Geron Forms Collaboration to Investigate GRNOPC1 in Alzheimer's Disease Models

Project is Awarded University of California Discovery Research and Training Grant

MENLO PARK, CA, USA | January 26, 2009 | Geron Corporation (Nasdaq: GERN) today announced that it has entered into a collaboration jointly funded by Geron and a University of California Discovery Research and Training Grant to investigate the therapeutic potential of its human embryonic stem cell (hESC)-based product, GRNOPC1, for Alzheimer's disease. The company is currently developing GRNOPC1 for the potential treatment of spinal cord injury.


Under the collaboration, GRNOPC1 will be evaluated in models of Alzheimer's disease. The study is designed to assess whether memory, which is impaired in the rodent models as it is in human patients, shows recovery after transplantation of GRNOPC1. The research will be led by Professor Frank M. LaFerla, 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," said Jane S. Lebkowski, Ph.D., Geron's senior vice president and chief scientific officer, regenerative medicine. "There are striking parallels between recent data on mouse stem cells in Alzheimer's disease models and what we know about GRNOPC1. We are looking forward to testing GRNOPC1 in Alzheimer's models."

In recent published studies, Professor LaFerla and his colleagues have 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's disease.

"We are excited about this collaboration with Geron," said Frank M. LaFerla, Ph.D. "Our recent studies with mouse neural stem cells have shown that a cell therapeutic approach may have application for the treatment of Alzheimer's disease. Additionally, 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. GRNOPC1 meets these criteria so we will now test this human cell product in Alzheimer's models."

The transgenic mouse models of Alzheimer's disease that will be used in the study were developed by Professor LaFerla's research group. Alzheimer's mouse models have been used extensively for investigating potential therapeutic approaches because they recapitulate key pathologies that are associated with progressive loss of memory in the human disease, including the extensive loss of neurons in the hippocampus.

Rationale for the Approach

The results from Professor LaFerla's previous studies provide a strong rationale for the potential application of GRNOPC1 in Alzheimer's disease. The most recent data were published in August of 2009 in the Proceedings of the National Academies of Sciences.

The studies were set up to investigate whether a stem cell-based therapy could potentially rescue or replace the cells that are lost in Alzheimer's disease and thereby improve memory. Since past studies have shown that murine neural stem cells can differentiate into all mature cell types of the central nervous system, in the LaFerla studies murine neural stem cells were transplanted into the brains of Alzheimer?s disease models. The models reproduce impaired memory, which can be assessed using standard behavioral tests.

Professor LaFerla's studies showed that memory improved in the models after neural stem cell transplantation. When the brains of the transplanted Alzheimer?s mice were analyzed, an increase in neuronal synaptic connections was found. Importantly, mature glial cells derived from the injected stem cells were found to be the predominant cell type in the transplanted area. New neurons were very rare. GRNOPC1 contains the precursors to human glial cells, which have been shown to mature and repair the lesion site in rodent models of spinal cord injury.

In addition, 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.

About 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. The disease starts with the loss of neural connections, called synapses, in the hippocampus, affecting memory, and progresses to the cortex affecting language and thought.

Currently there is no cure for Alzheimer's disease. Treatments can provide some relief of symptoms but do not stop progression or prevent the disease.

Alzheimer's disease is the most common cause of dementia in the elderly and the most significant and costly neurological disorder. An estimated 5 million people in the U.S. have Alzheimer's disease with nearly 500,000 new cases predicted each year. As our population ages, the number of new cases is expected to rise significantly.

About Geron

Geron is developing first-in-class biopharmaceuticals for the treatment of cancer and chronic degenerative diseases, including spinal cord injury, heart failure and diabetes. The company is advancing an anti-cancer drug and a cancer vaccine that target the enzyme telomerase through multiple clinical trials in different cancers. For more information about Geron, visit


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