Geron Corporation announced that the US Food and Drug Administration (FDA) has notified the company that the clinical hold placed on Geron's Investigational New Drug (IND) application has been lifted and the company's phase-I clinical trial of GRNOPC1 in patients with acute spinal cord injury may proceed.

The US FDA notification enables Geron to move forward with the world's first clinical trial of a human embryonic stem cell (hESC)-based therapy in man. 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.

"We are pleased with the US FDA's decision to allow our planned clinical trial of GRNOPC1 in spinal cord injury to proceed," said Thomas B Okarma, Geron's president and CEO. "Our goals for the application of GRNOPC1 in subacute spinal cord injury are unchanged - 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. Additionally, we are now formally exploring the utility of GRNOPC1 in other degenerative CNS disorders including Alzheimer's, multiple sclerosis and Canavan disease."

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

GRNOPC1, Geron's lead hESC-based therapeutic candidate, 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 neurosurgical community is ready to begin the clinical testing of this new approach to treating devastating spinal cord injury," said Richard Fessler, professor of neurological surgery at the Feinberg School of Medicine at Northwestern University. "We know that demyelination is central to the pathology of the injury, and its reversal by means of injecting oligodendrocyte progenitor cells would be revolutionary for the field. If found to be safe and effective, the therapy would provide a viable treatment option for thousands of patients who suffer severe spinal cord injuries each year."

Geron is developing first-in-class biopharmaceuticals for the treatment of cancer and chronic degenerative diseases, including spinal cord injury, heart failure and diabetes.