204 replies to this topic

[niner]

Over at Reason's site, he said that BioViva performed gene therapy for telomerase activation.  Presumably they used an AAV vector to add htert.  There's some info in the comments, stating the following:

 

Nicolai said:

 

 

You forgot to mention the other treatment they applied, the myostatin inhibitor AAV gene therapy

 

And Steve h said:

 

 

To clarify the two therapies in question are htert and a proprietary myostatin inhibitor that has in animal data and anecdotal human data shown to reduce atherosclerosis. The hope is the combination has synergy to rejuvenate stem cells and combat Atherosclerosis as well as muscle wastage.

 

I'm not sure what they mean by a myostatin "inhibitor".  Wouldn't they need to reduce the expression of myostatin?   Is there an endogenous protein inhibitor of myostatin that could be inserted?   Anyone know what they actually did?  "Anecdotal human data shown to reduce atherosclerosis?"   I wonder where that happened?

[roamer7]

Interesting stuff. It begs the question... if this works, how much would YOU pay to shave 20 years or more off of your bilogical age? Would you re-mortgage your home for it?.. cash in retirement savings?... I know at nearly 50 I'd be prepared to put off retirement for a decade or so to be 30 again...

 

 

[niner]

Interesting stuff. It begs the question... if this works, how much would YOU pay to shave 20 years or more off of your bilogical age? Would you re-mortgage your home for it?.. cash in retirement savings?... I know at nearly 50 I'd be prepared to put off retirement for a decade or so to be 30 again...

 

Just as a ballpark number, $50,000-100,000 per life-year is around the point where interventions are considered to be "worth it" when talking about things like screening programs.  I really doubt that you'd get 20 years from the current BioViva experiment, but assuming you had something that would give you 20 years, it could be logically priced at one to two million dollars.  I don't think that individual medical decisions are typically made this way, though.  These days, the pharmaceutical industry is pricing some small-volume therapies at levels of $100,000 to $300,000 per year.  On the other hand, people die all the time for want of basic medical care. 

 

A treatment that added 20 years would save governments a fortune in health care costs, and if it was coupled with a 20 year increase in the retirement age, would save them a fortune in pension payouts.  It would thus be worthwhile for the government to pick up the cost of the treatment, if it wasn't crazy expensive.  Since it would be a very high volume treatment (most people would want it), there would be a large economy of scale.  If it were something like the current BioViva experiment, I don't think it would be very expensive.

[resveratrol_guy]

I really don't think any of this is about mass market life extension. In order to do that, you need to attack all the common causes of aging; otherwise, it's just a "weakest link" problem. That said, I'd be thrilled if they could do so much as to restore skin elasticity and remove age spots.

 

I also don't think another a gene therapy disaster (like the tragic adenovirus gene therapy fatality, not to be confused with adeno-associated virus therapy) would cripple BioViva, because at least they seem to be intimately aware of the fact that the US is a nonstarter for biotech trials. I'm sure they would just turn to offshore options once again, after they thought that they had fixed the problem.

 

I agree that they're pretty light on technical details, but I don't think they're dumb enough to be as bombastic as they are without having the animal data to back them up. After all, they have no publicly traded stock to pump, and no product to sell, if it's all smoke and mirrors.

 

[Logic]

http://www.longecity.../77748-bioviva/

 

"...I feel that most here are missing a very important point in that the long telomere ends of the DNA strand curl round and affect genes closer to the center of the strand in a epigenetic way.
So its not simply a case of trying to keep the critically short telomeres from causing 'glitches in the program'.
There are also other positive effects from longer telomeres..."

[alc]

 "Nevertheless, alc, you rock for sharing this!"

 

I have NO merit in this - I just set up an alert on Google and once notified I posted here.

 

You can do it with whatever research interests you, just set up the alert and once new articles are available you will get the notification.

 

It is simple.

 

For me reverse aging became an "obsession" for past years and obvious I've set up an alert for that.

 

[alc]

"Just as a ballpark number, $50,000-100,000 per life-year is around the point where interventions are considered to be "worth it" when talking about things like screening programs.  I really doubt that you'd get 20 years from the current BioViva experiment, but assuming you had something that would give you 20 years, it could be logically priced at one to two million dollars.  I don't think that individual medical decisions are typically made this way, though.  These days, the pharmaceutical industry is pricing some small-volume therapies at levels of $100,000 to $300,000 per year.  On the other hand, people die all the time for want of basic medical care."

