By Donald Zuhn --
On Friday, Geron Corporation announced that the U.S. Food and Drug Administration has approved the biopharmaceutical company's Investigational New Drug (IND) application for the clinical trial of GRNOPC1 in patients with acute spinal cord injury. According to Geron's press release, the clinical trial, to be conducted on patients with complete American Spinal Injury Association (ASIA) grade A subacute thoracic spinal cord injuries, will constitute the world's first study of a human embryonic stem cell (hESC)-based therapy in man. The trial will utilize Geron's lead hESC-based therapeutic candidate, GRNOPC1, which consists of 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 (Kierstead et al., 2005, "Human embryonic stem cell-derived oligodendrocyte progenitor cell transplants remyelinate and restore locomotion after spinal cord injury," J. Neurosci. 25(19): 4694-705). Geron President and CEO Dr. Thomas Okarma called the approval "the beginning of what is potentially a new chapter in medical therapeutics -- 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."
Candidates for the trial must have a functionally complete spinal cord injury and agree to have GRNOPC1 injected into the lesion sites between seven and fourteen days after injury. While the goal of the trial is to determine the safety of GRNOPC1, the trial will also seek to assess efficacy. Once safety has been established, Geron intends to seek approval to increase the dose of GRNOPC1, enroll individuals with complete cervical injuries, and expand the trial to include patients with severe incomplete injuries (ASIA grade B or C). Additional information, including a video illustration of GRNOPC1 in an animal model of spinal cord injury, can be found here.
According to Geron's press release, the production and commercialization of GRNOPC1 is protected by a portfolio of patents owned by or exclusively licensed to Geron. Among the patents in its portfolio are those covering hESCs, which are exclusively licensed to Geron from the Wisconsin Alumni Research Foundation (WARF), and those covering the production of oligodendrocytes from hESCs, which are exclusively licensed to Geron from the University of California.
Human embryonic stem cell image: Ryddragyn, Wikipedia Commons
The FDA has a tough line to walk to try and get medications to people that can receive benefit, but not so early as to miss major problems.
Posted by: Ajlouny | May 31, 2009 at 09:39 PM