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Study Holds Promise for New Pediatric Brain Tumor Treatment

Sam Cheshier in his lab looking at a beaker

SALT LAKE CITY - New research published in Nature Communications from scientists at Huntsman Cancer Institute (HCI) at the University of Utah, in collaboration with the Stanford University School of Medicine, shows a specific protein regulates both the initiation of cancer spreading and the self-renewal of cancer cells in medulloblastoma, a type of pediatric brain cancer.

Among children’s cancers, brain cancer has the highest mortality for children, with medulloblastoma being one of the most common brain tumors in children. Medulloblastoma usually develops at the base of the brain and spreads through the cerebral spinal fluid (CSF) to the spinal cord or other parts of the brain.

"When medulloblastoma spreads, it’s much harder to treat and in many cases is incurable," said the study lead, Samuel Cheshier, MD, PhD, cancer researcher at HCI and associate professor of neurosurgery at the U of U. "The need to understand medulloblastoma and how it spreads is incredibly important in order to know how to treat the cancer and find how to stop the disease from spreading, or metastasizing."

To learn more about the metastasis of medulloblastoma, the research team gathered samples from patients that included a brain tumor sample and a spinal metastasis. Researchers then focused on studying different cancer cell markers and found that NOTCH 1, a protein that resides on the surface of the cells, played an important role in the metastasis of the cancer.

"NOTCH 1 is very important in development, as well as stem cell biology. It tells the stem cell to make another stem cell, or self-renew," said Cheshier. "NOTCH 1 has a unique attribute that makes the cancerous cells more likely to spread and form new tumors, as well as self-renew. Understanding this interaction from signals outside the cells is a major step."

Cheshier’s team used mouse models to research the NOTCH 1 pathway and test a monoclonal antibody treatment that targets the protein to stop the cancer from spreading.

Now, the research team is working on developing a clinical trial for medulloblastoma using the same treatment tested in the lab. This treatment would be administered directly into the CSF spaces of the brain and spinal cord where the tumors reside. Researchers hope this treatment will result in fewer side effects and lead to a more effective treatment option.

This work was in collaboration with, Suzana Kahn, PhD, Siddhartha Mitra, PhD, and Irving Weissman, MD, all at Stanford University School of Medicine, with support by the Pew Latin American Fellowship, Price Family Charitable Fund, Center for Children’s Brain Tumors, American Brain Tumor Foundation, Matthew Larson Foundation for Pediatric Brain Tumors, Virginia and D.K. Ludwig Fund for Cancer Research, Lucile Packard Foundation for Children’s Health, Child Health Research Institute at Stanford-Tashia and John Morgridge Faculty Scholarship in Pediatric Translational Medicine, NIH-NCATS-CTSA UL1 TR001085, German Cancer Aid (Deutsche Krebshilfe)-P-91650709 , Ty Louis Campbell Foundation-St. Baldrick’s Foundation Award, Gifts from Kathryn S.R. Lowry, George Landegger, Rider and Victoria McDowell, Charles Comey and Judith Huang, Colin and Jenna Fisher, Canadian NSERC Discovery Grant, Operating Grant 21089 of the Cancer Research Society of Canada, and R01 NS096368-02.

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Heather Simonsen
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Huntsman Cancer Institute
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About Huntsman Cancer Institute at the University of Utah

Huntsman Cancer Institute at the University of Utah (the U) is the National Cancer Institute-designated Comprehensive Cancer Center for Utah, Idaho, Montana, Nevada, and Wyoming. With a legacy of innovative cancer research, groundbreaking discoveries, and world-class patient care, we are transforming the way cancer is understood, prevented, diagnosed, treated, and survived. Huntsman Cancer Institute focuses on delivering a cancer-free frontier to all communities in the area we serve. We have more than 300 open clinical trials and 250 research teams studying cancer at any given time. More genes for inherited cancers have been discovered at Huntsman Cancer Institute than at any other cancer center. Our scientists are world-renowned for understanding how cancer begins and using that knowledge to develop innovative approaches to treat each patient’s unique disease. Huntsman Cancer Institute was founded by Jon M. and Karen Huntsman.

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