A Single Arm, Open-Label Phase 1 Study to Evaluate the Safety and Tolerability of ISC-hpNSC® Injected Into the Striatum and Substantia Nigra of Patients With Parkinson’s Disease
International Stem Cell Corporation is currently running a Phase I clinical trial using human parthenogenetic stem cell-derived neural stem cells (ISC-hpNSC®). The company, through its wholly-owned Australian subsidiary, Cyto Therapeutics, is currently enrolling patients for the treatment of moderate to severe Parkinson’s disease in Melbourne, Victoria, Australia at the Department of Neurology of the Royal Melbourne Hospital.
The study is designed to primarily determine safety and tolerability of ISC-hpNSC® with secondary efficacy measures. We are hoping that participants in this trial may benefit from this unique approach for the treatment of Parkinson’s disease which could alter disease progression. The Therapeutic Goods Administration (TGA), Department of Health and Ageing (Australian equivalent of the U.S. Food and Drug Administration) and the Melbourne Health Human Research Ethics Committee (HREC) approved the Phase I clinical trial in patients with moderate to severe Parkinson’s disease after reviewing the company’s extensive pre-clinical data.
Please refer to this study by its ClinicalTrials.gov identifier: NCT02452723
What is ISC-hpNSC®?
ISC-hpNSC® is a cellular therapeutic consisting of neural stem cells derived from human parthenogenetic stem cells (hpSC). The ability of ISC-hpNSC® to both differentiate into dopaminergic neurons and express brain-protecting neurotrophic factors offers a new approach to treating Parkinson’s disease. We believe that a one-time transplant of ISC-hpNSC® into the brain of Parkinson’s patients, replacing the dead and dying dopaminergic neurons and offering protection and neurotrophic support to the remaining neurons, could alleviate the current symptoms and prevent further deterioration.
ISCO’s preclinical studies show that administration of ISC-hpNSC® was safe and improved motor symptoms, increased dopamine levels, innervation and number of dopaminergic (DA) neurons in PD animal models compared to controls.
Purpose of the Clinical Trial
This clinical trial under way at the Royal Melbourne Hospital in Australia is designed to demonstrate the safety of ISC-hpNSC® as a treatment for Parkinson’s disease. In the phase I trial, three groups of four patients will each receive a specific dose of ISC-hpNSC®, ranging from 30,000,000 to 70,000,000 neural stem cells. Primary outcome measure in the first year will look for incidence of treatment-emergent adverse events (TEAEs). Secondary outcome measures will look for change in Unified Parkinson’s Disease Rating Scale (UPDRS) score and other neurological assessments from baseline.
Men or women aged 30 to 70 years are eligible for participation in the clinical trial. Additional criteria include a diagnosis of idiopathic PD of ≤ 13 years duration, as defined by the United Kingdom (UK) Parkinson’s Disease Society Brain Bank criteria, Hoehn and Yahr stage II-IV during “ON” time, UPDRS motor score (Part III) in the “OFF” state ≤ 49, and Montreal Cognitive Assessment (MOCA) score ≥ 26. More detailed inclusion and exclusion criteria are available at ClinicalTrials.gov, identifier NCT02452723.
Department of Neurology, Royal Melbourne Hospital
Melbourne, Victoria, Australia, 3050
To enroll in the trial contact: Lisa J Braumen, BA 613 9035 7231, Ibrauman@unimelb.edu.au
About Cyto Therapeutics
Cyto Therapeutics is the wholly owned subsidiary of International Stem Cell Corporation conducting the clinical trial for Parkinson’s disease in Australia. Based in Australia, Cyto Therapeutics has received regulatory approval for the clinical application of ISC-hpNSC® and is currently enrolling patients for its Phase I study.
The human parthenogenetic Neural Stem Cells (ISC-hpNSC®) used in the clinical trial have been extensively tested for safety and efficacy in preclinical models.
According to the Parkinson’s Disease Foundation, an estimated seven to ten million people worldwide live with Parkinson’s disease.
It is widely accepted that the degeneration of DA neurons causes a gradual dysfunction of the motor system leading to symptoms such as tremor, rigidity, and bradykinesia. There is currently no cure for PD and although treatments such as deep brain stimulation and levodopa can alleviate some of the symptoms, they tend to lose efficacy over time.
Grafting fetal neural tissue has shown significant biochemical and clinical improvements in some PD patients. However, fetal neural stem cells have limitations and are clinically impractical. ISCO has developed the alternative, ISC-hpNSC®, a renewable, clinical grade source of neural stem cells which could provide a solution to these problems.