Skip to content

Completed project

Crystal Mackall, MD

Stanford University

GD2-CAR T-cells for H3K27M Diffuse Intrinsic Pontine Glioma

CureSearch Catapult Award

2019–2022 · Diffuse intrinsic pontine glioma

Crystal Mackall, MD

Crystal Mackall, MD, will lead a novel trial examining GD2 CAR T-cell therapy as an approach to treat diffuse intrinsic pontine glioma (DIPG), a highly aggressive brain tumor in children that has a 5-year survival rate of less than 1%.

In the News

From loss comes hope: Pediatric brain tumor treatment shows promise SCOPE by Stanford Medicine

Revolutionary leukemia treatment offers ‘a hopeful moment’ in fight against solid tumors USA Today

Project at a glance

Program
Catapult Award
Institution
Stanford University
Focus
Diffuse intrinsic pontine glioma

Crystal Mackall, MD

Crystal Mackall, MD, is the Ernest and Amelia Gallo Family Professor of Pediatrics and Medicine at Stanford University. She serves as Founding Director of the Stanford Center for Cancer Cell Therapy, Associate Director of Stanford Cancer Institute, Director of the Parker Institute for Cancer Immunotherapy at Stanford, and Co-Executive Director of Stanford Laboratory for Cell & Gene Medicine. During a 27 year tenure culminating as Chief of the Pediatric Oncology Branch, NCI, and now at Stanford, she has led an internationally recognized translational research program. Her work is credited with defining the major pathways of T cell homeostasis and she has conducted numerous first-in-human and first-in-child clinical trials spanning dendritic cell vaccines, cytokines, and adoptive immunotherapy using NK cells and genetically modified T cells. Her group was among the first to demonstrate impressive activity of CD19-CAR in pediatric leukemia, developed a novel CD22-CAR that demonstrates impressive activity and has identified major pathways of resistance to CAR therapies. She has published over 185 manuscripts and serves in numerous national leadership positions, including Chair of the NCI Pediatric Cancer Immunotherapy Trial Network, and co-Leader of the St. Baldrick’s-StandUp2Cancer Pediatric Dream Team. She is Board Certified in Pediatrics, Pediatric Hematology-Oncology and Internal Medicine.

Mackall is a professor of pediatrics and of medicine at the Stanford University School of Medicine, where she leads the Cancer Immunology and Immunotherapy Program, serves as associate director of the Stanford Cancer Institute and is the director of the Parker Institute for Cancer Immunotherapy (PICI) at Stanford.

Follow the project’s progress.

Project Update

As of November 2022, Dr. Mackall’s team conducted large-scale genomic testing in human CAR-T cells to identify specific genes that limit T cell function.

They identified two genes, MED12 and CCNC, that were found to be key regulators of T-cell activation and the immune response. When these two genes were genetically inactivated in CAR-T cells, there was an increase in T-cell expansion, metabolic fitness, and tumor control. Technologies to inactivate genes are increasingly accessible, highlighting the potential for clinical translation of these findings.

Help move the next discovery forward

Research progress happens one milestone at a time.

Your support helps researchers test, refine, and advance better treatment ideas for children with cancer.

Ultimate Hike participants cheering together on the trail
Kids carrying a banner at a CureSearch Walk
Supporters holding a donation check from a local fundraiser
A young Ultimate Hike participant on the trail
A hiker wearing a backpack covered in CureSearch pins

Share Your Story

Every connection to CureSearch has a story. We’d love to hear yours.

Whether you’ve hiked, given, volunteered, or lived this journey yourself, your story is part of ours.

Share Your Story