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.
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, Geron 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.
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.
Other Potential Neurological Indications for GRNOPC1
In addition to spinal cord injury, GRNOPC1 may have therapeutic utility for other central nervous system indications. Geron has established a number of collaborations with academic groups to test GRNOPC1 in selected animal models of human disease for which there is a strong rationale for the approach.
Alzheimer's Disease: Alzheimer's disease is a progressive, fatal, degenerative disorder that attacks the neurons in the brain, resulting in loss of memory, cognitive function such as reasoning and language, and behavioral changes. According to the Alzheimer's Association an estimated five million people in the United States have Alzheimer's disease. GRNOPC1 is being evaluated in animal models of Alzheimer's disease in collaboration with Professor Frank M. LaFerla, Director of the Institute for Memory Impairments and Neurological Disorders (UCI MIND) at the University of California, Irvine.
Multiple Sclerosis (MS): MS is an autoimmune disease that causes demyelination of nerve axons in the brain and spinal cord often progressing to physical and cognitive disability. There is currently no known cure for the disease. According to the National Multiple Sclerosis Society there are about 400,000 people in the United States with MS. GRNOPC1 is being tested in a non-human primate model of MS in collaboration with Professor Jeffery D. Kocsis of the Departments of Neurology and Neurobiology at Yale University School of Medicine and the Department of Veterans Affairs.
Canavan Disease: Canavan disease is a fatal neurological disorder that belongs to a group of genetic disorders called leukodystrophies, characterized by the abnormal development or degeneration of myelin. Symptoms of Canavan disease present in the first six months of life and death usually occurs at 3 – 10 years of age. GRNOPC1 is being tested in a rodent model of Canavan disease in collaboration with Dr. Paola Leone, Director of the Cell and Gene Therapy Center, at the University of Medicine and Dentistry of New Jersey.