CGF166 – The Latest News

“There have been a couple patients with hearing improvement, so we are definitely encouraged.” – Dr. Lawrence Lustig

There is no cure for sensorineural hearing loss, the type that most of us with aging ears suffer from.  At least not yet.  But as I’ve written about in earlier posts, a pioneering treatment may be on the way.  It’s called CGF166.  It’s the only gene therapy for hearing loss now undergoing human trials in the U.S. and early reports are promising.  Here’s a progress report.

Hair Cells
National Institutes of Health

First, a little background.  The cause of most sensorineural hearing loss is fairly straightforward.  As we get older we lose hair cells, the tiny vibration detectors in the cochlea, and as we lose hair cells we lose hearing.  We are born with about 30,000 of them and we all lose them at different rates because of noise induced damage, ototoxic drugs,  or genes that make you predisposed towards early age-related hearing loss.

Once they are gone they do not grow back.  But CGF166 appears to be able to trigger the regeneration of hair cells.

It uses a gene know as Atoh1 which causes hair cells in mammals to grow in the embryo stage, and then shuts off.  With CGF166, Atoh1 is added to a harmless virus which binds to cells in the cochlea and tells them to begin growing hair cells again.

It’s worked for mice and monkeys.  Now Novartis is conducting trials to see if it also works for humans.  These are what’s called Phase 1/2 trials in which the priority is safety and determining it it works.  The trials are being conducted at three centres: Johns Hopkins in Baltimore, University of Kansas in Kansas City and Columbia University in New York.

Dr. Lawrence Lustig
Columbia University

I spoke with Dr. Lawrence Lustig who is conducting the Columbia trials, and he generously gave me an update on the progress.

The first patient received CFG166 in 2014 and they had planned on a total of 45 patients.  But so far only 11 people have undergone treatment.  For Dr. Lustig and his colleagues that is a disappointingly low number.  I asked him why.

“One of the issues that we’re confronting in terms of trying to find patients who are candidates is the very restrictive criteria that we’re using for inclusion.  Basically everybody who is a candidate is also a cochlear implant candidate. You have to have that level of hearing loss. But if it’s too severe then you’re no longer candidate either. So you have to be within a window of severity.

Number two, you can’t have certain rare genetic conditions  (such as Usher Syndrome) for your hearing loss.

And number three, most people who have hearing loss at that level have already received a cochlear implant.”

These restrictive criteria may be loosened if CGF166 enters wide clinical use but, for now, as Dr. Lustig points out, “The FDA is being appropriately cautionary because this is the first time anyone’s ever done a gene therapy study.”

He also reports that so far none of the patients has had any major problems.

The procedure involves going down the ear canal and detaching the tympanic membrane (ear drum) to reach the cochlea.  A laser is used to create a hole in the cochlea to deliver the CGF166.  Remarkably, it only takes 30 minutes and the patient is sent home the same day.

The results so far are promising.  “There have been a couple patients with hearing improvement, so we are definitely encouraged.”

Assuming that all goes well, when could CGF166 be approved for wider use?

“I would say it’s going to be at least a couple of years.  We have to do a Phase 3 study yet.  We’re hoping to add additional centres just to aid with the recruitment because patients are hard to find, and that will speed things up.”

Fingers crossed.  If you think you might be candidate for the trial, go to the U.S. National Institutes of Health site: CGF166 clinical trials.

As well, you can contact Novartis directly:  Novartis CGF166 trials. 

For the record, I didn’t qualify. My hearing is still too good.

25 thoughts on “CGF166 – The Latest News”

  1. Thank you so much for that update.! I am trying to see if I qualify but have had trouble getting a hold of the studg centers.

  2. They say there’s been a couple of people with hearing improvement, well this represents an absolute breakthrough so why not more information?? A ‘couple’???

    1. So far only 11 people have undergone the treatment and it takes time to assess their results so a couple is not surprising. I will update the trials as more info becomes available.

  3. I’m just curious on the delivery method. The method you describe is intratympanic. But according to Novartis website they are using IL-infusion which is a systemic delivery method. Have they changed the delivery method?

    1. As I understand it, access to the cochlea is via the ear canal through the tympanic membrane. CFG166 is then infused through an opening in the bony labyrinth. So the delivery is IL-infusion.

  4. If all goes well, let’s hope Novartis has the sense to make this thing affordable for everyone, since sooner or later everyone is affected by hearing loss. The only other gene therapy on the market, Glybera, costs $1 million at least, and in four years it’s only been purchased once, which has absolutely hurt the company making it. (Being marketed for an ultra-rare disease doesn’t help.)

  5. It’s amazing to be living in a time when it may soon be possible to restore one’s natural hearing. Repairing the cochlear will be far superior to hearing aid solutions, as helping as they are. Hearing aids tend to give tinny sound quality, do not fix distortions and lack of a sense of loudness in low frequencies caused by damage to the cochlear, not to mention frustrations with flat batteries, ear wax and not being able to wear them when swimming. Fixing the root cause of deafness has to be the forward. I wish this research could be accelerated.

  6. Why can’t they use it on people with mild/moderate hearing impairment? Wouldn’t that be so cool if those who’ve got a little hearing problem can have their hearing restored (even a little bit)? That would open much more the “restrictive window” and patients would tell quickly if there is any hearing improvement…
    Might be a stupid question… but I don’t understand why they want to test it only on severe deaf people when others with mild/moderate hearing impairment could benefit too…

    1. I think the reason for that is because the people with mild-to-moderate loss can be helped by hearing aids whereas the people with severe to profound can’t be helped as well with them.

      1. I guess because experimental cures are not risk-free, and things might worsen rather than improve. While for a severre-profound loss, a further deterioration would not yield a great difference, this is not the case for a mild-to-moderate loss, for which a further loss could make a huge difference.

      2. Many people with mild/moderate hearing loss get tinnitus. Is it not possible to help them get rid of that by improving their hearing even if they can’t get their hearing back to 100% ?

  7. Good question. I do know of several hearing aids that help with tinnutus usually by masking it with an overlay of “white noise”. I will investigate.

  8. There is cellular repair and there is everything else. Everything else is Mickey Mouse. Staecker will be up for a Nobel if his upcoming paper is the real deal.

  9. There are many people intend to parcipate for the trial overseas. As the article says people who already had cohlear implentation are not qualified for thes trail. Fortunately , if the trial is opened internationally, then there are, I assume, many candidates who can participate for the trial

  10. I have ototoxicity I got it in the hospital when I was 24 years old I’m 26 years old now I have tried to get my doctor get ahold of Novartis w/my hearing records I have severe hearing loss bilateral this is absolutely the worst thing that’s ever happened to me,I just hope and pray everyday for a cure.

  11. My daughter is 3 year old she is delayed milestone she got 80decibal of hearing loss is this treatment done on kids too actually she do not wear hearing aid and I’m really worried about she do not speak either can you please where this treatment is done and also the cost??

    1. I went to Seattle and talked to a doctor that knows about genvec and Novartis he said he would absolutely not want me to go to the trials at all genvec and Novartis he said that it could cause a tumor or ur auditory nerve can be completely damaged I would check out mit hearing loss research

  12. Do tennagers with Noonan syndrome and profound hearing loss who do not have cochlear implant but a candidate, quality for this treatment if and when it is approved ?

  13. I am korean. I woked shipyard for a month. at that time the place was very noise. after that I am experiencing big pain from tinnitus.
    sometimes I wanna die. if possible I wanna join Novartis CGF166 trials. I will go to U.S if Novartis allow me as clinical patients.
    I have U.S visa. Help me. I can’t stand this suffering anymore.

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