Last week Geron announced the enrollment of the first patient in the company’s clinical trial of human embryonic stem cell (hESC)-derived oligodendrocyte progenitor cells, GRNOPC1.
The primary objective of this Phase I study is to assess the safety and tolerability of GRNOPC1 in patients with complete American Spinal Injury Association (ASIA) Impairment Scale grade A thoracic spinal cord injuries. The study represents the word’s first clinical embryonic stem cell trial and occurs eleven years after the company started working with hESCs and twenty two months after the trial was first approved by the Food and Drug Administration.
On January 23, 2009 the FDA approved the world’s first test in people of a therapy derived from human embryonic stem cells. The phase I trial was approved for Geron Corp to inject stem cells into 8 to 10 spinal cord injury patients 7 to 14 days after the injury occurs. Geron’s therapy involves using various growth factors to turn embryonic stem cells into precursors of neural support cells called oligodendrocytes, which are then injected into the spinal cord at the site of the injury.
The therapy was developed in collaboration with Hans Keirstead of the University of California, Irvine who published a paper in the May 11 issue of The Journal of Neuroscience which demonstrated that human embryonic stem cells were successful in restoring the insulation tissue for neurons in rats treated seven days after spinal cord injury, which led to a recovery of motor skills.
Below is a video of Hans Keirstead’s reaction to news that the FDA approved the world’s first embryonic stem cell therapy study in humans.
According to Geron’s latest press release:
“Initiating the GRNOPC1 clinical trial is a milestone for the field of human embryonic stem cell-based therapies,” said Thomas B. Okarma, Ph.D., M.D., Geron’s president and CEO. “When we started working with hESCs in 1999, many predicted that it would be a number of decades before a cell therapy would be approved for human clinical trials. This accomplishment results from extensive research and development and a succession of inventive steps to enable production of cGMP master cell banks, scalable manufacture of differentiated cell product, and preclinical studies in vitro and in animal models of spinal cord injury, leading to concurrence by the FDA to initiate the clinical trial.”
For a great primer on stem cell research download the free guide:Stem Cell Basics for Life Science Researchers from Bio-Rad Laboratories.