The best that can be hoped for in a new clinical trial or new treatment for aggressive forms of cancer is that patients gain time. That’s what makes a recent study at the Mayo Clinic so impactful, not only for individual patients but also for the medical community seeking new discoveries to help patients live longer.
The Mayo Clinic study was the first clinical trial of its kind, investigating an innovative treatment for patients aged 65 years and older who were newly diagnosed with glioblastoma. The treatment, short-course hypofractionated proton beam therapy combined with advanced imaging techniques, showed significantly improved survival, giving hope to those who face an otherwise grim outlook for their cancer progression.
What Is Glioblastoma?
Glioblastoma is a type of brain cancer that is characterized by malignant grade 4 brain tumors. It is the most common type of primary brain cancer, with nearly 14.5k people in the US diagnosed annually. It is also the most aggressive type of brain cancer.
Glioblastoma tumors are composed of abnormal astrocytic cells and invade other areas of the brain. These tumors can be invasive and stretch to opposite ends of the brain through connection fibers or the ventricular system. As a result, glioblastoma is difficult to treat.
This cancer can grow quickly and is very difficult to remove since the tumor can invade nearby brain tissue. Glioblastoma tumors can have a tentacle-like structure that infiltrates the brain. Removing this cancer and even treating it can be problematic, as intervention also risks damaging healthy brain tissue. For example, glioblastoma can be near the areas of the brain that control language, movement, and coordination. Additionally, this cancer can be treatment-resistant, as the blood-brain barrier prevents the tumors from being reached, rendering treatment ineffective.
For patients who are diagnosed above the age of 40, the 5-year relative survival rate is only 5.6%. There is a great need for treatments and strategies that effectively eliminate these tumors and increase survival.
Enter: An Innovative Treatment
The Mayo Clinic study, a phase 2 trial, focused on patients who have the lowest relative survival rate. Published in Lancet Oncology, the findings signify progress in the treatment of glioblastoma, both for patient survival and quality of life measures.
In the trial, patients received dose-escalated hypofractionated proton beam therapy while taking oral temozolomide, another drug that treats glioblastoma. 39 patients enrolled and received treatment. After 12 months of therapy, 56% of patients were still alive, and the median survival length was 13.1 months.
To better illustrate the significance of these results, it’s important to know that other phase 3 studies of a similar patient population with glioblastoma might have only seen a median survival of 6 to 9 months. Additionally, when stratifying patients with tumors that have favorable genetics in this study, the median survival rose even higher to 22 months.
One of the goals of this new proton beam therapy was to treat the tumor without unintentionally exposing healthy brain tissue to radiation. Proton beam therapy offers precision to target cancer cells without damaging the surrounding healthy tissue.
To increase the precision of the treatment, researchers also used advanced imaging technology to map the brain, specifically F-DOPA PET and contrast-enhanced MRI. This imaging allowed them to identify the most metabolically active (ie, aggressive) regions of the glioblastoma.
Results: Offering hope for patients
In addition to living longer, patients who underwent advanced imaging combined with proton beam therapy were also able to tolerate treatment better and did not feel the side effects of their glioblastoma.
One patient in the study lived almost 2 years longer than his initial prognosis predicted. Before succumbing to his glioblastoma, he noted that he felt great and wouldn’t have even known he had cancer.
Now that the phase 2 study is complete, a larger, randomized clinical trial is underway at the Mayo Clinic. One of the patients participating in the next phase of the trial has seen no recurring glioblastoma after her fourth MRI, and has been able to live and travel with her family.
Though the results already seem promising, the larger study will help to better determine the safety and efficacy of the treatment and whether shorter courses of radiation will continue to be viable choice for treatment of glioblastoma in the future
Further support for researchers and drug developers
As treatments undergo research and development to treat diseases like glioblastoma, it’s important to continually evaluate the market and connect with the medical community and academic experts. How can life science and pharmaceutical leaders access these experts in a way that’s fast enough to keep up with the rapidly evolving healthcare industry? The answer lies in NoeticInsight.
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Sources:
1. https://www.abta.org/tumor_types/glioblastoma-gbm/
2. https://cancerblog.mayoclinic.org/2025/01/16/breakthrough-in-treatment-approach-showing-promise-in-the-fight-against-glioblastoma/
3. https://www.thelancet.com/journals/lanonc/article/PIIS1470-2045(24)00585-0/abstract