The Gatekeepers of Medical Regulation are Horrified by Freedom, Responsibility, and Progress

Much pearl-clutching is in evidence in a recent article on the existence of groups, such as Libella Gene Therapeutics, attempting to prototype telomerase gene therapies via patient paid trials, or such as Integrated Health Systems and BioViva, trying to develop markets for such therapies via medical tourism. The gatekeepers of medical regulation stand in opposition to the idea that patients and their supporters can make responsible decisions about risk, based on the available data. Medicine is somehow a privileged space, different from every other human endeavor, in which only the anointed priesthood are allowed to determine what is and is not allowed. It is reprehensible. Analysis has shown that this attitude, and the system of regulation that accompanies it, costs a great many lives by slowing the development of new therapies and limiting the opportunities for treatment.

Medicine, like every form of service, works best in an environment of review organizations, competition, and due diligence by customers. At the end of the day, it is always a matter of caveat emptor. And this already happens, as anyone who has been through a scheduled surgery can tell you. Patients absolutely shop the market to the degree that present regulation allows them to do so, and there is a robust system of legal culpability by which fraud and harm can be prosecuted. As is the case in every other industry, providers of medical services have financial and other incentives to produce good results for patients.

I personally do not have a strong opinion on whether the telomerase gene therapy that is the subject of this article will help with Alzheimer's disease; it seems indirect and compensatory, improving cell function rather than striking directly at the known causes. That said, several noted research groups are very much in favor of developing telomerase based therapies for human use, and at least one company in the longevity industry, Telocyte, is seeking to raise funding to run a formal trial of telomerase gene therapy for Alzheimer's disease within the existing regulatory system. It is an entirely mainstream goal.

At some point all new medical technologies must be tried for the first time in humans. Is that to be left to the anointed priesthood, with its impossible goal of zero patient risk, ever increasing costs of certification, and ever fewer approvals with each passing year? Or should we live in a more reasonable world in which more sensible and cost-effective choices regarding risk, pace of development, and availability of treatments are made by patients, supporters, and networks of review organizations, rather than by uncaring bureaucrats? I would vote for the latter of these two options.

Six patients with dementia went to Mexico for an unproven gene therapy, a biotech CEO claims

Six patients with dementia traveled to Mexico last year to be injected with a gene therapy not authorized for use in the U.S., according to the CEO of a Seattle-area startup that wants to accelerate testing of unproven anti-aging medicines and views U.S. drug safety regulations as a hindrance. At the heart of the project is a controversial biotech called BioViva, whose CEO had herself injected with an experimental gene therapy in Colombia and whose advisory board includes renowned Harvard geneticist George Church. It is part of a growing ecosystem of entrepreneurs and scientists, dreamers and schemers, who believe aging is not inevitable and aim to develop treatments to extend the human life span.

Last month, during a talk hosted by the National University of Singapore, the CEO, Elizabeth Parrish, divulged that she was eagerly awaiting data from a human study involving six patients who received an experimental gene therapy. On Friday, she told STAT the procedures were done last year in Mexico. If true, it would be the first known attempt to use the technique to treat age-related dementia, which is most often caused by Alzheimer's disease.

STAT set out to independently verify the accuracy of Parrish's claims. While many key details could not be confirmed, including the identities of these six patients and how the purported treatment affected them, STAT found evidence that BioViva and partners were recruiting patients. A newsletter emailed by BioViva in 2019 said 10 patients over age 50 with mild to moderate Alzheimer's were needed for a study of a gene therapy. The one-hour procedure, according to an FAQ linked from the email, would be done in Mexico City and involved a one-time injection. The effort raises the specter of an overseas medical tourism industry targeting patients desperate to lengthen their lives and offering unproven treatments that would permanently alter the genetic code inside recipients' cells.

Comments

I'm not as confident as you in the capability of the general public to "make responsible decisions based on available data" nor in the "robust system of legal culpability by which fraud and harm can be prosecuted". There are lots of homeopatic potions, herbal remedies, organic food and many other BS being sold as cures for medical problems. There is also a lot of mass hysteria and conspiracy theorists against approbed vaccines.

