Roswell Park's Brain Cancer Vaccine, SurVaxM, Shows Promise in Pediatric Brain Tumor Trial
Key Insights
SurVaxM, a brain cancer vaccine developed at Roswell Park (search), has shown promising results in adults by doubling survival time in glioblastoma (search) patients.
A pilot clinical trial is underway to evaluate SurVaxM's safety and efficacy in children and young adults with high-risk brain tumors (search), including DIPG.
The vaccine targets survivin (search), a protein crucial for tumor cell survival, and has demonstrated mild side effects in adult trials by stimulating the immune system.
Roswell Park (search) Comprehensive Cancer Center is expanding clinical trials for its innovative brain cancer vaccine, SurVaxM, to include children and young adults with high-risk brain tumors (search). This follows promising results in adult trials, where the vaccine doubled survival times for glioblastoma (search) patients. The pediatric trial aims to assess the safety and potential efficacy of SurVaxM in treating a range of aggressive brain tumors, including diffuse intrinsic pontine glioma (search) (DIPG), for which there is currently no curative therapy.
SurVaxM: How It Works
SurVaxM is designed to stimulate the patient's immune system to target and destroy tumor cells. The vaccine targets survivin (search), a protein that is essential for the survival of many tumor cells, including glioblastomas. The treatment regimen involves initial surgery to remove the tumor, followed by a series of vaccine injections. In adult trials, patients received four injections over six weeks, followed by maintenance doses every three months.
Blood tests from the initial adult clinical trial participants indicated that the vaccine effectively activated the immune system, leading to the production of T cells that target and kill tumor cells expressing survivin (search). Because survivin is not typically found on healthy cells, the vaccine has shown minimal side effects, mainly limited to pain or itching at the injection site, fatigue, and muscle aches.
Pediatric Brain Tumor Consortium Trial
The pilot clinical trial for pediatric patients is being conducted through the Pediatric Brain Tumor Consortium, with Roswell Park (search) serving as one of the 12 participating centers across the nation. The study aims to enroll 30 patients aged 1-21 years with various high-risk brain tumors (search), including glioblastoma (search) and DIPG. Unlike the adult trials where patients were treated immediately after initial surgery, the pediatric trial focuses on patients whose disease has recurred after standard therapies like chemotherapy and radiation.
According to Clare Twist, MD, Director of Pediatric Developmental Therapeutics at Roswell Park (search), this phase 1 trial will primarily evaluate the safety of SurVaxM in younger patients. Researchers are optimistic about the potential of immunotherapy in treating pediatric cancers, drawing parallels with successful antibody therapies in neuroblastoma (search).
Broader Applications and Future Research
Given that survivin (search) is present in various types of tumors, researchers are also investigating SurVaxM's potential effectiveness against other cancers. A clinical trial for multiple myeloma (search) patients has recently concluded at Roswell Park (search), and another trial for neuroendocrine tumors (search) is currently underway. Future research will focus on preclinical experiments to determine the prevalence of survivin in pediatric and young-adult tumors, such as neuroblastoma (search) and sarcoma (search), to inform the design of future clinical trials.
Robert Fenstermaker, MD, Chair of the Department of Neurosurgery and Director of the Neuro-Oncology Program at Roswell Park (search), along with Michael Ciesielski, PhD, Assistant Professor of Neurosurgery, are leading the SurVaxM research. Their work is supported by the Roswell Park Alliance Foundation, highlighting the institution's commitment to advancing cancer treatment through innovative approaches.