Glioblastoma is an aggressive brain cancer with limited treatment options. Patients face significant challenges, highlighting the urgent need for innovative therapeutic strategies. Researchers are continuously exploring new avenues to improve outcomes for those affected.
Understanding Glioblastoma
Glioblastoma is the most common and aggressive type of primary brain cancer, originating from star-shaped brain cells called astrocytes. These tumors are characterized by their rapid growth and tendency to spread microscopically into surrounding brain tissue, making complete surgical removal nearly impossible. The highly invasive nature and diverse cell types within a single tumor contribute to its resistance against conventional treatments like radiation and chemotherapy. This disease carries a poor prognosis, with a median survival time ranging from 12 to 18 months following diagnosis.
The brain’s natural defenses, such as the blood-brain barrier, further complicate treatment by restricting many drugs from reaching the tumor effectively. Despite decades of research, the survival rate for glioblastoma patients remains low, underscoring a significant unmet medical need. New therapies are continually being investigated to offer hope for improved outcomes against this challenging cancer.
What is SurVaxM
SurVaxM is an investigational immunotherapy, often described as a “vaccine-like” treatment, specifically designed to address glioblastoma. It functions by targeting a particular protein called survivin, which is found in high quantities in about 95% of glioblastoma cells. Survivin acts as a cell-survival protein, helping cancer cells evade programmed cell death, a natural process for eliminating damaged or unhealthy cells.
This therapeutic approach is distinct from traditional cancer treatments such as chemotherapy or radiation, which directly aim to destroy cancer cells or slow their growth. Instead, SurVaxM works to harness the body’s own defense mechanisms. SurVaxM consists of a portion of the survivin protein attached to an immunogenic carrier protein, keyhole limpet hemocyanin (KLH), which helps activate the immune system against the survivin protein.
How SurVaxM Targets Glioblastoma
SurVaxM operates by educating the patient’s immune system to identify and attack glioblastoma cells that produce survivin. When SurVaxM is administered, it introduces a peptide, a small part of the survivin protein, which the immune system recognizes as an antigen or foreign substance. This recognition triggers a targeted immune response.
Specifically, antigen-presenting cells (APCs) within the immune system process this survivin peptide. These APCs then activate T-cells, a type of white blood cell, to recognize and eliminate cancer cells expressing survivin on their surface. The goal is for the activated T-cells to seek out and destroy tumor cells while largely sparing healthy cells, as normal adult tissues do not produce survivin. This mechanism aims to control tumor growth and potentially prevent recurrence.
Clinical Trial Findings and Safety
Clinical trials for SurVaxM have shown promising results regarding both safety and preliminary efficacy in patients with glioblastoma. In a Phase 1 study, SurVaxM demonstrated safety and tolerability in patients with recurrent or progressive malignant glioma. A subsequent Phase 2a single-arm, multicenter trial involving 63 patients with newly diagnosed glioblastoma evaluated SurVaxM in combination with standard-of-care treatments like surgery, chemotherapy, and radiation.
The treatment was well-tolerated, with no serious adverse events attributed to SurVaxM. Common side effects were mild.
Preliminary indicators of efficacy from the Phase 2a study are encouraging: 95.2% of patients remained progression-free at six months from diagnosis. The median progression-free survival was 11.4 months, and the median overall survival was 25.9 months from the first dose. These outcomes compare favorably to historical data, where the median overall survival for glioblastoma patients receiving traditional treatment is around 15 months, and a 12-month overall survival rate is 60% to 65%. In contrast, the Phase 2a study reported a 12-month overall survival rate of 93.4% from diagnosis and 86% from the first immunization. Furthermore, 51% of participants survived at least two years, and 41% survived three years, rates higher than historically observed with standard care.
Outlook for SurVaxM
SurVaxM is currently in later-stage clinical trials, including a fully enrolled Phase 2b study known as SURVIVE, which continues to evaluate its safety and efficacy in newly diagnosed glioblastoma patients. An interim analysis of the SURVIVE trial data has supported its continuation without modification, indicating that the research met prespecified standards for potential benefit. This investigational therapy holds potential as an add-on treatment to existing standard care for glioblastoma.
The ongoing efforts aim to further confirm its ability to extend survival and improve the quality of life for patients. SurVaxM received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) in 2017, which recognizes its potential for treating a rare disease and facilitates its development and review process. The continued progress of SurVaxM offers a hopeful prospect for glioblastoma treatment.