Work on developing first generation autologous stem cell therapies is starting to look a lot like work on old school drug trials; this is to be expected under the present regulatory regime, and by the general way in which new technologies mature. Here's an example:
The Autologous Cellular Therapy CD34-Chronic Myocardial Ischemia (ACT34-CMI) Trial is the first human, Phase II adult stem cell therapy study in the U.S. designed to investigate the efficacy, tolerability, and safety of blood-derived selected CD34+ stem cells to improve symptoms and clinical outcomes in subjects with chronic myocardial ischemia (CMI), a severe form of coronary artery disease.
The baseline frequency and severity of anginal episodes are established as a first step for all study subjects. Next, all subjects receive a series of subcutaneous injections (needle shots, typically delivered under the skin in the arm, thigh or abdomen) of a commercially produced protein (granulocyte colony stimulating factor). The protein helps to release CD34+ stem cells (also known as endothelial progenitor cells) from a subject’s bone marrow into the bloodstream.
Then, investigators use a cell separation system, similar to the automated systems that are used with people who donate specific blood components such as platelets or red blood cells, to collect from the subject’s bloodstream, an enriched preparation of cells that contain CD34+ stem cells.
The researchers then use a special investigational catheter that functions like a "global positioning system" to precisely deliver CD34+ cells, or placebo, into the areas of the heart that have been identified as having poor blood flow.
Researchers are encouraged by reports that the therapy appeared to be well-tolerated and no serious adverse events directly related to the stem cell therapy in an earlier study. According to preliminary, anecdotal patient reports, 16 of the 24 total Phase I study subjects reported feeling better with reductions in chest pain and improved exercise capacity during the early stage of the trial.
The first phase of development in any new technology is a struggle to best the performance of old, mature technologies - here we have an early stem cell trial looking well placed (like others) to do at least as well as the best drugs and other well-developed medical technologies can do. The difference here is that stem cell researchers are just getting started; this is a matter of doing a little with the first steps into the pool. If we can gain benefits at this level through utilizing only the first laboratory steps of being able to identify, separate and culture stem cells, then just imagine what is to come soon, armed with the knowledge and techniques of controlling stem cell behavior.
Stem cell therapies and related areas such as tissue engineering will make up one pillar of the fight to eliminate age-related frailty and degeneration in the years ahead, replacing tissue and specialized cell populations that have been lost, diminished and damaged. What we see now are the early days, the first steps. Stem cell medicine will become much more impressive in just a few short years from now.
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