Is your stem cell treatment safe and legal?

Stem cells have the unique ability to replicate and develop into specialised tissues with specific functions. Currently stem cells are used to replenish blood cells that are depleted during chemo therapy for cancer. And although indications exist in numerous clinical trials that stem cells can also be used to treat diseases such as arthritis, heart attacks, multiple sclerosis, diabetes and spinal cord injuries, only one stem cell product, Prochymal from Osiris Therapeutics, has obtained market approval by Canadian and New Zealand regulatory authorities for the treatment of so called graft vs. host disease which presents as a complication of haematopoietic stem cell transplants. Thus, the development of stem cell treatments for the above mentioned diseases is still in pre-clinical and clinical trial phase and might remain there for quite some time before safety and efficacy thereof can be proven, considering the unpredictable nature of stem cells. Only then can such treatments be approved and registered for sale.

In South Africa, stem cell treatments are categorised as biological medicine in terms of the Medicine Control Council’s (MCC) Guidelines for the Registration of Medicines where the active ingredient or key excipients have been derived from living organisms or tissues, or manufactured using a biological process. The Medicine and Related Substances Control Act 101 of 1965 further subjects all medicines that are biological medicine to registration with the MCC and specifically prohibits the sale of any unregistered medicine which is subjected to registration in terms of Section 14(1).

Accordingly, in the absence of proven and registered stem cell treatments available for sale, the only other route to obtain stem cell treatment is by participating in a clinical trial, if compliant with the specific clinical trail’s inclusion criteria, or in rare circumstances, via medical innovation.

Unfortunately many of the promises that stem cell research holds for curing diseases, for which no treatment currently exists, is often translated as fact, resulting in the financial exploitation and physical harming of already desperately ill patients. In the matter of United States of America v Laura Brown and Stephen Mark van Rooyen decided on 28 March 2006 in the United States District Court for the Northern District of Georgia, Atlanta Division under case number 1:06-cr-00153-UNA, Criminal Indictment No: 1:06CR153, the defendants were criminally indicted after administering the same type and quantity of stem cells to different patients regardless of the diseases they were suffering from and charged between $10 000 and $32 000 for these unproven and unlicensed treatments. None of the patients undergoing these treatments were cured and many of them died during the course thereof.

In an effort to protect patients against similar exploitation and harm, the International Society for Stem Cell Research (ISSCR) issued Guidelines for the Clinical Translation of Stem Cells in 2008 which point out some important aspects a patient should carefully consider before engaging in stem cell treatment. These guidelines include the following:

  • There is a long process of laboratory studies and clinical research to show that a treatment is safe and effective. Like a new drug, stem cell treatments must be assessed and meet certain standards before national regulatory bodies approve their use;
  • Doctors have been transferring blood stem cells by bone marrow transplants for more than 50 years, and advanced techniques are used clinically to collect blood stem cells. Umbilical cord blood is often collected as a source of blood stem cells, for experimental use as an alternative to bone marrow in transplantation. Other tissue-specific stem cells, such as for skin and corneas, have played a role in tissue transplants for several years, contributing to long-term regeneration. Other stem cell treatments are still experimental;
  • Unlike drugs, stem cells cannot necessarily be produced and tested for quality in large batches, and treatments may even be patient specific. For most diseases, it is still being determined which cells best will repair a particular damaged or diseased tissue, and how to get them to the right place in the body. Side-effects and long-term safety must also be determined, since transplanted cells may remain in patients’ bodies for many years. Therefore, careful monitoring and extended follow-up of patients who receive stem cell treatments is extremely important;
  • To test whether and how a new intervention may work for a particular disease or injury, studies are done first in vitro and, if possible, in animals with a disease or injury similar to those in humans. These preclinical studies should be reviewed by other experts, published and repeated before the research is extended to human subjects. After demonstrating a reasonable expectation that the treatment will work and be safe, permission is sought to conduct a clinical trial in humans, starting with a few individuals. New experimental treatments may sometimes be carried out on a very small number of people before a clinical trial is started. As the safety and side-effects are better understood and methods for delivery of the treatment to the correct part of the body improve, the number of patients may be gradually increased and the new intervention compared with existing treatments. Once safety and effectiveness is demonstrated, the relevant regional regulatory agency should approve the use of the treatment for particular diseases or conditions;
  • No medical treatment can be described as completely safe as all may involve risk, albeit small. Even small risks should be explained clearly by a medical professional;
  • Patients must be sure that there is good scientific evidence that the treatment is safe and effective, and that their rights are respected;
  • Patients should be cautious if claims are based on anecdotal evidence; if multiple diseases are treated with the same cells; the source of the cells or how the treatment will be done is not clearly documented; practitioners claim there are no risks; or in cases of high or hidden treatment costs.

If any uncertainty exists when faced with the decision whether or not to engage in stem cell treatment, it is recommendable that the advice from a stem cell specialist and/or biotechnology attorney is obtained before committing to innovative life changing treatment.

by Marietjie Botes
marietjie@dyason.co.za