Biotech CEO claims she is the first to undergo gene therapy to reverse aging
technologyreview.com
technologyreview.com
The previous posters final comment is the critical one: let's hope she hasn't given herself cancer. Previous gene therapy trials were all stopped because of that risk. Current Crispr techniques (if that's what she used) aren't specific enough to avoid off target effects, those could easily cause cancer.
I tend to be quite pro DIY medicine, but this still seems quite rash to me because of those risks.
In your example, an immortal cell who cannot repair DNA or even identify damaged DNA wouldn't be that dangerous, since it doesn't have the signal to reproduce wildly.
See D. Hanahan and R. Weinberg "Hallmarks of Cancer: The Next Generation" for a scholarly (and quite readable) review http://www.sciencedirect.com/science/article/pii/S0092867411...
The gene therapies used are well exercised in animal studies. At some point you have to move to humans, but that costs money. This is one approach to raising the funding to perform small trials, you get the first trial member done first since that's what you have the funds for, and move on from there.
Parrish has been a member of the community interested in aging and biotech for some years. Look at the the advisory board of BioViva. You don't magically get George Church on your board without having paid your dues.
Meanwhile if you happen to be someone who disagrees with the existence and current practice of the FDA - and this may be a minority position, but it isn't a small minority position - then linking this all with regulatory arbitrage makes perfect sense. There is an enormous gap between what can be done safely (large set) and what the FDA permits (small set), and with the cost of biotechnology and medicine falling rapidly, sooner or later something has to give.
The FDA is all about maintaining the position of FDA bureaucrats. That's the only real way to interpret the 2.5x cost increase in trials over the past decade. They are willing to suppress as much medical progress as it takes in order to look like people who are doing a non-phony-baloney job. But medicine is inherently risky. There will always be people who die as a result of new therapies: it should be up to the patients to make informed decisions, not a matter of bureaucrats pushing the cost of development upwards to a point at which only a trickle of new treatments emerges on the other side. The public doesn't react to the invisible death toll of people who would have been saved by the medicines that are no longer cost-effective to develop.
It's a sad situation.
Given how conservative the FDA is in allowing companies to inform customers what's in their own bodies, it's unlikely companies will get any form of approval for modification.
Most people are not properly equipped to handle such information. I see this as a failure of education, that could probably be easily fixed if e.g. high-school curriculum taught basic statistics and probability theory as applied to interpreting medical results. It's the simple things, like "just because you got a positive test on X doesn't mean you have X", "think of horses before zebras" and "if you read 'X causes cancer' or 'X increases risk of cancer by 1000%' in the news, take note if anyone is providing the base level - an increase of 0.00001% probability to 0.0001% means you can ignore the news and keep enjoying X", and "if commonly-used-in-food-X was really dangerous, you'd see people dying like flies around you; if you don't, it means it's mostly safe so don't panic".
1: I don't doubt it's some people's underlying motivation, I just hope it's not overt enough that it's valid reasoning in official reports.
In the United States, pharmacologist Frances Oldham Kelsey M.D.
withstood pressure from the Richardson-Merrell company
and refused Food and Drug Administration (FDA) approval
to market thalidomide, saying further studies were needed.I feel it's important to judge weight of this story.
http://www.technologyreview.com/news/542371/a-tale-of-do-it-...
From the guidelines: "Please submit the original source. If a post reports on something found on another site, submit the latter."
> Telomerase ... is present in cells that can continue to divide indefinitely, like stem cells and tumors.
Your cells dividing indefinitely is not the opposite of aging, it's cancer. This is completely quackery.
Look at it this way: the Hayflick Limit on cellular replication limits act as a fail-safe in case other growth inhibition mechanisms fail. If a liver cell decides that it wants to be its own immortal organism but doesn't express telomerase, it won't get very far in its enterprise. The replication limit makes it more likely that we'll survive to reproductive age.
On the other hand, the Hayflick Limit kills us. Evolution doesn't care much about this side effect, but we do. If we can suppress cancer other ways --- or hell, even if we can't --- NOPing out the Hayflick Limit safety code is worthwhile.
No. As long as they divide on schedule in the amount they're supposed to, they're not cancer.
Dividing indefinitely is the opposite of aging at the cell level; aging at the whole-organism level has some other stuff to it. I think there's so much emphasis on the cell division clock because we know what it is.
Showmanship is not how we should evaluate clinical therapies.
[1] https://en.wikipedia.org/wiki/Seth_Roberts
[2] https://youtu.be/IuRLGdGnqSU?t=47s https://en.wikipedia.org/wiki/Hair_Club
http://discovermagazine.com/2010/mar/07-dr-drank-broth-gave-...
>Fossell, the anti-aging entrepreneur, says even if Parrish were to somehow succeed in slowing the aging of her body, an outcome he rated as unlikely, the experiment would have no impact because of how it was carried out. “The problem is that no one is going to believe them,” he says. “The credibility will be zero even if they are right on the money.”
Also:
> “We as a company have our own ethics,” she says, referring to what she calls the need for inexpensive gene therapy treatments. “I am certainly not going to ask someone’s permission to potentially create new industries and cures.”
