CIRM Awards UCI Researchers

Sue & Bill Gross Stem Cell Researchers Receive Private Funding to Develop New Stem Cell Lines in Hopes of Treating Alzheimer’s and Eye Disease.

During the most recent grant disbursements taking place on Sept. 5, two UC Irvine stem cell collaboration projects received a financial boost on behalf of the California Institute for Regenerative Medicine, also known as CIRM.

As a leading university in the field of embryonic stem cell research, UCI has several projects exploring the potential of embryonic stem cells that hold the possibility of differentiating into any cell.

CIRM awarded a total of $37.3 million to aid UCI’s efforts in treating advanced retinitis pigmentosa, a degenerative eye disease, and Alzheimer’s.

Private funding from organizations like CIRM are essential for the continued expansion of stem cell research, and the Sue & Bill Gross Stem Cell Research Center is a recipient and beneficiary of such funding.

After the passage of the Stem Cell Research Advancement Act by Congress, pushed through with the support of President Obama, government funding has been approved for the continued research of the original 21 existing stem cell lines that were created in 2001. This funding, however, is not allowed for the development of new embryonic stem cell lines because of the social implications of utilizing cryogenically frozen zygotes.

Under the passage of the new Act, researchers are  able to legally develop new embryonic stem cell lines from unused, donated zygotes from the “in vitro” fertilization process. However, they must be procured without federal funding.

Due to the fact that the embryonic stem cells are such an integral part of the zygote’s growth, with roughly 30 cells out of the 100-150 cell blastocyst being embryonic stem cells, government funding of developing new stem cell lines have faced social and religious ramifications as the extraction process means the destruction of the zygote.

This private funding from organizations like CIRM can allow for the development of new germal stem cell lines with new possibilities of curing diseases.

$17.3 million was awarded to the associate professor of ophthalmology, Dr. Henry Klassen, for his work at the Sue & Bill Gross Stem Cell Research Center.

Klassen and collaborators from UC Santa Barbara, UC Davis and Cedars-Sinai Medical center intend to create stem cells that can reverse the blindness caused by the degenerative eye disease, retinitis pigmentosa, and restore vision.

Due to the fact that embryonic stem cell lines are able to differentiate into any cell, Klassen and his team are working on restoring vision by replacing degenerated cells in the retina caused by retinitis pigmentosa.

The project will use the funding to develop cells in pharmaceutically correct conditions and will then test the cells to determine therapeutic effectiveness. It is the team’s goal to rejuvenate a significant number of cones, which provide color vision, in the worsening retina to help restore fading vision.

“We believe it’s possible to rejuvenate a portion of inactive cones in the degenerating retina,” Klassen said. “Our methods have been validated, and I’m optimistic that stem cell-based treatments can help restore clinically significant vision in people going blind due to retinal degeneration.”

The second project, which aims to treat the debilitating effects of Alzheimer’s, will use the funding to explore the use of neural stem cells to restore memory in Alzheimer patients.

$20 million was awarded to the longtime UCI research partner, StemCells Inc., in hopes of advancing stem cell-based treatments. The company will work with UCI neurobiologists Frank LaFerla and Mathew Blurton-Jones, from the university’s Institute for Memory Impairments and Neurological Disorders, also known as UCI MIND.

“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.

The funding will allow the project to delve deeper into the understanding of how human neural stem cells can treat Alzheimer’s disease. The team intends to use further animal studies after the development of the stem cell lines, to begin testing this therapeutic approach in vivo, in live patients, before beginning human testing. The support from CIRM aims to enhance and restore memory in Alzheimer’s patients.

“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.”

These two recent grants mark the third time CIRM has awarded the Sue & Bill Gross Stem Cell Research Center in their efforts to advance medicine. UCI and its collaborators hold promising work in the stem cell field as they work toward a goal of curing degenerative diseases through therapeutic uses of embryonic stem cells.