Inner Ear Hair Cell Regeneration — Maybe We Can Know More

[cornelius]

I seriously doubt if health insurance will ever pay for this in the Netherlands. If it is not life threatening you can pay for it yourself or take out extra insurance that costs so much that in the end you will have paid for it yourself anyway. This is also why I already started saving. When in 10 years time something will be available I can have the the money.
I don't know what the situation with health insurers is in other countries.

I believe the price could be the same as current most expensive hearing aids in the best case or the price of a cochlear implant in the worst case. I will save too, though for me it's more a luxury (regaining the overall 15-20 db loss to be cured, making normally music again, hearing in noise along with speech clarity and maybe smoothing tinnitus) than your and other's emergency.

 

[Reinier]

specifically differentiated stem cells

And that is just the complicated part that has not yet been accomplished. This is the reason why I believe the different stem cell clinics are not there yet. It is not just using stem cells. This is why HHF, Action On Hearing Loss, Stanford etc...are still busy doing research.

 

[Reinier]

I believe the price could be the same as current most expensive hearing aids in the best case or the price of a cochlear implant in the worst case.

I have no idea how much a cochlear implant procedure costs.
I would Imagen that is something will be available initial prices will be very high.
Assume the Novartis/GenVec trials prove good enough to produce a medicine. I think it will be very expensive to receive treatment. Than when competition catches up prices could come down.

 

[RB2014]

I have no idea how much a cochlear implant procedure costs.

I'm pretty sure a cochlear implant is $100k+ per ear.

I'm agreed with Nic, that the best strategy would be to get the stem cells directly into the inner ear. My concern with an iv is that the stem cells might go some place they are not supposed to go and cause issues. I have seen rats with huge tumors that were created when they were experimented on possibly with some similar type of therapy. I think thats why we don't experiment on humans right now.

I'm still hoping that new genvec publication was a tweak to the existing formula and maybe its something that they use to complete the trials with?? If not I'm sure we will see new trials.

 

[Aaron123]

I'm pretty sure a cochlear implant is $100k+ per ear.

Depends on what is counted. Looking at a number of sites, the implant and surgery appear to average closer to $50,000 per ear. However, follow-up care apparently can increase this significantly. Costs for adults may also be different than infants - who receive a significant number of implants.

If a treatment appeared tomorrow that is better than CIs, I would expect it to cost more than CIs.

 

[Reinier]

I think thats why we don't experiment on humans right now.

Is what the stem cell clinics do not experimenting with people? It is not exactly clear why people get improvements after stem cell therapy. Does that not imply that it is experimental?

I'm still hoping that new genvec publication was a tweak to the existing formula and maybe its something that they use to complete the trials with?? If not I'm sure we will see new trials.

Yes good point. To what extend are they able to change this CGF-166 compound during the trial?
I assume GenVec is still researching this CGF-166. The way knowledge is gaining every year it would seem disastrous to start a clinical trial that could take 5-10 years and not be able to change the initial formula of the medicine.
When you know through ongoing research the medicine is able to be improved a lot, but you are not able to change the original formula and you need to start a whole new process with animal testing etc..
It surely doesn't work like that?

 

[Silvio Sabo]

Is what the stem cell clinics do not experimenting with people? It is not exactly clear why people get improvements after stem cell therapy. Does that not imply that it is experimental?

There are lots of things about the human body we don't fully understand but still mess around with. We actually have a pretty poor understanding of all the mechanisms in the human body. Because we have learned so much we think we have come along way. But if it's something I've learned while studying molecular biology it's that lot's of times it's really just poking in the dark.

We have developed drugs and treatments that we know work but don't fully understand why. Or at least didn't when we started using them. Aspirin and antibiotics where a mystery when your GP's around the corner started prescribing them. It doesn't make them experimental.

Experimental is when you are uncertain of the outcome, both positive and negative. Primarily you test something to see if it actually hurts someone. This is why all drugs are being tested on healthy individuals first. If you know that the outcome isn't negative or at least isn't in a vast majority of cases it's not really experimental. Don't confuse experiments with natural evolution of drugs and treatments. That is that they tend to improve over time because we learn more about them when they are used by many doctors on many people.

If you know how for instance computer software is developed it's pretty much the same thing there. Microsoft released Windows 10 about a year ago. Before that there were a lot of beta releases. Those were experimental. Does that mean that Windows 10 was perfect at release? No! There are literately hundreds or even thousands of bugs and errors that will get fixed over time and even new features will be added.

