International Stem Cell Corporation is now enrolling patients in a Phase 1 clinical trial of its lead product candidate, ISC-hpNSC — human parthenogenetic stem cell-derived neural stem cells — for patients with moderate to severe Parkinson’s disease.
Parthenogenesis uses unfertilized eggs to create pluripotent human stem cells and hopes to significantly advance the field of regenerative medicine by addressing the problem of immune-rejection.
The Phase 1 approval in patients with moderate to severe Parkinson’s was granted by the the Melbourne Health Human Research Ethics Committee, and the study will be conducted at the Royal Melbourne Hospital in Australia.
“Enrollment in this trial is an important milestone. Promising preclinical results support our expectation that ISC-hpNSC will bring a long-needed solution for patients suffering from Parkinson’s disease,” Russell Kern, Ph.D., executive vice president and chief scientific officer of ISCO, said in a recent press release. “The ability of our approach to replace and protect dopaminergic neurons and restore neural function offers significant potential benefit to patients. We look forward to preliminary clinical data in Q4 2016.”
The company believes that a one-time transplant of hpNSCs into the mid-brain of Parkinson’s patients, replacing dead and dying neurons and offering protection to the remaining neurons, will alleviate disease symptoms and prevent further deterioration. Importantly, preclinical studies in rodents and non-human primates have shown improvement in Parkinson’s symptoms and increase in brain dopamine levels following intracranial administration of ISC-hpNSC.
The Phase 1 open-label, single center, uncontrolled clinical dose escalation safety and pilot efficacy clinical trial will evaluate ISC-hpNSC, which will be intracranially transplanted in 12 patients with moderate to severe Parkinson’s. The trial will assess three different dose regimens of 30,000,000 to 70,000,000 neural cells.
After transplantation, all patients will be followed for 12 months to evaluate the safety and biologic activity of ISC-hpNSC. At baseline, all patients will be assessed with a PET scan as well as at 6 and 12 months following surgical intervention. All patients will be evaluated with the Unified Parkinson Disease Rating Scale (UPDRS), Hoehn, and Yahr as well as other tools to assess the clinical outcomes.