 

A treatment that added 20 years would save governments a fortune in health care costs, and if it was coupled with a 20 year increase in the retirement age, would save them a fortune in pension payouts.  It would thus be worthwhile for the government to pick up the cost of the treatment, if it wasn't crazy expensive.  Since it would be a very high volume treatment (most people would want it), there would be a large economy of scale.  If it were something like the current BioViva experiment, I don't think it would be very expensive.

 

 

Liz Parish talks in one of her interviews about "how much will cost" once the platform is up and running. Figures are much more reasonable based on her comments and I do hope that is the case. 300k/year is not feasable and we will end up with a scenario like in the Elysium movie (which of course, can be the case).

 

One thing to keep in mind is that as technology evolve, this "reverse aging" will become more mainstream, including home-brew technologies.

 

And yes, if somebody in the government realizes that such treatment will actually reduce A LOT the health care costs and they actually will put MORE money in our reserve, than that will be a seller, and this field will ignite for good.

 

 

[corb]

Just as a ballpark number, $50,000-100,000 per life-year is around the point where interventions are considered to be "worth it" when talking about things like screening programs.

 

That's too steep considering what the therapy entails.

This is not something that can't be mass produced. If it proves useful it will become equitable soon enough.

[niner]

 

Just as a ballpark number, $50,000-100,000 per life-year is around the point where interventions are considered to be "worth it" when talking about things like screening programs.

 

That's too steep considering what the therapy entails.

This is not something that can't be mass produced. If it proves useful it will become equitable soon enough.

 

If a therapy amounted to nothing more than an injection of genetically modified viri, that would cost next to nothing in quantity.   I expect many life extension therapies to be relatively cheap infusions.   The current hand-wringing over "equity" (despite the fact that the world doesn't seem to mind that millions lose decades of life for want of basic medical care) will probably fade away once the therapies are being delivered in large numbers.

[alc]

I'm not sure what they mean by a myostatin "inhibitor".  

 

 

 

Just do a simple Google search:

 

https://www.google.c...ibitor"&tbm=nws

 

typically I look at "news" so recent results come up.

 

... and from PubMed.gov you get several articles:

 

"Myostatin inhibitors as therapies for muscle wasting associated with cancer and other disorders"

 

http://www.ncbi.nlm....les/PMC3819341/

 

"Antibody-directed myostatin inhibition enhances muscle mass and function in tumor-bearing mice."

 

http://www.ncbi.nlm....pubmed/21677277

 

... etc ...

 

There are lots of companies going this path:

 

like Acceleron Pharma

 

www.acceleronpharma.com

 

http://www.acceleron...-2494-wms-2015/

also, a while ago I was looking at Myos Corp. run by Robert Hariri:

http://www.tedmed.co...rs/show?id=6461

 

and I'm trying to understand better what path they are going:

 

http://www.myoscorp.com/

 

http://www.myoscorp....clinical-trials

 

 

[Steve H]

Myostatin gene therapy has also recently been used in kids with beckers muscular dystrophy and with good success. This I'd now moving to Duchenne muscular dystrophy. Both are at nationwide children's hospital and are on clinical trials . Gov.

There is some rodent data suggesting myostatin inhibiting (at least with the gene they use) helps with plaques, will the results translate to humans? Don't know but they want to find out. Allegedly their MD and another patient both took this therapy some time ago with positive results. I am trying to obtain the cardiogram data for this.

Will htert yield similar or better results than animal and human cell tests? If you agree with the Dr Michael fossel school of thought then very likely. If you are more sens then not so much bar mobilizing stem cells to spur some repair.

I will add that targeting the microglial cells with hrert is not as outlandish as it seems. Rodent data has shown association between htert and tau pathology. The best explanation as to why it might work is given by Dr fossel on his website.

I can tell you that bioviva went for whole body induction as the funding was offered. They use AAV 2 for delivery and they recently recruited Dr avi Roy who has considerable knowledge in regenerative medicine.

End of the day I think it could lead to regeneration and life extension but what I think isn't important, data is king. It's a waiting game now, I just hope they did everything right.

