The California Institute for Regenerative Medicine (CIRM) has awarded $20 million to UCI Institute of Memory Impairments and Neurological Disorders (UCI MIND) to advance stem cell treatment of Alzheimer’s disease in partnership with Stem Cell, Inc. of Newark, California. Led by a disease team that includes Dr. Frank LaFerla, a world-renowned researcher in Alzheimer’s disease who serves as director of UCI MIND and Dr. Mathew Blurton-Jones, Assistant Professor in the Department of Neurobiology and Behavior at UCI, the four-year project is designed to bring neural stem cell treatment into human clinical trials. Both researchers are part of the UCI Bill and Sue Gross Stem Cell Research Center.
Neural stem cell therapy for Alzheimer’s has shown promise in studies using mice that have been genetically programmed to develop the disease. When neural stem cells were injected into the hippocampus, a structure important for learning and memory compromised early in Alzheimer’s disease, the mice showed improvements in both memory and synaptic connectivity. In fact, the neural stem cells migrated to other areas of the brain, showing the potential to more broadly impact the extensive neuronal death that occurs in Alzheimer’s disease.
“The stem cells were releasing a factor,” explained Dr. LaFerla, “and that factor was sort of acting like a fertilizer for the brain, and causing the surviving neurons to put out more connections. So it was enhancing the health of the surviving neurons in the brain.”
Funds will support preclinical development of stem cell therapy for Alzheimer’s disease, that is, the additional studies using genetically engineered mice needed to move the treatment forward to human trials. By the end of the four-year grant period, the partners intend to submit a new investigational drug application to the Food and Drug Administration (FDA) for human trials.
Given the recent failures of multiple potential medications for Alzheimer’s in clinical trials, this innovative project gives new hope for a future free of this devastating disease to the 5.4 million Americans affected today and their families. Current medications provide only symptomatic relief, with no impact on the course of Alzheimer’s disease. With the number of Americans affected by Alzheimer’s expected to reach at least 13.5 million by 2050, at an estimated cost of care of $1.1 trillion, there is an unprecedented need to pursue neural stem therapy as a potential means of halting progression of the neuronal degeneration and memory decline that characterizes this disease.
Stem Cells, Inc. is engaged in the research, development, and commercialization of cell-based therapeutics and tools for use in stem cell-based research and drug discovery. Human neural stem cells for this project are provided by Stem Cells, Inc.
CIRM was established in November 2004 with the passage of Proposition 71, the California Stem Cell Research and Cures Act. Overwhelmingly approved by voters, the California Stem Cell Research and Cures Act called for establishment of an entity to make grants and provide loans for stem cell research, research facilities, and other vital research opportunities. With the current award, CIRM is enabling UCI MIND and Stem Cells, Inc. to lead the path towards a world in which “memories last a lifetime.”