Objective: To select the safest and most effective number of repeat doses of allogeneic mesenchymal stem cells (MSC) purified from bone marrow-derived from a healthy adult delivered intravenously to slow the progression of Parkinson’s disease (PD). Secondary outcomes for safety and tolerability were assessed by serious adverse reactions and immunologic responses in each active treatment arm versus placebo at 40 weeks follow-up.

Background: Considerable evidence supports the rationale for using an immunomodulatory therapy such as MSCs to restore neuronal-glial homeostasis. Findings from our Phase I trial demonstrated that a single intravenous infusion of MSCs was safe and well-tolerated¹.

Method: Forty-five subjects with iPD were recruited and randomized to 1 of the three treatment arms (Arm 0=14, Arm 1=15, Arm 2=16). The 3 treatment arms include: a) 2 infusions of 10 X 10⁶ MSC/kg & 1 placebo; b) 3 infusions of 10 X 10⁶ MSC/kg or c) 3 infusions of placebo. All subjects received 3 infusions at 4-month intervals and are currently followed to 88 weeks.

Results: *Baseline characteristics appeared similar across groups with no statistically significant differences between age, disease duration, MDS-UPDRS-III, MDS-UDRS-Total, LEDD (Table 1).

*Forty-three patients received 3 infusions of either placebo or MSC; 2 received 2 infusions due to one withdrawal and one immunologic reaction. All patients were followed up to 6 months after their last infusion.

*There were no serious adverse reactions in the first 24hrs after each infusion

*There were 78 adverse events, with 11 classified as treatment‐emergent adverse events (TEAE), Table 2. All TEAE have been mild/moderate and transient. 45 % of all TEAE were present in arm 1. The most common TEAE was fatigue (6.6%, N=3). The rest of the TEAE were present singularly (2.2%, N=1).

*There were no significant changes in CBC and CMP six months after the last infusion.

*One patient had a positive immunogenic reaction that could be related to the treatment. Reactivity resolved 6 months post-treatment.

Conclusion: Preliminary findings from this ongoing Phase II study demonstrated that allogeneic MSC infusions appear safe and well tolerated in subjects with mild to moderate PD. Our preliminary data warrant the completion of the 88-week study.

References: 1Schiess, M., Suescun, J., Doursout, M.-F., Adams, C., Green, C., Saltarrelli, J.G., Savitz, S. and Ellmore, T.M. (2021), Allogeneic BoneMarrow–Derived Mesenchymal Stem Cell Safety in Idiopathic Parkinson’s Disease. Mov Disord, 36: 1825-1834.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/preliminary-report-on-the-safety-and-tolerability-of-allogeneic-bone-marrow-derived-mesenchymal-stem-cells-as-a-disease-modifying-therapy-for-parkinsons-disease-phase-iia-double-blind-rando/