Amyotrophic lateral sclerosis (ALS) is a progressive and fatal neurodegenerative disease characterized by the loss of motor neurons, leading to paralysis and, ultimately, respiratory failure. Given the limitations of current therapies, researchers have been exploring innovative approaches. The objective of the study conducted by Barczewska et al. (2020) was to assess the efficacy and safety of therapy using mesenchymal stem cells derived from Wharton’s jelly in patients with ALS.
Intervention Used in the Study
The study was conducted as a case-control design, including 67 ALS patients who received intrathecal injections of mesenchymal stem cells obtained from Wharton’s jelly (WJ-MSCs). These patients were compared to 67 matched reference patients selected from the PRO-ACT database, an international repository of clinical data on ALS.
Each treated patient received a total of three injections, with a cell dose of 30 million cells per application, administered at two-month intervals.
The stem cells used in the study had rigorously controlled characteristics:
- Obtained from umbilical cords of healthy newborns.
- Processed without enzymatic digestion under controlled laboratory conditions.
- Expanded up to the fifth passage, ensuring maintenance of cellular properties.
- Evaluated for viability, morphology, immunophenotype, microbiological purity, and absence of endotoxins.
- Immunophenotyped according to the criteria established by Dominici et al.
- Cryopreserved and thawed prior to administration.
The effects of the intervention were monitored using the ALS Functional Rating Scale-Revised (ALSFRS-R), along with survival time analysis, with follow-up conducted up to March 2020.
Results Presented by the Study
The study’s findings indicated clinically relevant results:
- The median survival time was doubled across all evaluated groups.
- Disease progression, as measured by changes in the ALSFRS-R scale, presented three distinct patterns:
- A decreased progression rate in 31.3% of treated patients.
- Stabilization, with no change in progression rate, in 49.3% of cases.
- An increased progression rate in 19.4% of patients.
Furthermore, the treatment demonstrated a favorable risk-benefit profile, with no serious adverse reactions reported.
Authors’ Interpretation
The authors concluded that therapy with mesenchymal stem cells derived from Wharton’s jelly is safe and demonstrates positive therapeutic effects in some ALS patients, regardless of clinical and demographic characteristics—except for sex. Female sex and a positive response to the first administration were identified as significant predictive factors for therapeutic efficacy in subsequent applications.
Thus, the authors suggest that WJ-MSC therapy may represent a promising strategy in the management of ALS. However, they emphasize the need for further studies, especially randomized clinical trials, to confirm these findings and to explore in greater detail the underlying mechanisms, as well as the role of genetic factors in the therapeutic response.
Reference: Barczewska M, Maksymowicz S, Zdolińska-Malinowska I, Siwek T, Grudniak M. Umbilical Cord Mesenchymal Stem Cells in Amyotrophic Lateral Sclerosis: an Original Study. Stem Cell Rev Rep. 2020;16(5):922-932. doi:10.1007/s12015-020-10016-7