Claim of risky DIY gene therapy linked to unlicensed stem cell clinic

Elizabeth ParrishIn a strange, but fascinating tale recounted by Antonio Regalado in an article over at MIT Tech Review, we hear the first claim of a do-it-yourself (DIY) gene therapy.

The owner of a small biotech called BioViva, Elizabeth “Liz” Parrish, reportedly traveled from her home in Seattle to get an unapproved, experimental gene therapy in another country.

The article pointed to a Reddit AMA that Parrish recently did where she discussed these events in the context of her interest in anti-aging efforts.

Regalado is fairly blunt about his view of the anti-aging crowd, describing it this way:

“The field of anti-aging research is known for attracting a mix of serious scientists, vitamin entrepreneurs, futurists, and cranks peddling various paths to immortality, including brain freezing.”

In the introduction to the AMA, Parrish is described as, “”the woman who wants to genetically engineer you.” You can read more about her here on her LinkedIn profile.

Below is a video of Parrish discussing anti-aging therapy. She’s clearly very articulate and bright. In the talk she claims that the doctor that she works with at BioViva received an anti-heart disease gene therapy five years ago and is doing well. It’s an extraordinary claim.

She also talks about stem cells and cures for a variety of serious and even fatal diseases such as ALS.

Apparently Parrish’s DIY “therapy” involved two components.

One effort targeted muscle through introduction (presumably through injections of virus?) of the follastatin gene into her muscle. The rationale seems to be that it would inhibit myostatin and give her big muscles. The second intervention reportedly was an IV of “viruses that express telomerase”.

The second one sounds particularly risky given the association between elevated telomerase and cancer. Many things can go wrong with gene therapy and when you combine that with DIY administration it gets even scarier.

Who knows the possible consequences of either of these gene therapies? Even tightly-regulated, experimental gene therapies in the past given as parts of controlled clinical trials have led to deaths. I hope that doesn’t happen here, but if true this situation sounds radical and possibly dangerous.

Still BioViva has had some impressive scientists on board. One, George Martin of the UW, has terminated his link to the company after these recent events. Harvard geneticist George Church, a transhumanist and at times a proponent of human genetic modification, apparently remains on board with BioViva.

By getting an unapproved and potentially dangerous gene therapy abroad, Regalado reports that Parrish was intentionally circumventing the FDA:

“Parrish said in an interview she chose to bypass the U.S. Food and Drug Administration by trying the procedure overseas. The FDA requires costly trials, and aging itself is not generally recognized as a disease that can be addressed by drugs. “What we did is we moved forward to try to treat biological aging,” Parrish says. “We are attempting to reverse aging at a biological level.”

This sidestepping of the FDA was not viewed positively by Church, but he indicated he was inclined to believe that the gene therapy intervention was real. He also has at other times recently been enthusiastic about the concept of a telomerase intervention in people as an approach to aging:

“Church, the Harvard professor, says he thinks targeted DNA changes could in fact extend the normal human life span, which has a maximum length of about 120 years. Earlier this month, at a meeting of the National Academy of Sciences organized to weigh policy on genetic interventions, Church proposed telomerase as one bearing serious consideration. “I think we are very close. I think the world is close, so long as we don’t have a setback,” he says. “The extension of life span is quite dramatic in model organisms … it would be amazing in humans.”

Again, to me the cancer connection with telomerase makes this a dangerous shot in the dark.

This tale gets more puzzling as Regalado points out the involvement of Jason Williams, who is more well-known in the stem cell world for his dubious stem cell clinic (Precision StemCell) that had been located here in the US. He has since moved that abroad after a situation with the FDA. Apparently Williams is also the co-owner of BioViva and he indicated to Regalado that Parrish’s DIY gene therapy took place in Colombia, but not at his clinic.

Parrish seems to espouse a very libertarian view of biomedicine and bioethics:

“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.”

This sounds reminiscent of transhumanism and disruptive technological innovation, but sure seems extremely risky.

It also could set a dangerous precedent for DIY interventions or encourage others to follow this uncertain path. Is this a one-time thing or the start of a larger trend in the world of genetic interventions?

Leigh Turner Reports Company Precision StemCell To FDA citing numerous concerns including treatment of a minor

Precision StemCellWhat is up with the stem cell company, Precision StemCell, these days?

