Everolimus tablets, sold under the brand name Afinitor by Novartis, are approved by the U.S. Food and Drug Administration (FDA) to treat many breast cancers and advanced renal cell carcinomas, among others.

Everolimus is also being investigated as a treatment for myeloma.

How does everolimus work?

Myeloma is a type of cancer that affects immune cells called B-cells. The cancer starts in the bone marrow, where blood cells are made, but often spreads from one bone to another, which is why it’s often called multiple myeloma.

In healthy people, the growth and division of cells is tightly controlled. In the case of cancer, however, the signaling that normally regulates these processes is disrupted, and signaling pathways that increase cell growth and division are upregulated.

Everolimus is a small molecule that binds tightly to a protein called FK506 binding protein-12 (FKBP-12). Once bound to FKBP-12, the complex inhibits a signaling protein called mTOR, a central regulator of cell metabolism, growth, proliferation, and survival. The result is the disruption of all these biological events, leading ultimately to cell death.

Everolimus also reduces the production of a growth factor called vascular endothelial growth factor (VEGF). VEGF contributes to the formation of new blood vessels (a biological event called angiogenesis) around cancer cells, which is needed for nutrient supply. By reducing VEGF production, everolimus is thought to reduce angiogenesis and limit the growth of cancer.

Everolimus in clinical trials

A small open-label, multicenter, Phase 1 clinical trial (Eudra CT 2006-002675-41) was conducted to evaluate escalating doses of everolimus, taken once a day, in myeloma patients. The primary objective was to determine the maximum tolerated dose of everolimus. The secondary objectives were to assess the tolerability and efficacy of the treatment.

Results of the trial were published in the journal Haematologica. Patients in each treatment group received higher dosages of everolimus than the previous group. Of the 17 patients enrolled, one patient achieved partial remission, one had a partial response, and eight were stable for up to nine months. However, the median time to progression was 90 days, and so the authors suggested that a monotherapy approach may not be sufficient for treatment. One serious adverse event, a case of pneumonia, was reported during the trial.

A Phase 1/2 trial (NCT00918333) is studying the combination of Farydak (panobinostat, another treatment approved for myeloma) and everolimus in patients with recurrent myeloma or lymphoma. The first part of the study aims to establish the maximum safe dose of the two treatments, while in the second half, the treatment combination will be evaluated for safety and efficacy. The study is ongoing but no longer enrolling participants.

Other information

Everolimus can cause side effects, including bloating or swelling in the hands and feet, weight loss or gain, cough, diarrhea, fever, stomach pain, and back pain.

The treatment can also lead to the suppression of the immune system, making patients more susceptible to infections.


Last updated: Jan. 30, 2020


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