Targeted Drug Therapy for Adult Brain and Spinal Cord Tumors

Bevacizumab

Bevacizumab (Avastin, other brand names) 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 of bevacizumab can 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.

Drugs that target tumors with IDH gene changes

In people with some types of brain tumors, the tumor cells might have a change (mutation) in either the IDH1 or IDH2 gene. These genes help the cells make certain proteins, which are also called IDH1 and IDH2. Mutations in one of these genes can stop the tumor cells from maturing the way they normally would. 

Vorasidenib (Voranigo) is a type of targeted drug known as an IDH inhibitor. It blocks abnormal IDH1 and IDH2 proteins, which seems to help the tumor cells mature into more normal cells. 

This drug can be used after surgery in people with a grade 2 astrocytoma or oligodendroglioma, if the tumor cells are found to have an IDH1 or IDH2 gene mutation. Your doctor can get tests of the tumor cells to see if they have one of these mutations.

This drug is taken by mouth as tablets, once a day.

Common side effects of vorasidenib can include feeling very tired, headache, nausea, muscle aches or stiffness, diarrhea, seizures, and changes in lab tests showing the drug is affecting the liver.

Sometimes this drug might have more serious effects on the liver, which could lead to symptoms such as jaundice (yellowing of the eyes and skin), dark urine, loss of appetite, or pain in the upper right side of your belly. It’s important to let your health care team know if you have any of these symptoms.

Drugs that target tumors with H3 K27M changes

Brain tumors known as diffuse midline gliomas often have a genetic change known as an H3 K27M mutation, which affects how some genes in the cell work. 

Dordaviprone (Modeyso) is a type of targeted drug known as a protease activator. It seems to work by helping to restore more normal gene control within the cell.

Dordaviprone can be used to treat diffuse midline gliomas that have the H3 K27M mutation, if the tumor is still growing despite a prior treatment.

This drug is taken by mouth, typically once a week. It can be taken by capsules, or the content of the capsules can be dissolved into a liquid.

Common side effects of dordaviprone can include feeling very tired, headache, nausea and vomiting, muscle, joint, and bone pain. It can also lower your blood cell counts and lead to changes in lab tests showing the drug is affecting the liver.

Less common but more serious side effects can include:

• Allergic reactions, which can show up as hives, skin rash, fever, feeling faint or dizzy, wheezing or trouble breathing, or swelling of the face or throat
• Heart rhythm problems, which might show up as symptoms like feeling lightheaded or faint, feeling dizzy, having heart palpitations or a fast heartbeat, or shortness of breath

Everolimus

Everolimus (Afinitor) 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 of everolimus can 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.