Study: Novel combined vaccine therapy for malignant gliomas in kids and young adults

A phase 1, single institution clinical trial was recently launched by Anne Bendel, MD, director of neuro-oncology, and Maggie Skrypek, MD, pediatric neuro-oncologist at Children’s Minnesota, to determine the dose and safety of a novel combined vaccine therapy for newly diagnosed diffuse midline glioma (DMG) and recurrent high-grade glioma (HGG) in pediatric and young adult patients. Dr. Bendel is also the Harper Family Foundation Endowed Chair in Novel Therapies and Cancer Research. 

New treatment options are complex – and long overdue 

DMG and recurrent HGG are among the most difficult brain tumors to treat because of their invasive nature and tendency to spread quickly. DMG is the leading cause of brain tumor-related deaths in children, so finding new treatments to improve cure rates is paramount for researchers, health care providers and families alike.

“DMG is the deadliest pediatric cancer, with a median survival time of less than one year and an over-all survival rate of <5%,” said Dr. Bendel. “Unfortunately, these dismal statistics have not changed over the last 40 years, despite significant advances in medicine, and they are strikingly different than those seen in other pediatric cancers, where the survival rates have increased from 10% to 85% over the last four decades. DMG desperately needs new treatment approaches to overcome their inherent resistance to radiation and conventional chemotherapy.”   

Encouraging results with combined vaccine therapy studies 

In a previous pediatric clinical trial, the allogeneic tumor lysate vaccine (GBM6-AD) was found to be safe, and an appropriate pediatric dose was determined. However, the efficacy of the vaccine was diminished due to secretion of the CD200 protein by the patient’s DMG or HGG cells. Tumors actively shed CD200, which then binds to inhibitory receptors on immune cells and suppresses their response. Encouraging lab studies have shown a newly developed agent (CD200AR-L) binds to the CD200 activation receptor on immune cells and protects them from the suppressive effects of CD200.  

The new clinical trial by Drs. Bendel and Skrypek (NCT06305910) will assess the effects of combining CD200AR-L with the GBM6-AD vaccine, imiquimod cream and a single dose of radiation given on day 15 in children and young adults with DMG and malignant glioma. The clinical trial will enroll patients 2 to 25 years old; it will initially enroll and treat three patients older than 12 years in the first dosing cohort in order to acquire safety data before enrolling patients 2 to 11 years old.  

“We are very excited to be launching this trial, which uses an innovative combined vaccine approach to stimulate the patient’s own immune cells to recognize and destroy their DMG cells,” said Dr. Bendel. “We are very hopeful that this novel treatment will improve the cure rate of this devastating disease.”

Read more about the clinical trial here. To refer a patient for enrollment in the trial, please contact Children’s Minnesota Physican Access at 866-755-2121.