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Dr. Richard Burt of Northwestern University in Chicago reported interim results of an international clinical trial of HSCT (autologous hematopoietic stem cell transplant) at the European Society for Bone and Marrow Transplantation in Lisbon. 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. The trial is being performed at Northwestern University, Rush University Medical Center, University of Sao Paulo, Uppsala University and Sheffield Teaching Hospitals NHS Foundation Trust.

• This trial enrolled 110 people with active MS whose disease has not responded to disease-modifying medications. Half of the participants stayed on their medication and half underwent HSCT.
• So far, the team reports that one year after enrollment, no severe toxicities or deaths have occurred in those who received the transplants. One MS relapse occurred in the transplant group versus 39 relapses in those continuing their medication. The team also reported improvement in MS disability in those who had received the transplants.
• The trial is ongoing but no longer recruiting new participants.
• Further details will be available when the study is completed and results are published.
• Additional controlled trials, now in planning stages, will help determine who may respond best and which approach to HSCT is optimal for treating MS.
• 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.

“These preliminary results from one of the first well-controlled clinical trials of HSCT for MS are exciting,” said Bruce Bebo, PhD, Executive Vice President of Research at the National MS Society. “This trial adds to a growing body of knowledge that is helping to define the precise risks and benefits of HSCT and who is more likely to benefit.”

Read more about HSCT and MS