Risk Management & Treatment

Biomarkers, targeted and immunotherapies for pancreatic cancer

This section covers the following topics:

Genetic tests for inherited mutations for treatment selection

Any person diagnosed with pancreatic cancer meets national guidelines for genetic testing for an inherited mutation. Genetic testing may be used to guide treatment selection.  

  • People who test positive for an inherited BRCA1 or BRCA2 mutation may respond better to a treatment regimen that includes a type of chemotherapy known as platinum. They may also benefit from maintenance therapy with a type of targeted therapy known as a PARP inhibitor.  
  • People who test positive for other inherited gene mutations may qualify for clinical trials looking at targeted therapies. 
  • People who test positive for a Lynch syndrome gene mutation may benefit from treatment with an immunotherapy agent

Biomarker testing for treatment selection

Biomarker tests look at samples of blood, tumor or other tissue for changes or abnormalities caused by cancer. These tests can give doctors clues about the cancer, including:

  • how fast the cancer is growing
  • which treatments are most likely to work
  • whether or not the cancer is responding to treatment or growing
  • whether or not the cancer has come back after remission

Biomarker tests may be used to select treatments, and help patients avoid side effects from treatments that will not work for them. Biomarker tests used to select a specific treatment are sometimes called "companion diagnostic tests." These tests may be done on tumor tissue or (in many cases) on blood. See our Biomarker Testing section for more information. 

Examples of biomarker tests used in pancreatic cancer include:

  • Some pancreatic cancers will have an abnormality known as MSI-H (microsatellite instability high") also known as "mismatch repair deficiency" (dMMR or MMR-D). MSI-H cancers are common in people with a Lynch syndrome gene mutation. These cancers may respond well to a type of treatment known as an immune checkpoint inhibitor. One example of an immunotherapy agent used for MSI-high cancers is Keytruda (pembrolizumab).
  • Some cancers have a specific genetic change called an NTRK fusion, which can be found on tumor testing. People whose tumor test reveals an NTRK fusion may benefit from the targeted therapy Vitrakvi (larotrectinib).
  • Additional biomarker tests may help identify people who are elegible for certain clinical trials. 

PARP inhibitors 

PARP inhibitors work by blocking a protein used by cells to repair damaged DNA. They were initially developed to treat cancers in people with an inherited gene mutation BRCA1 or BRCA2 mutation. For people with pancreatic cancer, the PARP inhibitor Lynparza (olaparib) has been approved as maintenance therapy in patients with advanced pancreatic cancer whose cancer has stabilized after at least four months of chemotherapy.  Approximately 5-8% of patients with pancreatic cancers will have a mutation in BRCA or related genes.

Research is ongoing to learn if PARP inhibitors are also affective for treating cancer in other situations, including:  

  • people with an inherited mutation in a different gene that repairs DNA damage (for example: PALB2).
  • people who do not have an inherited gene mutation, but their tumor tested positive for an acquired mutation in a gene that repairs DNA damage.
  • in combination with immunotherapy or other agents.


Immunotherapies are cancer treatments that hlep the body’s immune system detect and attack cancer cells. There are several different categories of immunotherapies. 

  • Keytruda (pembrolizumab) is known as an immune checkpoint inhibitor. Keytruda is approved for treatment of patients with metastatic cancer with a biomarker known as microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR). Although this biomarker is not common in pancreatic cancer, it is often seen in people with a Lynch syndrome gene mutation who develop cancer. 

Other targeted therapies

  • Tarceva (erlotinib) is approved in combination with the chemotherapy gemcitabine as first-line therapy for locally advanced, unresectable or metastatic pancreatic cancer.  
  • Vitrakvi (larotrectinib) is approved for treatment of pancreatic cancer that is metastatic or cannot be removed with surgery and has worsened with other treatments. It targets a specific genetic change called an NTRK fusion. This type of genetic change is found in a range of cancers, including pancreatic cancer.
  • Afinitor (everolimus) is a type of targeted therapy known as an mTOR inhibitor that is FDA approved for treating people with advanced pancreatic neuroendocrine tumors.
  • Sutent is a targeted therapy that is FDA approved to treat patients with pancreatic neuroendocrine tumors that cannot be removed by surgery or that have metastasized.

Table of targeted and immunotherapies for pancreatic cancer

Name of drug Cancer stage Indication Biomarker Type of agent
Metastatic pancreatic cancer First-line maintenance therapy for patients whose disease has not progressed on at least 16 weeks of platinum-based chemotherapy  Inherited mutation in BRCA1 or BRCA2 PARP inhibitor
Tarceva (erlotinib) Locally advanced, unresectable or metastatic pancreatic cancer First-line therapy used in combination with gemcitabine No biomarker required EGFR inhibitor
Progressive pancreatic neuro-endocrine tumors (PNET)  Treatment of neuroendocrine tumors of pancreatic origin (PNET) that have progressed No biomarker required MTOR inhibitor (type of kinase inhibitor)
(sunitinib malate)
Unresectable, locally advanced or metastatic pancreatic neuro-endocrine tumors (PNET) For treatment of progressive, well-differentiated pancreatic neuroendocrine tumors (pNET) No biomarker required Multi-target kinase inhibitor
Keytruda (pembrolizumab) Metastatic or unresectable solid tumors For treatment of solid tumors that have progressed after treatment and for which there are no other treatment options MSI-H (Microsatellite Instability High) or MMR-D (Mismatch Repair Deficiency Immune checkpoint inhibitor
Keytruda (pembrolizumab) Metastatic or unresectable solid tumors

For the treatment of solid tumors that have progressed following prior treatment and for which there are no satisfactory alternative treatment options

Tumor Mutational Burden High (TMB-H) Immune checkpoint inhibitor
Vitrakvi (larotrectinib) Metastatic solid tumors For treatment in metastatic solid tumors where surgical resection is likely to result in severe morbidity, and  for which there are no satisfactory alternative treatments or the cancer progressed following treatment NTRK fusion Kinase inhibitor

The majority of public and private health insurance plans are required to cover cancer diagnosis and treatment; copays, coinsurance and deductibles often apply. Patient costs and coverage for specific doctors, facilities or treatments may vary based on your health plan. Visit our section on Insurance and Paying for Care: Treatment  for more information, links to sample appeal letters and other resources. 

Some pharmaceutical companies and laboratories have assistance programs that help cover the cost for their medications, tumor testing or genetic testing for an inherited mutation: 

Organizations that offer co-pay assistance:

Other resources:

  • The American Cancer Society provides information and resources on covering the cost of cancer care. Public assistance, such as Medicaid may be available if you are ineligible for other programs. 
  • Needy Meds: Assistance programs to help patients with cost of medications and other healthcare.
  • Triage Cancer offers tools and resources to help individuals cope with the financial aspects of a cancer diagnosis.

The following studies may be of interest to people with pancreatic cancer and an inherited mutation.  

Pancreatic cancer specific

Advanced solid tumors of any type 

Visit our Research Search and Enroll Tool to find additional studies.