 

[Reinier]

There are lots of things about the human body we don't fully understand but still mess around with. We actually have a pretty poor understanding of all the mechanisms in the human body. Because we have learned so much we think we have come along way. But if it's something I've learned while studying molecular biology it's that lot's of times it's really just poking in the dark.

I am starting to realise that too.
This is why I am still reading with interest the different experiences from people doing LLLT and stem cell therapy.

But is there not a pivotal point? A point where knowledge starts to cascade. If you look at the time line of human existence I suppose this is happening at the moment (at least for the last 100 years). Where so much is already explained/understood about mechanisms in the body that processes are able to be predicted better al the time.
I suppose this is exactly what scientists are trying to do with the different hearing regeneration projects.
But also existing paradigms are still changed al the time.

I don't want to underestimate what research scientists are doing. I believe a cure or part cure will come from regular science. When there is an accidental discovery, regular science still needs to confirm the validity of a therapy.

 

[Silvio Sabo]

I am starting to realise that too.
This is why I am still reading with interest the different experiences from people doing LLLT and stem cell therapy.

But is there not a pivotal point? A point where knowledge starts to cascade. If you look at the time line of human existence I suppose this is happening at the moment (at least for the last 100 years). Where so much is already explained/understood about mechanisms in the body that processes are able to be predicted better al the time.
I suppose this is exactly what scientists are trying to do with the different hearing regeneration projects.
But also existing paradigms are still changed al the time.

I don't want to underestimate what research scientists are doing. I believe a cure or part cure will come from regular science. When there is an accidental discovery, regular science still needs to confirm the validity of a therapy.

I didn't mean to downplay our accomplishments. We have come a long way. We know a lot. What I meant to say is that we are far away from fully understanding everything about the human body. But that is due to the fact that our body is an overwhelmingly complex biological machine.

50 or 100 years from now I bet that people will look at us in this age of time in the same way we look at people from the 19th century. They will look at our surgeons as butchers and our doctors as medieval medicine men. We do know a lot about the big stuff like how our organs function and so on. But at the cellular level we still have a lot to learn. However that doesn't have to mean we can't make useful drugs and treatments that work. You don't have to know everything about how a drug works at the cellular level. If you test it and it works and it doesn't kill anyone, then that's enough for now. Knowledge is overrated if you ask me.

Another good example of something we use every day but don't really understand is anesthesia. There is still no accurate explanation for why it works or how it works. But I wouldn't say that doctors experiment every time they put a patient to sleep before surgery.

 

[tomytl]

Novartis (Genvec) got an ok to continue with the cgf166 trial.
http://ir.genvec.com/news/detail/1800

 

[Reinier]

Novartis (Genvec) got an ok to continue with the cgf166 trial.

Excellent news. I needed that today. My hyperacusis is debilitating again.
I am trying to ignore it but am not succeeding.

Quote: This recommendation was based on a review of safety and efficacy data from the nine patients currently enrolled in the study.
End quote.

So can we assume that safety and efficacy data looks good enough to continue? I should think so. Surely, If the trial did not show any effect they would not have continued the trial? Same with safety.

I still wonder what you can change during a clinical trial. Is it important that the compound CGF-166 is exactly the same throughout the clinical trial? I can Imagen it should. Or can you "tweak" it?
Researchers continue to gain insight into the regeneration mechanisms. Or is this trial so far ahead from ongoing research?
It is also possible I really do not understand what the whole biological process entails.

 

[RB2014]

Excellent news. I needed that today. My hyperacusis is debilitating again.
I am trying to ignore it but am not succeeding.

Quote: This recommendation was based on a review of safety and efficacy data from the nine patients currently enrolled in the study.
End quote.

So can we assume that safety and efficacy data looks good enough to continue? I should think so. Surely, If the trial did not show any effect they would not have continued the trial? Same with safety.

I still wonder what you can change during a clinical trial. Is it important that the compound CGF-166 is exactly the same throughout the clinical trial? I can Imagen it should. Or can you "tweak" it?
Researchers continue to gain insight into the regeneration mechanisms. Or is this trial so far ahead from ongoing research?
It is also possible I really do not understand what the whole biological process entails.

Great news Tomytl. Great find.

I really think they will just complete the trial with the CGF-166 formula. I'm hoping they can tweak it, but I'm pretty sure they would have to start a whole new trial for that. Even if they could, they probably want to collect as much data as they can with this formula so they could compare it to another down the road. Who knows, if they have a new formula, we might see another trial alongside this one.

