Targeted Therapy

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Targeted therapies attack specific parts of the pancreatic cancer cell

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What Is Targeted Therapy?

Targeted therapy is cancer treatment that attacks unique aspects of cancer cells with little harm to healthy cells.

Targeted therapies block processes that change normal cells into cancer. Blocking these processes can stop the cancer cells from growing.

Doctors may use targeted therapies to treat patients based on their unique biology.

Targeted therapies generally do not harm healthy cells. So, they usually cause fewer side effects than other treatments.

Targeted therapy side effects are different for each treatment. Every patient will also have a different response to a treatment.

Talk to the healthcare team about any treatment side effects or related concerns. Supportive care may help.

The Pancreatic Cancer Action Network’s Patient Central can also give you information about side effects as well as supportive care and side effect management.

What Targeted Therapies Are Available for Pancreatic Cancer?

There are several targeted therapies approved by the Food and Drug Administration (FDA) for certain groups of pancreatic cancer patients:

  • Tarceva® (erlotinib)
  • Sutent® (sunitinib) for neuroendocrine tumors
  • Afinitor® (everolimus) for neuroendocrine tumors
  • Vitrakvi® (larotrectinib)
  • ROZLYTREK® (entrectinib)
  • Lynparza® (olaparib)

For many of these treatments, patients must have testing to see if they have mutations (changes) targeted by these treatments. This testing includes:

  • Genetic testing for an inherited mutation: testing of blood or saliva to find mutations the patient was born with, passed on from a parent
  • Molecular profiling: testing of tumor tissue to find mutations in the tumor (also known as tumor biomarker testing)

Patient Central can give you free information about genetic testing and molecular profiling, including how to get these tests through the Know Your Tumor® precision medicine service.

Tarceva® (erlotinib)

Tarceva (erlotinib), in combination with the chemotherapy gemcitabine (Gemzar®), is approved for use in advanced pancreatic adenocarcinoma (the most common type of pancreatic cancer) that cannot be removed by surgery.

Tarceva is not commonly used to treat pancreatic cancer. This is because only a small group of patients benefited in clinical trials.

Common side effects of Tarceva include acne-like skin rash, diarrhea, nausea, appetite loss and fatigue.

Lynparza® (olaparib)

Lynparza is FDA-approved as a maintenance therapy for patients who have:

A patient can learn if they have a germline BRCA mutation through genetic testing. About 4-7% of pancreatic cancer patients have germline BRCA mutations.

Doctor shows pancreatic cancer patient test results that mean targeted therapy is a treatment optionLynparza is a type of targeted therapy called a PARP inhibitor. It blocks the PARP protein, which helps cells repair DNA damage. This causes damage to build up.

Patients who have a BRCA mutation may respond well to treatment with a PARP inhibitor like Lynparza because these mutations already weaken the cells’ ability to fix damaged DNA. When too much DNA damage occurs, the cancer cells cannot survive.

Approved as a maintenance therapy, the goal Lynparza is to extend the time that a patient’s cancer does not progress after receiving a platinum-containing chemotherapy. Common platinum chemotherapies include oxaliplatin and cisplatin. Not all standard of care chemotherapies for pancreatic cancer contain platinum agents.

Chemotherapy can cause side effects that may make long-term use challenging. So, patients whose cancer has remained stable on chemotherapy sometimes stop treatment entirely to give their bodies a break or go on a maintenance therapy like Lynparza.

Common side effects of Lynparza include lowered blood cell count, nausea, vomiting, diarrhea, fatigue, upper respiratory tract infection and joint or muscle pain.

Vitrakvi® (larotrectinib) and ROZLYTREK® (entrectinib)

Two targeted therapies are FDA-approved for use in patients with any type of locally advanced or metastatic solid tumor with an NTRK gene fusion:

  • Vitrakvi (larotrectinib)
  • ROZLYTREK (entrectinib)

NTRK gene fusions are very rare in pancreatic cancer. Only about 0.5% of patients have them. Patients can learn if they have an NTRK gene fusion through molecular profiling.

Common side effects of these treatments include fatigue, vomiting, constipation, dizziness, diarrhea and nausea.

Targeted Therapies for Pancreatic Neuroendocrine Tumors (PNETS)

Two targeted therapy drugs are approved for the treatment of advanced pancreatic neuroendocrine tumors (PNETs) that have progressed and cannot be treated with surgery:

  • Sutent (sunitinib)
  • Afinitor (everolimus)

About 7% of pancreatic tumors are neuroendocrine tumors.

Both Sutent and Afinitor are taken as a daily pill.

Common side effects of Sutent include lowered blood cell counts, diarrhea, upset stomach, nausea, vomiting, mouth sores, loss of appetite, fatigue and congestive heart failure.

Common side effects of Afinitor include lowered blood cell counts, fatigue, nausea, diarrhea, cough, mouth sores, high blood sugar and pneumonitis (inflamed lung tissue).

Targeted Therapy in Clinical Trials

Other targeted therapies for pancreatic cancer are still being studied in the laboratory or in clinical trials.

Clinical trials are research studies that test new investigational treatments. The goal is to find better ways to treat pancreatic cancer.

Trials give patients the opportunity to receive a promising investigational drug or treatment and are the only way to make progress in treating the disease.

In clinical trials, patients usually get investigational targeted therapies along with other standard treatments like chemotherapy, radiation or surgery.

For a personalized list of targeted therapy clinical trials, contact Patient Central.

How Can I Access Targeted Therapies?

Targeted therapies are usually available in one of two ways:

Contact Patient Central for a personalized list of clinical trials, including any trials available based on the patient’s biology. Patient Central can also give you information about germline testing and molecular profiling.

The Pancreatic Cancer Action Network strongly recommends:

  • Clinical trials at diagnosis and during every treatment decision
  • Molecular profiling of the patient’s tumor to help determine the best treatment options
  • Genetic (germline) testing as well as genetic counseling, regardless of family history

Our Know Your Tumor precision medicine service gives eligible patients free access to germline testing and molecular profiling.

Patients who have already had testing identify genetic changes that may point to an approved targeted therapy can speak with their healthcare team about what options are available to them. Patient Central can also provide more information.

We’re Here to Help

For free, in-depth and personalized resources and information on pancreatic cancer treatment, contact Patient Central. Patient Central can also give you more information on genetic testing, molecular profiling and clinical trials.

Related Topics

  • Clinical Trials

    See how clinical trials benefit patients and how to find one.


Information reviewed by PanCAN’s Scientific and Medical Advisory Board, who are experts in the field from such institutions as University of Pennsylvania, Memorial Sloan-Kettering Cancer Center, Virginia Mason Medical Center and more.

Information provided by the Pancreatic Cancer Action Network, Inc. (“PanCAN”) is not a substitute for medical advice, diagnosis, treatment or other health care services. PanCAN may provide information to you about physicians, products, services, clinical trials or treatments related to pancreatic cancer, but PanCAN does not recommend nor endorse any particular health care resource. In addition, please note that any personal information you provide to PanCAN’s associates during telephone and/or email communications may be stored and used to help PanCAN achieve its mission of assisting patients with, and finding cures and treatments for, pancreatic cancer. Stored constituent information may be used to inform PanCAN programs and activities. Information also may be provided in aggregate or limited formats to third parties to guide future pancreatic cancer research and treatment efforts. PanCAN will not provide personal directly identifying information (such as your name or contact information) to such third parties without your prior written consent unless required or permitted by law to do so.

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