TY  - JOUR
AU  - Jamali, Fatima
AU  - Aldughmi, Mayis
AU  - Khasawneh, Mohammad W
AU  - Dahbour, Said
AU  - Salameh, Alaa A
AU  - Awidi, Abdalla
PY  - 2021
DA  - 2021/10/22
TI  - A New Tool for Safety Evaluation and a Combination of Measures for Efficacy Assessment of Cotransplanting Human Allogenic Neuronal Stem Cells and Mesenchymal Stem Cells for the Treatment of Parkinson Disease: Protocol for an Interventional Study
JO  - JMIR Res Protoc
SP  - e29695
VL  - 10
IS  - 10
KW  - Parkinson disease
KW  - neurodegenerative disease
KW  - regenerative medicine
KW  - mesenchymal stem cells
KW  - MSCs
KW  - neuronal stem cells
KW  - NSCs
KW  - Unified Parkinson Disease Rating Scale
KW  - UPDRS
KW  - Mobility Lab
KW  - α-synuclein
KW  - PARK-7
KW  - stem cells
KW  - stem cell therapy
KW  - therapeutics
KW  - Parkinson’s
KW  - neurological diseases
AB  - Background: Parkinson disease (PD) is a neurodegenerative disorder associated with a broad spectrum of motor and nonmotor symptoms. Any proposed cure needs to address the many aspects of the disease. Stem cell therapy may have potential in this regard as indicated in recent preclinical and clinical studies. Objective: This protocol aims to examine the safety and therapeutic benefit of human Wharton jelly-derived mesenchymal stem cells (WJ-MScs) and their derivatives, neuronal stem cells (NSCs) in PD. Methods: This clinical trial is a double-arm, single-blinded, phase I-II interventional study. Participants have been allocated to 1 of 2 groups: one receiving allogeneic WJ-MSCs alone, the other receiving NSCs and WJ-MScs. Participants are being followed-up and assessed over a period of 6 months. To assess safety, an incidence of treatment-emergent adverse events (TEAEs) tool tailored for PD is being used immediately and up to 6 months after treatment. For efficacy assessment, a number of factors are being used, including the gold standard severity test and the Unified Parkinson Disease Rating Scale. In addition, the following standardized assessments for different common symptoms in PD are being included: motor (both subjectively and objectively assessed with wearable sensors), sensory, quality of life and psychological well-being, cognition, and sleep quality. Furthermore, immune-modulatory cytokines and neuronal damage versus regeneration markers in PD, including the neuronal protein linked to PD, α-synuclein, are being monitored. Results: Ten patients have been enrolled in this study and thus participant recruitment has been completed. The study status is active and beyond the recruiting stage. Study chart implementation, data collection, and analysis are ongoing. Conclusions: The combination of NSCs and MSCs in PD may be useful for harnessing the best of the immunomodulation and neural repair characteristics of these cell types. The tailored comprehensive and scaled TEAEs and the variety of evaluation tools used enables a comprehensive assessment of this cellular therapy treatment protocol. A consideration of this expanded tool set is important in the design of future clinical studies for PD. Trial Registration: ClinicalTrials.gov NCT03684122; https://clinicaltrials.gov/ct2/show/NCT03684122 International Registered Report Identifier (IRRID): DERR1-10.2196/29695 
SN  - 1929-0748
UR  - https://www.researchprotocols.org/2021/10/e29695
UR  - https://doi.org/10.2196/29695
UR  - http://www.ncbi.nlm.nih.gov/pubmed/34677138
DO  - 10.2196/29695
ID  - info:doi/10.2196/29695
ER  -