I guess we really never figured out why they stopped the trial. I'm glad there were no adverse reactions. I'm pretty sure anything bad, related or not to the gene therapy, would have shut the entire clinical trial down as they really don't take chances anymore with Gene therapy.

 

[jdjd09]

Great news Tomytl. Great find.

I really think they will just complete the trial with the CGF-166 formula. I'm hoping they can tweak it, but I'm pretty sure they would have to start a whole new trial for that. Even if they could, they probably want to collect as much data as they can with this formula so they could compare it to another down the road. Who knows, if they have a new formula, we might see another trial alongside this one.

I guess we really never figured out why they stopped the trial. I'm glad there were no adverse reactions. I'm pretty sure anything bad, related or not to the gene therapy, would have shut the entire clinical trial down as they really don't take chances anymore with Gene therapy.

Seriously, I know someone around here has to be in contact with one of these participants.

I have randomly heard someone say that one of the participants may have gained 20-30db back in lost hearing.

Is this true? Can someone please give update on progress of one of the participants.

 

[RB2014]

Seriously, I know someone around here has to be in contact with one of these participants.

I have randomly heard someone say that one of the participants may have gained 20-30db back in lost hearing.

Is this true? Can someone please give update on progress of one of the participants.

I have been following this pretty closely and no numbers have been thrown out yet. I'm pretty sure the participants are going to be limited on what they are allowed to say as it would affect Genvec stock in a positive or negative way. We are all going to have to wait until 2017 unless someone spills the beans and I really don't think that will happen.

If you read back on this thread you will find some discussion on it, but its mostly one girl who said she is able to hear new sounds that she had not heard before. Other participants reported little to no change, but 20-30 db could be little to no change. No one can really tell the difference between a 40db loss and a 70db loss at 4k without an audiogram and no one has seen any of those.

They are doing thorough tests though and it will be exciting to see the results.

jdjd09, there were other companies too that have been discussed also doing work that got going around September/Oct of last year. No clinical trials yet, but the clock is ticking for them as they received funding and the investors are going to start wanting to see results quickly.

 

[Cillian]

@RB2014 spill the beans haha

 

[jdjd09]

I have been following this pretty closely and no numbers have been thrown out yet. I'm pretty sure the participants are going to be limited on what they are allowed to say as it would affect Genvec stock in a positive or negative way. We are all going to have to wait until 2017 unless someone spills the beans and I really don't think that will happen.

If you read back on this thread you will find some discussion on it, but its mostly one girl who said she is able to hear new sounds that she had not heard before. Other participants reported little to no change, but 20-30 db could be little to no change. No one can really tell the difference between a 40db loss and a 70db loss at 4kdbithout an audiogram and no one hdownen any of those.

They are doing thorough tests though and it will be exciting to see the results.

jdjd09, there were other companies too that have been discussed also doing work that got going around September/Oct of last year. No clinical trials yet, but the clock is ticking for them as they received funding and the investors are going to start wanting to see results quickly.

Can I ask what those companies names are? Is it really likely to have a hearing loss cure by 2025? One that can bring back hearing by 20-30db back?

Seriously , if someone could just point to some real evidence of that happening I would probably call down.

 

[RB2014]

Can I ask what those companies names are? Is it really likely to have a hearing loss cure by 2025? One that can bring back hearing by 20-30db back?

Seriously , if someone could just point to some real evidence of that happening I would probably call down.

https://decibeltx.com/
http://www.audiontherapeutics.com/
https://www.novartis.com/stories/discovery/can-we-unlock-bodys-ability-regenerate-lost-hearing

We know about the novartis trials going on right now.
The other two mentioned are actively working on hearing loss cures. Both got pretty serious at the end of last year both being backed by some major financing.
You still have all the work being done by Harvard, Stanford, the HHF, action on hearing loss, etc. It continues.

To me its really never looked brighter than it is today.

@RB2014 spill the beans haha

I wish I had beans to spill.

 

[jdjd09]

https://decibeltx.com/
http://www.audiontherapeutics.com/
https://www.novartis.com/stories/discovery/can-we-unlock-bodys-ability-regenerate-lost-hearing

We know about the novartis trials going on right now.
The other two mentioned are actively working on hearing loss cures. Both got pretty serious at the end of last year both being backed by some major financing.
You still have all the work being done by Harvard, Stanford, the HHF, action on hearing loss, etc. It continues.

To me its really never looked brighter than it is today.


