Ganitumab is an injectable medicine that recently received orphan drug designation from the U.S Food and Drug Administration (FDA) for the treatment of Ewing sarcoma, a type of bone cancer.

Ganitumab, now developed by the immuno-oncology start-up company NantCell (a subsidiary of NantWorks), was first produced by Amgen as a pancreatic cancer drug candidate. However, in 2012, the company terminated the late stage pancreatic cancer trial of ganitumab because it showed no significant improvement in patients’ survival.

In addition to Ewing sarcoma, ganitumab also is being investigated for the treatment of rhabdomyosarcoma, advanced solid tumors, and breast cancer when used in combination with other medicines.

How ganitumab works

Ganitumab works by attaching to cancer cells and blocking signals that are important for cell growth.

The drug is a fully human monoclonal antibody designed to attach to and block the insulin-like growth factor 1 receptor (IGF-1R).

IGF-1R is important in the development and progression of many cancers, possibly due to its properties of preventing cancer cells death, which allows those cells to resist chemotherapeutic drugs or radiation therapy.

By preventing the attachment of IGF-1R to its receptors and the subsequent activation of the PI3K/Akt signaling pathway, ganitumab inhibits tumor cell proliferation and induces the death of tumor cells.

Ganitumab in clinical trials

The safety and effectiveness of ganitumab monotherapy in patients with metastatic Ewing family tumors (EFT) were evaluated in an open-label Phase 2 study (NCT00563680). The results of the trial showed that ganitumab was well-tolerated and demonstrated modest, but encouraging, antitumor activity.

A randomized Phase 3 clinical trial (NCT02306161) is currently evaluating the use of ganitumab as an add-on therapy to multi-agent chemotherapy. The trial aims to recruit 330 participants with newly diagnosed metastatic Ewing sarcoma.

The effectiveness of ganitumab, in combination with other therapies, also is being examined in several clinical trials for the treatment of other types of cancer. For example, a Phase 1/2 study (NCT03041701) is evaluating whether combined ganitumab and Sprycel (dasatinib) is safe and able to shrink or slow the growth of tumors in people with rhabdomyosarcoma. That study is recruiting patients.

Another open-label Phase 1 trial (NCT01061788) is assessing the safety, tolerability, and effectiveness of the combined therapy of ganitumab, Afinitor (everolimus) and Vectibix (panitumumab) in patients with advanced solid cancers that did not respond to other treatments or for which no standard therapies exist. This study is also currently recruiting patients.

Finally, a Phase 2 trial (NCT01042379) is recruiting participants to test the combination of ganitumab plus metformin in breast cancer.

Other details

The most common adverse effects associated with the use of ganitumab include fatigue, low platelet counts, high blood sugar, and infusion site reactions.


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