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Researchers from Italy Report Long-Term Outcomes from Bone Marrow Transplants (aHSCT) to Treat MS

January 20, 2021

Researchers from across Italy have been conducting autologous hematopoietic stem cell transplantation (aHSCT, commonly known as bone marrow transplants) since the late 1990’s to treat individuals with multiple sclerosis who were not helped by standard disease-modifying therapies. Now those researchers have published a case series looking back at outcomes of 210 people with MS who received stem cell transplants from 1997 to 2019. Nearly all of them had relapsing forms of MS, including either relapsing-remitting MS or active secondary progressive MS
  • Background: AHSCT attempts to “reboot” the immune system, which is responsible for damaging the brain and spinal cord in MS. In aHSCT 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.
  • This study: The average age of those studied was 35. Most of those studied received an intense chemotherapy regimen prior to transplant (referred to as BEAM+ATG).
  • Findings: After ten years, 65.5% of the study group continued to experience no worsening of disability. This rose to 71% among those with relapsing-remitting MS. Three individuals died within 100 days of the procedure prior to 2007, and none since then. Those who received the BEAM+ATG chemotherapy regimen and those who had higher levels of relapse activity prior to transplant tended to have better chances of experiencing no worsening of disability at 10 years.
  • Comment: These results add to the increasing evidence that aHSCT can benefit people with a specific type of active MS who have not benefitted from disease-modifying therapies.
  • The National MS Society’s National Medical Advisory Committee recently released recommendations related to the optimal use of aHSCT in MS. To answer remaining questions about the best treatment approach for achieving lasting benefits, a large clinical trial called BEAT-MS is now underway, comparing aHSCT and the most effective disease-modifying therapies.
“Hematopoietic Stem Cell Transplants May Provide Long-Term Benefit for People with MS,” by Drs. Giacomo Boffa, Luca Massacesi, Matilde Inglese, and others on behalf of the Italian BMT-MS study group, was published early online on January 20, 2021 in Neurology, the journal of the American Academy of Neurology.
Read more about aHSCT for MS
Read FAQs about aHSCT for MS (.pdf)
Read about stem cell clinics

About Multiple Sclerosis

Multiple sclerosis is an unpredictable, often disabling disease of the central nervous system. Symptoms range from numbness and tingling to blindness and paralysis, and there is currently no cure for MS. The progress, severity and specific symptoms of MS in any one person cannot yet be predicted, but advances in research and treatment are leading to better understanding and moving us closer to a world free of MS. An estimated 1 million people live with MS in the United States. Most people with MS are diagnosed between the ages of 20 and 50, and it affects women three times more than men.


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