Objective: To select the safest and most effective number of repeat doses of allogeneic mesenchymal stem cells (MSC) delivered intravenously to slow the progression of Parkinson’s disease (PD). As the primary outcome, efficacy will be determined by the change in the MDS-UPDRS-III score (≥-5) in each active treatment arm versus placebo between baseline and 40 weeks follow-up.

Background: Considerable evidence supports the rationale for using an immunomodulatory therapy such as MSCs to restore neuronal-glial homeostasis. Our Phase I trial demonstrated a significant motor improvement based on a treatment effect that relies on paracrine immune modulation and increased blood flow¹.

Method: Forty-five subjects with iPD were randomized to 1 of the three treatment arms (Arm Zero=14, Arm One=15, and Arm Two= 16). The 3 treatment arms include: a) 2 infusions of 10 X 10⁶ MSC/kg and 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 being followed. Preliminary analysis using Bayesian statistics was performed one-month post final infusion (week 40). Generalized linear modeling was used to evaluate motor improvement as a function of treatment.

Results: *Baseline characteristics appeared similar across groups with no statistically significant differences.

*The estimated treatment effect for the MDS-UPDRS-III was 0.355 for arm Zero (95% CrL 0.141-0.631), 0.946 for arm One (95% CrL 0.752-0.998), and >0.99 for arm Two (95% CrL 0.933->0.99).

*The probability of a decrease of ≥ -5 in the MDS-UPDRS-III was 36% for arm Zero, 93% for arm One, and 100% for arm Two.

*Mean and percentage MDS-UPDRS-III change per treatment arm from baseline was -5.86 (9.5%)  for arm Zero, -12.20    (34.6%) for arm One, and -16.13 (43.4%) for arm Two.

*Mean and percentage MDS-UPDRS-Total change per treatment arm from baseline was -7.09 (11.3%)  for arm Zero, -18.87(28.6%) for arm One, and -26.50 (40.1%) for arm Two.

Conclusion: Preliminary findings from this ongoing Phase II study demonstrated a significant difference in treatment effect between the three arms for MDS-UPDRS-III and MDS-UDRS-Total. These results warrant completing the 88-week study and the final unblinded analysis to quantify treatment efficacy.

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-efficacy-of-allogeneic-bone-marrow-derived-mesenchymal-stem-cells-as-a-disease-modifying-therapy-for-idiopathic-parkinsons-disease-phase-iia-double-blind-randomized-contr/