A new study shows the use of chemotherapy followed by autologous haematopoietic stem cell transplantation (aHSCT) has fully halted clinical relapses and development of new brain lesions in 23 of 24 patients with multiple sclerosis for a prolonged period without the need for ongoing medication. This is the first treatment to produce this level of disease control or neurological recovery from MS, but treatment-related risks limit its widespread use.

Researchers from The Ottawa Hospital and the University of Ottawa, Ottawa, Canada, tested whether complete destruction, rather than suppression, of the immune system during aHSCT would reduce the relapse rate in patients and increase long-term disease remission. They enrolled 24 patients aged 18-50 from three Canadian hospitals who had all previously undergone standard immunosuppressive therapy which did not control the MS. All patients had poor prognosis and their disability ranged from moderate to requiring a walking aid to walk 100m, according to their Expanded Disability Status Scale scores.

The study resulted in MS activity-free survival at three years (as measured by relapses of MS symptoms, new brain lesions, and sustained progression of EDSS scores) which occurred in 69.6 percent of patients after transplantation. Eight of the 23 patients had a sustained improvement in their disability 7.5 years after treatment. Out of the 24 patients, one died from hepatic necrosis and sepsis caused by the chemotherapy. Prior to the treatment, patients experienced 1.2 relapses per year on average. After treatment, no relapses occurred during the follow up period (between 4 and 13 years) in the surviving 23 patients.

These clinical outcomes were mirrored by freedom from detectable new disease activity on MRI images taken after the treatment. The initial 24 MRI scans revealed 93 brain lesions, and after the treatment only one of the 327 scans showed a new lesion. Furthermore, progressive brain deterioration typical of MS slowed to a rate associated with normal aging in nine patients with the longest follow-up, and eight of 23 patients had a sustained improvement in their EDSS score at 7.5 years after treatment. At three years, six patients were able to reduce or stop receiving disability insurance and return to work or school. Eight of the 24 patients had a moderate toxic effect and 14 patients had only a mild toxic effect related to transplantation.

“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.” Dr. Mark S Freedman, a study author, said. “This procedure should be considered as a treatment option for people with early, aggressive MS. 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.”

The results were published in The Lancet medical journal.