Design and Baseline characteristic of Phase II clinical trial of allogeneic bone marrow-derived Mesenchymal Stem Cells as a disease-modifying therapy for idiopathic Parkinson’s disease (P1-11.004)

Abstract

<h3>Objective:</h3> 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). <h3>Background:</h3> Neuroinflammation plays a vital role in the pathogenesis of PD, thus supporting the rationale for using an immunomodulatory therapy such as MSCs for restoring homeostasis to the neuronal-glial microenvironment. <h3>Design/Methods:</h3> 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. <h3>Results:</h3> 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&amp;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. <h3>Conclusions:</h3> 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. <b>Disclosure:</b> Dr. Schiess has nothing to disclose. The institution of Prof. Ellmore has received research support from NIMH. The institution of Dr. Shahnawaz has received research support from American Parkinson Disease Association. The institution of Dr. Shahnawaz has received research support from NIH. The institution of Dr. Shahnawaz has received research support from Texas Alzheimer’s Research &amp; Care Consortium . Dr. Shahnawaz has received intellectual property interests from a discovery or technology relating to health care. Marie-Francoise Doursout has nothing to disclose. Dr. Adams has stock in Medtronic. Dr. Tharp has nothing to disclose. Dr. Suescun has nothing to disclose.