The ISSCR: Who Are We and Where Are We Going?

Abstract

Every week, I am asked whether a family should take their loved one with an incurable disease to clinics that promise cures with supposed stem cell therapies. Usually, the costs for such therapies are in the tens of thousands of dollars. When I go to the websites of the providers, usually very slick, the therapies are often transplantation of tissue or umbilical cord stem cells for a variety of nonhematopoietic or nonmesenchymal disorders (claiming transdifferentiation), or even claim to be ESC therapies. I tell those that ask that there is no scientific basis for such transdifferentiation to date and no approved therapies using ESCs. If an enquirer is in the biomedical field, I remind him/her that there is now a pretty good operational way to determine whether a phenomenon, such as transdifferentiation, is real:(1)The scientific basis (e.g., of transdifferentiation) must be published in a peer-reviewed journal, but that is not enough.(2)Several independent laboratories should have confirmed and published in peer-reviewed journals that the findings as first published are reproducible, but that is not enough.(3)The principle that underlies the discovery (e.g., transdifferentiation) is so robust that any way you approach the issue, the discovery is validated, but that is not enough when therapeutic cell therapies are the objective.(4)The regeneration of tissues is rapid, robust, tissue-appropriate, and curative, not simply rare cells of uncertain origin.But these are not issues the lay public calling for the cures promised can judge alone. My own advice is that they should attempt to obtain the following from the stem cell companies or clinics before they agree to a therapy:(1)The provider must be able to send peer-reviewed papers from independent practitioners that establish the therapy as possible.(2)The provider must send evidence of approval from an independent committee, such as an institutional review board (a document from the clinical entity where the therapy is being practiced that shows the steps taken to protect the patient and justifying the experimental or approved therapy for their specific disease).(3)The provider must send a document from an agency equivalent to the Food and Drug Administration showing the approval of the experiment or the therapy for human subjects with their disease.I advise them that without all three, they must assume they are receiving an unproven therapy and that the patient is in danger of risking their life or health, of losing their money, of being away needlessly from home, family, and friends, and of course, of not being therapeutically improved.Over the past 2 years, the ISSCR has taken further steps to support the responsible translation of stem cell research to the clinic, releasing the Guidelines for the Clinical Translation of Stem Cells and accompanying Patient Handbook on Stem Cell Therapies (www.isscr.org/clinical_trans). As President of the ISSCR, I propose that we develop a website and occasional press alert naming as unproven stem cell therapies where providers of so-called “therapies” cannot supply to the ISSCR, within 60 days of request, evidence of rigorous and independent scientific and ethical review along the lines of what I recommend patients seek.We should be aware that the entities that cannot prove safety and efficacy are at one extreme of an unfortunate continuum that has, at the other end, proven therapies. We must all be cautious in our own enthusiasms and also how we define the minimum criteria required to achieve nonlisting on the site. We will not be a judge of approved therapies. I hope you agree with me on this subject, and I welcome your comments on this proposal either way.I ask for your understanding of this long introduction to a short presidency. I felt it was important to establish stem cell biology as an independent field, to establish our research as central to the development of regenerative medicine and new types of cancer therapies, to try to summarize the politics of stem cell policies clearly, and to find some way to keep our legitimate efforts separable and separate from the charlatans who compromise the safety of citizens in need of hope and cure and ultimately the promise of the field.