International Stem Cell Corp. is inching closer to bringing its stem cell therapy for Parkinson's to the FDA in hopes of eventually reversing the disease's symptoms in people.
In collaboration with researchers at the Yale University Medical School, International Stem Cell Corp. has released positive data from a key preclinical trial that sets the Carlsbad, CA-based company on course for filing an investigational new drug application with the FDA by the end of the year.
Results from a first interim analysis of the ongoing pharmacology and toxicology primate study show that monkeys with Parkinson's treated with the company's stem cell therapy began to have improved behaviors beginning three to four months after they were given an injection of the therapy.
The study consists of 18 primates divided into three groups--a control group and two treatment groups receiving different doses of human neural stem cells (hPNSC) derived from its proprietary parthenogenetic stem cell line (hpSC). The hPNSCs are self-renewing multipotent cells that are precursors for the major cells of the central nervous system. These cells are able to differentiate into dopaminergic neurons and express neurotrophic factors, essentially replacing lost neurons with new neuronal cells.
More than half of the 9 primates that received the stem cell therapy have shown significant improvement in parkinsonism, including a return of many normal behaviors.
"Seeing behavioral improving is very encouraging," Simon Craw, executive vice president of International Stem Cell Corp., told FierceBiotechResearch.
The new data follow positive preclinical results of the company's stem cell therapy in rats unveiled in March 2013.
"This will be the first IND for Parkinson's using a pluripotent stem cell-based therapy," Craw said. Previous studies have used fetal neuronal cells to treat neurogenerative disorders like Parkinson's. But there are two major problems associated with this approach. First, the source of these cells presents an ethical issue since they must be taken from human fetuses, and second, fetal neuronal cell transplantation for Parkinson's has caused the movement disorder dyskinesia in some studies.
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