Nearly 14 years ago I wrote my first piece about stem cell treatment for multiple sclerosis (MS). In that post I described the procedure in these simple terms:
A patient is treated with a drug that helps the bone marrow produce extra stem cells (these are the cells in the body that can become any cell required at the time), and these cells are harvested and stored. The patient is then treated with a nasty cocktail of drugs to almost completely kill the immune system. They would like to kill the whole thing, but the immune system is pretty resilient.
Once the body is almost completely devoid of immune cells, the stored cells are replaced into the body. These cells will ‘look around’ and find that there are not enough immune cells. They then morph into the needed cells, hopefully without the memory to attack myelin in the central nervous system.
It’s a layman’s description, but a decade and a half on, it still holds water as a way of explaining autologous hematopoietic stem-cell transplantation (AHSCT).
Progress on Hematopoietic Stem-Cell Transplantation
The procedure has been in the research phases for a good long time now, and there is a mixed bag of results.
In addition, an unknown number of people have gone outside the research sphere and paid for stem cell transplants out of pocket at for-profit facilities in places like Mexico, Israel, Russia, and others.
The rates of success (and failure) in research settings are well-documented, while anecdotal reports from these non-research-based treatments are not. It makes it difficult to know what the risk-to-benefit ratio of the procedure is compared with that of the disease-modifying therapies available to people with MS.
Recruiting Now: 6-Year Study to Compare Treatments
This past January, however, the U.S. National Institutes of Health (NIH) announced the launch of a study that will directly compare AHSCT with the best available high-efficacy biologic drugs, and then follow the study’s subjects for six years to ascertain which may be the best treatment for early aggressive forms of MS.
The BEAT-MS study, which is currently enrolling 156 adults ages 18 to 55 at 19 sites in the United States and the United Kingdom, is a follow-up to the HALT-MS study, in which 25 people underwent AHSCT, but the stem cell transplants were not compared with drug therapy.
According to NIH, referring to BEAT-MS:
- The main outcome investigators will measure is how much time elapses between a participant’s assignment to a treatment strategy and MS relapse or death from any cause, if either of these occur, during the first three years of the follow-up period.
- The researchers also will examine the mechanisms of action of the two treatment strategies and will compare the newly developing immune systems of participants who receive AHSCT with the immunologic features of participants who receive the best available biologic drugs.
- In addition, investigators will compare the effects of the two treatment strategies on other measures of disease activity and severity, cost-effectiveness in terms of health care costs and individual productivity, and participants’ quality of life.
Participation in BEAT-MS Is No Small Commitment
It's a big ask for the research participants, and as a person living with MS, I thank all who consider taking part in this study. It will truly be one of the most important research steps for MS treatment in my lifetime with the disease.
It will take more than six years to get the final answers to the questions researchers are posing, and it’s been a long time coming to this point. Fingers crossed for the next generation of people with MS that a clear path forward is revealed.
Wishing you and your family the best of health.
Cheers,
Trevis
