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.
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. The issue of safety is not a simple one: Alzheimer’s disease is uniformly fatal, so safety might seem to take a distant back seat to efficacy. True, but we see no reason to try an experimental therapy on despairing patients if we inflict easily avoidable risks (by using safe manufacturing processes for the viral vectors). The issue of efficacy is also not simple: you might think that any therapy, even if remotely effective, might be worth trying. True, but we see no reason to use a minimally effective therapy if we can provide a maximally effective therapy with only a bit more forethought and care. The issue of credibility is also not simple: you might argue that if we can cure even one case of Alzheimer’s disease, that will in itself be sufficient. True, but not if no one will believe the clinical results. It may have cured one person, but what about the millions of patients that won’t be treated if no one believes the result?
At Telocyte, we intend to meet all three of those obligations. The therapy must be sufficiently safe to justify the risk in using it in patients who are already desperately ill, it must be sufficiently efficacious to offer more than simple solace or wishful-thinking, and our human trials must be sufficiently credible that our results can be translated into a therapy that can become the accepted standard of care for millions of patients, not simply for a few people.
We applaud Liz’s hopes, her courage, and her enthusiasm as she makes a splash in the news, but Telocyte will take the more difficult path. We don’t intend to create a splash, but a world without Alzheimer’s. It is easy to act, it can even be easy to act with genuine compassion, but it is hard to act effectively and harder still to ensure that compassion is not only the intent, but the final reality.