The Childhood Cancer Blog

Breakthrough: ALSF Innovation Grant Leads to FDA Breakthrough Therapy for Glioblastoma

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Read more about how one of our early Innovation Grants helped lead to a major breakthrough in the treatment of glioblastoma, an aggressive and often deadly brain tumor. 

In 2006, ALSF funded a project called “Targeting Pediatric Brainstem Glioma with Oncolytic Polioviruses,” led by Dr. Matthias Gromeier, a researcher at Duke University. His work centered upon using a modified version of the polio vaccine to attack malignant brain tumors. 

Earlier this month, Dr. Gromeier’s continued work has resulted in the FDA designating his treatment protocol as a “Breakthrough Therapy,” for recurrent glioblastoma, a deadly brain tumor. Now, hundreds of patients have hope; when before there was none.

Getting to this point took years and several thousand hours of research.

Shortly after the ASLF funding was complete, journal articles, like this one, started being published about the promise of the research and the breakthroughs coming from Dr. Gromeier’s team. As his project progressed, more stories of the success of a clinical trial using a modified version of the polio virus to treat recurrent glioblastoma, an aggressive brain tumor, began surfacing. 

One patient, a young woman battling relapsed glioblastoma, ran out of treatment options. She was the first person to try Dr. Gromeier’s polio virus treatment. In the trial, the patient’s brain tumor was infected with the polio virus. This triggered an immune response and the patient’s body began attacking the brain tumor. 

The tumor shriveled up and disappeared. The treatment worked and this 20-year-old patient was able to go away college to study nursing. Someday she plans on becoming a pediatric oncology nurse. 

This early Innovation Grant is truly a breakthrough. As a designated ‘Breakthrough Therapy,' Dr. Gromeier’s treatment will be fast tracked straight to the patients who need it.

Reaching this milestone for Dr. Gromeier is what the ALSF Innovation Grants are all about. Our Scientific Advisory Board uses a thorough review system to make sure the projects most likely to succeed receive funding. Even with this elaborate review system and with leading scientists guiding the process, it is still difficult to tell which projects will be successful. There are so many unknowns in science and science often takes a long time show results on whether or not it will work to help kids.

The Scientific Advisory Board is just one piece of the breakthrough process—it is also thanks to the many, many supporters that join together to fund research. 

Thank you to Dr. Gromeier for being so creative and developing this idea into a treatment and thanks to all the ALSF donors who made it possible for us to be a funder of this project.

You can learn more about Dr. Gromeier’s amazing breakthrough as featured on 60 Minutes earlier this month. Also, read more about the amazing researchers striving for more breakthroughs.