A potential treatment for multiple sclerosis

Excerpts from the report at JAMA Neurology (the new name for the Archives of Neurology):

High-dose immunosuppressive therapy (HDIT) with autologous hematopoietic cell transplant (HCT) may.. induce sustained remissions in early MS.

Results  Grafts were collected from 25 patients, and 24 of these individuals received HDIT/HCT. The median follow-up period was 186 weeks (interquartile range, 176-250) weeks). Overall event-free survival was 78.4% (90% CI, 60.1%-89.0%) at 3 years. Progression-free survival and clinical relapse-free survival were 90.9% (90% CI, 73.7%-97.1%) and 86.3% (90% CI, 68.1%-94.5%), respectively, at 3 years. Adverse events were consistent with expected toxic effects associated with HDIT/HCT, and no acute treatment-related neurologic adverse events were observed. Improvements were noted in neurologic disability, quality-of-life, and functional scores.


Conclusions and Relevance  At 3 years, HDIT/HCT without maintenance therapy was effective for inducing sustained remission of active RRMS and was associated with improvements in neurologic function. Treatment was associated with few serious early complications or unexpected adverse events.

In the present study, HDIT/HCT induced remission of MS disease activity for up to 3 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 immune-mediated diseases of the central nervous system. Most early toxic effects were hematologic and gastrointestinal and were expected and reversible. Longer follow-up is needed to determine the durability of the response... We have presented the 3-year interim analysis of the HALT-MS trial; the prespecified full duration of observation is 5 years, after which the final report on the study will be prepared.