Targeted therapy for adult brain and spinal cord tumors

As researchers have learned more about the inner workings of cells that cause cancer or help cancer cells grow, they have developed newer drugs that specifically target these changes. These targeted drugs work differently from standard chemotherapy drugs. They often have different (and less severe) side effects. These drugs do not yet play a large role in treating brain or spinal cord tumors, but some of them may be helpful for certain types of tumors.

Bevacizumab (Avastin)

Bevacizumab 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 to get nutrients (a process known as angiogenesis). Tumors need new blood vessels to grow.

Bevacizumab is given by intravenous (IV) infusion, usually once every 2 weeks. Some early studies have shown it might help shrink certain brain tumors, especially glioblastomas, but it is not yet clear if it can help people live longer.

More common side effects include high blood pressure, tiredness, bleeding, low white blood cell counts, headaches, mouth sores, loss of appetite, and diarrhea. Rare but possibly serious side effects include blood clots, internal bleeding, heart problems, holes (perforations) in the intestines, and slow wound healing.

Everolimus (Afinitor)

This drug works by blocking a cell protein known as mTOR, which normally promotes cell growth and division. For subependymal giant cell astrocytomas that can't be removed completely by surgery, it 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 taken as a pill once a day. Common side effects of this drug 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.