[Steve H]

I notice telocyte is now live. They are using the same proposed approach bioviva is using to treat the microglial cells. They are working with Dr Maria blasco from the cnio in Madrid, they have a lot of clinical and in Michael's case medical expertise too. Michael has talked about bioviva in interviews recently too the main difference being he wants to go via fda and take the harder road and bioviva want to get results sooner not in a decade.

http://www.telocyte.com
I notice telocyte is now live. They are using the same proposed approach bioviva is using to treat the microglial cells. They are working with Dr Maria blasco from the cnio in Madrid, they have a lot of clinical and in Michael's case medical expertise too. Michael has talked about bioviva in interviews recently too the main difference being he wants to go via fda and take the harder road and bioviva want to get results sooner not in a decade.

http://www.telocyte.com

[resveratrol_guy]

I notice telocyte is now live. They are using the same proposed approach bioviva is using to treat the microglial cells. They are working with Dr Maria blasco from the cnio in Madrid, they have a lot of clinical and in Michael's case medical expertise too. Michael has talked about bioviva in interviews recently too the main difference being he wants to go via fda and take the harder road and bioviva want to get results sooner not in a decade.

http://www.telocyte.com
 

 

"In animal trials, we can not only prevent, but reverse CNS dysfunction [via telomerase gene therapy on microglia]." Really? I don't see any references in their bibliography. And their front page investment solicitation doesn't give me good vibes. But as always, I won't throw them out just because they look scammy. But where's the rat study?

 

The closest I found was this study with this graph showing the effect of their broad-spectrum "telomerase-increasing compound", AGS499. What I haven't managed to dig up is something that says restoring telomere length in the microglia can reverse any particular degree of Alzheimer's in rats, or something like that.

 

If Telocyte is for real, then I don't blame them for scrambling to find investors. All that FDA paperwork is going to cost a mint (and prohibit the practical implementation of any such therapy, but that's another matter entirely). Sounds like someone needs to introduce them to BioViva so they can actually get some work done overseas.

[Steve H]

This very recent paper gives a hint of the protective quality of TERT against Tau Pathology.

 

http://www.ncbi.nlm....les/PMC4308607/

 

I can see why Dr Fossel thinks activating TERT and lengthening telomeres will help encourage the microglia to clean up the plaque. He explains the reasoning behind this well in his book cells, aging and Human disease which you can read free on google books. 

 

Telocyte are working with the Spanish CNIO (the national Government cancer lab in Madrid) and in particular with Dr Maria Blasco who among various things heads up the telomere biology department, she is developing a cancer treatment that unravels telomeres via Shelterin and is highly skilled in the field and has been involved in a number of telomerase regeneration therapies.

 

We have seen evidence in the Rando experiments that telomerase induction improves cognitive function, this could be due to this action or perhaps due to mobilized stem cells in the niche returning to function after dormancy.

 

I understand that Telocyte already has funding to get fairly far into clinical trials and are approaching the FDA for an IND application. 

 

Michael has indeed talked about Bioviva and is a friend of Liz Parrish but I think working together might damage Telocytes chances with the FDA so I think a collaboration is unlikely. 

I just found out there is an AMA with Liz Parrish on 11th on Reddit at 6pm UTC https://redd.it/3ncujs

 

 

[Logic]

https://plus.google....524942941/posts

 

Contains a good # of BioViva videos.

[resveratrol_guy]

This very recent paper gives a hint of the protective quality of TERT against Tau Pathology.

 

http://www.ncbi.nlm....les/PMC4308607/

 

I can see why Dr Fossel thinks activating TERT and lengthening telomeres will help encourage the microglia to clean up the plaque. He explains the reasoning behind this well in his book cells, aging and Human disease which you can read free on google books. 

 

Telocyte are working with the Spanish CNIO (the national Government cancer lab in Madrid) and in particular with Dr Maria Blasco who among various things heads up the telomere biology department, she is developing a cancer treatment that unravels telomeres via Shelterin and is highly skilled in the field and has been involved in a number of telomerase regeneration therapies.

 

We have seen evidence in the Rando experiments that telomerase induction improves cognitive function, this could be due to this action or perhaps due to mobilized stem cells in the niche returning to function after dormancy.

 

I understand that Telocyte already has funding to get fairly far into clinical trials and are approaching the FDA for an IND application. 

 

Michael has indeed talked about Bioviva and is a friend of Liz Parrish but I think working together might damage Telocytes chances with the FDA so I think a collaboration is unlikely. 

