Several years ago a friend in declining health due to pulmonary fibrosis traveled overseas to receive stem cell treatment. Unable to get the therapy in the U. S., she elected to spend the money and take a chance. The procedure left her feeling better and she did appear to have improved for a while. Ultimately her deteriorating condition drove her to try a second round of stem cells provided by a clinic located in the U. S. This time there was no respite from the disease and she passed away last year.
In just a few years the stem cell therapy situation in the U.S. has changed quickly and continues to evolve. Confusion as to whether stem cell therapies using the patient’s own cells require approval from the Food and Drug Administration (FDA) and lax oversight have combined to yield a situation where hundreds of clinics are now offering treatments (1). The problem is that many are unproven, sometimes downright questionable and can be dangerous (2). A recent blog post by Dr. Paul Knoepfler contrasts one well-conducted stem cell clinical trial that generated promising results with something that became a tragic debacle (3).
Creating and enforcing regulations that safeguard public health without stifling promising research will be a feat. Concerns about speeding up FDA approval processes have been expressed for years. Now ideas are being floated to allow drugs to be used before their efficacy has been proven; a sort of ‘buyer beware’ policy for clinical trial participants. In the case of life-threatening diseases without any available therapies it may make sense to allow clinical use of apparently promising treatments without proof they are truly curative. However, providing therapies of uncertain value based only on the hope or belief they might help is ethically questionable.
A new world-wide industry has sprung up in response to the hopes and needs of persons suffering with terrible illnesses. Our friend and her spouse were extremely intelligent and well-educated people who researched stem cell therapies thoroughly before reaching their decision to go forward. They were also desperate and convinced stem cells were their last hope. Their story reveals that even if the FDA clamps down on unproven stem cell therapies, some will travel far and pay dearly to get them.
(1) H. Ledford. 2016. Boom in Unproven Cell Therapies Intensifies Regulatory Debate. Nature, 7 September 2016. http://www.nature.com/news/boom-in-unproven-cell-therapies-intensifies-regulatory-debate-1.20545
(2) R. Stein. 2017. Three Women Blinded by Unproven Stem Cell Treatments. NPR, 15 March 2017. http://www.npr.org/sections/health-shots/2017/03/15/520118310/3-women-blinded-by-unproven-stem-cell-treatments
(3) P. Knoepfler. 2017. NEJM [New England Journal of Medicine] Paper Links 3 Blinded Patients to Publicly-Traded Stem Cell Clinic. The Niche blog, 15 March 2017. https://ipscell.com/2017/03/nejm-paper-links-3-patients-blinded-to-publicly-traded-stem-cell-clinic/#more-19979