Researchers aim to find a cure for a rare, fatal disease that affects infants
DUARTE, Calif. — City of Hope today announced that a research team led by Yanhong Shi, Ph.D., director of the Division of Stem Cell Biology Research, has received a $7.38 million grant from the California Institute for Regenerative Medicine (CIRM) to develop a novel treatment for Canavan disease, a rare and fatal neurological disease that afflicts infants.
Shi’s previous research on Canavan disease – also funded by a CIRM grant – collected stem cells from patients with the disease and corrected the genetic defect that is responsible for the disorder. The reconfigured cells also reduced the impact of the disease when they were transplanted into mice with the neurological condition. The next step of the research will focus on developing a process that leads to an in-human clinical trial, which will include stem cell transplantation in a patient, using their own reconfigured cells.
“As our research has demonstrated, City of Hope is dedicated to developing stem cell therapies that will ultimately lead to cures for life-threatening diseases,” said Shi, the research project’s principal investigator who is also a professor in the Department of Developmental and Stem Cell Biology within Beckman Research Institute of City of Hope. Her proposal received the highest score from CIRM’s Grants Working Group, which reviewed all the applications. “We are extremely grateful to CIRM for their previous and current support, and are inspired by the children and their families who are battling the emotional and physical pain caused by Canavan disease.”
Canavan disease causes a child’s brain to degenerate into spongy tissue riddled with microscopic fluid-filled spaces, according to the National Institute of Neurological Disorders & Stroke. Symptoms usually appear in the first three to six months of life and include lack of motor development, feeding difficulties, abnormal muscle tone, and a large, poorly-controlled head. Death usually occurs before age 10. Approximately 12 percent of all Canavan disease cases in the United States are in California.
“Our hope is that one day, parents of children with this devastating disease will be able to watch their children grow up,” Shi said.
She noted that City of Hope’s Center for Applied Technology was instrumental in helping the team secure the current CIRM grant. The center houses two biologics GMP (good manufacturing process) facilities, which follow strict U.S. Food and Drug Administration guidelines aimed at producing high quality and safe therapies for consumers.
“City of Hope is a recognized national leader in developing clinical grade products that can be tested in pioneering clinical trials,” said Larry A. Couture, senior vice president of the Sylvia R. & Isador A. Deutch Center for Applied Technology. “Finding a therapeutic cure for Canavan disease exemplifies City of Hope’s commitment to developing scientific research and therapeutics for devastating diseases.”
Jonathan Thomas, Ph.D., J.D., chair of the CIRM Board, said the goal of the agency’s translation program is to support the most promising stem cell-based projects and help them accelerate research out of the lab and into the real world, such as a clinical trial where they can be tested in people.
“The projects that our board approved today are a great example of work that take innovative approaches to developing new therapies for a wide variety of diseases,” he said.
The research project’s co-investigators are Couture and Behnam Badie, M.D., City of Hope’s chief of neurosurgery and director of its Brain Tumor Program. John Zaia, M.D., director of the Center for Gene Therapy at City of Hope’s Hematologic Malignancies and Stem Cell Transplantation Institute and the Aaron D. Miller and Edith Miller Chair in Gene Therapy, serves as clinical advisor.
About City of Hope
City of Hope is an independent research and treatment center for cancer, diabetes and other life-threatening diseases. Designated as one of only 45 comprehensive cancer centers, the highest recognition bestowed by the National Cancer Institute, City of Hope is also a founding member of the National Comprehensive Cancer Network, with research and treatment protocols that advance care throughout the world. City of Hope is located in Duarte, California, just northeast of Los Angeles, with community clinics throughout southern California. It is ranked as one of "America's Best Hospitals" in cancer by U.S. News & World Report. Founded in 1913, City of Hope is a pioneer in the fields of bone marrow transplantation, diabetes and numerous breakthrough cancer drugs based on technology developed at the institution.