Childhood brain tumors cause the most cancer-related deaths among kids and young adults ages 0–19, recently surpassing leukemia.

But, a profound breakthrough is on the brink of human clinical trial which may change the future of brain tumor treatment, thanks to University of Minnesota researchers Dr. Michael Olin, PhD and Dr. Christopher Moertel, MD, their team, and the donors who have helped fund their work.

“The 2018 Nobel Prize was given to the scientists who discovered key proteins that have led to development of effective immunotherapy for cancer,” said Dr. Moertel, Medical Director of Pediatric Neuro-Oncology and Neurofibromatosis Programs. “Our discovery compliments their discovery, and actually goes one up. We could have an extraordinary impact on cancer therapy.”

In 2014, CCRF awarded Dr. Moertel’s research partner, Dr. Olin, Assistant Professor in the Department of Pediatrics, a grant to test the hypothesis that a protein called CD200 interferes with a potentially lifesaving brain tumor vaccine.

Previously, the developed vaccine helped trigger a child’s immune response against the brain tumor, but it was not as effective as it should have been. Though it initially worked, the immune response was short-lived.

Dr. Olin discovered that the brain tumor shielded itself with a protein called CD200, preventing a child’s immune system from attacking it. His new goal was to “turn off” this shielding CD200 protein to give the vaccine and the child’s immune system a chance to attack the brain tumor.

With CCRF donations, he had enough funding to find a way to turn off the protein shield using what he calls a CD200 inhibitor. This inhibitor stopped the protein shield from shutting off the immune system, letting the immune system and the vaccine recognize and attack the tumor.

After this discovery, he worked with the veterinary school at the University of Minnesota to give the newly improved vaccine to dogs who already had brain tumors. Using Dr. Olin’s treatment, the dogs’ tumors disappeared, and many of the dogs remained tumor-free.

Because dog and human brain tumors are very similar, Dr. Moertel is confident that the new CD200 inhibitor/vaccine combination will be just as effective in humans.

In March 2017, Dr. Olin confirmed that human CD200 inhibitors work and designed five humanized versions to test for human clinical trial.

“The biggest surprise has been the revelation of the impact of our drug on the immunosuppressive environment created by tumors - especially malignant brain tumors,” said Dr. Moertel.  “We have the potential to truly disarm cancer - especially regarding its manipulation of the immune system to avoid detection and death.”

In the last year, Drs. Olin and Moertel confirmed which type of formula to use, making sure it is stable and preserves its function. Now, they are in the “queue” to have the drug vialed at the University of Iowa.

Once the drug is manufactured, researchers need to double-check to make sure that the drug does what they intended for it to do. But for Dr. Moertel and Dr. Olin, bringing treatment to patients is the most audacious goal.

After the University of Iowa vials the drug, the inhibitor will move through several approval processes such as FDA approval and the University of Minnesota’s IRB approval. Then, the inhibitor will move to a Phase I human clinical trial, giving children with fatal brain tumors another treatment and opportunity for hope.

Though the current project focuses on brain tumors in children, the drug combination could have significant impact on other cancers, like lung cancer and melanoma. Once the drug proves to be successful for brain tumors, Drs. Moertel and Olin hope to have the funding branch out and attack these other cancers.

“Every so often in the field of cancer therapy, these huge steps happen that make a big difference for a lot of people,” said Moertel. “We really think this has the promise to do that, and support from CCRF will help us get it done as quickly as possible.”