Targeted Drug Therapy for Adult Brain and Spinal Cord Tumors

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

Bevacizumab (Avastin, Mvasi, Zirabev)

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 (a process known as angiogenesis), which they need in order to grow.

This drug is used mainly to treat some types of gliomas (especially fast-growing ones such as glioblastomas) that come back after initial treatment. It might also be useful in treating recurrent meningiomas.

When used alone or added to chemotherapy, this drug can help shrink some tumors or extend the time until they start growing again, although it does not seem to help people live longer. It can also help lower the dose of the steroid drug dexamethasone needed to help reduce swelling in the brain, which is especially important for patients sensitive to steroid side effects.

Bevacizumab is given by intravenous (IV) infusion, usually once every 2 weeks.

Common side effects include high blood pressure, tiredness, bleeding, low white blood cell counts, headaches, mouth sores, loss of appetite, and diarrhea. Less common but possibly serious side effects include blood clots, internal bleeding, heart problems, and holes (perforations) in the intestines. This drug can also slow wound healing, so usually it can't be given within a few weeks of surgery.

Everolimus (Afinitor)

Everolimus works by blocking a cell protein known as mTOR, which normally helps cells grow and divide into new cells. For subependymal giant cell astrocytomas (SEGAs) that can’t be removed completely by surgery, This drug 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 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 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.