Abstract Details

Title Serum Neurofilament Light Chain Levels in Parkinson’s Disease Therapy: Preliminary Results from a Phase 2 Clinical Trial of Allogenic Bone Marrow-derived Mesenchymal Stem Cell

Topic Movement Disorders

Presentation(s) S37 - Movement Disorders: Clinical Trials (2:00 PM-2:12 PM)

Poster/Presentation
Number 006

Background

Serum NfL is a sensitive biomarker for neuroaxonal damage proposed as a potential biomarker for PD, as higher levels have been correlated with motor severity and disease progression. In other neurologic disorders, the pathologically elevated levels of serum NfL have been shown to improve after treatment with MSCs. Currently, there is no information on the impact of MSC on NfL in PD.

Objective To establish the impact of Allogenic Bone Marrow-derived Mesenchymal Stem Cells (allo-hMSCs) on serum Neurofilament Light Chain (NfL) in Parkinson’s disease (PD)

Design/Methods Data were retrieved from a Phase 2 trial investigating allo-hMSCs in PD (NCT04506073). Participants were randomized to either three infusions of 10×10⁶ allo-hMSCs/kg, one placebo and two infusions of 10×10⁶ allo-hMSCs/kg, or three infusions of placebo at four-month intervals. Movement Disorder Society-Unified Parkinson's Disease Rating Scale motor subscore (MDS-UPDRS-III) and serum NfL levels (Simoa™ NF-Light® kit) were collected at baseline and 3 months after the last infusion. Changes from the baseline in the MDS-UPDRS-III (ΔMDS-UPDRS-III) and the percentage change in serum NfL (%NfL) were calculated. Blinded adjusted multivariate linear correlations were performed using SPSS v25.

Results

44 patients with PD were included, with a mean age of 67 (SD ±7) and a baseline MDS-UPDRS-III score of 34 (IQR: 30-38), with no significant differences between treatment groups (p=0.56). The median decrease in MDS-UPDRS-III scores was -3.0 in Arm 0, -11.0 in Arm 1, and -16.0 in Arm 2 (p<0.001). All treatment groups showed an increased %NfL (Arm 0=15.8% vs. Arm 1=27.1% vs. Arm 2=19.7%, p=0.48). In Arm 2, %NfL positively correlated with ΔMDS-UPDRS-III (β =+0.11, R²=0.42, p=0.006).

Conclusions These data suggest that serum NfL may serve as a potential predictor of motor severity improvement in patients with PD treated with allo-hMSCs, making it a valuable biomarker for therapeutic follow-up. Unblinded analyses and larger trials are needed for its validation.

Authors/Disclosures
Juan D. Martinez Lemus, MD (Hospital Simón Bolívar) PRESENTER	Dr. Martinez Lemus has nothing to disclose.
Emily Tharp, MD	Dr. Tharp has nothing to disclose.
Timothy M. Ellmore, PhD (The City College of New York)	Prof. Ellmore has nothing to disclose.
Robert Ritter III	Mr. Ritter has nothing to disclose.
Tran B. Le, MD	Dr. Le has nothing to disclose.
Chiamaka C. Onuigbo, MD	Dr. Onuigbo has nothing to disclose.
Mya C. Schiess, MD, FAAN (Univ of Texas-Houston Med School)	Dr. Schiess has nothing to disclose.

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