To present the design, baseline data and initial safety of subjects enrolled in an ongoing Phase II randomized, double-blind, placebo-controlled trial of mesenchymal stem cells (MSCs) as a disease-modifying therapy for idiopathic Parkinson’s disease (PD).

A total of 45 subjects were enrolled and randomized to 1 of the three treatment arms. 14 patients were randomized to arm 0, 15 were assigned to arm 1, and 16 were allocated to arm 2. The study design includes 3 treatment arms: a) 2 infusions of 10 X 10 ⁶ MSC/kg and 1 placebo; b) 3 infusions of 10 X 10⁶ MSC/kg; and c) 3 infusions of placebo. All subjects have received 3 infusions at 4-month intervals and have entered a 1-year follow-up. The last subject is projected to complete the study in August 2023.

All treatment‐related adverse events have been mild/moderate and temporary. There were no significant changes in CBC and CMP one month after the last infusion. Only one patient had a positive transient immunogenic reaction that could be related to the treatment. All Baseline characteristics appeared similar across groups.  Subjects had a mean age of 66, an average modified H&Y of 2, a disease duration between 2 and 9 years, and mean total MDS-UPDRS total score of 64.9, and a mean MDS-UPDRS-III of 36.3. All patients used dopaminergic medications before baseline evaluation.

This study is the first FDA-approved mesenchymal stem cell study for PD. Preliminary findings showed that repeated doses of intravenous infusion of allogeneic bone marrow-derived MSCs are safe and well-tolerated in PD. Efficacy and preliminary mechanism of action data will be reported in December 2023.