Targeted Drug Therapy for Pancreatic Neuroendocrine Tumor

Targeted drugs work differently from standard chemotherapy (chemo) drugs. These drugs target specific parts of cancer cells. They are sometimes helpful when chemo is not, and they often have different side effects than chemo.

The targeted drugs used to treat pancreatic neuroendocrine tumors (NETs) work by blocking angiogenesis (the growth of new blood vessels that nourish cancers) or important proteins (called tyrosine kinases) in cancer cells that help them grow and survive.

Sunitinib (Sutent)

Sunitinib blocks several tyrosine kinases and attacks new blood vessel growth. It has been shown to help slow tumor growth. This drug is taken as a pill once a day.

The most common side effects are nausea, diarrhea, changes in skin or hair color, mouth sores, weakness, and low blood cell counts. Other possible effects include tiredness, high blood pressure, heart problems, bleeding, hand-foot syndrome (redness, pain, and skin peeling of the palms of the hands and the soles of the feet), and low thyroid hormone levels.

Everolimus (Afinitor)

Everolimus blocks a protein known as mTOR, which normally helps cells grow and divide. It has been shown to help treat advanced pancreatic NETs. Everolimus is a pill taken once a day.

Common side effects of this drug include mouth sores, 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.

Belzutifan (Welireg)

Belzutifan is a type of drug known as a HIF inhibitor. It blocks a protein called hypoxia-inducible factor 2 alpha (HIF-2a), which is involved in both cancer cell growth and the formation of new blood vessels in tumors. This drug is taken as pills, typically once a day.

Belzutifan can be used in people with von Hippel-Lindau (VHL) disease who have a pancreatic NET and don’t need surgery right away.

Common side effects of this drug include low red blood cell counts (anemia), feeling tired and/or dizzy, nausea, headache, increased blood sugar levels, and changes in lab tests showing the drug might be affecting the kidneys. Less common but more serious side effects can include very low red blood cell counts (severe anemia), which might require blood transfusions, and low oxygen levels in the body, for which you might need oxygen therapy or even be admitted to the hospital.