Treatment with Umbilical Cord Stem Cells Safe with Sustained Benefits for MS, Trial Shows
Treatment with umbilical cord stem cells was found to be safe and leads to sustained improvements in disability and brain lesions of multiple sclerosis (MS) patients, according to a clinical trial.
The study, “Clinical feasibility of umbilical cord tissue-derived mesenchymal stem cells in the treatment of multiple sclerosis,” was published in the Journal of Translational Medicine.
Although current treatments for MS are able to reduce the frequency of flare-ups and slow disease progression, they are not able to repair the damage to nerve cells or the myelin sheath, the protective layer around nerve fibers.
Mesenchymal stem cells (MSCs) are adult stem cells found in multiple tissues, such as umbilical cord, bone marrow, and fat. These cells are able to mature into bone, cartilage, muscle, and adipose tissue cells.
MSCs may inhibit immune-mediated alterations. In particular, MSCs derived from the umbilical cord have a high ability to grow and multiply, increase the production of growth factors, and possess superior therapeutic activity, compared with other MSCs.
Diverse clinical studies have shown that MSCs can safely treat certain immune and inflammatory conditions, including MS.
The research team had previously demonstrated that MSCs can also improve cognitive and motor function.
Recent results with placenta or umbilical cord MSCs showed few mild or moderate adverse events, as well improvements in patients’ level of disability.
Researchers at the Stem Cell Institute in Panama have now completed a one-year Phase 1/2 clinical study (NCT02034188) to test the effectiveness and safety of umbilical cord MSCs for the treatment of MS.
The trial included 20 MS patients with a mean age of 41 years, 60 percent of whom were women. Fifteen participants had relapsing-remitting MS, four had primary progressive MS, and one had secondary progressive MS. Patients’ disease duration was a mean of 7.7 years.
Participants received seven intravenous infusions of 20×106 umbilical cord MSCs over seven days. The treatment’s effectiveness was evaluated at the start, at one month, and at one year after treatment.
Assessments included evaluating brain lesions with magnetic resonance imaging (MRI) and disability based on the Kurtzke Expanded Disability Status Scale (EDSS), as well as validated MS tests for neurological function, hand function, mobility, and quality of life.
Patients did not report any serious adverse events. Most mild adverse events possibly related to treatment were headaches, which are common after MSC infusions, and fatigue, which is common in MS patients, the authors observed.
Improvements were most evident at one month after treatment, namely in the level of disability, nondominant hand function, and average walk time, as well as bladder, bowel, and sexual dysfunction. Patients also reported improved quality of life.
MRI scans at one year after treatment revealed inactive lesions in 15 of 18 evaluated patients. One patient showed almost complete elimination of lesions in the brain, which “is a particularly encouraging finding,” the researchers wrote.
At the one year point, improvements in disability levels were also still present, and could translate into improved ability to walk and work without assistance.
“The potential durable benefit of UCMSC [umbilical cord MSC] at 1 month, and sustained in some measures to 1 year, is in stark contrast to current MS drug therapies, which are required to be taken daily or weekly,” the researchers wrote.
The safety of the treatment is another advantage over available MS therapies, the team said.
They concluded that “treatment with UCMSC intravenous infusions for subjects with MS is safe, and potential therapeutic benefits should be further investigated.”