Targeted Therapy for Pancreatic Cancer

As researchers have learned more about the changes in pancreatic cancer cells that help them grow, they have developed newer drugs to specifically target these changes. These targeted drugs work differently from standard chemo drugs. Sometimes they work when standard chemo drugs don’t, and they often have different side effects. (See What’s New in Pancreatic Cancer Research? for more information.)

EGFR inhibitor

Erlotinib (Tarceva) is a drug that targets a protein on cancer cells called EGFR, which normally helps the cells grow. In people with advanced pancreatic cancer, this drug can be given along with the chemo drug gemcitabine. Some people may benefit more from this combination than others.

This drug is taken as a pill, once a day.

Common side effects of erlotinib include an acne-like rash on the face and neck, diarrhea, loss of appetite, and feeling tired. Less common but more serious side effects can include serious lung, liver, or kidney damage; holes (perforations) forming in the stomach or intestines; serious skin conditions; and bleeding or blood clotting problems.

PARP inhibitor

In a small number of pancreatic cancers, the cells have changes in one of the BRCA genes (BRCA1 or BRCA2). Changes in one of these genes can sometimes lead to cancer.

Olaparib (Lynparza) is a type of drug known as a PARP inhibitor. PARP enzymes are normally involved in a pathway that helps repair damaged DNA inside cells. The BRCA genes are normally involved in a different pathway of DNA repair, and mutations in one of these genes can block that pathway. By blocking the PARP pathway as well, this drug makes it very hard for tumor cells with a mutated BRCA gene to repair damaged DNA, which often leads to their death.

Olaparib can be used to treat advanced pancreatic cancer in people with a known or suspected BRCA gene mutation, whose cancer has not gotten worse after at least 4 months of chemo that included a platinum drug (such as oxaliplatin or cisplatin).

This drug has been shown to help shrink or slow the growth of some advanced pancreatic cancers, although so far it's not clear if it can help people live longer.

This drug is taken by mouth as pills, typically twice a day.

Side effects of this drug can include nausea, vomiting, diarrhea or constipation, fatigue, feeling dizzy, loss of appetite, taste changes, low red blood cell counts (anemia), low white blood cell counts (with an increased risk of infection), belly pain, and muscle and joint pain. Less common but more serious side effects can include inflammation in the lungs and the development of certain blood cancers, such as myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML).

NTRK inhibitors

A small number of pancreatic cancers have changes in one of the NTRK genes. These gene changes can sometimes lead to abnormal cell growth and cancer. 

Larotrectinib (Vitrakvi) and entrectinib (Rozlytrek) target the proteins made by the NTRK genes. These drugs can be used in people with advanced pancreatic cancer that has been found to have an NTRK gene change, typically when the cancer is still growing despite other treatments.

These drugs are taken as pills, once or twice daily.

Common side effects of these drugs can include dizziness, fatigue, nausea, vomiting, constipation, weight gain, and diarrhea. Less common but more serious side effects can include abnormal liver tests, heart problems, and confusion.