What the trial was testing
The trial enrolled 43 patients with glioblastoma. The study was sponsored by Mayo Clinic and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was mid-stage testing (phase 2/3). Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
56% of older adults with glioblastoma were alive at one year with this targeted proton therapy approach.
The Lancet. Oncology · 2024 · NCT03778294
These findings — that over half of older patients survived one year versus typical 6-9 month survival — were published in the The Lancet. Oncology and represent the headline result of the study.
Researchers tracked outcomes across 43 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with glioblastoma, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
This was a mid-stage study testing a newer approach to radiation therapy for glioblastoma in older adults. The treatment is not yet FDA-approved as a standard option. If you're 65 or older with newly diagnosed glioblastoma, ask your doctor about proton beam therapy availability and whether you might qualify for the follow-up study (NCT05781321) comparing this approach to standard treatment.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open glioblastoma trials
A Phase 0/1 Study of BDTX-1535 in Recurrent High-Grade Glioma (rHGG) and Newly Diagnosed Glioblastoma (nGBM) Participants With EGFR Alterations or Fusions
This study will administer the investigational drug, BDTX-1535 to eligible patients with recurrent high-grade glioma (HGG) and newly-diagnosed glioblastoma (nGBM). BDTX-1535 was designed to block a growth signal important to some cancers. BDTX-1535 is being tested in this study to see if it can be given safely to people who have tumors that can be dependent on that growth signal because of changes in a protein called EGFR. These gene changes are called amplifications, mutations, fusions or alterations and are found only in the tumors. The study design includes a Phase 0 component with PK/PD-trigger for participant enrollment into an Expansion Phase 1 component. The primary objective of the Phase 0 component is to evaluate the PK endpoints of BDTX-1535. The primary objective of the Phase 1 component is to establish the safe dose of BDTX-1535 to be used in participants with a specified treatment regimen, three of which include standard of care radiotherapy for nGBM participants.
Magnetic Resonance Imaging for Improving Knowledge of Brain Tumor Biology in Patients With Resectable Glioblastoma
This clinical trial uses a type of imaging scan called magnetic resonance imaging (MRI) to study brain tumor biology in patients with glioblastoma that can be removed by surgery (resectable). Malignant gliomas are the second leading cause of cancer mortality in people under the age of 35 in the United States. Glioblastoma is a type of malignant glioma with very poor patient prognosis. There are currently only about 3 drugs approved by the Food and Drug Administration (FDA) for the treatment of glioblastoma, one of them being administration of bevacizumab, which is very expensive. It is the most widely used treatment for glioblastoma with dramatic results. However, previous clinical trials have not demonstrated an overall survival benefit across all patient populations with glioblastoma that has returned after treatment (recurrent). The study aims to identify which patients who will benefit from bevacizumab therapy by observing MRI images and corresponding imaging biomarkers.