The FDA is allowing Geron to initiate a Phase I trial using its human embryonic stem cell (hESC) product, GRNOPC1, in patients with acute spinal cord injury. The decision lifts a clinical hold on the treatment, which the agency issued in May 2008.
This is reportedly the first time hESCs will be tested in humans. The cells are derived from the H1 human embryonic stem cell line, which was created before August 9, 2001. Studies using this line thus qualify for U.S. federal research funding, although Geron says that no such funding has been received thus far.
Geron reports that the Phase I study will be a multicenter investigation to establish the safety of GRNOPC1 in patients with complete American Spinal Injury Association (ASIA) grade A subacute thoracic spinal cord injuries. GRNOPC1 contains hESC-derived oligodendrocyte progenitor cells that have demonstrated remyelinating and nerve growth stimulating properties leading to restoration of function in animal models of acute spinal cord injury.
“The ultimate goal for the use of GRNOPC1 is to achieve restoration of spinal cord function by the injection of hESC-derived oligodendrocyte progenitor cells directly into the lesion site of the patient’s injured spinal cord,” explains Thomas B. Okarma, Ph.D., M.D., Geron’s president and CEO.
“This marks the beginning of what is potentially a new chapter in medical therapeutics,” notes Dr. Okarma. “One that reaches beyond pills to a new level of healing: the restoration of organ and tissue function achieved by the injection of healthy replacement cells."