Stem Cell-Derived Therapy Shows Promise in Progressive Multiple Sclerosis

Intrathecal dosing of neural progenitors appears to be safe and well tolerated in patients with progressive multiple sclerosis.

VANCOUVER, British Columbia — Intrathecal administration of autologous mesenchymal stem cell-derived neural progenitors (MSC-NPs) was safe and well tolerated as a regenerative multiple sclerosis (MS) therapy, concluded a first-of-its-kind study presented at the 2016 annual meeting of the American Academy of Neurology (AAN).

“In addition, the majority of patients showed improvement in their [Expanded Disability Status Scale (EDSS)] score, a well-recognized scale that measures disability,” said Saud A. Sadiq, MD, study investigator with the Tisch MS Research Center, New York. “The majority of patients with bladder dysfunction also noted improvement, both symptomatically and based on urodynamic testing.”

Dr Sadiq explained in an interview with Neurology Advisor that the catalyst for the study was the current lack of a treatment to reverse disability in disabled patients with progressive MS.

“Multiple intrathecal dosing of neural progenitors in an animal model reversed clinical disability,” he said. “We therefore wanted to establish the safety and tolerability of our approach in the phase 1 trial. Although the trial was not designed to determine efficacy, we measured a number of disability determinants.”

For the study, Dr Sadiq and colleagues administered autologous MSC-NPs, an autologous bone marrow-derived population with regenerative potential, in 3 doses (spaced 3 months apart) of up to 10 million cells per injection.

Researchers enrolled 20 patients with MS who met the following criteria: established disability (range, 3.5-8.5); relatively stable disease as demonstrated by less than 1-point change in EDSS score in the last year; and stable magnetic resonance imaging disease burden with no enhancing lesions in the previous 6 months.

Adverse event assessments served as the primary safety outcome measure, while EDSS score, timed 25-foot walk, peg test, paced auditory serial addition test, evoked potentials, and urodynamics testing served as secondary efficacy end points.

All 20 patients received intrathecal MSC-NPs, and 13 patients received all 3 doses and underwent 3-month follow-up.

Overall, no patients experienced serious adverse events. Minor adverse events were reported, however, including approximately 65% of patients who experienced transient headache and/or fever.

Following treatment, a subset of patients demonstrated neurologic improvement, including improved EDSS scores and peg tests, as well as better bladder function clinically and on urodynamic assessment.

“Our conclusion thus far is that multiple [intrathecal] dosing of neural progenitors is safe, well tolerated, and appears to be efficacious in reducing disability with progressive MS,” Dr Sadiq said. “The strong efficacy signal is somewhat unexpected and warrants a double-blind study to be performed.”

Specifically, he said there is a need for well-controlled double-blind studies using autologous stem cells to determine if long-standing disability can be reversed in MS and related neurologic diseases.

“The Tisch Center is currently planning a multicenter, phase 2, double-blind trial to determine the efficacy of intrathecal neural progenitor therapy to reverse disability in patients with MS,” Dr Sadiq said. “We hope to commence this trial in the later part of 2016.”

Click here for more coverage from the 68th Annual Meeting of the American Academy of Neurology, April 15-21, 2016, in Vancouver, British Columbia, Canada.


Sadiq S, Blackshear L, Joo G, et al. Multiple intrathecal dosing of neural progenitors administered to progressive MS patients with disability is safe and improves disability scores. Presented at: The 68th Annual Meeting of the American Academy of Neurology; April 15-21, 2016; Vancouver, British Columbia, Canada.  Abstract I10.008.