New hope for deadly pediatric brain tumors; opening the door to improved diagnosis and treatment of epilepsy in children; and advanced genetic testing to better understand the causes of autism and cancer in kids. These research projects are currently underway at Mattel Children's Hospital UCLA under the umbrella of the UCLA Children's Discovery and Innovation Institute (CDI), and aim to dramatically improve the lives of children with serious illnesses.
To help support this work with critically-needed research dollars, a group called Today's and Tomorrow's Children Fund (TTCF) presented a total of $348,000 to UCLA researchers during the 7th Annual Faculty Presentation and Awards Day on May 10.
"Not only does Mattel Children's Hospital UCLA provide world-class patient care, but our faculty members are also in the CDI's laboratories studying the causes of diseases that afflict our patients and searching for new treatments and cures," said Dr. Sherin Devaskar, professor of pediatrics, executive chair of the department of pediatrics, physician-in-chief at Mattel Children's Hospital UCLA, and assistant vice chancellor for children's health with the UCLA Health System. "However because of limited federal research dollars, we must rely on additional avenues for funding. TTCF is an innovative way for the community to embrace the work of our pediatric subspecialty physician researchers and actively participate in the exciting process of advancing science."
The grand prize of $140,000 was presented to Dr. Tom Belle Davidson, assistant professor of pediatric hematology/oncology, for her work in studying a potential therapy for one of the deadliest types of pediatric brain tumors called high-grade gliomas which have few treatment options and an overall five-year survival rate of only 10 to 30 percent.
Based on current UCLA research underway for adult brain tumors, as well as a pilot study conducted among a small number of pediatric patients, this phase-1 trial for children will study a vaccine created from immune system cells—called dendritic cells (DC)—taken from the patient's blood and treated with broken-down cells isolated from his or her tumor tissue during surgery. These stimulated DCs will then be injected back into the patient as a vaccine in order to teach the host immune system to identify the malignant brain tumor cells as "foreign" to the body. Researchers hope the treatment will show improved outcomes with fewer toxic side effects than current standard therapies.
Additionally, Davidson's team will investigate the use of a topical compound cream on the vaccination site that stimulates the innate immune system and has been shown to display antiviral and antitumor activity.
Dr. Joyce Wu, associate professor of pediatric neurology, won a prize of $118,000 to help advance her team's research into pediatric epilepsy, including types of the diseases that do not respond to medication. The researchers have already discovered in children with epilepsy that ultra-fast brain electrical activity called high-frequency oscillations (HFO) are found throughout the brain and are a potential biological marker—or biomarker—to help assess, monitor and predict the condition. This finding has been seen without the use of invasive surgical implants inside patients' heads for days to weeks as part of their epilepsy surgery evaluation and was correlated to surgical removal of seizure-causing zones resulting in seizure freedom.