Dr Michael Fossel comments on the recent Bioviva announcement of the first human gene therapy against aging.
The other day, a friend of mine, Liz Parrish, the CEO and founder of BioViva, made quite a splash when she injected herself with a viral vector containing genes for both telomerase and FST. Those in favor of what Liz did applaud her for her courage and her ability to move quickly and effectively in a landscape where red tape and regulatory concerns have – in the minds of some – impeded innovation and medical care. Those opposed to what Liz did have criticized her for moving too rapidly without sufficient concern for safety, ethics, or (from some critics) scientific rationale.
Many people have asked me to comment, both as an individual and as the founder of Telocyte. This occurs for two reasons. For one thing, I was the first person to ever advocate the use of telomerase as a clinical intervention, in discussions, in published journal articles, and in published books. My original JAMA articles (1997 and 1998), my first book on the topic (1996), and my textbook (2004) all clearly explained both the rational of and the implications for using telomerase as a therapeutic intervention to treat age-related disease. For another thing, Liz knew that our biotech firm, Telocyte, intends to do almost the same thing, but with a few crucial differences: we will only be using telomerase (hTERT) and we intend to pursue human trials that have FDA clearance, have full IRB agreement, and meet GMP (“Good Medical Production”) standards.
We cannot help but applaud Liz’s courage in using herself as a subject, a procedure with a long (and occasionally checkered) history in medical science. Using herself as the subject undercuts much of the ethical criticism that would be more pointed if she used other patients. Like many others, we also fully understand the urgent need for more effective therapeutic interventions: patients are not only suffering, but dying as we try to move ahead. In the case of Alzheimer’s disease, for example (our primary therapeutic target at Telocyte), there are NO currently effective therapies, a history of universal failure in human trials for experimental therapies, and an enormous population of patients who are currently losing their souls and their lives to this disease. A slow, measured approach to finding a cure is scarcely welcome in such a context.
Wow! Seems like someone is quite jealous Liz got there first.
Interesting quote: “We have elected to follow the standard approach – with FDA-sanctioned human trials – for three reasons that we see as crucial: 1) we want to ensure safety, 2) we want to ensure efficacy, and 3) we want to ensure credibility.”
All complete nonsense that holds no credence in 2015
- Safety
One need only look at the sheer number of withdrawals, as well as adverse drug events (ADE) and deaths, associated with FDA approved, marketed drugs (2 million ADE and 100,000 fatalities annually in U.S. alone) which have gone through decades of human testing and use.
Animal models remain poorly predictive for humans, yet remain a mandatory cornerstone behind years and millions of dollars of early drug development activities. Penicillin kills guinea pigs and produces birth defects in rats, aspirin is poisonous to cats, cancer has been “cured in mice” thousands of times, and dozens of drugs found safe in animals are later withdrawn from market due to adverse drug events in humans.
- Efficacy
We spend billions of dollars on traditional “gold standard”, population level clinical studies, despite the fact that they use definitions of disease that are excessively inclusive (based upon disease characterizations from literally decades ago), while at the same time are excessively exclusive of major segments of the patient populace.
As a result, important differences among individuals with common clinical presentations are obscured, and we end up completely ignoring important underlying pharmacogenomic and toxicogenomic variability, to the extent that we even know about them in 2015 (which we don’t). The more we learn, the more we realize that every patient’s disease is a rare disease.
- Credibility
From a drug perspective, one only need look at all the FDA approved products that can: prolong QT and/or Cause Torsades De Pointes resulting in sudden cardiac death; which are IARC Group 1 and 2 carcinogens; which have significant ”black boxed” SAE warnings; not to mention their written positions on off-label use, “animal rule only” development, botanical drugs, and personal importation, all which highlight examples of various degrees of clinical, “let the patient beware” freedom that the agency is quietly willing to grant subjects today.
Further afield from pharmaceuticals, markets such as dietary supplements, cosmetics, processed ingredient foods/beverages, tobacco, legalized marijuana, and pesticide/herbicide usage, all technically represent large, uncontrolled clinical experiments on the general public, all of which NEVER have to be tested in humans, that we all are quite willing to accept today as personal choices.
A whole new era is evolving and will continue to grow into two distinct defined camps in the global healthcare realm
One, that more and more believes that the current system has failed them, and that the only important issue is how something works (or fails to work), in oneself
And another, that is more comfortable with the status quo, and is comforted by how something works (or fails to work), in a sample of patients that may or may not represent the target population (and what happens in a range of non-human species)
I doubt that Dr Fossel is jealous. Rather I think as a pioneer in the field he is as excited as the rest of us. Ms Parrish has taken an approach we all applaud. Sadly if telomerase therapy which in point of fact is the topic of Dr Fossel’s most recent book and all the books before is not brought through a “typical trial” it will remain untapped for many more decades. I think Ms Parrish will answer a lot of questions and jump the potential for anti-aging therapy ahead by decades with her own therapy. All of us hope it is successful in such a way that it shuts up a lot of naysayers.
That said an FDA approved trial is the ONLY thing that may eventually get Big Pharma and other interests like Calico (currently focusing on mTOR) to look at what many of us think is the obvious choice for combatting disease AND aging. As far as “geting there first” are you serious? Without Dr Fossel, Dr Harley and Dr Blasco there would be no therapy for Ms Parrish to try. This is not a unilateral effort. Both a single isolated trial with the idea of a commercial product AND an FDA trial are needed for this kind of therapy to actually move from the fringes of science to the mainstream. People need to understand that.
I applaud Dr Fossel’s even handed posting. BioViva’s bold strategy is contrasted by Telocyte’s more measured strategy. The United States regulatory agency, the Food and Drug Administration (FDA), is notorious for their red tape; tremendously expensive and extra long regulatory road to approval. Frankly, the FDA outdated procedures are the cause of a massive amount of pain, suffering, and expense. Unfortunately, the US politically system is ossified — tied up in knots by special interests (like Big Pharma), so not reform can be expected anytime soon. As a consequence, the natural bypass is going to a more regulatorily friendly environment, and thus creating a medical tourism industry to offer the latest cutting edge treatments to the consumer.
I applaud Dr Fossel for working within the system, but I especially applaud Ms Parrish (CEO of BioViva) for working outside the system. I am 54 and am dying of a genetic disease called aging — I am desperate to find treatments to delay and possibly reverse this genetic death sentence. The FDA will quite possibly be the death of me, because they want to protect me. That makes them evil in my eyes.
Thank you all for your comments. I repeat: Liz is a friend and I applaud her, although Telocyte and I will take a different path. The question is NOT who gets there first, but who saves lives. Good luck to Liz and good luck to anyone who (by whatever pathway) succeeds in stopping Alzheimer’s and other age-associated diseases. God only knows, no previous approach has worked. Liz and I share the hope that our approach will create a clinical revolution.
Thank you Michael it means a lot to me to see both groups working on the same problem, saving lives! I think both paths have merit and I admire the work you are both doing to that end.
I highly recommend getting Michaels new book “The Telomerase Revolution” if you are interested in learning more about the work and why both companies agree that telomeres are so important.
Best of luck to Liz and Michael!