Biomarkers, targeted and immunotherapies for endometrial cancer

This section covers the following topics:

• Tumor biomarker testing for treatment selection 
• Immunotherapy
• Targeted therapy

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 endometrial 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 endometrial cancer may respond well to a type of immunotherapy known as an immune checkpoint inhibitor. 
  • People with advanced, recurrent endometrial cancer that is not MSI-H, may benefit from a combination of the targeted therapy, Lenvima (lenvatinib) and the immunotherapy agent Keytruda.
• Examples of additional biomarker tests used in endometrial cancer include:
  • A rare type of endometrial cancer—known as a uterine sarcoma—may have a genetic change called an NTRK fusion, which can be found on tumor testing. Endometrial with an NTRK fusion may benefit from the targeted therapy Vitrakvi (larotrectinib).
  • A biomarker known as an NTRK fusion is rare in colorectal cancer. 
  • Estrogen receptor testing is used for advanced and recurrent endometrial cancers. 
  • Her2neu testing is used to find advanced or recurrent endometrial cancers that may respond to Herceptin.
  • Additional tumor biomarker testing may help identify people who are elegible for certain clinical trials. 

Immunotherapies 

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

• Keytruda (pembrolizumab) is FDA approved to treat tumors that have MSI-H or dMMR. It can also be used along with the targeted drug lenvatinib in women with advanced endometrial cancers that are not dMMR or MSI-H, usually after other treatments have been tried.

Targeted therapies

Targeted therapy is still fairly new in the treatment of endometrial cancer. Currently, these agents are only prescribed if the cancer has recurred or as part of a clinical trial. Targeted therapies for endometrial cancer include:

• Vitrakvi (larotrectinib) is approved for treatment of endometrial 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 a rare type of uterine cancer known as uterine sarcoma.
• Lenvima (lenvatinib) helps block tumors from forming new blood vessels. Lenvima can be used along with the immunotherapy drug Keytruda to treat some advanced endometrial cancers, typically after at least one other drug treatment has been tried.
• Afinitor (everolimus) is a type of targeted therapy known as an mTOR inhibitor that has been used (off label) to treat some people with advanced endometrial cancer. Afinitor does not have FDA approval for use in endometrial cancer. 
• Avastin (bevacizumab) helps block tumors from forming new blood vessels. Avastin does not have FDA approval for use in endometrial cancer. 

Table of targeted and immunotherapies for endometrial cancer