Today a Clinical Trial, Tomorrow a Standard Treatment

"When do clinical trials cease and when does it become a therapy?"

I get this question from my team whenever I discuss the latest trials that are going on for stem cells in other parts of the world, and I just got asked this question again today from my colleague.

I took the opportunity (during the last BIO 2005 conference I attended in Philadelphia) to pose the question to the prominent panel of scientists consisting of Prof. Wise Young of Rutgers, State Univ. of New Jersey and NIH policy advisor, Prof. Ruth Faden of the Phoebe R. Berman Bioethics Institute at Johns Hopkins University and Prof. Darwin Prockop, Director of Gene Therapy at the Tulane Univeristy Health Science Centre.The topic discussed was Stem Cells, the policy and legislation surrounding the issues of research and clinical trials.

Despite the accumulated brain capacity and years of experience in stem cells exceeding the entire collective of Malaysia, I gathered up my courage to ask them my question. "Sirs, in your talk, you mention many clinical trials that have been done and are on-going. May I ask -in your view of adult stem cell therapy- when is a clinical trial considered a therapy?"

The panel smiled at me kindly, and Prof. Prockop took up the microphone. He said "Young lady from Malaysia (I introduced myself before asking the question), a clinical trial is never just a trial, if it provides a cure for a disease which is incurable, it is not a trial. It is a therapy."

Many people are misled into thinking that by participating in a clinial trial, that the treatment is thought not to work. There are basically 3 stages of clinical trials, take for example cancer. The earliest stage, called phase I trials, are aimed at finding out about the side effects and the acceptable doses of new treatments. Phase II trials aim to find out how well the new treatment works. Phase III trials compare the new treatments with the standard one.

A treatment is better either because it is more effective against the cancer or because it is equally effective and has fewer unpleasant side effects. It is very likely that the treatment being given has already been tested for its effectiveness in a previous trial.

Today's stem cell trial treatment may be tomorrow's standard treatment, whether it be for cancer, heart disease* or diabetic foot ulcers. As long as the treatment does not do worse than the conventional therapy being offered (e.g. stem cell improving ejection fraction vs. nothing else offered by the doctor), it may be labelled a trial for the doctor, but as Prof. Prockop said- it is already a therapy for the patient.


*See previous entry on stem cells for heart disease- patients have already benefitted (therapeutic effect).