Osteosarcoma Survivor
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Brain Tumor Survivor
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Leukemia Survivor
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Leukemia Survivor
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Wilms Tumor Survivor
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Leukemia Survivor
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Retinoblastoma Survivor
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Brain Tumor Research

Groundbreaking approaches are stopping tumors

Brain tumors are the leading cause of cancer-related deaths in children. Our researchers are steadfastly working to change that. Established by our key contributors, the Brain Tumor Program at the University of Minnesota works to understand, treat, and cure brain tumors. It is actively investigating new, safer therapies, including:

Drug Delivery
Drug delivery into the brain is difficult because of the blood-brain barrier. Work is being done to deliver therapeutic drugs directly into a brain tumor behind the blood-brain barrier through hollow fiber catheters. A second approach involves efflux inhibitors that temporarily suspend this barrier response.

Basic Biology

Work is underway in areas of gene discovery, stem cell biology, and mouse models. A gene manipulation tool called “Sleeping Beauty” enables scientists to find genetic fingerprints in each tumor to uncover what causes a cell to initiate cancer.

Gene Therapy
Proteins secreted by cells stimulate an anti-tumor immune response that either inhibits the tumor cell growth itself or hinders the growth of blood vessels that support the tumor.

Small Molecule Drug Therapy
Receptor tyrosine kinases, which are expressed at high levels by brain tumor cells, also signal the tumors to grow. Inhibitors in combination with other therapies can be used to attack brain tumor cells.

Working with man’s Best friend
In a unique, multi-disciplinary partnership between the Brain Tumor Program and the University of Minnesota’s Veterinary Medical Center, researchers are taking advantage of the similarities between human and canine brain tumors in an effort to discover new therapies. Started in 2005, the program first focused on the safety and efficacy of adenoviral and nonviral gene transfer into a dog’s brain. Subsequent work has included immunogene therapy. Funding from the Children’s Cancer Research Fund enables the continuation of this unique program and may lead to human clinical trials in the near future.

Children’s Cancer Research Fund: A committed partner

At Children’s Cancer Research Fund, we support the passionate work of the University of Minnesota researchers who are seeking to prevent, treat, and cure brain tumors in children. We also have an endowed a chair for brain tumor research.

Future work

The University of Minnesota Brain Tumor Program has no shortage of goals. In the next five years, its leaders hope to recruit additional faculty and scientists, establish a pediatric neuro-oncology fellowship program, initiate the first brain tumor vaccine trial, develop a bone marrow transplant trial for patients whose brain tumor has relapsed and establish a Brain Tumor Comprehensive Care Clinic.

“We have a strong and committed partner in Children’s Cancer Research Fund,” added David Largaespada, Ph.D. “The investments it has made in our program have funded proof-of-principal experiments for new projects. The best of these ideas have already attracted large grants from the National Institutes of Health! We believe that brain tumor research needs much more attention, and Children’s Cancer Research Fund is making this happen at the University of Minnesota.”

A cure is possible

The cause of most brain tumors is unknown, yet these tumors continue to kill young children at an alarming rate. Researchers at the University of Minnesota are impatient. “I want to see the day, in my career, when we will cure nervous system tumors using safer and more effective therapies,” said Largaespada.


Proteins secreted by cells stimulate an anti-tumor immune response that either inhibits the tumor

Pediatric Brain Tumor Program’s key successes include:

  • Recently launching a vaccine to prevent cancer recurrence to increase the long-term cure rate for brain tumors  
  • Bypassing the blood brain barrier to deliver drugs into the brain more effectively
  • Understanding the effects of diet to understand the connection between the diet of a pregnant woman and her child’s risk of developing cancer
  • Working with man’s best friends to take advantage of the similarities between human and canine brain tumors


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