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Targeted Drug Therapy for Adult Brain and Spinal Cord Tumors

As researchers have learned more about the inner workings of cells that cause tumors or help tumor 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

Bevacizumab (Avastin, other brand names) 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 (a process known as angiogenesis), which they need in order to grow.

This drug is used mainly to treat some types of gliomas (especially fast-growing ones such as glioblastomas) that come back after initial treatment. It might also be useful in treating recurrent meningiomas.

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 of bevacizumab can 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.

Drugs that target tumors with IDH gene changes

In people with some types of brain tumors, the tumor cells might have a change (mutation) in either the IDH1 or IDH2 gene. These genes help the cells make certain proteins, which are also called IDH1 and IDH2. Mutations in one of these genes can stop the tumor cells from maturing the way they normally would. 

Vorasidenib (Voranigo) is a type of targeted drug known as an IDH inhibitor. It blocks abnormal IDH1 and IDH2 proteins, which seems to help the tumor cells mature into more normal cells. 

This drug can be used after surgery in people with a grade 2 astrocytoma or oligodendroglioma, if the tumor cells are found to have an IDH1 or IDH2 gene mutation. Your doctor can get tests of the tumor cells to see if they have one of these mutations.

This drug is taken by mouth as tablets, once a day.

Common side effects of vorasidenib can include feeling very tired, headache, nausea, muscle aches or stiffness, diarrhea, seizures, and changes in lab tests showing the drug is affecting the liver.

Sometimes this drug might have more serious effects on the liver, which could lead to symptoms such as jaundice (yellowing of the eyes and skin), dark urine, loss of appetite, or pain in the upper right side of your belly. It’s important to let your health care team know if you have any of these symptoms.

Everolimus

Everolimus (Afinitor) 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 of everolimus can 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 editors and translators with extensive experience in medical writing.

Dorsey JF, Salinas RD, Dang M, et al. Chapter 63: Cancer of the central nervous system. In: Niederhuber JE, Armitage JO, Doroshow JH, Kastan MB, Tepper JE, eds. Abeloff’s Clinical Oncology. 6th ed. Philadelphia, Pa: Elsevier; 2020.

National Cancer Institute Physician Data Query (PDQ). Adult Central Nervous System Tumors Treatment. 2020. Accessed at www.cancer.gov/types/brain/hp/adult-brain-treatment-pdq on February 17, 2020.

National Comprehensive Cancer Network. NCCN Clinical Practice Guidelines in Oncology: Central Nervous System Cancers. V.3.2019. Accessed at www.nccn.org/professionals/physician_gls/pdf/cns.pdf on February 17, 2020.

Wen PY. Systemic treatment of recurrent meningioma. UpToDate. 2020. Accessed at https://www.uptodate.com/contents/systemic-treatment-of-recurrent-meningioma on February 17, 2020.

Last Revised: August 8, 2024

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