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Results Published from Australian HSCT Bone Marrow Stem Cell Transplantation Trial in MS

December 13, 2018

Drs. John Moore, David Ma (St. Vincent’s Hospital, Darlinghurst, NSW, Australia) and colleagues reported results of a small clinical trial of HSCT (autologous hematopoietic stem cell transplant) conducted at a single medical center in Australia. HSCT is a procedure that attempts to “reboot” the immune system, which is believed to launch attacks on the brain and spinal cord in people with MS.
  • This trial enrolled 35 people with relapsing-remitting MS or secondary progressive MS whose disease had not responded well to disease-modifying medications. There was no control group or blinding; all participants underwent the HSCT procedure.
  • The team reported on results after following participants from 12 to 66 months after transplantation. After 12 months, 82% remained free of relapses, MRI-detected new or enlarging lesions, and progression (called “Event-Free Survival” or EFS). At two years after transplant, 65% of the group had EFS, and at three years 60%. EFS was better in those who had relapsing MS. Of 8 who experienced MS progression after transplantation, 2 had relapsing-remitting MS and 6 had secondary progressive MS. Twelve of thirteen whose disability scores improved after transplantation had relapsing-remitting MS.
  • At this center, which has a long experience with bone marrow transplants, there were no transplant-related deaths. Many experienced complications expected from the chemotherapy cocktail (called “BEAMS”) used to deplete their bone marrow cells in preparation for the transplant.
  • The results were published early online on December 11, 2018 in the Journal of Neurology, Neurosurgery and Psychiatry.
  • This trial adds to a growing body of knowledge needed to define the precise risks and benefits of HSCT, the best approach to this procedure, and who is most likely to benefit. Additional controlled trials, now in planning stages, will help answer these and other questions.
  • Research to date suggests that those most likely to benefit are 50 years or younger, have had MS for 5 or fewer years, have active relapsing-remitting MS but are still walking, and whose disease is not adequately treated by available disease-modifying therapies.
Read more about HSCT and MS

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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