Controversial Clinical Trial Requires Patients Pay $1M Cash & Travel to Colombia for Experimental Gene Therapy

Dec 13, 2019 | Aging, Anti-Aging, Colombia, Gene Therapy, Libella Gene Therapeutics, Telomeres

Controversial Clinical Trial Requires Patients Pay $1m Cash, Travel to Colombia for Experimental Gene Therapy

Patients seeking to “reverse the aging process” are invited to participate in a Phase I gene therapy clinical trial conducted at an obscure physician practice in Colombia. Seeking 5 patients, the American-based biotech sponsoring the study seeks to expedite the clinical trials process by avoiding FDA jurisdictional rules and guidance. The participants must pay $1 million to join this study. The experimental gene therapy premise: extend one’s telomeres to prevent, delay, or even reverse aging.

What is a Telomere and Why is it Relevant?

It is a kind of structure that actually caps the tips of chromosomes and serves to prevent the genetic material inside from fraying. As telomeres grow shorter each time a cell divides, the structures reach a critical length and cells stop dividing or expire, according to a Stanford study. In that study, it was reported that telomeres represent a form of internal clock that make it hard to keep most cells growing in a laboratory for more than a few cell doublings. Apparently, the study uncovered a method to lengthen human telomeres up to 1,000 nucleotides, hence “turning back the internal clock in these cells by the equivalent of many years of human life” stated Helen Blau, PhD and director of Stanford’s Baxter Laboratory for Stem Cell Biology. The Stanford researchers objective was to increase the number of cells used for studies, such as drug testing or disease monitoring.

The Millionaire’s Clinical Trial

Kansas City-based startup biotech venture called Libella Gene Therapeutics, the study’s sponsor, is enrolling five (5) patients to participate in this Phase I controversial trial using gene therapy to express active telomerase (hTERT). During the study, the principal investigator, a physician named Winston Pernet, MD, will treat subjects with hTERT delivered via transduction using AAV. The goal of the study is to extend the telomeres to prevent, delay or even reverse aging, as reported in Clinicaltrials.gov. The study commenced in October 2019 and is planned for completion in January 2021. Patients must pay the sponsor $1 million cash due to the sponsor’s claim that the cost to produce the hTERT is in the hundreds of thousands of dollars. Obviously, few people have $1 million to spare but there are considerable concerns with this study and the decision to locate the trial in Columbia—away from the FDA’s reach—is notable.

The Libella Mission—Truly Experimental but Inspirational

Based on legitimate experimentation and study, such as the 2015 study at Stanford, Libella’s premise is that if one could rebuild their shortened telomeres, they might be able to reverse certain signs of aging in animals. There are several pre-clinical research studies in mice, such as one conducted in Spain, suggesting that the use of gene therapy to lengthen telomeres can reverse certain signs of aging in the animals.

Libella Gene Therapeutics was formed in 2017 based on research done many years earlier by William Andrews, their head scientific officer. A molecular biologist by training, Andrews, undoubtedly a brilliant, charismatic and driven character, was involved in the identification of the telomerase enzyme at Geron, a biotech company. He apparently was involved with the licensing of a gene therapy based on these findings for Libella, as reported in OneZero. In an interview with the OneZero publication, Andrews declared, “I can’t say [telomere shortening is] the only cause of aging, but it plays a role in humans.”

Their other founder, Jeff Mathis, serves as president of Libella and runs a very successful private practice focusing on genetic testing and specialized therapies in the Midwest. Mathis formed Libella in 2017 with the sole goal of completing a human trial using AAV/hTERT to treat Alzheimer’s disease. The Mathis and Andrews duo is formidable—the researcher and clinician paired for such an ambitious undertaking—inspired by the vision and quest to take on aging—is surely exciting and the promise for the human species is monumental. With such potential, is the pair taking the right approach?

Concerns & Possible Danger

A 2018 study raised considerable concern about high-dose gene therapy. Many gene therapy-based biotech ventures use adeno-associated virus 9 (AAV9)—typically a harmless virus that infects neurons—increasingly used to ferry therapeutic genes into cells to treat neuromuscular genes. However, according to this report, after high doses some of the animals within days developed severe liver and neuron damage, according to Jocelyn Kaiser with Science Magazine. As reported by OneZero in their interview with Andrews, when asked about gene therapy dose amounts he noted, “All I can say is, it’s a lot.” Why can’t Andrews be more specific? Doesn’t the protocol break all of this down for review and discussion?

Research has pointed to a relationship between telomere length and cancer. For example, a study conducted by King Abdulaziz University, Saudi Arabia, and UT Southwestern Medical Center found that telomere length and telomerase activity are crucial for cancer initiation and the survival of tumors. Note, TrialSite News lists the principal investigators/study authors for that research for the benefit of our readers later.

Libella acknowledges that based on early stage research, there is some evidence that telomerase induction may cause cancer. However, they take the convenient position that telomerase therapy doesn’t cause cancer. Their evidence: a link to “multiple research articles.” But upon selecting the link, the webpage simply refreshed. This was hardly reassuring for the TrialSite News research team. The team will update links when Libella fixes the website link.

Why Colombia?

First, we start this section by reminding all that Colombia is a beautiful, fantastic country—one that has been transforming its economy at a rapid pace. It is a perfectly acceptable location to conduct a clinical trial. For purposes of this inquiry, we delve more into underlying strategy and intentions for site location.

