UCI scientists receive CIRM grants to improve stem cell treatments

CIRM grants will advance work on blindness, Huntington's disease and brain injury

UC Irvine scientists will receive grants totaling $9.35 million to help create stem cell treatments for retinitis pigmentosa, Huntington's disease and traumatic brain injury.

The grants from the California Institute for Regenerative Medicine were awarded Thursday, Oct. 21, to Dr. Henry Klassen, Leslie M. Thompson, Brian Cummings and Aileen Anderson - all members of the Sue and Bill Gross Stem Cell Research Center at UCI - to support early-stage translational research.

CIRM's governing board gave 19 such grants worth $67 million to 12 institutions statewide. The research funded by these awards is considered critical to CIRM's mission of translating basic discoveries into clinical cures. The selected projects are expected to either result in candidate drugs or cell therapies or make significant strides toward such treatments, which can then be developed for submission to the Food & Drug Administration for clinical trial.

These grants bring total CIRM funding for UCI to $71.85 million.

"I am delighted that CIRM has made these awards to my colleagues. It speaks to the breadth of outstanding stem cell research at UC Irvine," said Peter Donovan, director of the Sue and Bill Gross Stem Cell Research Center. "The studies they will carry out on eye diseases, Huntington's and traumatic brain injury could have wider implications for the use of stem cells to treat a variety of human diseases, disorders and injuries and could have a major impact."

With a $3.85 million grant, Klassen, assistant professor of ophthalmology, will target a stem cell treatment for retinitis pigmentosa, an inherited form of blindness in which the light-detecting cells in the retina are destroyed. To counteract this, Klassen plans to standardize a method to create photoreceptor progenitor stem cells from immature retinas and then transplant these cells into the eye to repair or replace damaged light-sensing cells. The CIRM grant reviewers gave Klassen's proposal the highest scientific score - 93 on a scale of 100 - among all applicants for early translational grants. "The eye is an important proving ground for stem cell-based therapies and provides a stepping stone to many otherwise incurable diseases of the brain and spinal cord," he said.

With a $3.8 million grant, Thompson, professor of psychiatry & human behavior and neurobiology & behavior, will develop a technique using stem cells to support areas of the brain susceptible to Huntington's disease, an inherited, incurable and fatal neurodegenerative disorder. "These cells offer a possible long-term treatment approach that could relieve the tremendous suffering experienced by patients and their families," she said.

In 2008, Thompson received a $1.4 million CIRM grant to create human embryonic and adult-derived stem cell lines from individuals carrying the Huntington's genetic mutation in order to study the disease.

With a $1.7 million grant, Cummings and Anderson, both associate professors of physical medicine & rehabilitation and anatomy & neurobiology, will explore utilizing neural stem cells to treat traumatic brain injury, which devastates 1.4 million Americans each year. The researchers believe these cells, when introduced into the injury site, will grow into new neurons, replacing damaged and dead ones, and facilitate recovery. They plan to develop a number of stem cell lines for addressing traumatic brain injury. "If successful, these lines would be potentially useful for treating a variety of other central nervous system disorders, including Alzheimer's, Parkinson's, stroke, spinal cord injury and multiple sclerosis," Cummings said.

Earlier this year, he, Anderson and UCI neurobiology researcher Hal Nguyen were awarded a CIRM basic biology grant of $1.3 million to study the properties of induced pluripotent stem cells to learn whether these cells could treat central nervous system injury and disease.

The four researchers' work will take place in Sue and Bill Gross Hall: A CIRM Institute, an $80 million, 100,000-square-foot structure that opened on campus in May, becoming the first major stem cell facility in Southern California.