8 things to consider at decisionmaking crunch time

Are you considering going for "stem cell therapy"? Have you beensurfing the internet for websites that market stem cell therapy?

We recently had to assist a patient who hasmuscular dystrophy and who was enquiring about stem cell therapy for thisdisease. We would like to share with you some of the salient points which aroseduring our interaction. Our hope is that you might find some usefulguidelines to assist you in making your own decisions. At the outset, wewould like to confirm that:

  • the use of cells for therapeutic purposes isgaining momentum world-wide
  • cell-based therapy, including but notlimited to stem cells, will have a major impact on medicine in the years tocome
  • the indications for these applications areas valid for South African patients as they are elsewhere
  • it is unethical to administer a treatmentfor which the safety and efficacy has not been established unless it isadministered as part of a valid clinical trial
  • it is considered unethical to ask patients to pay for a treatment which is part of a valid clinical trial.

Here are several important questionsthat you might want to ask the people at the clinic you are consideringvisiting:

1. The most important question to askis whether there have there been any clinical trials, or otherrecognized studies, which show that the proposed treatment has thepotential to beneficially effect your condition.

2. Has the clinic treated other patientswith your condition using the same proposed therapy and, if so, whatwas the outcome? In other words, does the clinic have any proof thatthis therapy is effective for your condition?

3. Are  you entering a clinical trial inorder to obtain the proposed therapy? Remember that a clinical trial isa research study that aims to determine fi rstly the safety, andsecondly, the effi cacy of a proposed treatment. Clinical trials areusually a requirement before a treatment strategy can be suppliedcommercially.

4. Are there measures in place in theevent that something goes wrong during the treatment period?

5. Has a long-term follow-up study beenconducted to determine safety?

6. Will there be a follow-up process tomeasure the effect of the cells and monitor potential side effects? Willside effects originating from the therapy (both during and after thetreatment period) be treated and, if so, who will bear the cost?

7. You have been told that your condition can be improved/treated with stem cells

  1. What will be the route of administration?
  2. How many treatments/applications willbe required?
  3. How many cells will be administered perdose?
  4. Assuming the cells are of human origin (which is not always the case), what is the source of the cells? Ifthe cells are not yours, will they be assessed for a match with your owntissues?
  5. If you are not receiving your own cells,will you receive immunosuppressive therapy to prevent your body fromrejecting the cells?

8. Will the cells be grown/expanded in atissue culture facility after isolation? If so,

  1. Will this be done in a clean room facility?
  2. What growth medium and supplements will beused to culture cells?
  3. Will serum supplementation be used withthe growth media and from what source will it be derived? (Bovine serum isnormally used for experimental work, but this may carry bovine diseasesand could cause an immune response in human patients)
  4. How long will the cells be grown and atwhat stage in the process will they be used for therapeutic purposes?
  5. Will the cells be characterized todetermine whether they are still stem cells and, if not, to determine whatthey have become?

 The above questions are by no means exhaustive,but they do raise some of the most important points that need tobe considered before embarking on a course of cell based therapy. Hope isa wonderfully powerful tool in our survival kit, and it forms an essentialcomponent of the so-called “power of positive thinking”. However, difficult asit may be to believe, there are people who exploit this very positivefacet of our lives for their own commercial gain, without having proofthat the therapy that is being proposed has any chance of success. So, beforeyou decide to embark on a long and challenging trip, and are prepared tolay out large sums of money (often in excess of R 150,000 per treatment), westrongly advise that you ensure that your emotional vulnerability andpositive frame of mind are not being exploited.

Dr Marnie Potgieter & Prof. Michael Pepper

Department of Immunology, Faculty of Health Sciences, University of Pretoria