ScienceDaily: Report on remission in patients with MS three years after stem cell
transplant
December 29, 2014
The JAMA Network Journals
Three years after a small number of patients with multiple sclerosis (MS) were treated with highdose
immunosuppressive therapy (HDIT) and then transplanted with their own hematopoietic stem cells, most of the patients
sustained remission of active relapsingremitting MS (RRMS) and had improvements in neurological function, according
to a study published online by JAMA Neurology.
MS is a degenerative disease and most patients with RRMS who received diseasemodifying therapies experience
breakthrough disease. Autologous (using a patient’s own cells) hematopoietic cell transplant (HCT) has been studied in
MS with the goal of removing diseasecausing immune cells and resetting the immune system, according to the study
background.
The Hematopoietic Cell Transplantation for RelapsingRemitting Multiple Sclerosis (HALTMS) study examines the
effectiveness of early intervention with HDIT/HCT for patients with RRMS and breakthrough disease. The article by
Richard A. Nash, M.D., of the Colorado Blood Cancer Institute at Presbyterian/St. Luke’s Medical Center, Denver, and
coauthors reports on the safety, efficacy and sustainability of MS disease stabilization though three years after the
procedures. Patients were evaluated through five years.
Study results indicate that of the 24 patients who received HDIT/HCT, the overall rate of eventfree survival was 78.4
percent at three years, which was defined as survival without death or disease from a loss of neurologic function,
clinical relapse or new lesions observed on imaging. Progressionfree survival and clinical relapsefree survival were
90.9 percent and 86.3 percent, respectively, at three years. The authors note that adverse events were consistent with
the expected toxic effect of HDIT/HCT and that no acute treatmentrelated neurologic adverse events were seen.
Improvements in neurologic disability, qualityoflife and functional scores also were noted.
“In the present study, HDIT/HCT induced remission of MS disease activity up to three years in most participants. It
may therefore represent a potential therapeutic option for patients with MS in whom conventional immunotherapy fails,
as well as for other severe immunemediated diseases of the central nervous system. Most early toxic effects were
hematologic and gastrointestinal and were expected and reversible. Longer followup is needed to determine the
durability of the response,” the authors conclude.
Editorial: Moving Targets for Stem Cell Transplantation for Patients with MS
In a related editorial, M. Mateo Paz Soldán, M.D., Ph.D., of the University of Utah, Salt Lake City, and Brian G.
Weinshenker, M.D., of the Mayo Clinic, Rochester, Minn., write: “This study and another phase 2 singlearm study
leave little doubt that highdose immunotherapy is able to substantially suppress inflammatory disease activity in
patients with MS who have active disease in the short term. There is some evidence for longterm suppression of MS.
Lessons have been learned about how treatmentrelated morbidity and mortality may be reduced. However, deaths
have occurred, even in small studies, and aggressive regimens have resulted in lymphomas associated with EpsteinBarr
virus.”
“Nash et al show evidence of prolonged depletion of memory CD4+ cells, depletion of CD4+dominant Tcell receptor
clones and evidence of ‘immune reset’; however, clinical or radiologic evidence of relapse trumps immunologic evidence
of immune reset, and this study raises concern that those end points have not been adequately achieved. The jury is
still out regarding the appropriateness and indication of HCT for MS,” the authors conclude.
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ScienceDaily: Report on remission in patients with MS three years after stem cell transplant
