Read about cancer treatment options listed by gene mutation, type of cancer and type of treatment.

Biomarkers, targeted and immunotherapies for colorectal cancer

This section covers the following topics:


Biomarker tests

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


Biomarkers for treatment selection

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. 

  • Experts recommend testing all colorectal cancers for an abnormality known as MSI-H (microsatellite instability high") also known as "mismatch repair deficiency" (dMMR or MMR-D).
    • MSI-high cancers are common in people with a Lynch syndrome gene mutation. People with advanced or metastatic MSI-high colorectal cancer may respond well to a type of immunotherapy known as an immune checkpoint inhibitor
  • Experts recommend testing metastatic colorectal cancers for the following biomarkers:
    • A specific mutation in a gene known as BRAF.  Tumors with the mutation called V600E, may respond to targeted therapies known as BRAF inhibitors
    • HER2 (also known as HER2/neu or ERBB2) is a biomarker commonly used in breast cancer to select patients who would benefit from anti-HER2 therapy (for example Herceptin (trastuzumab). In advanced colorectal cancer, tumors that test negative for RAS and BRAF mutations may be tested for HER2. Tumors that test positive for HER2 may be treated with Herceptin, usually in combination with standard chemotherapy.
  • Additional biomarker tests that may be used in colorectal cancer:
    • A biomarker known as an NTRK fusion is rare in colorectal cancer. Advanced/metastatic colorectal cancer with an NTRK fusion may benefit from the targeted therapy Vitrakvi (larotrectinib).
    • Additional tumor biomarker testing may help people learn if they are eligible for certain clinical trials. 


Immunotherapies 

Immunotherapies help the body’s immune system detect and attack cancer cells.

  • Immune checkpoint inhibitors are often used to treat advanced or metastatic colorectal cancer that have MSI-H or dMMR; usually after other treatments have been tried. The most common immune checkpoint inhibitors used for colorectal cancer are:
    • Keytruda (pembrolizumab)
    • Opdivo (nivolumab)
    • Yervoy (ipilimumab)


Targeted therapies

Several targeted therapies are used to treat advanced or metastatic colorectal cancer.  Some work best for people with or without a certain biomarker

  • Anti VEGF therapies block tumors from forming blood vessels. Anti VEGF drugs used to treat colorectal cancer include:
    • Avastin (bevacizumab) 
    • Cyramza (ramucirumab)
    • Zaltrap (ziv-aflibercept)
  • Anti EGFR therapies slow down tumor cell growth. Anti EGFR drugs used to treat colorectal cancer include:
    • Erbitux (cetuximab)
    • Vectibix (panitumumab)
  • BRAF inhibitors are oral medications that help slow down tumor growth in advanced colorectal cancers that test positive for the tumor biomarker called a BRAF V600E mutation. BRAF inhibitors are usually given in combination with other oral targeted drugs called MEK inhibitors (e.g., trametinib [Mekinist] or binimetinib [Mektovi]). BRAF inhibitors include:
    • Tafinlar (dabrafenib)
    • Braftovi (encorafenib)
  • Stivarga (regorafenib) blocks several different types of proteins in the body that tumors use to grow. Stivarga may be used to treat metastatic colorectal cancer that has come back after treatment with other drugs. 
  • Herceptin (trastuzumab) is used to treat advanced or recurrent HER2-positive colorectal cancers that do not haveBRAF, KRAS or NRAS mutation.
  • Vitrakvi (larotrectinib) is approved for treatment of advanced cancers that have worsened with other treatments. It targets a genetic change called an NTRK fusion. This type of genetic change is found in a range of cancers, including some colon cancers.

 

Table of targeted and immunotherapies for colorectal cancer

Name of drug Type of agent Cancer stage Indication Biomarker
Keytruda (pembrolizumab) Immune checkpoint inhibitor Metastatic or  unresectable colorectal cancer For first-line treatment of metastatic or unresectable colorectal cancer Microsatellite Instability High (MSI-H) or Mismatch Repair Deficiency (MMR-D
Metastatic or  unresectable solid tumors For treatment of solid tumors that have progressed after treatment and for which there are no other treatment options Microsatellite Instability High (MSI-H) or Mismatch Repair Deficiency (MMR-D
Metastatic or  unresectable solid tumors For the treatment of solid tumors that have progressed after treatment and for which there are no other treatment options Tumor Mutational Burden High (TMB-H)
Metastatic or unresectable colorectal cancer Cancer that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan Microsatellite Instability High (MSI-H) or Mismatch Repair Deficiency (MMR-D
Opdivo 
(nivolumab)
Immune checkpoint inhibitor Metastatic colorectal cancer

As a single agent or in combination with Yervoy (ipilimumab) for cancer that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan

Microsatellite Instability High (MSI-H) or Mismatch Repair Deficiency (MMR-D
Yervoy (ipilumumab) Immune checkpoint inhibitor Metastatic colorectal cancer Combined with Opdivo (nivolumab) for cancer that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan Microsatellite Instability High (MSI-H) or Mismatch Repair Deficiency (MMR-D
Avastin
(bevacizumab)
Monoclonal antibody for vascular endothelial growth factor (VEGF) Metastatic colorectal cancer Combined with intravenous 5-fluorouracil-based chemotherapy for first- or second-line treatment No biomarker required
Metastatic colorectal cancer In combination with chemotherapy for second-line treatment in patients who have progressed on a first-line Avastin-containing regimen No biomarker required
Cyramza
(ramucirumab)
Monoclonal antibody for vascular endothelial growth factor (VEGF) Metastatic colorectal cancer Combined with FOLFIRI chemotherapy, for treatment after disease progression on, or after prior therapy with bevacizumab, oxaliplatin, and a fluoropyrimidine No biomarker required
Erbitux
(cetuximab)
Monoclonal antibody for  epidermal growth factor receptor (EGFR) Metastatic colorectal cancer Combined with FOLFIRI for first-line treatment, or combined with irinotecan for cancers that no longer respond to irinotecan-based chemotherapy or as a single agent in patients who have progressed after oxaliplatin- and irinotecan-based chemotherapy EGFR positive and KRAS mutation negative
Vectibix
(panitumumab)
Monoclonal antibody for  epidermal growth factor receptor (EGFR) Metastatic colorectal cancer Combined with FOLFOX for first-line treatment Negative for KRAS and NRAS tumor mutations
Metastatic colorectal cancer As a single therapy following disease progression after prior treatment with fluoropyrimidine, oxaliplatin, and irinotecan-containing chemotherapy Negative for KRAS and NRAS tumor mutations

 

key-facts
  • All colorectal cancers should be tested for a biomarker known as MSI-H (also called MSI-high, dMMR or MMR-D)
  • Advanced cancers that are MSI-high may respond well to immunotherapy called immune checkpoint inhibitors. 
  • Cancers in people with Lynch syndrome are often MSI-high
paying-for-service

The majority of public and private health insurance plans are required to cover cancer diagnosis and treatment; copays, coinsurance and deductibles often apply. 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.
clinical-trials

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

Colorectal cancer specific

Advanced solid tumors of any type, including colorectal cancer