Targeted Drug Therapy for Pancreatic Neuroendocrine Tumors (pNET)

Targeted drugs work differently from standard chemotherapy (chemo) drugs. These drugs target specific parts of cancer cells.

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

Cabozantinib (Cabometyx) for pNETs

Cabozantinib blocks several tyrosine kinases (cell function regulators such as RET, MET, and VEGFR2) and can help slow tumor growth in different ways. This drug is a pill taken once a day.

The most common side effects are nausea, vomiting, diarrhea, constipation, fatigue, poor appetite, and high blood pressure. Rare, but serious side effects include bleeding, clotting, severe high blood pressure, palmar-plantar erythrodysesthesia (PPE, which is redness, pain, and skin peeling of the palms of the hands and the soles of the feet), liver damage, difficulty with wound healing, and harm to a fetus.

Sunitinib (Sutent) for pNETs

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

The most common side effects are tiredness (fatigue), nausea, vomiting, diarrhea, constipation, abdominal pain, mouth sores, problems breathing, cough, and changes in skin or hair color.

Other possible effects include 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) for pNETs

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

Common side effects of this drug include mouth sores, infections, loss of appetite, diarrhea, tiredness, cough, 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 (dyspnea) or other problems.

Belzutifan (Welireg)

Belzutifan is an HIF inhibitor. It blocks a protein called hypoxia-inducible factor 2 alpha (HIF-2a), which affects how a cell responds to low oxygen levels (hypoxia) and how new blood vessels form in tumors. This drug is a pill, taken typically once a day.

Belzutifan can be used in people with von Hippel-Lindau (VHL) disease who have an advanced pNET.

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.