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Hot Topics in MS: Stem Cell Treatment in MS

With current disease modifying therapies (DMTs) we have several highly effective treatments that work for many people, but there is a percentage of the population for whom these medications do not work. Depending on your age and how long you’ve had MS, stem cells may eventually offer an alternative.

When you develop MS, there is a disturbance in the immune system that causes it to attack the central nervous system. So, what would happen if we got rid of the improperly functioning immune system and “rebooted” it without that disturbance? Would that stop MS from further impacts on the central nervous system?

Autologous hematopoietic stem cell transplant (aHSCT) — the stem cell treatment being studied as a possibility to fight MS — involves cells being collected from a patient’s own bone marrow. The patient is then treated with high-dose chemotherapy, after which the harvested bone marrow is reintroduced, effectively re-setting the immune system.

The premise behind using stem cells to fight MS is just that — shutting off the immune attack by rebooting the immune system. The specific type of stem cell therapy is called autologous hematopoietic stem cell transplant (aHSCT).

Autologous means that the cells come from the person themselves. Hematopoietic stem cells are pluripotent (stem cells that can be induced to make every part of a person except a fetus), come from your own bone marrow, and can become white or red blood cells or platelets. Once the right cells are collected from the patient’s bone marrow, the next step is to destroy the remaining bone marrow in the body with high dose chemotherapy.

This is a complicated and risky process and the MS community has not yet come to a consensus on the best way to achieve it. High dose chemotherapy has a lot of potential side effects, including risk of infections which could result in hair loss, lengthy hospital stays, nausea, and possibly even death. The more intense and lengthy a treatment is, the more risk there is; but a shorter and less intense course of treatment may not be as effective for eliminating MS from the body. There is also a risk of an MS relapse while you are undergoing chemotherapy.

Assuming both the collection of cells and the following chemotherapy are successful, stem cells are then injected back into the patient’s blood with the hopes that they will not manifest MS again. This stem cell transplant or “reboot” of your immune system is meant to shorten the length of time that it takes your body to recover from the high dose chemotherapy, however, it is still an arduous process for your immune system to reestablish, and you are at risk for infections in the meantime.

Patients will need to be closely monitored by a neurologist and hematologist after a stem cell transplant because the process is not a guarantee. This procedure should only be considered when the clinic you’re working with has expertise and experience in both MS and stem cell transplants.

According to Dr. Jeffrey Cohen, a prominent researcher in the field of stem cell therapy and MS working at Cleveland Clinic, the largest benefit of aHSCT has been seen in younger patients who have active relapsing-remitting MS that has not responded well to highly effective traditional therapies (such as ocrelizumab (Ocrevus), rituximab (Rituxan), ofatumumab (Kesimpta) fingolimod (Gilenya), and natalizumab (Tysabri).

aHSCT accomplishes essentially the same thing as many already approved DMTs, which have a much stronger safety profile – namely, it can stop new lesions and halt the accumulation of disability. Therefore, patients should consider traditional therapies before exploring aHSCT.

aHSCT is less helpful for progression resulting from neurodegeneration and is less safe for older adults who have more accumulated disability and/or other comorbidities.
Stem cell transplants in MS are in an experimental stage currently, and we’re still working to find out answers related to the safety and to develop a standard procedure, so this alternative should not be considered lightly. There are many concerns to think through when considering aHSCT, namely:

  • There is no consensus in the MS community for exactly how to wipe out your immune system.
  • Every step of aHSCT involves risks and may have complications.
  • Due to the nature of the medications used to eliminate your old immune system, you are at high risk of infections and even death.
  • At a minimum, you will be required to have a lengthy hospital stay of several weeks, or up to a month or more.

In addition, most of the time, it’s difficult or impossible to get insurance to cover the procedure, which on average costs around $150,000. On top of that, there are many unregulated clinics offering “miracle treatments” ­— you should be wary when a clinic advertises stem cell treatment for many medical conditions instead of specializing in a particular condition, if they are a free-standing clinic not associated with a legitimate medical program, or if they have not published their results in reputable scientific journals.

One important fact to note is that aHSCT does not reverse the damage already caused by MS. It has the same treatment goals as the already-approved MS therapies that are easier to get and safer to manage. But, very early studies are underway looking at transplanting cells of the nervous system lineage. These would work by simply replacing damaged cells, or, more likely, providing growth factors to cells already in the brain and spinal cord that could aid in repair and regeneration of nerves.

BEAT-MS: Comparing aHSCT to More Traditional Therapies

There is a currently enrolling trial being conducted by the Immune Tolerance Network and sponsored by the National Institute of Allergy and Infectious Diseases (NIH funded), in collaboration with The Blood and Marrow Transplant Clinical Trials Network called BEAT-MS.

The purpose of the BEAT-MS study is to compare chemotherapy followed by autologous hematopoietic stem cell transplant (aHSCT) to the most effective medicines regularly used to treat relapsing MS (“best available therapy”).

Eligible participants will be randomly assigned to either aHSCT or best available therapy. Both arms of the trial will be followed by a study neurologist for six years. You may be eligible to participate in the BEAT-MS Study if you are aged 18 to 55, have been diagnosed with relapsing-remitting or secondary progressive multiple sclerosis, and have had incomplete benefit (continued relapses or active MRI) while taking prescription medicine for MS within the last three years.

The Principal Investigator for this study is Dr. Jeffrey Cohen at the Cleveland Clinic, however, there are many study locations around the United States including at the Rocky Mountain MS Center at University of Colorado. For more information, please visit www.beat-ms.org or reach out to the RMMSC Research Department at 303-724-4644 or neuroresearch@cuanschutz.edu.

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