International Stem Cell Corporation announce the completion of discussions with the U.S. Food and Drug Administration (FDA) regarding its Parkinson's disease (PD) cell therapy program through a pre-Investigational New Drug (pre-IND) meeting. The meeting provided formal feedback and guidance from the FDA on the proposed pre-clinical plan, manufacturing approach, and clinical study design necessary to file the IND.
"We're pleased with the outcomes of the meeting with the FDA," said Yale School of Medicine Professor D. Eugene Redmond Jr. MD, who is leading ISCO's pharmacology/toxicology study. "The FDA's feedback has provided clear direction for the remaining studies required for the IND."
Following this meeting, ISCO will be working to complete the Good Laboratory Practice (GLP) pharmacology and safety studies, including tumorigenicity, toxicology and tolerability, using the human parthenogenetic neural stem cell (hPNSC) product, a type of self-renewing multipotent cell that is a precursor for the major cells of the central nervous system. To date pre-clinical pilot studies in rodents and primates have shown the hPNSC to be safe, well tolerated, and provide therapeutic benefit by differentiating into dopaminergic neurons and expressing specific neurotrophic factors to protect the nigrostriatal system of the brain. We anticipate being able to report interim primate data before the end of March 2014 and top line final results from these GLP studies later in 2014 and expect to file the IND soon thereafter.
"As this will be the first IND for the Company, we're pleased to have completed this critical milestone," said Dr. Ruslan Semechkin, Chief Scientific Officer of ISCO. "Based on data from our pilot studies, along with the guidance from the FDA, we believe that we're well positioned to successfully transition this program to the clinical stage."
According to the Parkinson's Disease Foundation, an estimated seven to 10 million people worldwide live with PD, with as many as one million of those in the United States alone, more than the combined total of people diagnosed with multiple sclerosis, muscular distrophy, and Lou Gehrig's disease. The total direct and indirect cost of Parkinson's disease is estimated to be nearly $25 billion per year in the United States alone.
A more complete description of the hPNSC product and the GMP (21CFR211) manufacturing method was published together with Prof. Evan Y. Snyder, Director of Stem Cell Research Center at Sanford-Burnham Medical Research Institute in early 2013. Importantly, unlike many other stem cell therapy products, ISCO's proprietary hPNSC are cryopreservable, meaning they can be delivered in a frozen state to hospitals and clinics.