Results From Phase II Trial Suggest Stem Cell Treatment May Improve Certain Symptoms of Multiple Sclerosis

Researchers led by Saud A. Sadiq, M.D., director and chief research scientist of the Tisch Multiple Sclerosis Research Center of New York, completed a phase 2 clinical trial investigating the use of autologous stem cell-derived neural progenitors as a treatment option in patients with progressive forms of multiple sclerosis (MS).

The study results were published last month in Stem Cell Research & Therapy.

The National Multiple Sclerosis Society provided $1 million in partial funding for the trial.

The study included 54 patients with secondary-progressive or primary-progressive MS and an Expanded Disability Status Scale (EDSS) score between 3.0 and 6.5. EDSS scores range from 0 to 10, with larger numbers indicating a greater level of disability.

Participants were randomized to receive autologous bone marrow mesenchymal stem cell (MSC)-derived neural progenitors (MSC-NP) or saline by intrathecal injection. MSC-NPs are a type of MSCs that have an enhanced expression of neural and cell signaling genes.

Half of the participants received MSC-NP injections every two months for one year, and the other half received saline injections. The two groups were switched during the second year, so each participant received the study treatment for one year.

The primary study outcome was improvement in the EDSS Plus score. This is a composite score of the EDSS scale, timed 25-foot walk, and nine-hole peg test, which measures upper limb function.

The study did not meet the primary endpoint.

However, the researchers noted that some secondary outcomes were achieved. These included improvements in bladder function and the six-minute walk test.

The participants in the MSC-NP groups also had reduced brain gray matter atrophy and changes in biomarkers that indicated a potential for reduced inflammation and tissue repair.

No serious adverse effects were reported.

The researchers concluded, “Although the primary outcome of EDSS-based improvement was not met, the significant improvement in secondary walking outcomes addresses an unmet need in MS patients with progressive disability.”

They added, “[Intrathecal] MSC-NP injection was associated with improved bladder function which is a relevant quality of life issue in people with MS. In addition, we found indirect evidence of a neuroprotective effect as seen by brain MRI cortical gray matter volume changes.”

Sadiq and his colleagues recommend further studies with endpoints measuring ambulatory abilities and optimal dosing of MSC-NPs.