Geron and researchers from the University of California have decided to work together to assess the company’s human embryonic stem cell (hESC) product, GRNOPC1, for Alzheimer disease. The work will be jointly funded by the firm and a university discovery research and training grant.
Geron’s clinical development program with GRNOPC1 has been on again and off again. It has been designed as a treatment for spinal cord injury but has not yet been tested in humans. The Phase I trial with GRNOPC1 was first put on hold in May 2008, then given the go-ahead in January 2009, but by August of last year, FDA had once again revoked its trial sanction. Dose-escalation data from animal studies prompted the last hold.
Geron and the University of California team will now evaluate GRNOPC1 in models of Alzheimer disease. The study is designed to assess whether memory shows recovery after transplantation of GRNOPC1. The research will be led by Frank M. LaFerla, Ph.D., director of the Institute for Brain Aging and Dementia at the University of California, Irvine.
“We are exploring a number of potential applications for GRNOPC1 in neurological disease in addition to spinal cord injury,” says Jane S. Lebkowski, Ph.D., Geron’s svp and CSO, regenerative medicine. “There are striking parallels between recent data on mouse stem cells in Alzheimer’s disease models and what we know about GRNOPC1.”
In recent published studies including data that was published in August 2009 in Proceedings of the National Academy of Sciences, Dr. LaFerla and his colleagues demonstrated that defects in memory were improved by glial cells derived from mouse neural stem cells transplanted into the hippocampus of rodent models of Alzheimer disease.
“Our studies have provided insight into the potential reparative mechanism and the properties that are likely to be required of a human therapeutic cell population,” states Dr. LaFerla. “GRNOPC1 meets these criteria so we will now test this human cell product in Alzheimer’s models.”
GRNOPC1 contains the precursors to human glial cells, which matured and repaired the lesion site in rodent models of spinal cord injury. Additionally, the improvement in memory and the increase in synaptic density observed after injection of neural stem cells were found to be mediated, at least in part, by the neurotrophic factor BDNF, which is secreted from the transplanted cells. GRNOPC1 has been found to secrete BDNF as well as other neurotrophic factors.