On Friday (7/30/10), GERN said the clinical hold on its application has been lifted. The 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.
In 8/09, the GRNOPC1 trial experienced safety problems during the animal study stage, prompting the FDA to place a clinical hold on its investigational new drug application for the therapy.
The Phase I multi-center trial is designed to establish the safety of GRNOPC1 in patients with “complete” American Spinal Injury Association (NASDAQ:ASIA) Impairment Scale grade A sub-acute thoracic spinal cord injuries.
- GRNOPC1, GERN’s lead hESC-based therapeutic candidate, contains hESC-derived oligodendrocyte progenitor cells that have demonstrated re-myelinating and nerve growth stimulating properties leading to restoration of function in animal models of acute spinal cord injury.
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.
- Once safety in this patient population has been established, GERN plans to seek FDA approval to extend the study to increase the dose of GRNOPC1, enroll subjects with complete cervical injuries and expand the trial to include patients with severe incomplete (ASIA Impairment Scale grade B or C) injuries to enable access to the therapy for as broad a population of severe spinal cord-injured patients as is medically appropriate,
- 7 U.S. medical centers were selected as candidates to participate in this study and in planned protocol extensions.
Shares of the stem cell research company rose 17% to $5.60 in pre-market trade. They closed at $4.80 Thursday (7/29/10).