Mesenchymal Stem Cells Transplantation to Patients With Parkinson's Disease

Phase 1

• Conditions: Parkinson's Disease
• Interventions: Biological: bone marrow derived mesenchymal stem cells

• Registration Number: NCT01446614
• Lead Sponsor: Guangzhou General Hospital of Guangzhou Military Command

Brief Summary

The study is a phase I/II trial designed to establish the safety and efficacy of intravenous administration of autologous bone marrow derived mesenchymal stem cells to patients with Parkinson's disease.

Detailed Description

Parkinson's disease (PD) is a common progressive neurodegenerative disorder caused by the loss of dopaminergic neurons in the substantia nigra. A combination of genetic and environmental factors is likely to be important in producing abnormal protein aggregation within select groups of neurones, leading to cell dysfunction and then death. A large number of agents together with surgical interventions are now available to treat early and late complications of PD, but they are suffer from two main drawbacks: side effects and loss of efficacy with disease progression.

Bone marrow (BM) derived mesenchymal stem cells (MSCs) an differentiate under certain circumstances into cells from various neuronal and glial type lineages; they also exert immunomodulatory effects. PD-derived MSCs are similar to normal MSCs in phenotype, morphology, and multidifferentiation capacity. Moreover, PD-derived MSCs are capable of differentiating into neurons in a specific medium with up to 30% having the characteristics of dopamine cells. These findings indicate that MSCs derived from PD patients' bone marrow may be a promising cell type for cellular therapy.

BM-MSCs cultured with a cocktail of growth factors (containing FGF and BDNF) differentiate into neuronal/glial lineage cells with a predominance of cells expressing astrocytes' markers. They were effective in suppression of chronic EAE in mice and induced neuroprotection, preserving most of the axons in the CNS of successfully-treated animals. Histopathological studies revealed that MSCs could efficiently migrate into the CNS inflamed tissue (both when administered intravenously and intraventricularly) and differentiated into cells expressing neural-glial lineage markers. Such an approach may provide a feasible and practical way for PD.

Study Timeline

• Status Verified: 2011-10
• Study Start: 2011-10
• Study First Submitted: 2011-10-04
• Study First Submitted QC: 2011-10-04
• Last Update Submitted: 2011-10-04
• Study First Posted: 2011-10-05
• Last Update Posted: 2011-10-05
• Primary Completion: 2013-06
• Study Completion: 2014-06

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

• Patient with current diagnosis of idiopathic Parkinson's disease.
• Age 30 to 65.
• Experiencing motor complications despite optimized levodopa treatment.
• PD of Stage 2，2.5，3 or 4 of Hoehn-Yahr staging.
• Time between diagnosis and enrollment greater than 2 years.
• No significant cognitive impairment. MMSE > 24.
• Ability to understand and the willingness to sign a written informed consent document.

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Exclusion Criteria

• Patients may not be receiving any other investigational agents within 4 weeks of study entry.
• History of allergic reactions attributed to compounds of similar biologic composition to mesenchymal stem cells.
• Primary hematologic diseases.
• Patients undergo intracranial surgeries or implantation of a device for Parkinson's disease.
• Psychiatric, addictive or any other disorder that compromises ability to give a truly informed consent and perform all study assessments.
• Atypical or secondary parkinsonism.
• Malignancy within the last 5 years.
• Any other serious medical illness that might preclude safe participation in the study.
• Pregnant or breastfeeding women.
• HIV-positive patients.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
MSC	bone marrow derived mesenchymal stem cells	Intravenous autologous bone marrow derived mesenchymal stem cells infusion to patients with Parkinson's disease.

Primary Outcome Measures

Name	Time	Method
Number of participants with adverse events	1 month after transplantation

Secondary Outcome Measures

Name	Time	Method
Effect assessment	12 months after transplantation	Assessed by UPDRS

Trial Locations

Locations (1)

Guangzhou General Hospital of Guangzhou Military Command; 🇨🇳 Guangzhou, Guangdong, China