Objective: To assess the efficacy of repeated intravenous (IV) allogeneic bone marrow-derived mesenchymal stem cell (allo-hMSC) infusions in reducing motor and non-motor symptoms in people with Parkinson’s disease (PwPD).

Background: Neuroinflammation has a critical role in the pathogenesis and the relentless degeneration of neurons in Parkinson’s Disease (PD). allo-hMSC therapy has the potential  to slow disease progression due to its immunomodulatory, anti-inflammatory, and neurotrophic activity[1-3]

Method: Our investigator-initiated, randomized, double-blind, placebo-controlled phase 2 trial was conducted between November 2020 and July 2023 at a single academic center in USA. Participants included men and women aged 50-79 years with mild-to-moderate PD (Hoehn & Yahr stages 1-3, disease duration of 2-10 years). They were randomized into one of three groups: (1) three IV infusions of 10×10⁶ allo-hMSCs/kg (N=16), (2) an IV placebo infusion followed by two IV infusions of 10×10⁶ allo-hMSCs/kg (N=14), or (3) three IV placebo infusions (N=15). Infusions were administered at 18-week intervals, with follow-ups conducted at 62 and 88 weeks. Secondary outcomes included the Movement Disorders Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) Total Score (sum of parts I-IV) in the OFF-medicine state at baseline, 62, and 88 weeks. Bayesian statistical analysis was used to compare the posterior probability (PP) of achieving an improvement in the average clinical progression of PD patients, defined as a ≥12-point increase, between the treatment arms and placebo[4]. Data was analyzed using R.v.4.2.0.

Results: 45 participants were enrolled, with 42 completing the week 62 follow-up and 40 completing the week 88 follow-up (figure 1). Demographic and clinical characteristics were similar between groups at baseline (table1). Patients receiving three allo-hMSC infusions demonstrated 96.3% probability of ≥12-point improvement on the MDS-UPDRS Total Score compared to placebo at week 62, and ≥99.9% probability at week 88. In contrast, patients receiving two allo-hMSC infusions failed to show improvement of ≥12 points on the MDS-UPDRS Total Score at either 62 or 88 weeks compared to placebo (figure2).

Conclusion: Three repeated IV doses of allo-hMSCs/kg reduced motor and non-motor symptoms in PwPD, highlighting the potential of allo-hMSC therapy to slow disease progression.

Table 1. Baseline Characteristics

Figure 1. Recruitment and Randomization

Figure 2. MDS-UPDRS-Total

References: 1. Unnisa A, Dua K, Kamal MA. Mechanism of Mesenchymal Stem Cells as a Multitarget Disease- Modifying Therapy for Parkinson’s Disease. Curr Neuropharmacol. 2023;21(4):988-1000. doi: 10.2174/1570159X20666220327212414. PubMed PMID: 35339180; PMCID: PMC10227913.
2. Heris RM, Shirvaliloo M, Abbaspour-Aghdam S, Hazrati A, Shariati A, Youshanlouei HR, Niaragh FJ, Valizadeh H, Ahmadi M. The potential use of mesenchymal stem cells and their exosomes in Parkinson’s disease treatment. Stem Cell Res Ther. 2022;13(1):371. Epub 20220728. doi: 10.1186/s13287-022-03050-PubMed PMID: 35902981; PMCID: PMC9331055.
3. Schiess M, Suescun J, Doursout MF, Adams C, Green C, Saltarrelli JG, Savitz S, Ellmore TM. Allogeneic Bone Marrow-Derived Mesenchymal Stem Cell Safety in Idiopathic Parkinson’s Disease. Mov Disord. 2021;36(8):1825-34. Epub 20210327. doi: 10.1002/mds.28582. PubMed PMID: 33772873; PMCID: PMC8451899
4. Schrag A, Sampaio C, Counsell N, Poewe W. Minimal clinically important change on the unified Parkinson’s disease rating scale. Mov Disord. 2006 Aug;21(8):1200-7. doi: 10.1002/mds.20914. PMID: 16673410.

« Back to 2025 International Congress

MDS Abstracts - https://www.mdsabstracts.org/abstract/impact-of-allogeneic-mesenchymal-stem-cells-in-motor-and-non-motor-symptoms-of-parkinsons-disease-a-phase-2-randomized-trial/