Stanford/Packard Children’s Study Of Vaccine For Children’s Brain Cancer Seeks Participants

-- Scientists at the Stanford University School of Medicine and Lucile Packard Children’s Hospital are starting a phase-1 clinical trial of a vaccine-based treatment for the deadliest childhood cancer, a rare brain tumor called Diffuse Intrinsic Pontine Glioma. Today, just 1 percent of DIPG patients survive five years past diagnosis. No cure exists.

“Thirty years of numerous clinical trials have all failed,” said  Gordon Li, MD, who is an acting assistant professor of neurosurgery at Stanford and one of the study’s co-investigators. The tumor, seen mostly in school-aged children, is impossible to remove surgically because it grows tangled into a region of the brain stem essential for life. It does not respond to chemotherapy, and radiation gives only temporary remission.

Li and his colleagues are testing an approach never tried before for DIPG: They are giving an anti-cancer vaccine to try to make the patient’s own body attack the tumor. In the new trial, funded by a $1.5 million grant from the National Institutes of Health, patients will receive regular doses of a therapeutic vaccine against EGFRvIII, a cell marker that Li recently discovered is found on about half of DIPG tumors. Unlike prophylactic vaccines given to prevent disease, the goal of this treatment is to alert the body’s immune system that disease is already present. The researchers hope the vaccine will cause the body to produce immune cells that recognize and destroy tumor cells carrying the EGFRvIII marker.

“If a vaccine trial works, it could advance the concept of treatment for all types of tumors with immune therapy,” Li said. Immune therapies have been tested with mixed success in a few adult cancers, but only one, a prostate cancer treatment, has been approved by the U.S. Food and Drug Administration. The EGFRvIII vaccine is promising because its target — a molecular flag on the exterior of cancer cells — is a very different molecular shape than the markers on healthy cells, the researchers said.

The researchers plan to enroll 15 children with recent DIPG diagnoses for the trial.

“The study subjects will be getting conventional radiation therapy and then a series of monthly vaccinations to mount an immune response against the tumor,” said  Paul Fisher, MD, a pediatric oncologist at Packard Children’s who is a principal investigator on the study. Because it is a phase-1 trial, the study’s main purpose is to establish the vaccine’s safety, but the vaccine’s efficacy will also be monitored. Patients will receive up to 12 months of vaccine injections and will be monitored with MRIs, immunologic exams and physical exams. After the 12-month vaccine period ends, they will be followed by monitoring for as long as they remain alive.

Albert Wong, MD, professor of neurosurgery at Stanford, is leading the study with Fisher. Wong is one of the patent holders on the discovery of the EGFRvIII receptor and for using EGFRvIII as an anti-tumor vaccine. He also holds stock in Celldex, the company supplying vaccine for the trial.


About Stanford Children’s Health and Lucile Packard Children’s Hospital Stanford

Stanford Children’s Health, with Lucile Packard Children’s Hospital at its core, is the Bay Area’s largest health care enterprise exclusively dedicated to children and expectant mothers. As the top-ranked children’s hospital in Northern California, and one of just 11 nationwide to be named on the 2016-17 U.S. News & World Report Best Children’s Hospitals Honor Roll, Packard Children’s Hospital is a leader in world-class, nurturing care and extraordinary outcomes in every pediatric and obstetric specialty. Stanford Children’s Health offers care ranging from the routine to rare, regardless of a family’s ability to pay. Together with Stanford Medicine physicians, nurses, and staff, Stanford Children’s Health can be accessed through partnerships, collaborations, outreach, specialty clinics and primary care practices at more than 60 locations across Northern California and 100 locations in the U.S. western region. As a non-profit, Stanford Children’s Health is committed to supporting the community – from caring for uninsured or underinsured kids, homeless teens and pregnant moms, to training the next generation of doctors and medical professionals. Celebrating the 25th anniversary of Lucile Packard Children’s Hospital in 2016, Stanford Children’s Health looks forward to the fall 2017 debut of its expanded pediatric and obstetric hospital campus. Discover more at and on the Healthier, Happy Lives blog. Join Stanford Children’s Health on Facebook, Twitter, LinkedIn and YouTube.


About Stanford University School of Medicine

The Stanford University School of Medicine consistently ranks among the nation’s top medical schools, integrating research, medical education, patient care and community service. For more news about the school, please visit The medical school is part of Stanford Medicine, which includes Stanford Health Care and Stanford Children’s Health. For information about all three, please visit