FDA Gives Green Light To the First Gene Therapy For Deafness (npr.org) 33
An anonymous reader quotes a report from NPR: The Food and Drug Administration approved the first gene therapy to restore hearing for people who were born deaf. The decision, while only immediately affecting people born with a very rare form of genetic deafness, is being hailed as a milestone in the quest to treat hearing loss. "It's the first time in history there's a new drug for hearing loss," says Zheng-Yi Chen, an associate scientist at Mass Eye and Ear in Boston who was not involved in the development of the therapy approved by the FDA Thursday. But his research team reported very promising results with a similar approach Wednesday. "I think it's an historical event, a landmark, a great development for the whole field," he says of the approval. [...] The FDA's decision was based on the results from the treatment of 20 patients born with a defective version of a gene known as OTOF, which is necessary to transmit sound from the ears to the brain.
Doctors infused billions of adeno-associated viruses into the patients' ears by making a small incision behind the ear to open a small hole in the skull. The viruses carried a healthy version of the OTOF gene that had been split in half to fit inside the virus. The gene provides instructions to make the otoferlin protein, which is necessary for hair cells in the inner ear to transmit sound to the brain. Most of the patients began to hear for the first time within weeks, with the quality of their hearing improving over the following months, according to [Regeneron Pharmaceuticals, which developed the gene therapy and plans to offer it for free in the U.S. It should be available within weeks.]. The amount of hearing patients gained varied, but 80% achieved at least some significant hearing restoration and 42% ended up with normal hearing, which included the ability to hear whispers, Regeneron says. The hearing ability has lasted at least two years so far.
The treatment can only help patients with the very rare form of deafness that Smith was born with, which only affects about 50 children each year in the U.S. But similar gene therapies are showing promise for other forms of genetic deafness. And researchers hope someday gene therapy may help with common types of hearing loss, like from aging and loud noise.
Doctors infused billions of adeno-associated viruses into the patients' ears by making a small incision behind the ear to open a small hole in the skull. The viruses carried a healthy version of the OTOF gene that had been split in half to fit inside the virus. The gene provides instructions to make the otoferlin protein, which is necessary for hair cells in the inner ear to transmit sound to the brain. Most of the patients began to hear for the first time within weeks, with the quality of their hearing improving over the following months, according to [Regeneron Pharmaceuticals, which developed the gene therapy and plans to offer it for free in the U.S. It should be available within weeks.]. The amount of hearing patients gained varied, but 80% achieved at least some significant hearing restoration and 42% ended up with normal hearing, which included the ability to hear whispers, Regeneron says. The hearing ability has lasted at least two years so far.
The treatment can only help patients with the very rare form of deafness that Smith was born with, which only affects about 50 children each year in the U.S. But similar gene therapies are showing promise for other forms of genetic deafness. And researchers hope someday gene therapy may help with common types of hearing loss, like from aging and loud noise.
Not much fun to be deaf (Score:1, Insightful)
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Forever guessing what people said is not much fun.
No, it's not. Nor is having people seem to suddenly materialize on your deaf side, lol
I'm very glad that I got my cochlear implant for single sided deafness. Although who knows; if they ever figure out something similar to the treatment in this story for it, maybe I blew my chances for true recovery. But I sure can hear a lot better now, with the implant, than without.
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Forever guessing what people said is not much fun.
No, it's not. Nor is having people seem to suddenly materialize on your deaf side, lol
I'm very glad that I got my cochlear implant for single sided deafness. Although who knows; if they ever figure out something similar to the treatment in this story for it, maybe I blew my chances for true recovery. But I sure can hear a lot better now, with the implant, than without.
Out of curiosity, do implants help with tinnitus? I have what can best be described as raging tinnitus, along with little hearing above 3 KHz.
My brain goes into interpretation mode, and My wife, who has really acute hearing tells people if I say something that sounds insane or funny they just need to stop and say it a little louder, and don't be afraid to laugh then tell me if I accidentally say something hilarious or a non-sequitur.
But hearing aids only sorta work - after 15 minutes, they are terribly
invisible impediments (Score:3)
The world is unforgiving if you or one of your family members has an invisible impediment. One does not get the benefit of the doubt or consideration.
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Out of curiosity, do implants help with tinnitus? I have what can best be described as raging tinnitus, along with little hearing above 3 KHz.
From what I've read of others' experience, some get better, some worse, when it comes to tinnitus. I don't know what studies there are about it; you might want to look.
For me personally, it's better. Much less tinnitus. Not sure why. Maybe actually hearing stuff on that side again stops my brain from wanting to make stuff up? Dunno ...
How long... (Score:4, Insightful)
... before some extremist clowns from the deaf "community" start protesting about how this diminishes deaf "culture". Think I'm joking? This happened when cochlear implants first came along. Never mind the reduced quality of life people - particularly children - suffer being deaf, far more important is some kind of specious group identity being maintained.
Re: How long... (Score:1)
Finding out this is apparently a-thing has been truly bewildering... the internet gives too much of a platform to too many of the absolutely most insane and extreme people out there.
