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What is everolimus used for and how does it work?

Medically reviewed by Carmen Pope, BPharm. Last updated on May 22, 2024.

Official answer

  • Everolimus may be used to treat certain cancers, neuroendocrine tumors, seizures or other conditions associated with TSC, and organ rejection after a kidney or liver transplant, depending on the brand.
  • Everolimus is an mTOR inhibitor that is also a macrolide immunosuppressant.
  • It works by preventing the actions of mTOR, which plays a vital role in cell growth and proliferation.
  • Everolimus can slow or stop the growth of certain cells and prevent tumors from growing their own blood vessels.
  • Everolimus can also reduce the volume of lipomas and prevent the immune system from rejecting transplanted organs.

Everolimus is an oral medication that, depending on the brand, may be used to treat certain types of cancer or to suppress the immune system.

Everolimus is approved to treat:

  • Advanced breast cancer in postmenopausal women who have taken letrozole or anastrozole but the cancer has returned. Usually given in combination with exemestane (Afinitor only)
  • Certain advanced neuroendocrine tumors of pancreatic, gastrointestinal or lung origin (Afinitor only)
  • Advanced renal cell carcinoma (RCC) that has not responded to sunitinib or sorafenib (Afinitor only)
  • Tuberous sclerosis complex (TSC)-associated partial-onset seizures in adults and children aged two or older (Afinitor Disperz only). TSC is a genetic disorder
  • TSC-associated renal angiomyolipoma that does not require immediate surgery (Afinitor only)
  • Subependymal giant cell astrocytoma (SEGA) associated with TSC in adults and children aged one year and older that requires treatment but which can’t be removed surgically
  • Organ rejection after a kidney or liver transplant (Zortress only).

Everolimus may also be used off-label for other types of cancer, such as carcinoid tumors or Hodgkin lymphoma, lung or heart transplantation, or Waldenström macroglobulinemia.

Everolimus may be referred to as a chemotherapy medicine (cancer medication) or an immunosuppressant agent.

How does everolimus work?

Everolimus works by slowing or stopping the growth of cancer or tumor cells, or other growths.

It belongs to the class of medicines called the mechanistic target of rapamycin (mTOR) inhibitors. It may also be called a macrolide immunosuppressant because its structure includes a macrolide ring.

mTOR is a type of enzyme called a kinase that assists with the transfer of a phosphate group from ATP (adenosine triphosphate),which is the energy-carrying molecule found in the cells of all living things to other molecules. mTOR plays a vital role in regulating cell growth and proliferation. This means that mTOR is a good target for conditions, such as cancer, that are characterized by extensive cell proliferation.

mTOR inhibitors, such as everolimus, inhibit mTOR which is more active in certain cancers. This reduces cell proliferation, slowing the growth of cancer cells or other overly prolific cells. Everolimus also prevents tumors from growing their own blood vessels.

mTOR inhibitors can reduce the volume of lipomas – these are lumps under the skin that occur due to an overgrowth of fat cells. Angiomyolipomas (AML) occur in people with TSC and are lipomas that occur within the kidneys. Everolimus can reduce the growth of these.

When used for organ transplants, the Zortress brand of everolimus reduces the activity of the body’s immune system to stop it from fighting the transplanted organ. This is because our immune system can reject a transplanted organ such as a liver or a kidney because it perceives the new organ as an invader. Zortress is used together with cyclosporine, steroids, and other medications.

The benefits of mTOR inhibitors are enhanced when they are combined with other chemotherapy agents.


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