I wish I had beans to spill.

What year should a teatable cure come out? I just want someone to give a timeline....I'm just tired of doctors playing it so safe. They have a timeline internally. All of them do. You don't invest all this money in a treatment with no goal or timeline.

 

[RB2014]

What year should a teatable cure come out? I just want someone to give a timeline....I'm just tired of doctors playing it so safe. They have a timeline internally. All of them do. You don't invest all this money in a treatment with no goal or timeline.

There are no timelines right now, but greed makes the world go around. The first one to the market with any type of a benefit or a cure is going to reap some pretty big rewards. This is capitalism at its best right now. I'd say 10 years or less is dooable right now at least for a partial cure. A lot of these companies only have funding for maybe 2-6 years so something has to happen quickly for them, and I think there is some pressure for them to get this figured out. Investors arent patient when it comes to getting back their money on an investment.

 

[Reinier]

No one can really tell the difference between a 40db loss and a 70db loss at 4k without an audiogram and no one has seen any of those.

From experience I can tell you will notice
I know I am fixated at the moment and I always have been when it relates to hearing and sounds.
6 dB being double, 20-30 dB people will notice!
I realise if it is one ear it will not be so noticeable in everyday life. But if it is two ears or you plug the good ear I am certain you will notice this gain (or loss) in threshold.

 

[tomytl]

What year should a teatable cure come out? I just want someone to give a timeline....I'm just tired of doctors playing it so safe. They have a timeline internally. All of them do. You don't invest all this money in a treatment with no goal or timeline.

Hi, there are no timetables likebothers said before. All the biotechs got financial investment, but they are relatively small ones.
You can see it with Genvec and Novartis partnership, at least, Novartis can just win if they clould transfer those findings in cfg166 trial into a standard therapy.
Novartis injected some few milionsinto Genvec, that's almost nothing for conpany with this market power.

Also decibeltx and audiontx got relatively small financial, this is all on an experimental stage. If the there is a proof if concept, the big pharmas will start heavy to invest.
I can imagine, if they have a blockbuster, alone the marketing and the "get most out of it" strategy will take some years...
But as always, this is my view of things and might be conpletly wrong, because I'm not a professional, not in research and not in pharma market. I am just a sufferer.
But good Novartis can go ahead....

 

[Silvio Sabo]

Hi, there are no timetables likebothers said before. All the biotechs got financial investment, but they are relatively small ones.
You can see it with Genvec and Novartis partnership, at least, Novartis can just win if they clould transfer those findings in cfg166 trial into a standard therapy.
Novartis injected some few milionsinto Genvec, that's almost nothing for conpany with this market power.

Also decibeltx and audiontx got relatively small financial, this is all on an experimental stage. If the there is a proof if concept, the big pharmas will start heavy to invest.
I can imagine, if they have a blockbuster, alone the marketing and the "get most out of it" strategy will take some years...
But as always, this is my view of things and might be conpletly wrong, because I'm not a professional, not in research and not in pharma market. I am just a sufferer.
But good Novartis can go ahead....

Decibel Therapeutics (http://decibeltx.com/) are quite well funded. They have obtained $52 million from investors about 6 months ago. The main investor is Third Rock Ventures (http://www.thirdrockventures.com/).

 

[RB2014]

Decibel Therapeutics (http://decibeltx.com/) are quite well funded. They have obtained $52 million from investors about 6 months ago. The main investor is Third Rock Ventures (http://www.thirdrockventures.com/).

I don't think 52 million dollars gets you to far these days. If you take 100k a year salaries with a 3x multiplier for benefits and only 20 employees thats a 6 million dollar burn rate a year, plus office space, plus expensive equipment, plus lab supplies. Maybe 10 million a year burn rate a year. I think they said 3 years for clinical trials, which will take 2-3 years to complete. They might have 5-6 years.
I remember at some point an article said Novartis put 500 million into their hearing loss project, but we really haven't heard anything new from them. I have a feeling they might have pulled back a bit to let research keep advancing, but who really knows.

 

[Silvio Sabo]

I don't think 52 million dollars gets you to far these days. If you take 100k a year salaries with a 3x multiplier for benefits and only 20 employees thats a 6 million dollar burn rate a year, plus office space, plus expensive equipment, plus lab supplies. Maybe 10 million a year burn rate a year. I think they said 3 years for clinical trials, which will take 2-3 years to complete. They might have 5-6 years.
I remember at some point an article said Novartis put 500 million into their hearing loss project, but we really haven't heard anything new from them. I have a feeling they might have pulled back a bit to let research keep advancing, but who really knows.

