In a significant advancement for pediatric brain cancer treatment, Children's Minnesota has launched a clinical trial testing an innovative vaccine combination therapy targeting diffuse midline glioma (DMG), one of the most aggressive and lethal childhood cancers.
The devastating nature of DMG has long challenged medical professionals, with current statistics painting a grim picture. "The cure rate for DMG is less than 1%, and the median overall survival is nine to 11 months," explains Dr. Anne Bendel, the principal investigator leading the trial at Children's Minnesota. "And that statistic has not changed since the 1950s."
Novel Therapeutic Approach
The experimental treatment combines two key components developed by University of Minnesota researchers. The first is a vaccine designed to stimulate the immune system to attack cancer cells. The second is a synthetic peptide that counteracts the cancer's immune-suppressing defense mechanisms, which were discovered during earlier clinical trials.
Both components have been licensed to Minneapolis-based OX2 Therapeutics, a biotechnology company founded by University of Minnesota researchers Michael Olin and Dr. Chris Moertel. The company selected Children's Minnesota as an independent research organization to validate the combination therapy's effectiveness.
Trial Design and Patient Population
The early-stage trial focuses on establishing safety parameters and optimal dosing regimens. It is currently recruiting patients between ages 2 and 25 diagnosed with either early-stage DMG or related high-grade gliomas (HGGs). The first patient enrollment occurred two weeks ago.
DMG, a rare subtype of glioma affecting only 200-300 children annually, develops in the glial cells that protect the spinal cord and neurons. Traditional treatment approaches have proven largely ineffective, as the cancer's rapid spread prevents surgical removal, and the blood-brain barrier blocks chemotherapy drugs from reaching the tumor.
Promising Early Results
The foundation for this pediatric trial builds on encouraging results from an earlier adult trial. While the median survival in that study was 11 months, a subset of patients achieved survival of up to 26 months. Dr. Moertel emphasizes the significance of these outcomes: "If we can break down the tumor's barrier to allow the immune system to do its job, then we are well on the way to helping cure those kids."
Impact on Families
The potential impact of extended survival cannot be understated. Amanda Bekric from Andover shared her experience with her daughter Lejla, who lived for two years after her DMG diagnosis thanks to experimental treatment. Despite ultimately losing her battle at age 4 in 2017, Lejla's extended survival allowed her to experience crucial developmental milestones, including walking, talking, and attending preschool.
"Our goal is to give these families more time with their children," Dr. Moertel states. "If we can get to the point we have cured them, hip-hip hooray... but more time translates into all sorts of wonderful things."
Vaccine Mechanism
Unlike preventive vaccines, this therapeutic vaccine targets existing cancer. Dr. Bendel explains the mechanism: "If you can give a patient a vaccine that has the proteins that are on the surface of brain tumors, then the immune system will be trained to recognize those proteins as foreign and then attack anything in the body that has those proteins on the surface."
As the trial progresses, researchers hope to understand why certain patients respond better to the treatment, potentially paving the way for more effective therapeutic strategies against this devastating childhood cancer.
