Targeted Drug Therapy for Uterine Sarcomas

Targeted drug therapy uses medicines that target or are directed at proteins on cancer cells that help them grow, spread, and live longer. Research has shown that some uterine sarcomas make certain proteins or have gene changes that can be targeted with specific drugs to destroy cancer cells or slow their growth. Many of these drugs can be taken as pills and their side effects are different from those of chemotherapy (sometimes less severe).

Some targeted drugs, for example, monoclonal antibodies, work in more than one way to control cancer cells and may also be considered immunotherapy because they boost the immune system.

Kinase inhibitors

Kinases are proteins in the cell (or on its surface) that normally send signals to the rest of the cell, such as telling the cell to grow. Drugs that block certain kinases (kinase inhibitors) can help stop or slow the growth of some tumors.

Pazopanib (Votrient) is a targeted drug that might be used to treat a leiomyosarcoma that has spread or come back after treatment. 

Side effects include high blood pressure, diarrhea, nausea, headache, vomiting, and skin changes. More serious side effects can include bleeding in the lung or getting a hole in the bowel.

Targeted therapy is used to treat many types of cancer, but it's still new for treating uterine sarcoma.

TRK inhibitors

Some uterine sarcomas have changes in one of the NTRK genes. This gene change causes them to make abnormal TRK proteins, which can lead to abnormal cell growth and cancer. 

Larotrectinib (Vitrakvi) and entrectinib (Rozlytrek) are drugs that target the TRK proteins. These drugs can be used to treat advanced or recurrent (cancer that has come back) uterine sarcomas with NTRK gene changes.

These drugs are taken as pills, once or twice a day.

Common side effects of TRK inhibitors include muscle and joint pain, cough, dizziness, fatigue, nausea, vomiting, constipation, fever, abdominal pain, and diarrhea.

PARP inhibitors

Olaparib (Lynparza), rucaparib (Rubraca), and niraparib (Zejula) are PARP inhibitors. By blocking the PARP pathway, these drugs make it very hard for tumor cells with an abnormal BRCA gene to repair damaged DNA, which often leads to the death of these cells. If you are not known to have a BRCA mutation, your doctor might test your blood or saliva to be sure you have one before starting treatment with one of these drugs.

Less than 10% of women with uterine leiomyosarcomas will have a BRCA2 mutation. Those that do might benefit from one of these PARP inhibitors.

Olaparib (Lynparza), rucaparib (Rubraca), and niraparib (Zejula) might be used to treat advanced uterine leiomyosarcomas, typically after chemotherapy has been tried. 

All of these drugs are taken daily by mouth, as pills or capsules.

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.

Alektiar KM, Abu-Rustum NR, and Fleming GF. Chapter 75- Cancer of the Uterine Body. In: DeVita VT, Lawrence TS, Rosenberg SA, eds. DeVita, Hellman, and Rosenberg’s Cancer: Principles and Practice of Oncology. 11th ed. Philadelphia, Pa: Lippincott Williams & Wilkins; 2019.

Baek MH, Park JY, Rhim CC, et al. Investigation of New Therapeutic Targets in Undifferentiated Endometrial Sarcoma. Gynecol Obstet Invest. 2017;82(4):329-339.

Cuppens T, Annibali D, Coosemans A, et al. Potential Targets' Analysis Reveals Dual PI3K/mTOR Pathway Inhibition as a Promising Therapeutic Strategy for Uterine Leiomyosarcomas-an ENITEC Group Initiative. Clin Cancer Res. 2017;23(5):1274-1285. 

Hensley ML, Chavan SS, Solit DB, et al. Genomic Landscape of Uterine Sarcomas Defined Through Prospective Clinical Sequencing. Clin Cancer Res. 2020;26(14):3881-3888. doi:10.1158/1078-0432.CCR-19-3959.

Hensley ML and Leitao MM. Treatment and prognosis of uterine leiomyosarcoma. In: Chakrabarti A and Vora SR, eds. UpToDate. Waltham, Mass.: UpToDate, 2021. https://www.uptodate.com. Accessed June 13, 2022.

National Comprehensive Cancer Network (NCCN). NCCN Clinical Practice Guidelines in Oncology. Uterine Neoplasms, Version 1.2022 – November 4, 2021. Accessed at https://www.nccn.org/professionals/physician_gls/pdf/uterine.pdf on June 13, 2022.

Ricci S, Stone RL, Fader AN. Uterine leiomyosarcoma: Epidemiology, contemporary treatment 
strategies and the impact of uterine morcellation. Gynecol Oncol. 2017;145(1):208-216.

References

Alektiar KM, Abu-Rustum NR, and Fleming GF. Chapter 75- Cancer of the Uterine Body. In: DeVita VT, Lawrence TS, Rosenberg SA, eds. DeVita, Hellman, and Rosenberg’s Cancer: Principles and Practice of Oncology. 11th ed. Philadelphia, Pa: Lippincott Williams & Wilkins; 2019.

Baek MH, Park JY, Rhim CC, et al. Investigation of New Therapeutic Targets in Undifferentiated Endometrial Sarcoma. Gynecol Obstet Invest. 2017;82(4):329-339.

Cuppens T, Annibali D, Coosemans A, et al. Potential Targets' Analysis Reveals Dual PI3K/mTOR Pathway Inhibition as a Promising Therapeutic Strategy for Uterine Leiomyosarcomas-an ENITEC Group Initiative. Clin Cancer Res. 2017;23(5):1274-1285. 

Hensley ML, Chavan SS, Solit DB, et al. Genomic Landscape of Uterine Sarcomas Defined Through Prospective Clinical Sequencing. Clin Cancer Res. 2020;26(14):3881-3888. doi:10.1158/1078-0432.CCR-19-3959.

Hensley ML and Leitao MM. Treatment and prognosis of uterine leiomyosarcoma. In: Chakrabarti A and Vora SR, eds. UpToDate. Waltham, Mass.: UpToDate, 2021. https://www.uptodate.com. Accessed June 13, 2022.

National Comprehensive Cancer Network (NCCN). NCCN Clinical Practice Guidelines in Oncology. Uterine Neoplasms, Version 1.2022 – November 4, 2021. Accessed at https://www.nccn.org/professionals/physician_gls/pdf/uterine.pdf on June 13, 2022.

Ricci S, Stone RL, Fader AN. Uterine leiomyosarcoma: Epidemiology, contemporary treatment 
strategies and the impact of uterine morcellation. Gynecol Oncol. 2017;145(1):208-216.

Last Revised: September 20, 2022

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