Clinical Trial of Embryonic Stem Cell Therapy Authorized

Geron Corporation of Menlo Park, California said today that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold placed on the company’s investigational new drug and its Phase I clinical trial of GRNOPC1 in patients with acute spinal cord injury may proceed.

GRNOPC1 is Geron’s lead human embryonic stem cell (hESC)-based candidate. The company says it contains hESC-derived oligodendrocyte progenitor cells that have demonstrated remyelinating (re-generation of the nerve’s myelin sheath) and nerve growth stimulating properties leading to restoration of function in animal models of acute spinal cord injury.

The Phase I multi-center trial is designed to establish the safety of GRNOPC1 in patients with “complete” American Spinal Injury Association (ASIA) Impairment Scale grade A subacute thoracic spinal cord injuries, defined as having no motor and sensory function in the anal and perineal region representing the lowest sacral cord (S4-S5).

According to Geron, the clinical hold was placed following results from a single preclinical animal study in which a higher frequency of small cysts was observed within the injury site in the spinal cords of animals injected with GRNOPC1 than had previously been noted. In response, Geron developed new markers and assays as additional release specifications for GRNOPC1. The company completed an additional preclinical animal study to test the new markers and assays, and submitted a request to the FDA for the clinical hold to be lifted.