I just found out there is an AMA with Liz Parrish on 11th on Reddit at 6pm UTC https://redd.it/3ncujs

 

Let me echo that: check out the Liz Parrish link quoted above. It's an upcoming live Q&A on Reddit.

 

Awesome paper! A few highlights...

 

"Intriguingly, hippocampal neurons expressing TERT did not contain hyperphosphorylated tau. Vice versa, neurons that expressed high levels of pathological tau did not appear to express TERT protein." That's amazing!

 

But... "However, there is little information regarding TERT expression in human brain tissue, aside from its strong upregulation in brain tumors."

 

This is back to the old enough-growth-but-not-too-much problem. The takeaway I get is (1) use TERT as soon as you manifest memory loss (i.e. while it's still sort of repairable) but (2) coadminister anticancer compounds which target brain tumors, e.g. c60oo, pterostilbene, ashitaba, etc.

 

Also fascinating: "Bands were quantified and normalized to neuronal βIII-tubulin and revealed that TERT expression did not differ between controls and cases with Braak Stage I-VI... these data suggest that there does not seem to be a change in TERT protein levels in hippocampal neurons in the early and intermediate Braak stages of tau pathology or in AD... Interestingly, we did not find any overlapping of the TERT and AT8 signals, suggesting that TERT expression and the presence of tau pathology are mutually exclusive in neurons of the hippocampus." -- Wow! You can see this visually here, where there are red spots (TERT) and green ones (AT8), but no yellow ones (overlaps). I think this is saying that once you're an adult, if not at birth, the percentage of your neurons expressing TERT is essentially fixed; even Alzheimer's does not change this. So if I have fewer TERT-expressing neurons, I'm at higher risk of AD. But conversely, if I just dial up that percentage using BioViva's or Telocyte's nonexistent therapy, I downthrottle my risk of AD rather effortlessly, if not actually reverse some of the disease in progress. The only price is the murky risk of a brain tumor.

[resveratrol_guy]

https://plus.google....524942941/posts

 

Contains a good # of BioViva videos.

 

Thanks, Logic. This interview in particular has convinced me that Liz Parrish has the makings of a biotech Elon Musk. It's a highly recommend use of your next free hour and 17 minutes. Rich friends, please consider investing in their Alzheimer's trial as described in the video.

Edited by resveratrol_guy, 09 October 2015 - 01:32 AM.

[Steve H]

You may also find this excellent review from this year fairly interesting again showing the link between AD and telomerase/TERT.

http://www.researchg...mere_shortening

 

The referenced studies in the review are very interesting though reading it all is a major undertaking. The conclusion is a somewhat mixed bag but to me suggests there is merit in exploring TERT/Telomerase as a therapy. It also highlights the varied functions of telomerase and why average telomere length, critically short telomeres, telomerase interaction with non telomere mechanics and telomeres influence on gene expression could all be various drivers of ageing. It also picks up on the P53-PCG-1-telomere axis of ageing indentified by DePhino, this pathway interacts with mitochondria and metabolism and ultimately leads to stem cell decline via dysfunction telomeres.

It makes me wonder if rejuvenating resident stem cells with telomerase followed up by importing of fresh stem cells in artificial niche (ala RepleniSENS) would yield good results and allow the new stem cells to engraft and overcome the old system inhibiting them. Two new artificial stem cell niche technologies have recently been demonstrated and they showed a significant increase in engrafting rate and resistance to inhibition by native aged stem cells resulting in rejuvenation. 

 

 

Edited by Steve H, 09 October 2015 - 06:45 AM.

[resveratrol_guy]

You may also find this excellent review from this year fairly interesting again showing the link between AD and telomerase/TERT.

http://www.researchg...mere_shortening

 

The referenced studies in the review are very interesting though reading it all is a major undertaking. The conclusion is a somewhat mixed bag but to me suggests there is merit in exploring TERT/Telomerase as a therapy. It also highlights the varied functions of telomerase and why average telomere length, critically short telomeres, telomerase interaction with non telomere mechanics and telomeres influence on gene expression could all be various drivers of ageing. It also picks up on the P53-PCG-1-telomere axis of ageing indentified by DePhino, this pathway interacts with mitochondria and metabolism and ultimately leads to stem cell decline via dysfunction telomeres.

