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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 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. It is often used for glioblastoma. The medicine is given as an infusion into a vein. This is done usually once every 2 weeks. Common side effects can include:

  • Feeling tired

  • Bleeding

  • High blood pressure

  • Headaches

  • Diarrhea

In some cases, 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. The medicine can help treat a tumor known as subependymal giant cell astrocytoma (SEGA) if it can’t be treated with surgery. It is taken daily as a pill. Common side effects include:

  • Mouth sores

  • Nausea

  • Loss of appetite

  • Infections

  • Fatigue

  • Diarrhea

  • Skin rashes

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

More 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 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: Alteri, Rick, MD
Online Medical Reviewer: Jasmin, Luc, MD
Date Last Reviewed: 1/1/2018
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