Patient enrollment and dosing is now complete for International Stem Cell Corporation’s (ISCO) Phase 1 trial testing its ISC-hpNSC neural stem cells — a cellular therapy that offers a new approach for treating Parkinson’s disease.
“We are excited to announce the completion of enrollment of the world’s first approved human pluripotent stem cell-based clinical trial for the treatment of Parkinson’s disease. This is a major milestone for the Company and we expect to announce complete clinical results of this phase 1 clinical trial in the first half of 2020,” Andrey Semechkin, PhD, ISCO’s co-chairman and CEO, said in a press release.
ISC-hpNSC, which stands for human parthenogenetic stem cell-derived neural stem cells, is a cellular therapeutic that can not only differentiate into dopaminergic neurons, but can also release brain-protecting agents, offering a new approach for Parkinson’s treatment.
A one-time transplant of ISC-hpNSC into the brains of Parkinson’s patients can replace the dead and dying dopaminergic neurons and can offer protection to the remaining neurons, reducing disease symptoms and preventing further deterioration, according to the company.
The open-label, single center, Phase 1 trial (NCT02452723) — ongoing at The Royal Melbourne Hospital in Australia — is evaluating the safety, tolerability and preliminary effectiveness of transplanting ISC-hpNSC into Parkinson’s patients over a 12-month period, with a five-year, long-term follow-up.
A total of 12 patients are divided into three groups, each injected with 30 million, 50 million, or 70 million ISC-hpNSC. The stem cells are injected directly into the striatum and substantia nigra regions of the brain, which are directly affected in Parkinson’s disease.
The 12th and final trial participant recently received the transplant, with the highest dose of ISC-hpNSC. Eight participants are already in the five-year follow-up phase.
The trial’s interim results will be presented at the 2019 American Academy of Neurology 71st Annual Meeting, taking place in Philadelphia May 4-11.
At six and 12 months after the cell transplants, patients undergo a positron emission tomography (PET) scan, a non-invasive imaging technique that allows clinicians to visualize the metabolic processes in the body.
Researchers also will compare the participants’ clinical response before and one year after receiving ISC-hpNSC, using the Unified Parkinson Disease Rating Scale (UPDRS), which measures the course of disease; the Parkinson’s Disease Quality of Life Questionnair-39, which assesses difficulties in daily living; and patient motor diaries.
Results from a six-month analysis after cell transplants revealed the therapy was safe, with no serious adverse events. The company called this “a very significant achievement due to the invasive nature of the transplantation procedure.”
“[N]ow that we have completed the most expensive stage of the phase 1 clinical trial, ISCO will have more resources available to invest in growing and developing its commercial business, where we have recently made significant progress,” Semechkin said.