Note: CTSI supported study - former KL2 Scholar Sabine Mueller along with former Catalyst awardee Hideho Okada are leading the phase I clinical trial of this research.
UCSF Research Leads to a Clinical Trial of New Cancer Vaccine and Development of Cell Therapy Approaches
Children with an extremely deadly form of brain cancer might benefit from a new treatment that aims to direct an immune response against a mutant form of a protein found exclusively on cancer cells, according to a new study led by UC San Francisco researchers.
The focus of the study, published online Dec. 4 in the Journal of Experimental Medicine, is diffuse intrinsic pontine glioma (DIPG), an aggressive pediatric brain cancer. DIPG is rare — estimates suggest that about 300 new cases occur in the United States each year — but almost always fatal.
Because DIPG occurs in a difficult-to-access area of the brain stem that controls vital functions such as breathing, blood pressure, and heart rate, these tumors are almost impossible to remove surgically. Radiation therapy is the current standard treatment, but is rarely effective for long, according to Hideho Okada, MD, PhD, professor of neurological surgery and director of the Brain Tumor Immunotherapy Center at UCSF, and the senior author of the study.
“It is important to develop more innovative treatment approaches for childhood brain cancers, which now are the leading cause of cancer death in children. DIPG is a very deadly type of brain cancer, and not many children survive beyond 12 months from the time of diagnosis.” said Okada, also a member of the Helen Diller Family Comprehensive Cancer Center (HDFCC) at UCSF and of the Parker Institute for Cancer Immunotherapy.
Okada, along with Sabine Mueller, MD, PhD, MAS, an assistant professor of clinical neurology at UCSF and a HDFCCC member, already are leading a phase I clinical trial in children with DIPG and closely related gliomas, in order to evaluate a new anti-tumor vaccine based on the new target identified by Okada’s research group.