An international study for people with highly active relapsing multiple sclerosis has reported that those having stem cell treatment had slower disability progression.
The study recruited 110 people with relapsing MS who had two or more relapses in the previous year whilst taking a disease modifying drug (DMD). Half were randomly assigned to have autologous haematopoietic stem cell transplantation (AHSCT) and half assigned to take a different type or stronger DMD than they had taken the year before. The study allowed anyone taking a DMD to switch to AHSCT if they had disability progression after one year in the study.
The main measure used in the study was disability progression after at least 1 year, defined as a worsening of EDSS score of 1 point.
Of the 110 participants, 103 remained in the trial with 98 assessed at year 1 and 23 evaluated for five years. The average follow-up period was 2.8 years.
Significantly fewer people in the AHSCT group experienced disability progression after 1 year compared to those in the DMD group: progression occurred in 3 out of 52 people in the AHSCT group and in 34 out of 51 people in the DMD group. Progression increased over time, but significantly fewer progressed over time in the AHSCT group compared to the DMD group.
During the first year, EDSS scores decreased (improved) in the AHSCT group by a little over one point (from 3.38 to 2.36) and increased (worsened) by 0.67 in the DMD group (3.31 to 3.98).
There were no deaths in either group. There were no serious events immediately after AHSCT. Infection post-transplant was the most common side effect. Idiopathic thrombocytopenic purpura, a serious bleeding disorder, occurred in two people in the AHSCT group, and four in the AHSCT group developed autoimmune thyroid disease.
What does it mean?
The results suggest AHSCT is an effective treatment for people with highly active relapsing MS.
The investigators acknowledge the limitations of the study, including the small number of participants available to evaluate longer-term outcomes, and the fact that some of the more effective DMDs such as Lemtrada (alemtuzumab), Ocrevus (ocrelizumab) and Mavenclad (cladribine) were not among the therapies offered to those in the DMD group. The investigators consider this study preliminary, and recommend that further research is needed to confirm these findings and to determine longer-term outcomes and safety.
Burt RK, et al.
Effect of nonmyeloablative hematopoietic stem cell transplantation vs continued disease-modifying therapy on disease progression in patients with relapsing-remitting multiple sclerosis. A randomized clinical trial.
JAMA 2019; 321: 165-174.
Results from this study were previously presented at a scientific meeting in Lisbon, March 2018.
More about autologous haematopoietic stem cell transplantation (AHSCT)
Autologous haematopoietic stem cell transplantation (AHSCT) uses high doses of cancer chemotherapy drugs to wipe out harmful cells in your immune system. Your own stem cells are used to ‘regrow’ your immune system so that it no longer attacks myelin or causes inflammation in the brain and spinal cord. We caught up with consultant neurologist Eli Silber and asked him to tell us more about stem cell treatments for MS.