Stem cells halt CNS inflammatory activity for years in PhII MS trial

MS Demyelinisation
(By Marvin 101 (Own work) [GFDL ( or CC-BY-SA-3.0 (], via Wikimedia Commons)

A Phase II trial of autologous hematopoietic stem-cell transplantation (aHSCT) has reported long-term suppression of all inflammatory activity in patients with aggressive multiple sclerosis, an outcome that emboldened one researcher to dare to utter the word "cure."

The treatment, which entails immunosuppressing patients with chemotherapy and antibodies before performing aHSCT, is the immune system equivalent of restoring a computer to factory settings. In the first phase of the treatment, destructive components of the immune system and immunological memory are wiped out, leaving the patient with something approaching a blank slate. The aHSCT, a procedure that entails harvesting bone marrow, processing it to remove mature immune cells and infusing it back into the patient, is then performed to rebuild the immune system.

Researchers hoped this would free patients with particularly aggressive forms of multiple sclerosis from the debilitating autoimmune effects of their conditions. And, while the trial carries the caveats typical of small, single-arm studies, the data appear to back up the hypothesis. Disease progression halted in 70% of the 24 participants in the Phase II trial. One-third of participants improved on the Expanded Disability Status Scale (EDSS) in the three years following treatment. A further one-third stayed at baseline, a notable result in itself given the aggressive nature of their conditions.

The data have prompted some big claims. “Everyone is hesitating to use the 'c word,' but these patients are cured," Michael Rudnicki, director of the Regenerative Medicine Program and the Sprott Centre for Stem Cell Research at the Ottawa Health Research Institute, told Vox. "Jennifer Molson [one of the study participants] was in a wheelchair in a rehab center unable to work. And now she's skiing, she's working, she got married, got her driver's license. I think this is going to be the new standard of care for progressive MS." Rudnicki wasn’t involved in the research project.

Dr. Harold Atkins, who led the study, was more circumspect, noting that the study has followed the participants in the trial for, at most, 13 years. It is unclear how the patients’ conditions may change in the years ahead, both in terms of their multiple sclerosis and the other ailments that could arise as a result of their transplants. Other reasons for caution include the intensity of the treatment--one of the participants died--and the fact that around one-third of the subjects continued to progress on EDSS in the three years after the intervention.

The researchers behind the project think around 5% of multiple sclerosis patients will be eligible for the procedure, and that such restrictive inclusion/exclusion criteria were a major factor in the success of the study. “The strength of these data lies in the careful selection of patients with aggressive inflammatory disease who were deemed to have a very poor prognosis,” the researchers wrote in The Lancet.

- read the Lancet abstract
- and Vox’s piece

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