$7.38 million grant will benefit new treatments for fatal childhood disease

March 17, 2016 | by City of Hope

Canavan disease is a rare yet fatal neurological condition that can strike in the first three to four months of a child’s life, damaging the ability of nerve cells in the brain to send and receive messages. The disease, which ultimately degenerates the child’s brain into spongy tissue, afflicts an estimated one in 6,400 infants each year, with nearly 12 percent of all cases occurring in California. 
 
To better fight this fatal disease, scientists must first better understand it. 
 
A group of City of Hope researchers is committed to doing just that, and their efforts have now gotten a $7.38 million boost from the California Institute for Regenerative Medicine (CIRM). The grant was awarded to the research team led by Yanhong Shi, Ph.D., director of City of Hope’s Division of Stem Cell Biology Research. The funding will be used to develop a novel treatment and potential drug for Canavan disease.
 
“Our hope is that one day, parents of children with this devastating disease will be able to watch their children grow up to become healthy and productive adults,” said Shi, whose division is part of Beckman Research Institute of City of Hope. 
 
Shi’s previous research on Canavan disease – also funded by a CIRM grant – collected stem cells from patients with the condition and corrected the genetic defect that is responsible. The reconfigured cells reduced the disease's impact when they were transplanted into infected mice. 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.
 
The symptoms for Canavan disease usually appear in the first three to six months of a child's life, and include lack of motor development, feeding difficulties, abnormal muscle tone, and a large, poorly controlled head. Death usually occurs before age 10.
 
Shi noted that City of Hope’s Sylvia R. & Isador A. Deutch Center for Applied Technology Development was instrumental in helping the team secure the current CIRM grant. The center houses two biologics Good Manufacturing Process (GMP) facilities, which follow strict U.S. Food and Drug Administration guidelines aimed at producing high quality and safe therapies.
 
“Shi represents a new crop of researchers who are pushing the boundaries in stem cell therapy,” said Steven T. Rosen, M.D., City of Hope’s provost and chief scientific officer. “We expect her to make some amazing progress in treating this devastating disease.”
 
In addition to conducting the Canavan disease project, Shi is also researching new therapies in glioblastoma and schizophrenia.
 
The Canavan disease project’s co-investigators are Larry Couture, Ph.D., vice president and senior vice president of the Center for Applied Technology, 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. 
 
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