There are very few nations where many of its residents have sufficient amounts of wealth required to spend $1 million to participate in a study.  Should such a drug ever be approved, such a treatment would come with an expensive price tag. We don’t think that Colombia is first in line for a target market, should this gene therapy treatment ever be approved by regulatory authorities.

Frankly, for the study alone, only multi-millionaires could participate—there are many more potential subjects that fit into such a socio-demographic category in America or some countries in Europe or, for that matter, China or Japan. Why Colombia? Clearly, one answer could be that this is where the sponsor could secure approval for such an experimental human clinical trial. So, although Libella is based in the U.S., this specific study evades U.S. federal law and other authorities that model much of their regulations on the FDA’s approach.

Libella’s position, of course, is one of confidence and pragmatism: their president, Dr. Mathis, was on the record noting that “traditional clinical trials in the U.S. can take years and millions, or even billions, of dollars. The research techniques that have been proven to work are ready now.” Mathis continued, “We believe we have the scientist, the technology, the physicians, and the lab partners that are necessary to get this trial done faster and at lower cost.” 

Of course, what Mathis forgets to remind the audience that 1) this trial is costing millions too—the only difference is that they are counting on wealthy patients’ unrealistic desires and urges to live forever to underwrite the initiative; 2) a more “flexible” regulatory order that may exist in Colombia doesn’t necessarily mean it  benefits patients; and 3) the FDA mandate for existence is to protect patients—hence why its rules and guidance represents the world’s gold standard for drug development research oversight. For a science with such potential—and the market to support more sales for such a miracle treatment than anywhere else—why not file an investigator new drug (IND) application with the FDA?

INVIMA (Instituto Nacional de Vigilancia de Medicamentos y Alimentos) regulates clinical trials and drugs in Colombia. It is a decentralized agency of the Ministry of Health created in 1993. The TrialSite News research team is seeking the investigator new drug application there for review.

Other Concerns from Peers

Apparently, there are other ventures pursing gene-based therapies for anti-aging or to combat neurodegenerative disease. One example would be Telocyte—also on a mission to utilize telomere-lengthening science. Their founder and CEO told OneZero that although their experimental gene therapy is similar to Libella, they are working to seek approval through the FDA. Founder Michael Fossel noted, “We’re afraid that something will go wrong [with the Libella trials], whether it’s from safety or efficacy standpoint.”

The Investigator

The study is being conducted by Wiston Pernet, MD. TrialSite News research conducted a search to learn more about this principal investigator—after all, this is a $1 million study and any patient asked to spend that kind of money should have more details on their attending physician/investigator.

There was little publicly available information via search engines, such as Google or some accessible proprietary physician databases. However, under the name Dr. Wiston Pernet Cantillo, we found what we believe to be the same physician—he was director of the Unidad de Cuidados Intensivos de la Clinical Niss. Apparently, back in 2010, upon an influenza outbreak in a Colombian prison known as La Vega de Sincelejo, one of the patients that died had visited Dr. Wiston Pernet Cantillo’s clinic, according to his quote in the local press

As it turns out, this Dr. Pernet Cantillo owns the Nissi Clinic based on his LinkedIn profileTrialSite News cannot 100% conclusively state that the Dr. Wiston Pernet that serves as principal investigator for the Libella aging reversal gene therapy-based clinical trial in Colombia is the same physician, but we strongly suspect this is the case. We will continue to monitor to verify and, of course, should we be incorrect we’ll update appropriately. 

The Libella study will be conducted by Dr. Pernet at the clinical research site IPS Arcasalud SAS, which appears to be a small medical clinic based in Zipaquira, Colombia—near Bogota. When reviewing their Facebook site, what appears to be a disgruntled ex-employee notes, ”they are used to hiring and not paying is always a lack of respect saying they will pay. And they never do.” Early this year, another comment reiterated the theme of not paying employees and naming the owner of the clinic as Mr. Jose Luis Mallorca as being apparently involved with multiple clinics. Of course, these are just comments on a social website and cannot be verified, but we can’t help but feel a little disturbed about the selection of the research site. 

Lead Research/Investigator

Wiston Pernet, MD, IPS Arcasalud, SAS

The researchers looking at Telomeres and cancer connections includ Mohammad A. Jafri, Shakeel A. Ansari and Mohammed H. Alqahtani from King Abdulaziz University, Saudi Arabia and Jerry W. Shay from UT Southwestern Medical Center.

Call to Action: On the one hand, we are truly thankful for individuals such as William Andrews and Jeff Mathis. Combining deep academic and industry research pedigree with skilled clinical practice, the two are a great fit. We commend Andrews and his life-long quest to find ways to stop aging, and we believe Mathis to be truly an American success story. Without people like Andres and Mathis, there isn’t progress. They push the envelope, and we believe that has to be done for all scientific progress.

On the other hand, something just doesn’t feel right here. And TrialSite News exists to bring transparency to the world of clinical research with an emphasis on the research sites and investigative staff involved. So, we are thankful for Libella Gene Therapeutics and Sierra Sciences (Andrews), but we believe the intense drive and zeal behind this initiative could compromise their clinical operational strategy in actually progressing this potential gene therapy.

We believe they are trying to push the envelope faster than what is reasonable. We believe that, for example, their choice of research site is purely for convenience and not for the high quality, deep experience, pedigree and integrity of the research operation—which we believe is important for all drugs—whether they end up costing $1 or $1 million. This is what the market expects—FDA-based standards. We hope we are wrong and the study goes smashingly well and that the pair commercialize an anti-aging therapy. But it is our duty to call this out and comment—we welcome critical feedback, which we would gladly publish.  

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