Overview
This is a phase I, interventional, single arm, open-label, clinical study to evaluate the safety and efficacy of the striatal transplantation of autologous induced neural stem cell-derived DA precursor cells in Parkinson's Disease patients.
Description
Parkinson's Disease (PD) is the second most common neurodegenerative disease, caused by progressive depletion of midbrain dopaminergic neurons in the substantia nigra pars compacta. This clinical study will include the preparation of dopaminergic neural precursor cells derived from neural stem cells through reprogramming of patient's peripheral blood mononuclear cells (PBMCs), and transplantation of the obtained cells into the brains of PD patients by stereotaxic injection. Safety, tolerability, evidence of cell survival (using PET scan), and the efficacy on PD symptoms will be assessed at different time points up to 12 months post-transplantation.
Eligibility
Inclusion Criteria:
Ages between 30 and 85 years, males or females; Diagnosed to be Parkinson's disease
patients according to MDS Parkinson's disease diagnostic criteria; Disease history over 3
years; Hoehn and Yahr Stage less than or equal to 4 during the medication "on" time;
Responsive to levodopa treatment (Maximum rate of improvement in MDS-UPDRS, part 3, is over
30%).
Exclusion Criteria:
Atypical Parkinsonian syndrome or secondary Parkinsonian syndrome; Accompanied with other
central nervous system diseases; With other severe systemic diseases or dysfunction; With
severe psychiatric disorders; Subjects are using hormone or cytotoxic drugs and cannot stop
taking the drug during the trial; With cognitive disorders (MMSE<24); With severe
dyskinesia (MDS-UPDRS part 4, score in 4.1/4.2 ≥ 2); Subjects have undergone previous brain
surgery; Subjects are long-term user of anticoagulant; Subjects have intracranial lesions
which may affect the surgery or follow-up studies as assessed by imaging; Subjects are
unable to undergo MRI or AV133 PET examination; Pregnancy or in preparation for pregnancy;
Not suitable to participate in this clinical trial as assessed by the study
investigators/physicians.