Gene Therapy for Reversing Hearing Loss

Here is an encouraging article about reversing hearing loss:

Novartis AG (NOVN) is developing a gene therapy that may reverse hearing loss by stimulating the regrowth of microscopic hair cells in the inner ear, allowing people to hear. The hairs are destroyed by prolonged exposure to loud noise, and don’t take root again naturally. Novartis treated the first patient in October after successful tests on rats.

Novartis plans to test its treatment on 45 patients in the US, with results expected by 2017, according to a description of the trial on clinicaltrials.gov.

The therapy uses a disabled cold causing virus that enters so-called supporting cells in the inner ear, where it delivers its payload--a gene called atonal-1 that tells the cells to grow in to hair cells.

 

Cool! I wrote about one of the problems with gene therapy in another thread. The problem is knowing where the gene you are introducing will set int the DNA. If it ends up on the wrong place you can have cancer on your hands. But I'm sure they've worked this stuff out.

 

Oh snap! I never even thought about cancer occurring. Hopefully over time they'll come up with something more precise...

 

Gene therapy 1- 0 Stem cell therapy

 

Yes the technology isn't that new. They preformed gene therapy 25 years ago. They actually managed to cure a genetic disease called ADA deficiency. However at least one of the people involved in the study developed cancer.

The gene is inserted by using a virus (called a vector). The virus usually works by introducing it's own DNA to the cell (if it's a DNA virus). If we alter the virus we can make it introduce what ever gene we want. There is a problem however. The gene can splice anywhere in the genome. And if you're unlucky it can settle and "overwrite" another gene that is important for something.

There are some ways to make it target some regions of the DNA but it's not bullet proof so to say. Or it least it wasn't a few years ago when I studied the subject (I've been out of the game for a while).

If they've managed to work this issue out then there is a tremendous potential. We could basically cure more or less any genetic disorder there is. Not all but almost. Huntington's disease is a genetic disorder that we for example probably couldn't cure with this technique. That's because Huntington's is not a disorder where there is a gene that is not working. It's due to a repetitive sequence on chromosome 4. The repetition is CAG and normally we have up to 150 repetitions. And if you have more then 150 you're screwed (i think it's 150, or maybe 160, but somewhere around that).

Here is a little info about the risks with gene therapy: http://www.mayoclinic.org/tests-procedures/gene-therapy/basics/risks/prc-20014778

 

Sounds like the plot to the 2007 movie adaptation of I Am Legend.

 

Haha yea they probably did some reading before the movie. But in the movie the virus mutated by it self and something happened. Although it is possible that a virus might mutate the consequences wouldn't be as they were in the movie. The virus would probably just not work as intended and the body would take care of the rest.

Usually a virus for the common cold or something like that is used. So the worst that could happen is that you sneeze for a few days. Using the HIV or Ebola virus as a vector might be a bad idea

Edit: when I think of it HIV is a RNA virus if I remember corectly so it wouldn't work anyhow.

But a lot if viruses copy their genome into the genome of the cell. The herpes virus does this. This is why you can never get rid of it. It copies it self into the DNA of nerve cells and can stay inactive until a signal activates it's genes (usually avtivation by a transcription factor protein due to a response to weakening of the immune system). Then you get the blisters.

 

I'd gladly risk cancer, if I could hear clearly without T, for even 4 months. The T I have, is worse than cancer. (And I don't want any of you saying, that's not possible)
Because it is! Hell would be a cake walk at this point.

 

This appears to be the clinicaltrials.gov entry: https://clinicaltrials.gov/ct2/show/NCT02132130?term=Novartis+gene+therapy+hearing+loss&rank=1 It is a combined phase 1 and phase 2 trial. I'm presuming this is so because you can't really do a phase 1 trial on healthy volunteers for something like this.

Unless I have gotten these things mixed up, it looks like this is the same thing as reported earlier: https://www.tinnitustalk.com/threads/denver-man-gets-gene-therapy-to-restore-hearing.7312/ However, the OP mentions the first human treatment taking place in October so maybe they are different.

If you google gene therapy and hearing loss, it does seem like there is a lot going on.

 

Sounds promising, hopefully it brings relief for T too.

 

Yes Aaron123. Its the same link that you mentioned. It started with Rob Gerk and then the trial expanded. We have gotton some info from another participant at the end of that thread.

 

Lol. God knows how the T ridden brain will react when those hairs are restored... "Holy shit! That annoying ringing sound I've been making for the past decade has just been phantom noise! Those hair cells were dead and now they're alive again!"

 

Bump, bringing this back to life. It's almost 2017, folks. I wonder if there's any updates on this. I've recently stumbled across this thingy here as well: https://www.sciencedaily.com/releases/2016/09/160901102734.htm

 

You have to read the inner ear regeneration thread, we speak about Novartis.

 

I want to be in this trial:

CGF166 Inner Ear Gene Therapy Trial - Corp...

 

Well they're recruiting...

https://clinicaltrials.gov/ct2/show/study/NCT02132130#contacts

 

Guess I could. Doesnt say one has to be an American as far as I can see. But i dont thnk my hearingloss is severe enough, even though my T is.

Tinnitus is not even mentioned once in their trail-sheet, but surly it most be something they will monitor aswell!

 

One guy from the trial wrote about it and said he has had no improvement in his hearing. It's maybe even worse and he's going for a cochlear implant. He was almost totally deaf before, like all the participants I guess.

I think you can find it in the regeneration thread.

I believe too that they monitor T, as it can be an interesting sign of changes in the inner ear.

 

Is it possible that he got placebo ?

 

No.