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By Robyn White
Nature Reporter
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Scientists have discovered a new weapon against an incurable brain tumor found in children.
Researchers led by the University of Michigan Health Rogel Cancer Center and the Chad Carr Pediatric Brain Tumor Center have found for the first time, how one drug may improve the survival rate for a type of childhood brain tumor. Their findings have been published in Cancer Discovery, a journal of the American Association for Cancer Research.
The tumor, known as diffuse midline gliomas (DMG), is a particularly aggressive form of cancer usually found in young adults or children. When this cancer also has a mutation called H3K27M, the treatment becomes even harder.
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The average survival rate for a person with this type of tumor is 11 to 15 months. It is currently treated with radiation, but this is not an easy method as the tumors are located in a critical area of the brain. But now, researchers found that a drug called ONC201 nearly doubled the survival rate for those with the tumor, when compared to those who had not taken the drug.
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ONC201 is an experimental drug that selectively kills cancer cells. Scientists already knew the drug could be effective in tackling this type of cancer, but the new study shows just how much it extends life expectancy, and also underlines what causes the drug's success. This means the drug could be a viable treatment method for this incurable cancer.
"Quite honestly, this tumor is so hard to treat and so many treatments haven't worked, that the bar has become low for any kind of therapeutic success. So to see this kind of signal in an early phase trial for DMG is very exciting," senior author of the study Carl Koschmann, M.D., associate professor of pediatric neuro-oncology and clinical scientific director of the Chad Carr Pediatric Brain Tumor Center at Michigan Medicine told Newsweek.
Before this study, there have been over 250 clinical trials that were not able to improve the outcomes of this brain tumor, Koschmann said, making these new findings a "major crack in the armor."
To study this, researchers undertook two clinical trials, testing the drug in 71 patients with diffuse midline gliomas, with the H3K27M mutation. The participants all took ONC201. The researchers found that a third of them lived over two years. There was an average survival rate of 22 months among all patients.
Researchers found that the drug "passes the blood-brain barrier"—a system in the brain that protects the organ from toxic substances. Finding treatments that surpass this barrier is one of the main challenges in treating brain tumors, the study said.
The next step was to find out how it worked in patients. Using fluid from the brain and spine, researchers analyzed the patients' metabolic changes and found that the drug targets a gene group called mitochondria.
"DMGs are epigenetically driven tumors due to a mutation in an epigenetic gene. No treatments for DMG have ever reversed this hallmark epigenetic feature in patient tumor samples. We were not expecting to observe this, but it helped us understand how the drug is working and why we may be seeing a response in this patient population," co-author of the study Sriram Venneti told Newsweek.
Although these findings are hopeful, the tumor remains "very lethal," Koschmann said. "A near-doubling of survival is not enough for families of patients with this diagnosis."
But the breakthrough could lead to bigger steps toward treatment in the future.
"For now we have this patient population that didn't have a drug before, and now we see many of the tumors responding. We have a platform to build on and we can also explain why it's working," Koschmann said.
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