A groundbreaking phase 1 clinical trial has demonstrated promising results for an experimental dendritic cell therapy in treating glioblastoma, the most aggressive form of brain cancer. The treatment, called DOC1021 (dubondencel), showed positive immune responses and improved survival outcomes in patients with this devastating disease that typically offers only 14 to 16 months of survival even with surgery.
Novel Immunotherapy Approach
The investigational therapy represents a significant departure from traditional glioblastoma treatment by creating an individualized vaccine directly from each patient's tumor tissue. Dr. Joseph Georges, a neurosurgeon at Banner University Hospital in Phoenix, Arizona, who led the clinical trial, explained that the treatment addresses a fundamental challenge in glioblastoma care.
"It's a highly mutated tumor and there are different cell populations for each patient," Georges said. "And the tumor is also very good at silencing the body's immune system from attacking it."
The dendritic cell therapy works by collecting tumor tissue during surgery and using it to prime the patient's immune system. "We're picking up all these various tumor cell types, and then we're teaching the immune system how to attack the tumor — even the small cells that evade surgical resection," Georges explained.
Phase 1 Trial Results
The phase 1 clinical trial enrolled 16 patients between ages 47 and 73 who were newly diagnosed with glioblastoma. Following standard surgery, chemotherapy, and radiation treatment, patients received three courses of the DOC1021 injection every two weeks, along with weekly injections of pegylated interferon medication to help regulate the immune system.
According to a press release from Diakonos Oncology, the company that developed the treatment, the trial showed overall positive results with noted positive immune responses and improved survival after treatment. The therapy was found to be safe with minimal side effects, even at high doses.
Most remarkably, researchers observed tumor disappearance on MRI scans in some patients. "At best, we hope to keep it stable and hope that we don't see it grow, but we are actually seeing tumors disappear for some of these people on their MRIs," Georges said, describing this as "something you don't see in glioblastoma."
Patient Success Story
Among the trial participants was Pamela Goldberger, a 65-year-old New Jersey woman who was diagnosed with glioblastoma in 2023. Her initial symptoms were subtle, including severe nausea and confusion during a dinner where she used her utensils incorrectly. Neurological testing revealed a brain tumor, leading to her devastating diagnosis.
After participating in the clinical trial and completing the full treatment regimen, Goldberger is now 2½ years post-diagnosis and reports feeling "good" while maintaining an active lifestyle that includes playing tennis several times a week. "I'm living my best life right now, and I'm not restricted by not being able to do anything that I would want to do," she said.
Addressing an Unmet Medical Need
The promising results come at a critical time for glioblastoma patients, as survival rates and treatments for this cancer have remained unchanged for two decades. Georges emphasized the urgent need for new therapeutic approaches, noting that current standard treatments have reached their limitations.
The individualized nature of the therapy addresses the unique challenge posed by glioblastoma's highly heterogeneous tumor cell populations, which vary significantly between patients and contribute to treatment resistance.
Moving Forward
Based on the encouraging phase 1 results, Diakonos Oncology announced on July 22 that the first patient has been dosed in a phase 2 clinical trial of DOC1021. Georges shared that this expanded trial will be available at 20 sites across the country, offering broader access to this potentially life-saving treatment.
For patients facing glioblastoma diagnoses, both Georges and Goldberger recommend actively seeking clinical trial opportunities when available and appropriate. Goldberger attributed her success not only to the experimental treatment but also to her commitment to following medical advice, maintaining physical activity, proper nutrition, adequate sleep, and surrounding herself with positive influences.
