City of Hope Gets $7.38M Research for Canavan Disease Research

DUARTE, CA - City of Hope announced Wednesday that it has received a $7.3 million grant to develop a treatment for Canavan disease, a rare and fatal neurological disease that afflicts infants.

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. About 12 percent of all Canavan disease cases in the United States are in California.

The grant, from the California Institute for Regenerative Medicine, will help fund a research team led by Dr. Yanhong Shi, a professor in the department of developmental and stem cell biology at City of Hope's Beckman Research Institute in Duarte.

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," Shi said.

"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," she said. "Our hope is that one day, parents of children with this devastating disease will be able to watch their children grow up."

--City News Service, photo courtesy of City of Hope