Breakthrough Treatment in Multiple Sclerosis Replaces Diseased Immune System

Intensive stem cell transplant cleared all signs of brain inflammation in multiple sclerosis patients.

Patients with early, aggressive multiple sclerosis (MS) who underwent immunoablation and autologous hematopoietic stem cell transplantation (IAHSCT) were cleared of all signs of damaging brain inflammation during a recent study.

The $6.47 million trial published in The Lancet followed 24 patients with aggressive, relapsing MS for 13 years with a median post-treatment follow up of 6.7 years. The results of the study showed that there were zero patients who had a clinical relapse, compared with the average of 1.2 relapses per year before treatment.

Furthermore, there were no new active inflammatory lesions detected in the brains of the patients (0 lesions on 327 MRI scans), compared with 188 lesions on 48 scans found in the participants prior to the treatment. Additionally, 70% of patients saw disease progression completely stop.

“Our trial is the first to show the complete, long-term suppression of all inflammatory activity in people with MS,” said lead researcher Harold Atkins. “This is very exciting. However, it is important to note that this therapy can have serious side effects and risks, and would only be appropriate for a small proportion of people with very active MS. People with MS who have had significant disability for a long time would likely not benefit.”

The study also showed that none of the patients required any MS drugs to manage their disease; the average rate of brain shrinkage returned to normal aging levels; 40% of patients had lasting reversal of symptoms, such as vision loss, balance issues, and muscle weakness; and some study patients were able to return to work or school, and regain the ability to drive, get married, and have children.

“This procedure should be considered as a treatment option for people with early, aggressive MS,” said study co-leader Mark S. Freedman. “Although this trial was relatively small, it was intensive, with the longest prospective follow-up of any such treatment group to date, and that is what makes the results so convincing. However, this is a very complex procedure that should only be performed at very specialized centers with expertise in both the management of MS patients and blood stem cell transplantation.”

Currently, the procedure is only being used in patients who have very severe cases due to the treatment’s side effects, which include a significant risk of infection and death. In the study, one patient died during the treatment from liver failure.

Although the regimen was modified mid- study to reduce toxicity, all of the participants still developed fevers.

“Several recent clinical trials from other groups have examined this procedure in people with MS,” Freedman said. “Our study is unique in that we used a stronger cocktail of drugs to eliminate the immune system, we followed the participants for a very long time, and the majority of our participants have had significant, long-lasting responses.”