Glioblastoma Awareness Day: Innovative Solutions for the Most Aggressive Brain Tumor

Glioblastoma, also known as glioblastoma multiforme (GBM), is both the most common type of brain tumor in adults and the most aggressive. The 5-year survival rate in the U.S. is only 6.4%, with most patients surviving fewer than 3 years after diagnosis. These figures have changed little over the past 2 decades, highlighting the urgent need for more effective treatments. In recognition of Glioblastoma Awareness Day today, ACCC invites its members to support ongoing advocacy and research aimed at transforming the outlook for patients with this devastating disease. 

Glioblastoma arises from astrocytes, the star-shaped glial cells that support the structure and function of the nervous system. Symptoms vary widely depending on tumor location, but may include headaches, seizures, or neurological deficits. For example, if the tumor develops in the occipital lobe where the brain processes vision, the patient may experience a visual field defect as a primary symptom. 

Surgical resection is the first-line intervention, but it is not curative. Unlike some brain tumors with well-defined borders, glioblastoma infiltrates surrounding healthy brain tissue through microscopic extensions that cannot be fully removed. What remains after surgery is often inaccessible to chemotherapy or immunotherapy, in part because of the protective blood-brain barrier. These characteristics contribute to rapid tumor recurrence and the disease’s historically poor prognosis. 

Despite the significant therapeutic challenges, glioblastoma research continues to advance. While survival rates have remained largely unchanged, recent scientific efforts are yielding innovative strategies to target the disease’s most resistant features. From advanced drug delivery systems to cutting-edge immunotherapies and refined radiotherapy techniques, researchers are making meaningful strides toward more effective treatment options. 

When Tumors Infiltrate Brain Tissue, So Must Treatment 

Surgically removing glioblastomas that infiltrate healthy brain tissue is often extremely challenging—and in many cases, impossible. To treat residual tumor cells, the standard of care includes external beam radiation therapy (EBRT), which uses high-energy photons (x-rays) to penetrate deep into the brain. However, the EBRT approach lacks precision; the radiation passes beyond tumor margins, increasing the risk of damage to surrounding healthy tissue. 

A phase 2 clinical trial run by researchers from the Mayo Clinic evaluated proton beam therapy as a more targeted alternative. Unlike photons, protons are charged particles that deposit most of their energy directly into the tumor and then stop, sparing nearby healthy tissue. This makes proton therapy both potentially more effective and less harmful to critical areas of the brain.  

The trial results were published in The Lancet Oncology in January 2025, demonstrating improved survival for patients with glioblastoma aged 65 and older, the group with the worst prognosis from the disease. Building on these findings, the Mayo Clinic is now recruiting for a second phase 2 trial, this time evaluating proton beam therapy in patients of all ages. 

Disrupting the Blood-Brain Barrier Using Ultrasound Technology 

The blood–brain barrier is a dense network of endothelial cells that serves as a critical defense against pathogens and toxins. However, this same protective function often limits the delivery of chemotherapy and immunotherapy agents to brain tumors such as glioblastoma. 

Instead of modifying drugs to better penetrate the blood-brain barrier, researchers at Northwestern University Feinberg School of Medicine are targeting the barrier itself. In a study published in Nature Communications in June 2024, investigators implanted an ultrasound-emitting device into the skull to transiently disrupt the blood-brain barrier. The device delivers low-intensity ultrasound waves that stimulate the endothelial cells lining the barrier. When paired with intravenous microbubbles, the vibrations cause temporary openings that allow greater drug penetration into tumor tissue. 

Although the initial study involved only 4 patients, a follow-up trial is now underway to evaluate this approach in a larger cohort. This technique holds promise not only for enhancing the efficacy of existing therapies but also for enabling the use of agents previously deemed ineffective due to limited blood-brain barrier permeability. 

When Time is Limited, Treatment Must Be Strategic 

Glioblastoma’s rapid progression leaves little time for trial and error. Patients often cannot afford the delays associated with navigating traditional clinical trial screening and enrollment processes, especially when the therapeutic landscape is fragmented across numerous investigational approaches. 

Recognizing that the traditional model of drug development and clinical trials is poorly suited to glioblastoma, the nonprofit Global Coalition for Adaptive Research created Glioblastoma Adaptive Global Innovative Learning Environment (GBM AGILE). This innovative and collaborative platform trial is evaluating multiple treatments simultaneously (currently 9) across 62 cancer centers worldwide. 

Through biomarker-driven matching, GBM AGILE assigns patients to the most suitable experimental treatment at their nearest participating site. Importantly, if a therapy proves ineffective, or a patient becomes eligible for a different treatment arm, they can transition seamlessly within the trial framework—without the delays of re-enrollment or additional screening. 

By consolidating patients within a single adaptive trial, GBM AGILE accelerates data collection and sharing across institutions, fostering collaboration and scientific agility. The ultimate goal is to streamline access to promising therapies and expedite the drug development process for this aggressive tumor type. 

Hope Amid Uncertainty 

These innovative approaches represent just a fraction of the ongoing global effort to improve outcomes for patients with glioblastoma. Though the disease remains extraordinarily difficult to treat, patients and families are not alone. They are supported by a collaborative ecosystem of clinicians, researchers, drug developers, and advocates—all working to move the needle on treatment efficacy and survival. 

On this Glioblastoma Awareness Day, ACCC encourages its members to explore opportunities to engage in advocacy and support research. Resources such as the National Brain Tumor Society’s Patient and Caregiver Toolkit are available to help patients navigate their journey and connect with emerging clinical evidence. Together, we can help ensure that progress continues—for today’s patients and tomorrow’s breakthroughs.