Targeted Therapy Drugs for Brain and Spinal Cord Tumors in Children

As researchers have learned more about the changes in the inner workings of cells that cause cancer or help cancer cells grow, they have developed newer drugs that 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 do not yet play a large role in treating brain or spinal cord tumors, but some of them might be helpful for certain types of tumors.

Everolimus (Afinitor)

For subependymal giant cell astrocytomas (SEGAs) that can’t be removed completely by surgery, everolimus may shrink the tumor or slow its growth for some time. This drug works by blocking a cell protein known as mTOR, which normally helps cells grow and divide into new cells.

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 lung damage, which can cause shortness of breath or other problems.

Many other targeted drugs are now being developed and studied in clinical trials. Some of these are described in What’s New in Research and Treatment for Brain and Spinal Cord Tumors in Children?

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Last Medical Review: June 20, 2018 Last Revised: June 20, 2018

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