Anybody saying "we have our own ethics" is way way out there, it casts doubt on all of the rest of their endeavor.
Everybody has slightly different ethics. She's just pointing out that she views the advancement in this field and the millions of people it could give longer life to if it comes about sooner as more important than the arguments against a human trial, and to make sure someone isn't being taken advantage of, she's doing it on herself. I'm not sure how to fault that.
That's not really true, is it? If she lives to 120, or she lives a normal life span with dramatically "younger" looks and energy, it will have a huge impact. Even if the treatment can't be replicated exactly they'll know where to start looking.
Meddling with growth factors and chromosome structure systemically is hardly going to be reversible in anywhere near as controlled a way, and could well end up hurting her (and others who may decide to follow the example), potentially hurting her in a life threatening way by inducing cancer.
Then there's the abstract damage from reckless human experimentation on attitudes and community reactions. Early failures with gene therapy probably delayed the clinical use about a decade. We don't need more of that, and we don't need more excuses for people to dismiss life extension as a crackpot venture.
I hope she turns out at least unharmed. Got to admit the case study could prove to be interesting.
Is it legal to offer a product that hasn't gone through FDA as long as consumers sign their acknowledgement?
It might be necessary to be a little careful with what entity is promoting the treatment and so on.
Medical tourism for price reasons is already a big thing. People are also traveling to Mexico or South Korea for stem-cell treatments unavailable in the US. Startups might also find US jurisdictions that want to carve out more experimental freedom than the FDA's defaults, as with stem cells in Texas:
http://www.bloomberg.com/bw/articles/2013-01-03/stem-cell-sh...
Consider another technology: safe, effective, and reversible male birth control [1]. It's been used in India for decades, but happens to be illegal in the US right now due to the slow and expensive nature of the approval process.
[1] https://en.wikipedia.org/wiki/Reversible_inhibition_of_sperm...
It looks like (science funding) politics is the only reason we are not testing how these genes work in large mammals and primates. The public is scared of genetic engineering.
And nuclear power, and geo-engineering. Public hysteria is placing us at great risk on numerous fronts. Ultimately, though, I'm optimistic. Not once in human history has anyone been able to suppress technology once it became both possible to make and profitable to produce. In this instance, the Chinese will probably beat us to gene therapy technology, and we'll desperately play catch-up once we see the difference it makes in life outcome.
How do you feel about Japanese suppression of firearms?
I should say that at any given time there is a fairly large gap between what can be done in human medicine, the technology that actually exists and works, and what is being done in trials. Most of this gap is due to regulation, and the rest of it because development groups want to have a reasonable certainty that what they are doing actually works, does more good than harm, and so forth. The regulatory process might last a decade, while the actually useful part of that testing (does it basically work, and is the risk profile sufficiently defined and acceptable to patients) is only a few years. As the cost of research and development in the life sciences falls, it will become increasingly untenable that a huge ball and chain slows progress thanks to regulatory risk aversion, and a growing number of initiatives will forge ahead and build anyway. Some years ago I proposed the Vegas Group fable, something that I think will happen in the fullness of time: alternative roads that bypass official regulation in favor of faster progress, an inevitability in an environment of low-cost research. Also, I think, a necessity.
What about the science here? I've never been a big fan of telomere lengthening approaches, as average telomere length as it is measured today in immune cells looks very much like a marker of the progress of aging, an end stage consequence far removed from root causes. Telomeres shorten with cell division and new long-telomere cells are delivered into tissues by stem cell populations. Thus average telomere length in immune cells reflects some combination of immune health and stem cell activity, both of which are known to decline with age. You can't argue with the fact that telomerase gene therapy has been shown to extend life in mice, however, though you can certainly note that the size of the effect has been getting smaller as the research groups have refined their data and approaches.
How does this work to slow aging in mice? At this point I lump enhanced telomerase activity into the general category of approaches that either probably work or intend to work by boosting the activation of old stem cell populations, resulting in increased repair and tissue maintenance and thus a slower decline into frailty and organ failure. More telomerase doesn't seem to raise cancer risk in mice, but mice have very different telomere dynamics and cancer risk profiles than we humans. The fastest way to figure out what is going to happen in humans is of course to try it, and kudos to anyone volunteering at this stage, but I'd be waiting for a few more years of testing first in animal or tissue models closer to human telomere dynamics. In part that decision would be driven by the fact that I don't think that this is the best approach to move ahead with practical applications, to push ahead and get things done. I absolutely agree that pushing ahead to get things done needs to happen, but I'd rather see this sort of boldness for SENS treatments like senescent cell clearance.
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Digest of the Reddit AMA with the BioViva CEO: https://www.reddit.com/r/Futurology/comments/3ocsbi/ama_my_n...
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.
I believe that regulations are holding back therapy development, especially in any area that is not already popular.
I think this is an issue with government in general, and we will need a totally new type of government if we are going to get a moral society that integrates with technology.
Yay, thank god. Otherwise it might take us whole aeons to heat the planet beyond the point we can live on it.
Personal rudeness is not ok on HN, even when you think someone's view is wrong.