Stem cell therapy GRNOPC1 for spinal cord break injury goes on human testing: Geron

GRNOPC1, a human embryonic stem cell (hESC)-based therapy to treat spinal cord injury, will soon go on human testing.

US FDA has given approval for Geron Corporation to conduct phase 1 clinical studies using GRNOPC1 in humans removing the restriction imposed on for the last few months.

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 FDA’s decision to allow our planned clinical trial of GRNOPC1 in spinal cord injury to proceed,” said Thomas B. Okarma, Ph.D., M.D., Geron’s president and CEO in a company release.

To achieve restoration of spinal cord function the hESC-derived oligodendrocyte progenitor cells GRNOPC1 will be injected directly into the lesion site of the patient’s injured spinal cord.

Geron is also exploring the utility of GRNOPC1 in other degenerative CNS disorders including Alzheimer’s, multiple sclerosis and Canavan disease, Mr Okarma said

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 US 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.

Patients eligible for the Phase I trial must have documented evidence of functionally complete spinal cord injury with a neurological level of T3 to T10 spinal segments and agree to have GRNOPC1 injected into the lesion sites between seven and 14 days after injury, Geron said.

Although the primary endpoint of the trial is safety, the protocol includes secondary endpoints to assess efficacy, such as improved neuromuscular control or sensation in the trunk or lower extremities.

Geron has selected up to seven U.S. medical centers as candidates to participate in this study and in planned protocol extensions. The sites will be identified as they come online and are ready to enroll subjects into the study.