It makes me wonder if rejuvenating resident stem cells with telomerase followed up by importing of fresh stem cells in artificial niche (ala RepleniSENS) would yield good results and allow the new stem cells to engraft and overcome the old system inhibiting them. Two new artificial stem cell niche technologies have recently been demonstrated and they showed a significant increase in engrafting rate and resistance to inhibition by native aged stem cells resulting in rejuvenation. 

 

 

You've presented a wealth of compelling evidence. So I can see the connection between AD and telomeres. But one thing I don't get about the Telocyte and BioViva approaches is how they intend to create a wide effect radius with a few localized injections. If the viral vector doesn't aggressively replicate, then how could it be expected to influence a volume of brain tissue much larger than the size of the injected vehicle fluid? This is actually a problem with to the CERE-110 virally encoded NGF trial; they "solved" the problem by injecting only the hippocampus, in the hopes that the NGF produced would influence the entire brain by diffusion. While they might be right, it's much harder to see this with TERT, because we're not looking for TERT to migrate around the CNS by diffusion; rather, as I understand it, we want to edit neuronal DNA so as to cause the expression of TERT on the cell membrane. So does this mean that even if the trials succeed, they will only affect as much of the brain as can be reached by the injected fluid? What am I missing here?

[Steve H]

It is a good question why not go to the AMA on the 11th October and ask? 6pm UTC or 11am PDT.

https://www.reddit.c...py_to/#AMApromo

[resveratrol_guy]

It is a good question why not go to the AMA on the 11th October and ask? 6pm UTC or 11am PDT.

https://www.reddit.c...py_to/#AMApromo

 

I'm now more confused about their proposed approach than I was before the AMA. The only certainty is that you can replace "First Patient" with "Liz Parrish" in the title of this thread.

 

Cut to the chase: the only result reported thus far is: "I have actually been happier, more active and sleep better." As in, we're squarely within the placebo envelope. It's going to be months of worse sleep for the rest of us, while we wait to see if this pays off for her.

 

Nevertheless, it's well worth a read. Here is the transcript.

Edited by resveratrol_guy, 11 October 2015 - 08:35 PM.

[Steve H]

Cut to the chase: the only result reported thus far is: "I have actually been happier, more active and sleep better." As in, we're squarely within the placebo envelope. It's going to be months of worse sleep for the rest of us, while we wait to see if this pays off for her.

 

 

Tell me about it, going to be six months to a year to process initial data to see if it works. Seat of the pants flying as they say!

[corb]

 

Cut to the chase: the only result reported thus far is: "I have actually been happier, more active and sleep better." As in, we're squarely within the placebo envelope. It's going to be months of worse sleep for the rest of us, while we wait to see if this pays off for her.

 

 

This is more or less "trialing" for safety anyway.
They've proven she didn't drop dead the second she got the therapy so that's about good as it can get in the short term.

[niner]

Well, that was a surprise!  When you think about it though, she was the only logical choice to be patient zero.  If they'd used anyone else, the story would have been "heartless company risks life of desperate volunteer".  Instead, now it's "Brave, plucky businesswoman risks her life for the benefit of humankind."  Surprise number two:  Liz is 45?  Wow, I thought she was in her twenties.  I expect the media to get hold of this.  I wonder what's going to happen there?  It's a great story and Liz is telegenic and relateable.

[reason]

BioViva is a small group that recently announced they have moved ahead with a human test of telomerase and myostatin-related gene therapies as a potential method to modestly slow the effects of the aging process. Their initial goals are to get things moving in this part of the field by taking this step forward, observing the results, and raising funding for further development efforts to try to lower the costs of this sort of approach. The BioViva CEO Liz Parrish, who is also the initial test subject, recently hosted an AMA (ask me anything) event at Reddit's /r/futurology community. Her comments below are lightly edited for continuity, since they are pulled from numerous distinct answers to questions posted by the community:

I am patient zero. I will be 45 in January. I have aging as a disease. To take on this role myself was the only ethical choice. I am happy to step up. I do feel we can use these therapies in compassionate care scenarios now but we will have to work them back into healthier people as we see they work as preventive medicine.

