Multiple sclerosis (MS) is a chronic disease that affects the central nervous system, leading to a range of physical and cognitive impairments. There are different types of MS, with the most common being relapsing-remitting MS (RRMS) and secondary progressive MS (SPMS). RRMS is primarily an inflammatory condition, while SPMS involves a progressive decline due to neurodegeneration.
While significant advancements have been made in treating RRMS with new medications that target inflammation, treatment options for SPMS remain limited.
In this study, Fernández et al. aimed to evaluate the safety and feasibility of using adipose-derived mesenchymal stem cells (AdMSCs) in patients with SPMS.
Investigating Stem Cell Therapy for SPMS
Currently, the only approved treatments for SPMS with disease activity are interferon β and mitoxantrone, but their effectiveness is either uncertain or associated with serious side effects. Ocrelizumab has recently been approved for treating PPMS, but effective treatments for SPMS are still lacking.
Recently, stem cells have shown promise in various medical applications, particularly in regenerating damaged tissues and modulating the immune system. Currently, stem cell treatments are approved for conditions like blood disorders and severe burns, but mesenchymal stem cells (MSCs) are still being investigated for other uses.
Fernandez et al.’s study was conducted in two hospitals in Spain and involved a placebo-controlled, randomized trial. Patients were divided into three groups: one receiving a placebo, another receiving a lower dose of stem cells, and the third receiving a higher dose. To ensure unbiased results, the study was triple-blinded, meaning neither the patients, treating physicians, nor the statisticians analyzing the data knew which treatment each patient received.
The study also required participants to provide 30 grams of adipose tissue for stem cell preparation. Once enrolled, patients received their assigned treatment and were monitored for a year with follow-up visits at 30 days, six months, and twelve months after treatment.
Study Results and Safety Evaluation
A total of 34 patients were initially enrolled in the study. However, four patients were excluded before receiving the treatment due to abnormalities in their stem cell samples. The remaining 30 patients completed the study, with 29 undergoing at least one follow-up assessment. Unfortunately, two patients in the placebo group passed away during the trial—neither death was linked to the stem cell treatment.
Throughout the study, 70 adverse events were reported among 22 patients, with the most common being urinary infections, respiratory infections, and anemia. Four serious adverse events occurred, but only one (a urinary infection) was in a patient who received stem cells.
Overall, the authors report that there were no major safety concerns related to the treatment.
Evaluating the Effectiveness of Stem Cell Therapy
To determine whether stem cell therapy had any effect on disease progression, the authors also analyzed various clinical measures, including the Expanded Disability Status Scale (EDSS), MRI scans, and evoked potentials (tests measuring nerve function).
At the end of the 12-month follow-up, no significant differences were found between the placebo and treatment groups in terms of disability progression. The EDSS scores remained relatively stable in all groups, and MRI scans showed no significant reduction in active lesions or brain volume loss compared to placebo.
Fernández et al. report some positive trends in nerve function tests, particularly in visual evoked potentials, which measure the brain’s response to visual stimuli. Some patients in the treatment groups showed improvements in the speed of their nerve signals, suggesting possible stabilization or mild improvement. However, these changes were not statistically significant when compared to the placebo group.
AdMSC Therapy in SPMS: Findings, Challenges, and Future Directions
This study provides valuable insights into the safety of using AdMSCs in SPMS patients. The treatment was well tolerated, with no major safety concerns.
While there were some encouraging trends in nerve function, Fernández et al. call for larger and longer-term studies to determine whether these changes translate into meaningful clinical benefits. Considering this, the authors call for future research focusing on optimizing stem cell delivery methods, exploring combination therapies, and identifying patient subgroups that may benefit the most from stem cell treatments.
Source: Fernández O, Izquierdo G, Fernández V, Leyva L, Reyes V, Guerrero M, et al. (2018) Adipose-derived mesenchymal stem cells (AdMSC) for the treatment of secondary-progressive multiple sclerosis: A triple blinded, placebo controlled, randomized phase I/II safety and feasibility study. PLoS ONE 13(5): e0195891. https://doi.org/10.1371/journal.pone.0195891
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