‘We have a very rich resource here’

From Jia-Ray Yu’s perspective, pediatric brain cancer research is just the beginning. Yu was the Fralin Biomedical Research Institute at VTC’s first faculty member to set up a lab at the Children’s National Research & Innovation Campus in Washington, D.C., on Sept. 1, 2021.

Establishing a presence in uncharted territory poses formidable challenges, even with help provided by the research institute and Virginia Tech and the warm reception from colleagues at Children's National.

Today, the institute is laying the groundwork to expand, not only its D.C.-based research team but Virginia Tech’s health research footprint, with an even bigger personnel presence and double the space.

Yu had a glimpse of what that might look like when more than 100 researchers convened in May at the Children’s National Research & Innovation Campus to share research, forge new collaborations, and learn from experts spanning veterinary and human oncology and biomedical cancer research.

He sees great potential in the partnership between Virginia Tech and Children’s National and in expanding beyond pediatric brain cancer. He looks forward to a critical mass of faculty and labs filled with graduate students, postdoctoral scholars and technicians.

“We have a very resource rich resource here, and we can potentially provide some potential therapeutics or even cures for some of these patients who suffer,” said Yu, who is an assistant professor of the Fralin Biomedical Research Institute and biomedical sciences and pathobiology of the Virginia-Maryland College of Veterinary Medicine.

May was both Brain Tumor Awareness Month and National Cancer Research Month. The American Cancer Society projects that more than 1.9 million new cancer cases will be diagnosed in the U.S. in 2023. 

Yu’s focus on diffuse midline glioma puts him in an even smaller cohort of scientists looking for clues to treat fatal pediatric brain tumors. It is the most aggressive form of pediatric brain cancer. Approximately 200 to 300 new cases are diagnosed each year. It is the leading cause of brain tumor death in children, with a five-year survival rate of less than 1 percent. 

“That number has not changed in the past 20 years. So it is really a devastating disease with no cure, and not even good therapeutic options,” Yu said.

In fall 2022, Yu was part of a panel on tumor biology at the BrainStorm Summit, which brings together affected families as well as experts in pediatric brain cancer. 

One mother approached him in tears about her son’s diagnosis. He listened, heartbroken. Yu knows that there is promise in what’s happening at his lab bench, but the pace of science means answers won’t come soon enough.

“I don’t want to give false hope, because we're not really there yet,” Yu said. “There’s still a long way to go.” Despite the emotional toll, he still works to raise public awareness of pediatric cancer research and the need for investment.

Children’s health is underfunded and underresearched, particularly when it comes to rare diseases. 

Diffuse midline glioma tumors are in the brain stem and can’t be treated as if they were a solid mass. Symptoms vary based on size and location: vision problems, facial muscles working improperly, uncontrolled body movements, lack of coordination, inability to speak clearly, diminished strength, and twitching or spasms.

Surgery is not an option because of the risk of damage to the brain stem, which controls the body’s critical functions. The tumors also are resistant to chemotherapy. While radiation shows promise, it works best when a tumor grows as a solid mass and has provided marginal benefit. While it can slow the process of the disease, these tumors can still extend into other regions of the brain or spinal cord.

“With a very small population of patients being affected, they have less funding and fewer proper diagnostic tools and limited research,” Yu said. “Children’s health is a space where we need to be doing more.”

Yu was one of eight scientists selected to receive an Emerging Scientist Award through the Children’s Cancer Research Fund. He also received a grant from Cure Childhood Cancer for research into drug therapies that could potentially prove to be an off switch to selectively suppress cancer growth and another from The Children’s Cancer Foundation for research into acute myeloid leukemia and diffuse midline glioma.

Yu also was one of eight researchers named to the 2022-24 cohort of iTHRIV scholars. The integrated Translational Research Institute of Virginia is a research training and mentorship program for promising early career clinical and translational researchers that spans Virginia Tech, Carilion Clinic and the University of Virginia.

More recently, Yu received a grant from the Fralin Biomedical Research Institute's Seale Innovation fund. He is partnering with scientists from the Children's National Research Institute, Wei Li and Javad Nazarian, to battle the disease. The fund helps jump-start research projects that have the potential to transform health care.

Mutations make it difficult to develop drug therapy to battle the tumors. Two enzymes show promise as targets for combination therapies, however, so Yu and his collaborators are using the grant to investigate the biology of those enzymes and uncover potential targets for combination therapies to treat the disease.

Yu is looking forward to what comes next. He has been joined on the Children’s National Research & Innovation Campus by Kathleen Mulvaney, assistant professor of the Fralin Biomedical Research Institute with the Department of Biomedical Sciences and Pathobiology. More are certain to follow.

But he’ll always remember how it began. “It’s kind of exciting to be the first one.”