Targeted Therapy for Endometrial Cancer

Targeted therapy is treatment with drugs that are made to target certain changes in the cancer cells. Some people group them with chemotherapy, but they aren't the same. Targeted therapies leave most healthy cells alone. They often cause fewer and different side effects than chemo.

Targeted therapy is used to treat many types of cancer, but it's very new in the treatment of certain types of endometrial cancer. Only a few of these drugs are in use at this time, and are only given as part of a clinical trial, but many more are being studied. As doctors learn more about endometrial cancer cells, targeted therapy could become an important part of treatment. Today, these drugs are mostly used to treat high-risk endometrial cancers and those that have spread (metastasized) or come back (recurred) after treatment.  

Bevacizumab

Bevacizumab (Avastin®) belongs to a class of drugs called angiogenesis inhibitors. For cancers to grow and spread, they need to make new blood vessels to nourish themselves (a process called angiogenesis). This drug attaches to a protein called VEGF (which signals new blood vessels to form) and slows or stops cancer growth.

Bevacizumab is often given along with chemotherapy, but it can be given alone. It's given as an infusion into the vein (IV) every 2 to 3 weeks.

Common side effects include high blood pressure, tiredness, bleeding, low white blood cell counts, headaches, mouth sores, loss of appetite, and diarrhea. Rare, but possibly serious side effects include blood clots, severe bleeding, slow wound healing, holes forming in the colon (perforations), and the formation of abnormal connections between the bowel and the skin or bladder (fistulas). If a perforation or fistula forms, it can lead to severe infection and surgery may be needed.

mTOR inhibitors

These drugs block a cell protein known as mTOR, which normally helps cells grow and divide into new cells. These drugs might be given alone or added to chemo or hormone therapy to treat advanced (higher stage ) endometrial cancers, or those that come back after treatment.

Everolimus (Afinitor®) is taken as a pill once a day.

Common side effects include mouth sores, diarrhea, nausea, feeling weak or tired, shortness of breath, and cough. Everolimus can also cause low blood counts, increase blood lipids (cholesterol and triglycerides), and raise your blood sugar, so your doctor will check your blood work often while you are taking this drug. 

Temsirolimus (Torisel®) is given as an intravenous (IV) infusion, typically once a week. It can be given alone.

The most common side effects of this drug are skin rash, weakness, mouth sores, diarrhea, nausea, loss of appetite, fluid build-up in the face or legs, and increases in blood sugar and cholesterol levels. Rarely, more serious side effects have been reported.

Ridaforolimus can be taken as a pill or given as an IV infusion. So far, is has been studied in women with endometrial cancer that has spread or come back after treatment.

Common side effects of ridaforolimus include diarrhea, mouth sores, loss of appetite, weakness, nausea, high blood sugars, and vomiting.

More information about targeted therapy

To learn more about how targeted drugs are used to treat cancer, see Targeted Cancer Therapy.

To learn about some of the side effects listed here and how to manage them, see Managing Cancer-related Side Effects.

The American Cancer Society medical and editorial content team

Our team is made up of doctors and oncology certified nurses with deep knowledge of cancer care as well as journalists, editors, and translators with extensive experience in medical writing.

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Chellappan DK, Leng KH, Jia LJ, et al. The role of bevacizumab on tumour angiogenesis and in the management of gynaecological cancers: A review. Biomed Pharmacother. 2018;102:1127-1144.

Kassem L, Abdel-Rahman O. Targeting mTOR pathway in gynecological malignancies: Biological rationale and systematic review of published data. Crit Rev Oncol Hematol. 2016;108:1-12.  

McDonald ME, Bender DP.  Endometrial Cancer: Obesity, Genetics, and Targeted Agents. Obstet Gynecol Clin North Am. 2019;46(1):89-105.

National Cancer Institute. Endometrial Cancer Treatment (PDQ®)–Patient Version. April 26, 2018. Accessed at www.cancer.gov/types/uterine/patient/endometrial-treatment-pdq on February 20, 2019.

National Comprehensive Cancer Network, Clinical Practice Guidelines in Oncology (NCCN Guidelines®), Uterine Neoplasms, Version 1.2019 -- October 17, 2018. Accessed at www.nccn.org/professionals/physician_gls/pdf/uterine.pdf on February 20, 2019.

Oza AM, Pignata S, Poveda A, et al. Randomized Phase II Trial of Ridaforolimus in Advanced Endometrial Carcinoma. J Clin Oncol. 2015;33(31):3576-3582.

Last Medical Review: March 27, 2019 Last Revised: March 27, 2019

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