Efforts to begin human clinical trials using stem cells to treat Alzheimer's disease and retinitis pigmentosa received a $37.3 million boost from the California Institute for Regenerative Medicine during its most recent round of funding on Sept. 5.
UC Irvine scientists will be part of two research teams garnering CIRM Disease Team Therapy Development Awards, which are designed to accelerate collaborative translational research leading to human clinical trials. In one, Dr. Henry Klassen, an associate professor of ophthalmology in UC Irvine's Sue & Bill Gross Stem Cell Research Center, and his collaborators at UC Santa Barbara and Cedars-Sinai Medical Center, received $17.3 million to cultivate therapeutically potent retinal progenitor stem cells to treat the blinding effects of retinitis pigmentosa.
In the other, StemCells, Inc. in Newark, Calif., received $20 million and will collaborate with Frank LaFerla and Mathew Blurton-Jones - neurobiologists with the stem cell research center and the Institute for Memory Impairments and Neurological Disorders (UCI MIND) - to advance research using the company's proprietary purified human neural stem cells to improve memory in people with Alzheimer's disease.
"CIRM's support for UC Irvine's efforts to advance stem cell-based treatments for a variety of diseases is extremely gratifying," said Peter Donovan, director of the Sue & Bill Gross Stem Cell Research Center. "Henry's work on retinitis pigmentosa and Frank and Mathew's on Alzheimer's disease hold great promise, and we are delighted that they have the support to see their work move toward the clinic."
Klassen's objective is to introduce stem cells that rescue and reactivate damaged and dying photoreceptor rods and cones, thus reversing the course of RP even at relatively advanced stages. The current CIRM funding will allow Klassen and his collaborators to grow these cells under conditions ensuring that pharmaceutical standards are met. The resulting cells will be tested in animals for safety and to make certain that they are therapeutically potent. Then the team will seek FDA approval for the use of these cells in early clinical trials, in which a small number of patients with severe RP will be injected with cells in their worse-seeing eye and followed clinically for a specified period of time to determine the safety and effectiveness of the treatment.
"We believe it's possible to rejuvenate a clinically significant number of cones in the degenerating retina," said Klassen, whose work also has received long-standing support from the Discovery Eye Foundation. "Our methods have been validated, and I'm optimistic that stem cell-based treatments can help restore fading vision in people with eye diseases."
The CIRM award will further LaFerla and Blurton-Jones's efforts with StemCells, Inc. to understand how human neural stem cells can treat Alzheimer's disease, the leading cause of dementia in the U.S. Earlier this year, the researchers reported findings showing that neural stem cells restored memory and enhanced synaptic function in two animal models relevant to Alzheimer's disease, possibly by providing growth factors that protect neurons from degeneration. With these studies establishing proof of concept, the team intends to conduct further animal studies necessary to seek FDA approval to start testing this therapeutic approach in human patients.
"Our goal is to research ways to make memories last a lifetime, and we're excited to investigate the potential efficacy of stem cells for Alzheimer's disease," said LaFerla, the UCI MIND director and Chancellor's Professor and chair of neurobiology & behavior.