N.C., who is deaf, told the New York Times: “There is an internal Deaf community that doesn’t see itself as needing to be cured."