I can’t imagine they are exactly celebrating after having been reported to the FDA. They also have apparently moved operations from the US to Colombia at some point recently.

What’s the deal here? First, a little background is helpful.

In 2012, University of Minnesota Professor and Bioethicist Dr. Leigh Turner wrote an open letter to the FDA raising concerns about Texas stem cell company, Celltex.

You can read that February 21, 2012 letter here. I believe it was a key milestone event in the history of stem cells.

I was not a fan of Celltex’s practices either and had written a piece on them and RNL Bio on February 29, 2012 (I’m not sure, but I believe I had not yet learned at that time about Turner’s letter).

A few months after Turner’s letter the FDA paid a visit to Celltex (actually a two week audit) and within months of that all clinical transplants of stem cells related to Celltex were shutdown after a sternly worded FDA warning letter. It is very important to note that Celltex threatened Turner and the University of Minnesota with litigation as a result of the letter written by Turner.

Turner and his institution did not back down.

In the stem cell field, Turner is widely considered a hero for having the guts to stand up to a stem cell business that he believed was putting patients at risk. For example, he won runner up for my blog’s 2012 Stem Cell Person of the Year Award. On March 5th and March 12th of 2012 I wrote pieces strongly supporting Turner and his colleague Carl Elliott.

I had never met them at that time, but admired what they were doing.

Now, Turner has published a piece about a letter he recently wrote to the FDA about another stem cell company, Precision StemCell, that to his way of thinking may warrant some regulatory attention from the FDA. I highly recommend this piece to you as worth your time to read, but I will summarize key points of it and the letter to the FDA.

Turner writes in his letter to the FDA that the leader of Precision StemCell, Dr. Jason Williams, promotes stem cell interventions for a wide range of conditions.

In the FDA letter Turner says:

Many individuals have undergone adipose-derived stem cell interventions at Precision StemCell. It is imperative that such purported stem cell-based interventions are confirmed to be safe, efficacious, and in compliance with federal regulations before they enter the commercial marketplace and are used to treat patients. I am therefore hoping that you will investigate the regulatory status of stem cells administered to patients at Precision StemCell.

Turner also raises concerns about non-homologous use of stem cells by Precision StemCell and the treatment of a minor, a little boy. It seems that the treatment of this 5-year old ended up in what seems to be a promotional video for the company.

Remarkably, Turner in addition reports that  the company has therapeutically employed some kind of “stem cell reprogramming technique” using a drug called Seligilene to treat a 74-year old man with ALS. The details of this “reprogramming” are scarce, but it is alleged that the process changes fat stem cells into neural stem cells. If correct, such a process would surely seem to be beyond the definition of “minimal manipulation” established by the FDA and hence make the end product a drug subject to thorough FDA oversight PRIOR to administration to any patient.

Turner also goes on in the letter to the FDA to highlight a number of other concerns including the medical devices and kits used by the company.

In Turner’s blog piece he notes that Dr. Williams and Precision StemCell should come under the scrutiny of the FDA and the Alabama State medical board. Turner also argues that Williams was essentially engaging in non-compliant, unlicensed human experimentation:

If Dr. Williams wants to conduct research addressing safety and efficacy of adipose-derived stem cell injections for ALS he should have first submitted an Investigational New Drug application to the FDA and then waited until the FDA let the proposed study proceed or imposed a clinical hold. Instead, it appears that Dr. Williams began injecting adult stem cells into individuals without first submitting an IND application to the FDA or seeking to have a research protocol approved by an IRB. For over a year Dr. Williams has engaged in unapproved, unauthorized human subjects research. Over twenty individuals paid substantial amounts of money to Dr. Williams for these unproven, experimental stem cell interventions.

What comes next?

If history with the Celltex experience is any indication, it would seem that Dr. Williams and Precision StemCell could have possibly expected an FDA visit (no indication itself of any wrongdoing necessarily of course) in the coming months, but now that possibility is unclear with the company’s exit from the US.

Dr. Turner may get some blowback from the company, which could even include threats of litigation. However, it is difficult to see on what legal basis the company could successfully sue him.

More generally, frequently companies in this kind of situation do not in fact litigate against critics because they are afraid of the discovery phase producing inconvenient facts about their business. Of course in this case I have no specific knowledge one way or another.

Stay tuned as this story is likely to heat up more in the near future.