The Therapeutics Goods Administration of Australia has cleared a regulatory submission of Cyto Therapeutics, a subsidiary of International Stem Cell Corporation ([ISCO], CA, USA), to initiate a Phase I/IIa dose escalation trial of human parthenogenetic stem cell-derived neural stem cells (ISC-hpNSC) in patients with moderate to severe Parkinson’s disease (PD).
There remains no cure for PD, the second most common neurodegenerative disease globally, which affects over 7 million people worldwide. “We are very pleased to start the first human study of ISC-hpNSCs for the treatment of this debilitating disease. There is a large unmet medical need for new treatments that may halt or reverse the progression of Parkinson’s disease and we believe our human neural stem cells may fill this need for the millions of people with this disease” explained ISCO CEO Andrey Semechkin.
In 2014, ISCO announced positive results from preclinical studies into the ISC-hpNSC therapeutic candidate. In these studies, the cell therapy demonstrated an improvement in PD symptoms and increase in brain dopamine levels following intracranial administration. It was further noted that the ISC-hpNSCs provided neurotrophic support and cell replacement to dying dopaminergic neurons.
The newly approved Phase I/IIa clinical study is a dose escalation safety and preliminary efficacy study of ISC-hpNSCs intracranially transplanted into patients with moderate to severe PD. The open-label, single center, uncontrolled clinical trial will evaluate three different dose regimens of 30 to 70 million neural cells. A total of 12 PD patients will be treated. Following transplantation, patients will be monitored for 12 months to evaluate the therapy’s safety and biologic activity. PET scan will be performed at baseline, as part of the screening assessment, and at 6 and 12 months after surgical intervention. Clinical responses following treatment will be evaluated using various neurological assessments and compared with baseline values. The study will be performed at Royal Melbourne Hospital in Melbourne, Australia.
“We are the first company in the world to conduct clinical trials of a human pluripotent stem cell based product for the treatment of PD. We believe the outcome of the study will produce findings in-line with our preclinical studies, where we demonstrated not only safety of our proprietary neural stem cells, but also their functional efficacy. The cells were able to successfully integrate into the brain and provide a significant increase of dopamine levels in the nigrostriatal system,” summarized Russell Kern ISCO’s Executive Vice President and chief scientific officer.
Originally posted on RegMedNet