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Brain Tumors: Targeted Therapy

Targeted therapy is the use of medicines that target the parts of cancer cells that make them unlike normal cells. Or the medicines can target other cells that help tumors grow. Targeted therapy might be used to treat a brain tumor when other treatments are not working well. These medicines tend to have less-severe side effects than standard chemotherapy medicines. There are currently 2 targeted therapy medicines used to treat brain tumors: bevacizumab and everolimus.


This medicine is a type known as a monoclonal antibody. It’s a lab-made version of an immune protein. Antibodies can be made to affect very specific targets.

Bevacizumab targets a protein called VEGF. This protein helps tumors create the new blood vessels they need to keep growing. Blocking VEGF helps limit the size of the tumor. When added to chemotherapy, this medicine can help slow the growth of some types of tumors. It's often used for gliomas and glioblastomas. The medicine is given as an infusion into a vein once every 2 weeks. Common side effects can include:

  • Feeling tired

  • Mouth sores

  • High blood pressure

  • Headaches

  • Diarrhea

In some cases, more serious side effects can occur. These include:

  • Blood clots

  • Internal bleeding (bleeding inside the body)

  • Heart problems

  • Holes (perforations) in the digestive tract


This medicine targets a protein known as mTOR. This protein helps cells grow and make new cells. Everolimus can help treat a type of brain tumor called a subependymal giant cell astrocytoma (SEGA) if it can’t be treated with surgery. It's taken daily as a pill. Common side effects include:

  • Mouth sores

  • Nausea

  • Loss of appetite

  • Infections

  • Feeling tired or weak

  • Diarrhea

  • Skin rashes

In some cases, it can damage the lungs. This can lead to breathing problems. 

More medicines being tested

Few targeted medicines are currently used to treat brain tumors. But researchers continue to work on new medicines to treat these cancers. These new medicines are tested in clinical trials. If you want to be part of a clinical trial, talk with your healthcare provider. He or she can help you find out if a clinical trial would be right for you.

Online Medical Reviewer: Kimberly Stump-Sutliff RN MSN AOCNS
Online Medical Reviewer: Louise Cunningham RN BSN
Online Medical Reviewer: Richard LoCicero MD
Date Last Reviewed: 9/1/2020
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