State stem cell research funding agency awards $37.3 million to aid UC Irvine efforts

Collaborations set to advance Alzheimers disease and retinitis pigmentosa treatments

Irvine, Calif., September 06, 2012

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 Irvines Sue & Bill Gross Stem Cell Research Center, and his collaborators at UC Santa Barbara, UC Davis 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 companys proprietary purified human neural stem cells to improve memory in people with Alzheimers disease.

CIRMs support for UC Irvines 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. Henrys work on retinitis pigmentosa and Frank and Mathews on Alzheimers disease hold great promise, and we are delighted that they have the support to see their work move toward the clinic.

Klassens 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, which has partnered with investigators at the NIH, 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 its possible to rejuvenate a portion of inactive 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 Im optimistic that stem cell-based treatments can help restore clinically significant vision in people going blind due to retinal degeneration. The CIRM award will further LaFerla and Blurton-Joness efforts with StemCells, Inc. to understand how human neural stem cells can treat Alzheimers 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 Alzheimers 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 were excited to investigate the potential efficacy of stem cells for Alzheimers disease, said LaFerla, the UCI MIND director and Chancellors Professor and chair of neurobiology & behavior.

CIRMs governing board gave $63 million to four institutions and companies statewide on Wednesday. The funded projects are considered critical to the institutes mission of translating basic stem cell discoveries into clinical cures. UCIs portion of the awards it shares is $5.6 million for the Alzheimers disease effort and $6 million for the RP program, bringing the campuss total CIRM funding to $96.25 million.

The two grants are the second and third CIRM Disease Team Therapy Development Awards given to Sue & Bill Gross Stem Cell Research Center scientists. In July, Aileen Anderson and Brian Cummings and StemCells, Inc. received a $20 million commitment to fund the collection of data necessary to establish human clinical trials in the U.S. for cervical spinal cord injury.

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State stem cell research funding agency awards $37.3 million to aid UC Irvine efforts