National MS Society Releases Recommendations for aHSCT-Bone Marrow Transplant for MS

The National Medical Advisory Committee of the National MS Society has written an article reviewing evidence related to the optimal use of autologous hematopoietic stem cell transplantation (aHSCT, commonly known as bone marrow transplants) for the treatment of specific types of relapsing multiple sclerosis. The committee’s recommendations are now published (JAMA Neurology, online October 26, 2020).

• There is growing evidence that aHSCT is not for everyone with MS but may be highly effective for people with relapsing MS who meet very specific characteristics.
• The committee outlines what is known and not known about aHSCT in MS, and makes recommendations for its use (preferably in clinical trials), and for further research – see details below.
• The committee’s findings are published in JAMA Neurology (online October 26, 2020).

 DETAILS

Background: Despite the availability of disease-modifying pharmaceutical therapies for multiple sclerosis, some people continue to experience disease activity. There is growing evidence that autologous hematopoietic stem cell transplantation (aHSCT, commonly known as bone marrow transplants) can be highly effective for people with relapsing MS who meet specific characteristics. This once risky procedure appears to be getting safer when done by experienced and certified practitioners. AHSCT attempts to “reboot” the immune system, which is responsible for damaging the brain and spinal cord in MS. In HSCT for MS, hematopoietic (blood cell-producing) stem cells, which are derived from a person’s own (scientifically referred to as “autologous”) blood or bone marrow, are collected and stored, prior to depleting much of the immune system using chemotherapy drugs. Then the stored stem cells are reintroduced to the body. The new stem cells migrate to the bone marrow and over time reconstitute the immune system. There are differences in the way this procedure has been carried out, and so it has been difficult to compare the results of different studies. These differences include characteristics of those who underwent the procedure, differences in how the stem cells were stimulated, in the extent the immune system is depleted, and in whether the stored stem cells are sorted or given whole. Recommendations: The Society’s National Medical Advisory Committee, chaired by Dr. Aaron Miller (Corinne Goldsmith Dickinson Center for Multiple Sclerosis, Icahn School of Medicine at Mt. Sinai, New York), reviewed the current evidence to provide recommendations on the appropriate use of this aHSCT to treat MS. They also recommended areas needing further research. Recommendations include: WHO

• The best candidates for this procedure are people who
  • are less than 50 years of age
  • have had MS for 10 years or less
  • have relapsing MS and have had “breakthrough” disease activity (new inflammatory MS lesions in the central nervous system, and/or clinical relapses) despite treatment with a highly effective disease-modifying therapy (or for whom the use of a highly effective disease-modifying therapy is contraindicated).

WHERE

• That people with MS considering this procedure enlist in a clinical trial if available
  • In the U.S., a phase 3 clinical trial, called BEAT-MS, has recently begun.
• If not in connection with a clinical trial, individuals should have it performed by medical teams with experience with both aHSCT and MS. Seek transplant specialists whose centers are certified by the Foundation for the Accreditation of Cellular Therapies (in the U.S.), or (in Europe) the European Society for Blood and Marrow Transplantation.

 Further research: The committee indicated the need for further research that will determine the optimum protocols for this procedure, and for clinical trials that compare aHSCT to the best available disease-modifying therapies. They also recommend that a single registry be used to track individuals’ longer-term outcomes so that this treatment can be optimized for safety and efficacy. NOTE RELATED TO COVID-19: The MS International Federation has posted guidance related to aHSCT suggesting that people who have recently undergone treatment should consider extending the period they remain in isolation during the COVID-19 outbreak to at least six months. People who are due to undergo treatment should consider postponing the procedure in consultation with their healthcare professional. If aHSCT treatment is given, chemotherapy should be administered in rooms isolated from other hospital patients. “Autologous Hematopoietic Stem Cell Transplant in Multiple Sclerosis: Recommendations of the National Multiple Sclerosis Society,” by Aaron Miller, MD, Tanuja Chitnis, MD, and others, was published in JAMA Neurology on October 26, 2020.

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