That said, current trialing system could probably be made less slow and expensive, and more friendly to combination therapies (like antiamyloid + antitau antibodies).

Posted by: Antonio at May 4th, 2021 4:32 PM

"and ever fewer approvals with each passing year?"

Actually, there has been a meaningful increase in approvals in the last decade, but still quite few compared to, say, the number of medical papers published every year.

https://www.mdpi.com/1420-3049/26/3/627

Posted by: Antonio at May 4th, 2021 4:46 PM

Liz parish is hardly impartial here. On the other hand, If I had Alzheimer's I would grasp every straw and would not worry to much about "permanent gene modification" of my soon to be irrelevant cells. Since the situation is ripe for abuse it is absolutely certain that the will be a few horror stories and a pushback against medical tourism.

I was expecting Mexico to have very similar to US legal hurdles.

Posted by: Cuberat at May 4th, 2021 8:02 PM

Spot on, Reason. Obvious gatekeeping is obvious.

Posted by: Oliver at May 5th, 2021 3:54 AM

Mitigating the risk should involve holding the payment for this in escrow until it has a proven effect.

Posted by: Robert Read at May 5th, 2021 5:54 AM

When people like Ms. Parrish become the face of longevity biotechnology, this signals the collapse of the whole segment

It signal that this is no longer a serious place

Posted by: quan li at May 5th, 2021 7:08 AM

Reading the whole article George Church seems to trying to keep the whole trial at arms length, but is also failing to condem it. Perhaps he is glad to get some in human data at a reasonable cost?

The bioethisitc Turner sounds like a pain the neck. But critisism can be useful. BioViva could provide the dosages and other trial details up front. Being a heretic is hard work after all.

I'd like to see them do a trial against frailty using CRISPRoff against myostatin. Maybe combined with a senoltyic, with a gene therapy + senolytic, gene therapy only, senoltyic only, and placebo arms.

Posted by: jimofoz at May 5th, 2021 1:37 PM

The fundamental issue here is self-ownership. We accept the principle of self-ownership when it comes to making ones own financial decisions. Yet, for some inexplicable reason, we deny it with regards to making ones own medical decisions. It is good to prevent fraud. But the restrictions on self-choice in medicine go way beyond the mere prevention of fraud. This is unacceptable for a free society. At the end of the day, the individual is sovereign over one's self.

Liz Parrish's work is legitimate bio-engineering. By legitimate, I mean that her technology does work in the way that she describes it (it extends telemeres). Whether it will actually extends her life is an open question. However, I would not go for here therapy because I do not consider telomere extension to be the proper way to pursue life extension. A much better approach is to do the mito fission/fusion thing, then do senolytics with stem cell replacement. This latter should only be done if you are actually geriatric (e.g. you are over 60 and you look and feel like it).

Posted by: Abelard Lindsey at May 5th, 2021 2:35 PM

@Abelard Lindsey
The average human being is quite gullible regardless of the intellectual level. There are whole classes of financial instrument not available to the general public for protection purposes. The knowledge asymmetry is even greater for all things medical. And we all have much greater disgust and hate for medical malpractice than financial crooks. It is not only FDA that avoids the risks. Out is the whole government structure all over the world.

This particular case would benefitted of there were first some studies in aged diggs and cats pricing the efficacy before switching to humans. Here I am less concerned with the safety as the target group is hard to make much worse

Posted by: Cuberat at May 5th, 2021 2:57 PM

@quan li - I don't get all the hate. What are *you* doing for the longevity industry?! Please share.

Posted by: Joe Da Silva at May 5th, 2021 4:03 PM

@Joe Da Silva

I am a venture partner in a $450 million biotech fund with offices in Silicon Valley and Taiwan

We fund real companies

Ms. Parrish and her charade is a disgrace to the whole space

Posted by: quan li at May 5th, 2021 7:44 PM

"The knowledge asymmetry is even greater for all things medical. And we all have much greater disgust and hate for medical malpractice than financial crooks. It is not only FDA that avoids the risks. Out is the whole government structure all over the world."

