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Q&A: Understanding Brain Tumor Treatment, Impact, and Research

, by NCI-CONNECT Staff

Two doctors examine a patient in a hospital room

NCI-CONNECT providers collaborate with your doctor to devise a comprehensive care plan.


Our neuro-oncology providers share answers to important questions about brain tumor care, treatment, impact, and innovative research.

A primary brain tumor is a mass of abnormal cells that arises in the brain. An estimated 24,000 adults in the United States are diagnosed each year with a cancerous brain tumor. And brain cancers are less than two percent of all cancers diagnosed. This makes brain cancer more challenging to treat and study than other cancers.

To better understand brain tumor care, treatment, impact, and research, we talked to the NCI Center for Cancer Research's Neuro-Oncology Branch chief, Mark Gilbert, M.D., and deputy chief, Terri Armstrong, Ph.D.

Q: How Are Brain Tumors Diagnosed?

A: People most often have a sudden symptom like a seizure, migraine, or cognitive issue, and go to the emergency room or doctor. There, imaging tests, such as a magnetic resonance imaging (MRI) scan or computerized tomography (CT) scan, can detect a brain tumor. Then, a biopsy is performed to remove a piece of tumor tissue for examining by a neuropathologist who determines the grade, tumor type, and genetic information. This is critically important to make an accurate diagnosis.

There are more than 100 different types of primary brain tumors, and treatment is dependent upon the tumor type. A misdiagnosis can result in an inaccurate treatment plan. So, we strongly encourage patients to seek a second opinion to find a neuro-oncologist with experience treating their tumor type (see Guiding Questions to Ask Your Doctor).  

A good time to get a second opinion is when the tumor is first diagnosed—or if there is recurrence and patients are thinking about other treatment options. This helps patients feel comfortable about their treatment decisions. If at patient is seen at NIH, we always contact the referring doctor to let them know our thoughts and partner with them. To make an appointment or refer a patient, contact us at or call 1-866-251-9686.

Q: What Are Brain Tumor Treatment Options?

A: There is no cookbook, as we say, that covers all brain tumors and how we treat them. We need very important information, starting with the tumor type and grade. And as our knowledge is expanding, we can rely on genetic changes within specific tumors to guide treatment. We also individualize treatment based on patient factors, including functional status and concurrent illness. This helps optimize the therapy for each patient.

That said, surgery to remove as much of the tumor as possible is typically the first treatment for brain tumors. The risks and benefits of surgery depend on the location of the tumor in the brain. We encourage patients to seek a neurosurgeon who is an expert in brain tumor neurosurgeries. Radiation therapy and chemotherapy are other treatments considered after surgery and can depend on the tumor grade—grade1 tumors (also written as grade I) are usually not cancer and can be treated with surgery. Grade 2, 3, and 4 tumors (also written as grade II, III, and IV) are cancerous and either have a higher chance of coming back or are fast-growing, so they often need more treatment after surgery.

Q: What New Brain Tumor Treatment Approaches Are You Studying? 

A: There have been tremendous advances in our ability to investigate cancers. We can do genetic analysis to better understand the biology of the cancer to start asking questions about specific treatments. We consider these treatments targeted therapies—meaning we can personalize treatment based on the specific characteristics of the cancer. We are discovering that many cancers, even though they fall under the same name, have individual differences we can target.

Immunotherapy has been a fantastic advancement in treating other cancers. Physicians are now putting patients with diseases that were previously incurable into long-term remission with immunotherapy. And we would like to see the same type of success in brain tumors. But we also recognize that treating brain tumors is more complicated than other cancers. It will likely turn out that it won't be a single immunotherapy that works. It will have to be a combination of strategies to overcome some of the difficulties in getting an immune reaction in the brain.

We have active clinical trials to better understand immunotherapy, as well as other therapies. Clinical trials are important to advance our knowledge and help us develop better and better treatments for patients with brain tumors. We need to focus our energy and participation into clinical trials.

Q: How Can Brain Tumors Affect Patients?

A: The impact of a brain tumor on patients can be great. It's estimated that over 50 percent of low- and high-grade tumor patients have difficultly even returning to work from the time of diagnosis. We know that symptoms can occur at the time of diagnosis, but some can also occur throughout the time the person is dealing with the illness.

Researchers like us, are focusing on understanding what that impact is on the patient and trying to improve it. Specifically, we want to understand the impact of our treatments. Within our clinical trials, we try to identify that and monitor patients throughout the trial. We are learning more about people living with the diagnosis of a brain tumor and the issues they face, including care issues, financial issues, and the impact on the extended family.

At NIH, we are focused on helping patients cope with their illness and deal with the issues they may face as a result of it.

Q: What Is the Future of Brain Tumor Research?

A: There are a couple areas of investigation. We are working to understand the cell of origin of brain cancers. We don’t understand (in most patients) why the cancer formed, because these cancers are so uncommon. We are working with our colleagues in epidemiology to understand if some patients may have a susceptibility to cancer.

We have also discovered that cancer cells have a unique metabolism. There are active studies trying to target that metabolism to specifically target the cancer without causing side effects. This is called metabolomics.

We are excited about the fact that we are making progress both at the genetic level to find targeted therapies and in immunotherapy. We are using techniques and approaches to study immunotherapy that are really cutting edge, which will hopefully lead to a better understanding of immunotherapy for brain tumors and translate into much better treatment.

There is also a level of international collaboration that is unprecedented. Everybody recognizes that there needs to be a collective effort to make advancements. And so the scale, scope, and sophistication of our clinical trials is increasing. We are integrating laboratory science with clinical research, and—just as importantly—including patient outcomes measures in this research. This gives us insights into what our clinical trials really mean and how to build on them in a way that's never been done before.

To that end, building partnerships with providers, patients, and advocates to help patients with brain and spine tumors is the mission of NCI-CONNECT, our program supported by the Cancer Moonshot℠.  We are focusing on building the infrastructure for rare central nervous system tumors to improve approaches to care and treatment.

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