Irvine, Calif., March 26, 2015 -- Leslie Thompson of the Sue & Bill Gross Stem Cell Research Center at UC Irvine has been awarded $5 million by the California Institute for Regenerative Medicine to continue her CIRM-funded effort to develop stem cell treatments for Huntington's disease.
The grant supports her next step: identifying and testing stem cell-based treatments for HD, an inherited, incurable and fatal neurodegenerative disorder. In this project, Thompson and her colleagues will establish an HD therapy employing human embryonic stem cells that can be evaluated in clinical trials.
Over the past seven years, Thompson, a UCI professor of psychiatry & human behavior and neurobiology & behavior, and her team have used CIRM funding to produce stem cell lines "reprogrammed" from the skin cells of individuals carrying the Huntington's genetic mutation in order to study the disease. In addition, they conducted basic and early-stage transitional studies to develop a stem cell-based technique to treat areas of the brain susceptible to HD.
"These stem cells offer a possible long-term treatment approach that could relieve the tremendous suffering experienced by HD patients and their families," said Thompson, who's also affiliated with UCI's Institute for Memory Impairments and Neurological Disorders (UCI MIND). "We appreciate CIRM and the millions of people in the state of California for generously supporting breakthrough stem cell research."
With this award, CIRM has granted Thompson $10.3 million for her HD work. Overall, UCI has received $105 million from the state-funded agency.
Thompson said that her group has identified a highly promising neural stem cell line that shows disease-modifying activity in HD mice. These neural stem cells were grown from human embryonic stem cells at UC Davis. The researchers also will conduct essential preclinical efficacy and safety studies in HD mice with these cells.
Over the span of the 2½-year grant, Thompson said, the goal is to finalize work that will lead to a pre-investigational-new-drug meeting with the Food & Drug Administration and a path forward for clinical trials with the neural stem cells.
"This investment will let us further test the early promise shown by these projects," said Jonathan Thomas, chair of the CIRM governing board. "Preclinical work is vital in examining the feasibility, potential effectiveness and safety of a therapy before we try it on people. These projects all showed compelling evidence that they could be tremendously beneficial to patients. We want to help them build on that earlier research and move the projects to the next level."
HD is a devastating degenerative brain disorder with no disease-modifying treatment or cure. Current approaches only address certain symptoms of HD and do not change its course.
Participating in the project are: Mathew Blurton-Jones, Ed Monuki, Steven Cramer and Neal Hermanowicz of UCI; Michael Levine and Marie-Francoise Chesselet of UCLA; and Clive Svendsen of Cedars-Sinai Medical Center.
Also involved is UC Davis' good-manufacturing-practice laboratory, led by Gerhard Bauer, which will generate GMP-grade cells for the effort.
The group is partnering with Terumo BCT, a global biotechnology company that employs a system for expediting the growth of stem cells. BioTime Inc., a biotechnology company in the field of regenerative medicine that's headquartered in Alameda, Calif., is providing the human embryonic stem cells.
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