Targeted Therapy for Adult Brain and Spinal Cord Tumors

As researchers have learned more about the inner workings of cells that cause cancer or help cancer cells grow, they have developed newer drugs that specifically target these changes. These targeted drugs work differently from standard chemotherapy drugs. They sometimes work when chemo drugs don’t, and they often have different side effects. Targeted drugs don't yet play a large role in treating brain or spinal cord tumors, but some of them might be helpful for certain types of tumors.

Bevacizumab (Avastin, Mvasi)

Bevacizumab is a man-made version of an immune system protein called a monoclonal antibody. This antibody targets vascular endothelial growth factor (VEGF), a protein that helps tumors form new blood vessels to get nutrients (a process known as angiogenesis). Tumors need new blood vessels to grow.

This drug is used mainly to treat some types of gliomas (especially glioblastomas) that come back after initial treatment, When used alone or added to chemotherapy, this drug can help shrink some tumors or extend the time until they start growing again, although it does not seem to help people live longer.  It can also help lower the dose of the steroid drug dexamethasone needed to help reduce swelling in the brain, which is especially important for patients sensitive to steroid side effects.

Bevacizumab is given by intravenous (IV) infusion, usually once every 2 weeks.

Common side effects include high blood pressure, tiredness, bleeding, low white blood cell counts, headaches, mouth sores, loss of appetite, and diarrhea. Less common but possibly serious side effects include blood clots, internal bleeding, heart problems, and holes (perforations) in the intestines. This drug can also slow wound healing, so usually it can't be given within a few weeks of surgery.

Everolimus (Afinitor)

Everolimus works by blocking a cell protein known as mTOR, which normally helps cells grow and divide into new cells. For subependymal giant cell astrocytomas (SEGAs) that can’t be removed completely by surgery, this drug may shrink the tumor or slow its growth for some time, although it’s not clear if it can help people with these tumors live longer.

Everolimus is a pill taken once a day. Common side effects include mouth sores, increased risk of infections, nausea, loss of appetite, diarrhea, skin rash, feeling tired or weak, fluid buildup (usually in the legs), and increases in blood sugar and cholesterol levels. A less common but serious side effect is damage to the lungs, which can cause shortness of breath or other problems.

Other targeted therapies are now being developed and studied in clinical trials.

More information about targeted therapy

To learn more about how targeted drugs are used to treat cancer, see Targeted Cancer Therapy.

To learn about some of the side effects listed here and how to manage them, see Managing Cancer-related Side Effects.

The American Cancer Society medical and editorial content team

Our team is made up of doctors and oncology certified nurses with deep knowledge of cancer care as well as journalists, editors, and translators with extensive experience in medical writing.

Chang SM, Mehta MP, Vogelbaum MA, Taylor MD, Ahluwalia MS. Chapter 97: Neoplasms of the central nervous system. In: DeVita VT, Lawrence TS, Rosenberg SA, eds. DeVita, Hellman, and Rosenberg’s Cancer: Principles and Practice of Oncology. 10th ed. Philadelphia, Pa: Lippincott Williams & Wilkins; 2015.

Dorsey JF, Hollander AB, Alonso-Basanta M, et al. Chapter 66: Cancer of the central nervous system. In: Abeloff MD, Armitage JO, Niederhuber JE. Kastan MB, McKenna WG, eds. Abeloff’s Clinical Oncology. 5th ed. Philadelphia, Pa: Elsevier; 2014.

National Comprehensive Cancer Network. NCCN Clinical Practice Guidelines in Oncology: Central Nervous System Cancers. V.1.2017. Accessed at on September 19, 2017.

Last Medical Review: September 30, 2017 Last Revised: November 8, 2017

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