Re: How long... (Score:5, Insightful)
too much of a platform to too many of the absolutely most insane and extreme people out there
Before internet
>i want to fuck toasters
>dont be a fucking retard
>grow up
After internet
>I want to fuck a toaster
>google
>find a community with 1000+ members about people wanting to fuck toasters
>fuck up your life
Re: How long... (Score:1)
Sadly it is going MUCH further than that...
> Got the community? Now bully and harass where ever you can online for recognition and a fake, forced and compelled sense of acceptance, preferably by absolutely everyone, to normalize what should only ever have been a 0.001% fringe extremism
> Latch on to some tribalism of other, larger, more tribal communities
> Bully even more people together.
> If the bullying goes well enough, branch out into calling any opposing opinions to literally be GENOCIDE, an
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How can you be talking about a genocide when there is a genocide going on!
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Alexander Graham Bell was a major proponent of eugenics and wanted to eliminate deaf people.
You're being unfair to Bell, and most of your statements are factually incorrect. See the Wikipedia biography [wikipedia.org] for background. There's really too much to quote all the relevant passages here.
"Historians have noted that Bell explicitly opposed laws regulating marriage, and never mentioned sterilization in any of his writings. Even after Bell agreed to engage with scientists conducting eugenic research, he consistently refused to support public policy that limited the rights or privileges of the deaf."
Bell:
Re:How long... (Score:5, Interesting)
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I can see how being born hearing and then going deaf causes suffering, but I don't see how being born deaf does.
"Suffering" is probably not the correct word here - if you're born deaf, not hearing is normal for you. However, a deaf person is excluded - not by their fault, of course - from large areas of global culture; somebody born deaf will never be able to enjoy Beethoven's Ode to Joy (or whatever your favorite song is), never hear their child laugh, never go asleep to the sound of rain. As a deaf person, maybe you never had a chance to live those experiences. Would you choose to deny them to your child if you c
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Imagine to be deaf, but having Tinnitus ...
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There are ways to enjoy music other than hearing it. Beethoven is an ironic example since he went deaf (though I don't know enough about him to know if Ode to Joy was composed before or after that).
But, I understand your overall point. However, there are aspects to life that a deaf person experiences, that hearing people do not. To by default choose to deny your child the chance to experience life THAT way, assumes that hearing life is
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There are ways to enjoy music other than hearing it. Beethoven is an ironic example since he went deaf (though I don't know enough about him to know if Ode to Joy was composed before or after that).
Before, but he had performed and written most of his music while he still had his hearing.
DAME Evelyn Glennie [wikipedia.org] is a professional concert percussionist who "has been profoundly deaf since childhood, having started to lose her hearing at the age of 8. This does not inhibit her ability to perform. She regularly plays barefoot during live performances and studio recordings to feel the music." I've seen her perform, and she was barefoot on that occasion. She has a Web site, a TED talk, a book, and a podcast.
I don't know what the right choice is, nor what choice I would make for my child in that position.
Th
Re: How long... (Score:2)
Try a using a standard telephone then get back to us.
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Re: How long... (Score:2)
So Bell was anti deaf because he developed an audio device? Wow, talk about losing the plot. Are radio stations anti deaf too?
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They are not referencing the telephone. Look up the parts of life involving the teaching of deaf children, especially opinions on lip reading and oralism.
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They suffer from getting surprised by things a hearing person would hear coming long before ...
Or get an extreme example: neighbours are looking strange at them, because they are loud in bed, and do not notice themselves.
Or invent your own extrem example ...
A guy shouting at you: "damn idiot, can you not move out of the way, are you deaf??!!"
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Arguable, some deaf people have enhanced other senses, and I am frequently surprised by things I should have heard...
Some deaf people can feel when they are making sounds. But either way, any suffering from that is not caused by being born deaf, it is caused by a social stigma about being
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Not having to hear such a person seems like a benefit to me. :D
True
coming soon.... (Score:2)
To a new drug to treat male pattern baldness. It's all about monetization of new drugs. Curing 50 people a year for deafness isn't profitable enough for big pharma.
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To a new drug to treat male pattern baldness. It's all about monetization of new drugs. Curing 50 people a year for deafness isn't profitable enough for big pharma.
Yeah, and some forms of deafness just won't be cured. The only way so far to cure my tinnitus is to sever my auditory nerves.
I'd love a cure for MPB as well. My hair thinned, then stopped. A real pain in the ass because the wind plays hell with it, but the wife isn't at all wild about the idea of shaving my head. She says I'd look like my neck was blowing bubble gum.
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Cutting the nerves would not cure it.
That is a brain dead idea.
Tinnitus happens in the brain ... wrong trained signal patterns.
Re: coming soon.... (Score:2)
Only partially true.
Tinnitus can happen in the ear or auditory nerve as well as in the brain.
Stepdaughter had a growth on her ear drum -> tinnitus.
Friend was in a major car accident. Damaged his cochlear -> tinnitus.
However, severing the auditory nerve is actually likely to cause tinnitus, both from misfiring nerve endings and also by the overcompensating of the brain to the sudden loss of signal.
What?? (Score:2)