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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, it targets other cells that help tumors grow. Targeted therapy can help when other treatments are not working as well. They can also have less-severe side effects than standard chemotherapy medicine. There are currently 2 medicines to treat brain tumors. They are bevacizumab and everolimus.


This medicine is a type of targeted therapy known as a monoclonal antibody. It’s a lab-made version of an immune protein. Antibodies can be made to affect very specific targets. This medicine targets a protein called VEGF. The protein normally helps tumors create the new blood vessels they need to keep growing. Blocking this protein helps limit the size of the tumor. When added to chemotherapy, this medicine can help slow the growth of some types of tumors, especially glioblastoma. The medicine is given as an infusion into a vein, usually once every 2 weeks. Common side effects can include:

  • Feeling tired

  • Bleeding

  • High blood pressure

  • Headaches

  • Diarrhea

Less often, more serious side effects can occur. These include:

  • Blood clots

  • Bleeding inside the body

  • Heart problems

  • Holes (perforations) in the digestive tract


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

  • Mouth sores

  • Nausea

  • Loss of appetite

  • Infections

  • Fatigue

  • Diarrhea

  • Skin rashes

Less often it can damage the lungs, which can lead to breathing problems. 

More targeted medicines being tested

Only a small number of targeted medicines are currently used to treat brain tumors. But researchers continue to work on new medicines to treat cancer. These new medicines are being tested in clinical trials. If you’re interested in a medicine that is 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: Alteri, Rick, MD
Online Medical Reviewer: Cunningham, Louise, RN
Date Last Reviewed: 7/14/2015
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