The problem with this mentality is that it is often used as a smokescreen to maintain monopoly privilege as well as to maintain political ideologies that are hostile to radical life extension as well as liberty in general, both personal and economic. In our case (the U.S.) the gatekeepers have proven time and time again their utter corruption and parasitism. This is why they have to be eliminated.

In any case, this is no justification to restrict self-experimentation and DIY efforts to cure aging in cases where people are freely sharing information and not seeking to profit from others (presummably on the basis of fraud). I believe the best analogy would be to establish the DIY medical equivalent to the FAA's experimental and "kit-build" airplane regulatory regime. Many libertarians have argued this for several decades.

Posted by: Abelard Lindsey at May 5th, 2021 9:48 PM

@quan li - Any links? Would love to see the details.

Posted by: Joe Da Silva at May 6th, 2021 10:09 AM

Dear @Reason, thank you very much for your position! And people like Liz Parrish or biohacker Josiah Zayner are really in need because they pave the way for new regenerative medicine! If some commentators don't wish risk in using experemental therapy, well, -- just don't use them!

Posted by: Ariel at May 6th, 2021 3:57 PM

I wonder if Quan Li's VC group are funding AgeX and Turn.bio, both start ups in the cellular reprogramming space.

Posted by: Abelard Lindsey at May 6th, 2021 5:23 PM

I saw the article, and they made it sound as if they had murdered children. :)

Posted by: bmack500 at May 7th, 2021 1:03 PM

The FDA kills 100 people for every 1 they save. Simple math that nobody wants to admit.

Sure, their ultra-slow, 1960s-style ancient, corrupt, horrifyingly obtuse paperwork process makes drugs safer... eventually. But it takes so long, that hundreds of great drugs that could save lives never make it to market, or take 5 extra years. Net result: American citizens die while waiting. Thousands or tens of thousands per year, because of the FDA.

Why is the FDA so corrupt? Because FDA employees are corrupt. They have only one goal: to constantly ratchet up the amount of legal fees and consulting fees and paperwork for any given drug, so that when THEY retire, they will be able to become consultants charging 300 bucks an hour to help you navigate the screwed up system they created.

The FDA is a scam, my friends. Just watch how they arbitrarily decided that self-transplanted stem cells were "their territory". Next they will try to weasel their way into the business of organ transplants (starting yet another battle with the doctor groups who, no surprise, detest the FDA even more than I do).

What's the solution? Well, for a start, maybe we consider getting rid of a 1960-s style, ancient, corrupt, outdated, horribly inefficient and badly-run government agency that is literally crushing the life out of our medical system? Wow! What a thought!

Our modern medical world is completely different from what the FDA was set up for. We have a thousand patient-advocacy groups, Internet information centers, and foundations that are driving pharma. We have rare disease groups funded by parents, hiring teams to create drugs. We have massive information "efficiency" growing daily on the Internet. People are simply tired and disgusted by the incompetance and they are bypassing the system. The 6 people who went to Mexico are just the start of a flood. Japan, Singapore, Panama, Switzerland, etc. The United States is already a backwater.

The FDA is literally a lead weight of corrupt crap, dragging down and obstructing the growth of our entire healthcare system. It sucks in every way it is possible to suck. Amazing that people still defend it.

It's long past possible to fix it. Dump it...NOW. Push those responsibilities to states, doctor groups, and universities where they belong. Create a new regulatory regime that isn't based on the days when people used typewriters and slide rules.

Posted by: Jim Bonnyville at May 9th, 2021 7:18 PM

Most honest solution for longevity interventions probably : Intelligent, responsible, experienced therapists show up on a purpose-built platform clients can choose from to assist them looking for a tailor made intervention and therapists offering clients to monitor their interventions and results. As whole medicine is more or less based on trial and error liability of therapists limited to that truth. Everybody is the single owner of her/his body, so logically any "longevity" decision under educated circumstances can only be with the owner.
REASON: would that be a reasonable way forward ?

Posted by: Art Finch at May 9th, 2021 9:21 PM

Post a comment; thoughtful, considered opinions are valued. New comments can be edited for a few minutes following submission. Comments incorporating ad hominem attacks, advertising, and other forms of inappropriate behavior are likely to be deleted.

Note that there is a comment feed for those who like to keep up with conversations.