There is never a week that goes by when we at CIRM don’t get asked by someone battling a deadly disease if they should go to a clinic, usually overseas, for a stem cell therapy. It’s hard to just bluntly say “no” to someone who is running out of options and running out of time. But there is an alternative, and that was the focus of a panel discussion at the World Stem Cell Summit in San Diego titled “What to know before considering non-complaint cell therapies.”

Lee Buckler of the Cell Therapy Group described the goal of the panel as searching for practical solutions, things that would be helpful to patients in the real-life context of their decision about whether or not to get a non-complaint cell therapy (by non-compliant he means something that hasn’t gone through the approval process and/or undergone clinical trials to prove it is safe and effective).

This wasn’t going to be an outright condemnation of any therapy in any country that hadn’t been approved by the FDA. Instead it was an attempt to give patients and patient advocates, and their physicians, some things to think about, and some questions to ask, to help them make an informed decision about their health. “Our efforts should focus on steering patients away from the worst offenders,” Buckler said.

“This is not a black and white issue, there are many shades of gray involved in making the decision. Step one is finding out if the treatment is compliant or non compliant (has it been approved as a result of a clinical trial for example). Step two is distinguishing between non-complaint treatments, trying to identify if they are more or less risky, if they have more or less scientific and/or medical credibility.” 

Allan Wu, MD, a cosmetic surgeon with the Morrow Institute, says there are certain things to look out for when considering a treatment:

• If someone says they are “Board certified in Stem Cell Therapy” be wary, there is no official Board certification in this field right now so this is a claim without any real substance
• Ask them what their training and certification in this field is. If they can’t show any don’t go to them
• Exhaust all options; if you have a kidney complaint see a kidney specialist first before turning to someone who claim they can “fix” you with stem cells
• Find out if getting one of these non-compliant therapies might exclude you from some other NIH experimental therapies. Some clinics will not enroll you in a particular trial if you have undergone a therapy that has not been approved by the FDA
• Has the treatment been approved by an IRB (Institutional Review Board) which makes sure that the therapy meets certain medical, scientific and ethical standards
• If someone tells you that the stem cell treatment can be billed to your insurance, go to your insurance company and check. Most of these therapies would not be covered
• If you are told this therapy will “permanently cure” your problem don’t go to them; there are no guarantees in medicine and if something sounds too good to be true it probably is

Allan says there are some good sites online to go to where you can get well researched, reliable information. Happily one of those is our own website  and others include the FDA, NIH and the ISSCR.

Paul Knoepfler, Ph.D., a stem cell researcher at U.C. Davis (and CIRM grantee) and avid blogger advises people to ask lots of questions when someone is proposing any therapy, but particularly when it involves something that has not been through the approval process:

• Ask “Is this treatment an experiment?” if it’s not then ask for data to show it’s effective and/or approved
• Ask “what are your standards for preventing contamination of your stem cells?” If they can’t demonstrate high quality manufacturing conditions you could be at risk
• Ask if the treatment has any possible side effects, if they say no be careful because anything that has the potential to have a medical benefit also can cause side effects, and there is almost no science on long term side effects of stem cell treatments
• Ask “what will the stem cells do in me after the transplant” because there is not a lot of long term information about what happens after a transplant

Allan Wu says he has one last piece of advice for his patients “if the benefits are low and the risk is high, then don’t have the procedure” and he says if you don’t really know what kind of cells you are getting and there’s no available evidence they will help you, then the risks will almost always outweigh the benefits.

Kevin McCormack