This is startup money. I imagine they will acquire more money in the future. Autifony got about 8 million to do the trial for AUT0063. The minds behind Decibel Therapeutics are all Harvard professors that have published a lot in the field of tinnitus and hearing loss. I expect big things coming out from there in the future.

 

[Aaron123]

Only half of the founders are Harvard affiliated:

M. Charles Liberman, Ph.D.
Schuknecht Professor of Otology and Laryngology at Harvard Medical School and Director of the Eaton-Peabody Laboratory at Massachusetts Eye and Ear

Gabriel Corfas, Ph.D.

Director of the Kresge Hearing Research Institute, University of Michigan

Ulrich Müller, Ph.D.

Kershaw Professor of Neuroscience; Chair, Department of Molecular & Cellular Neuroscience; Director, Dorris Neuroscience Center; and Member, Skaggs Institute for Chemical Biology at theScripps Research Institute

Albert Edge, Ph.D.

Professor of Otology and Larynology, Harvard Medical School and Director, Tillotson Cell Biology Unit, Massachusetts Eye and Ear

 

[RB2014]

This is startup money. I imagine they will acquire more money in the future. Autifony got about 8 million to do the trial for AUT0063. The minds behind Decibel Therapeutics are all Harvard professors that have published a lot in the field of tinnitus and hearing loss. I expect big things coming out from there in the future.

I am totally agreed with you. I have high hopes for them as well. I'm looking forward to seeing what they come up with. As long as they can get more money later, then yes I see great things happening. If they set up a donation page, I would be giving to them as well.

Its good to see this sizable of an investment, but was only commenting that 52 million can be spent pretty quickly.

I'm strongly considering signing up for their clinical trials once they get going unless some really good news comes out of the Novartis trials.

 

[jdjd09]

I am totally agreed with you. I have high hopes for them as well. I'm looking forward to seeing what they come up with. As long as they can get more money later, then yes I see great things happening. If they set up a donation page, I would be giving to them as well.

Its good to see this sizable of an investment, but was only commenting that 52 million can be spent pretty quickly.

I'm strongly considering signing up for their clinical trials once they get going unless some really good news comes out of the Novartis trials.

Anyways, do they have a goal of curing hearing loss as well due to noise? What companies are aimed at this and realistically could bring back hearing in 10 years.

 

[Silvio Sabo]

Only half of the founders are Harvard affiliated:

M. Charles Liberman, Ph.D.
Schuknecht Professor of Otology and Laryngology at Harvard Medical School and Director of the Eaton-Peabody Laboratory at Massachusetts Eye and Ear

Gabriel Corfas, Ph.D.

Director of the Kresge Hearing Research Institute, University of Michigan

Ulrich Müller, Ph.D.

Kershaw Professor of Neuroscience; Chair, Department of Molecular & Cellular Neuroscience; Director, Dorris Neuroscience Center; and Member, Skaggs Institute for Chemical Biology at theScripps Research Institute

Albert Edge, Ph.D.

Professor of Otology and Larynology, Harvard Medical School and Director, Tillotson Cell Biology Unit, Massachusetts Eye and Ear

I didn't say all of them were. I believe my statement was "the minds behind it". Now we can bicker about the percentage of staff and their previous or current affiliations or we can just say that since most of them (excuse me, HALF of them) are from Harvard and the lead researcher is Charles Liberman it's most likely that research that originates from what they have done at Harvard is what will be their main platform.

And btw if you google Gabriel Corfas you will find that he indeed is a former member of the Harvard faculty as well. Have a look at this page: http://www.hms.harvard.edu/dms/neuroscience/fac/corfas.php

 

[Aaron123]

Now we can bicker about the percentage of staff and their previous or current affiliations or we can just say that since most of them (excuse me, HALF of them) are from Harvard and the lead researcher is Charles Liberman it's most likely that research that originates from what they have done at Harvard is what will be their main platform.

Sorry, I didn't mean to come across as critical. Just wanted to note current affiliations. Thanks for the link about Corfas. Do you know if Müller has a past link to Harvard?

 

[Silvio Sabo]

Sorry, I didn't mean to come across as critical. Just wanted to note current affiliations. Thanks for the link about Corfas. Do you know if Müller has a past link to Harvard?

I didn't find anything about him being employed at Harvard however I found that he had held lectures there so he has had some connection to Harvard as well from what I could tell.