The genes targeted are human telomerase reverse transcriptase (hTERT) and follistatin (FST). In animal models neither FST nor hTERT have increased the risk of cancer. We expect to see the same result on myself, and to that effect we are measuring all known cancer biomarkers. The gene therapies on my body are to measure the effects on humans. There is plenty of animal research to support these gene therapies but no one was conducting human tests. We are using both visual biomarkers, MRI and a panel of blood and tissue testing including work on telomere length and epigenetic testing. We are collecting as much data as we can, but unfortunately we currently don't have the coverage rate for this therapy, how much of the tissue of the body is affected. Depending on the tissue and vector used we ultimately expect to see similar rates of transfection as seen in mice, which is somewhere between 5 to 60%.

We are working as hard as we can to bring it to the world as quickly and safely as possible. We will will evaluate monthly and within 12 months we will have more data. If the results are good we hope to have something to the general public, that is cost acceptable, in 3-5 years. Our goal is to build laboratories that will have the mission of a gene therapy product at a reduced cost. Gene therapy technology is much like computing technology. We had to build the super computer which cost $8 million in 1960. Now everyone has technologies that work predictably and at a cost the average person can afford. We need to do the same with these therapies. What you will get in 3-5 years will be vastly more predictable and effective that what we are doing today and at a cost you or your insurance can cover .

We need a lab that works solely to bringing those costs down. We would need about $1 - 1.5 million to build one lab to focus on this. We can expand as needed. I would love to crowdfund this project but I do not know how to get good results at that scale - I think the price tag is high for that modality. We are raising investment to do offshore clinical trials. Many USA companies do this. If we can cut costs we will be able to bring back a treatment that people can afford.

Link: https://www.reddit.c...of_bioviva_the/

View the full article at FightAging

[Steve H]

Agreed Niner. I am thinking if the data is positive its something Longecity may want to support too. I know they want to test TERT with Longecity so this might be the data that convinces them its a good idea. She is certainly very brave to do this but she is willing to put her life on the line for progress which is admirable. I imagine if the results are good a larger test trial would be the next step.

[resveratrol_guy]

 I know they want to test TERT with Longecity so this might be the data that convinces them its a good idea.

 

Can you elaborate on this? Is there a thread somewhere where this discussion took place? Where's the signup form?

 

One of the central questions in TERT therapy, as BioViva is proposing it, is tissue locality. If you read the transcript, Parrish says that the effects are systemic; however, systemic delivery requires several different virusses, presumably because different ones localize more to different tissues. I assume this is why, for instance, they need a special Alzheimer's procedure to penetrate the CNS, even though it's essentially the same treatment. It remains to be seen which of her organs receives the most benefit.

 

Yes, she looks very young relative to 44. (She's a vegetarian.) Obviously she's not an ideal candidate for assessing the benefits of this procedure, although I understand the ethical and political reasons why she would want to be the guinea pig.

[sthira]

I'm sure many of us would take these supervised, controlled, documented injections ourselves ("put our lives on the line") if provided the opportunity. I know I would. So I'm not sure how brave she is. More like she's lucky. She seems cool, though, answering all those Reddit questions. Hopefully we'll all learn about slowing down the aging process from her experiences, and the company will be honest, upfront, and keep costs down for the rest of us. Maybe even in less than the proverbial 2 - 5 years?

Unsure if anyone else has posted this, but she's good :-)

Edited by sthira, 12 October 2015 - 05:58 PM.

[corb]

One of the central questions in TERT therapy, as BioViva is proposing it, is tissue locality. If you read the transcript, Parrish says that the effects are systemic; however, systemic delivery requires several different virusses, presumably because different ones localize more to different tissues. I assume this is why, for instance, they need a special Alzheimer's procedure to penetrate the CNS, even though it's essentially the same treatment. It remains to be seen which of her organs receives the most benefit.

 

The delivery method is far from ideal and it will change in the future. The reliance on viral vectors is the main problem with current gene therapies in general.

[niner]

Maybe even in less than the proverbial 2 - 5 years?

 

I think the proverbial timespan is closer to 10 years.  Maybe they could do it in less if the treatment includes a plane ticket to another country, for those of us in the US.  But they are a long way from showing that the treatment is safe in humans and that it does something useful.  This is kind of a Phase Zero trial.  The early-adopter customers probably wouldn't care if it was FDA approved or not, but they will want to have some evidence that it works at least reasonably well and isn't going to kill them.  It's going to take a lot of work and money to get from here to there.  Specifically, I think they will ultimately need to take it through the equivalent of FDA's phase 2, as well as probably doing a lot of work on optimizing the protocol.  To do all of this they will need some luck and a lot of money.