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New funding will benefit UCLA pediatric research in cancer, epilepsy, and autism

Robin Wulffson MD's picture
pediatric research, epilepsy, brain tumors, autism, cancer

Los Angeles is home to Mattel Children’s Hospital, which provides state-of-the art care to children in Los Angeles and throughout the globe. It is also a world-renowned research facility for pediatric research. On May 30, the facility announced that it had received a generous donation of $348,000 from the Today's and Tomorrow's Children Fund (TTCF).

The funds are earmarked for research for projects currently underway by the UCLA Children’s Discovery and Innovation Institute (CDI): deadly pediatric brain tumors, pediatric epilepsy, and advanced genetic testing for the causes of autism and cancer in children.

“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,” noted Dr. Sherin Devaskar, professor of pediatrics, executive chair of the department of pediatrics, physician-in-chief at Mattel Children's Hospital UCLA. She added, “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 funds were allocated to several investigators. 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 10-30%. The study is focused on a vaccine created from immune system cells, known as 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. In addition Dr. Davidson and her 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.

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Another beneficiary of the funding is Dr. Joyce Wu, associate professor of pediatric neurology. She received $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.

In addition, the funds will allow Dr. Wu and her team to expand on the concept of HFO. Two goals include correlating the presence of HFO in children who are at high-risk of developing epilepsy later in life due to an inadequate oxygen supply at birth, a traumatic brain injury or tuberous sclerosis complex—formation of tumors in the brain or other vital organs. Additionally, with the ability to interpret HFO “live” in the operating room and immediately study affected brain tissue, this could potentially lead to a better understanding of the development of epilepsy and new antiepileptic drugs. The research holds promise for future clinical trials for better surgical brain mapping and potential disease-modifying therapy to prevent the development of epilepsy altogether, not merely stopping seizures with anticonvulsant medications.

An award of $90,000 was presented to Dr. Julian Martinez-Agosto, assistant professor of human genetics and pediatrics, for his research focused on the genetic risk factors of autism and cancer predisposition. Dr. Martinez-Agosto’s research focuses on genetic conditions in which children experience overgrowth: a situation in which their bodies grow faster than their peers. A subset of these children has autism. By using the latest technologies, they are able to identify the genetic changes in these children in order to expand their understanding of the causes of autism and, in some cases, a predisposition to cancer.

In addition, the funds will enable Dr. Martinez-Agosto and his team to use a new, efficient way to establish a personalized genetic-based diagnosis called “next generation whole exome sequencing” to identify mutations in pediatric patients with overgrowth syndromes. This technology will allow them to examine each letter in the patient’s genes, sequencing DNA to identify rare mutations that are predicted to cause autism, overgrowth, and/or cancer predisposition. The findings are expected to enhance the understanding of the genetic risk factors for autism and the biology of pediatric growth disorders, as well as provide new potential targets for therapies.

The Today's and Tomorrow's Children Fund was founded in 2006 based on the belief that, by pooling their donations, a group of committed individuals could award an annual gift to support the research of talented pediatric faculty members at Mattel Children's Hospital UCLA. Each year, TTCF members vote on a ballot of select research projects and the three faculty members receiving the most votes present their research to the group, who then select the winning presentations. TTCF currently includes 67 members from the community and continues to grow each year. To date, the group has donated a total of $1.5 million to 13 researchers at UCLA. Ultimately, the group hopes to expand to 200 members in order to provide an annual award of $1 million.

Reference: UCLA Health System