Understanding Brain Cancer

Joining a Clinical Trial for Brain Cancer

Last updated and reviewed on June 16, 2026.

For many people with brain cancer, particularly those with high-grade tumors like glioblastoma, clinical trials are not just an option to consider if other treatments fail. They are often one of the most important tools available, and some brain tumor specialists recommend discussing them from the very beginning of treatment. Many of the treatments that brain cancer patients receive today, including the temozolomide chemotherapy protocol for glioblastoma and the use of tumor treating fields (TTFields), became standard of care because patients before you participated in clinical trials and helped researchers prove that these treatments worked.

A clinical trial is a carefully designed research study that tests new medical treatments in people. Clinical trials are how new drugs, devices, combinations of therapies, and treatment approaches are tested to find out whether they are safe and whether they work better than what is already available. For people with brain cancer, especially aggressive tumors like glioblastoma, where current treatments have limitations, clinical trials can offer access to cutting-edge therapies that are not yet available outside of a research setting. All of the treatments that are now standard care for brain cancer (including temozolomide chemotherapy and tumor treating fields) were first tested in clinical trials before becoming widely available. Participating in a clinical trial can benefit both the individual patient and future patients by helping science move forward.

What Are the Reasons to Join a Clinical Trial?

• Access to new treatments before they are widely available. Clinical trials often offer access to promising therapies that are not yet part of standard care, including new immunotherapies, targeted drugs, advanced radiation techniques, and experimental surgical approaches. For patients with glioblastoma, where standard treatments have remained largely unchanged for two decades, clinical trials represent the primary pathway to new and potentially more effective options.
• Expert care and closer monitoring. People enrolled in clinical trials are typically seen more frequently, have more imaging done, and are followed more closely than patients in standard care. You are cared for by research teams with deep expertise in your tumor type. Many clinical trials are offered at major academic brain tumor centers, and participation can be a pathway to accessing that specialized expertise.
• Contributing to progress for future patients, brain cancer is rare enough and diverse enough in its tumor subtypes that it desperately needs patient participation in research to make progress. When you join a trial, your experience directly contributes to the body of knowledge that will one day help future patients. Many brain tumor survivors and advocates describe participation in research as one of the most meaningful things they could do.
• Possible cost benefits: The experimental drug or device in a clinical trial is typically provided at no cost to participants. Some trials also cover costs for related imaging, lab tests, and visits. However, standard care costs (like hospitalization) may still be billed to your insurance. Always ask the trial team about costs before enrolling and speak with your insurance company about coverage.

How Do Clinical Trials Work?

Clinical trials are conducted in phases, each with a different purpose:

• Phase I trials test a new treatment for the first time in humans. They usually enroll a small number of people (often 15–30) and focus primarily on safety and finding the right dose. Phase I brain tumor trials often enroll patients who have already tried standard treatments.
• Phase II trials test whether the treatment works for a specific tumor type in a larger group of patients. They evaluate effectiveness along with safety.
• Phase III trials compare the new treatment to the current standard treatment. These trials involve larger numbers of patients and are often randomized, meaning patients are assigned by chance to receive either the experimental treatment or the standard one. Phase III trials are how researchers establish whether a new treatment truly improves outcomes.
• Phase IV trials continue after a treatment is FDA-approved, monitoring long-term safety and effectiveness in broader populations.

How to Find Brain Cancer Clinical Trials

By connecting your medical records to HealthTree's Clinical Trial Finder, it can match your specific diagnosis and molecular profile to provide you with a personalized list of open trials.

Connect Your Medical Records

When searching for trials, having your complete pathology report, including your tumor type, grade, IDH status, MGMT methylation status, and other molecular markers, will help identify trials you may be eligible for, since eligibility criteria often specify molecular subtypes. If you need help, you can call our support team at 1-800-709-1113 or email us at support@healthtree.org.

Questions to Ask Before Joining a Trial

Before deciding, ask the research team:

• What is the goal of this trial, and what phase is it in?

• What treatment will I receive, and how does it compare to standard treatment?

• Is there any chance I could receive a placebo instead of active treatment?

• What are the possible side effects and risks?

• How many visits and tests will the trial require beyond standard care?

• What costs will the trial cover, and what might I owe?

• Can I continue seeing my regular neuro-oncologist while enrolled?

• What happens if the treatment is not working or causes me harm?

• What happens to my data and tissue samples after the trial?

Sources:

1. National Cancer Institute. Understanding Clinical Trials. https://www.cancer.gov/about-cancer/treatment/clinical-trials/what-are-trials