Adding GM-CSF to Ipilimumab Lengthens the Lives of Patients with Metastatic Melanoma

ASCO Annual Meeting
June 1, 2013

In a recent study, combining a high dose of ipilimumab (Yervoy) with GM-CSF (Sargramostim, Leukine) helped patients with metastatic melanoma live longer than those who received ipilimumab alone. Both ipilimumab and GM-CSF are types of immunotherapy, a treatment designed to boost the body’s natural defenses to fight the cancer. Specifically, ipilimumab works to take the brakes off the immune system by targeting CTLA-4, a protein found on the surface of tumor cells that keeps the immune system from destroying the cancer. GM-CSF, on the other hand, is a growth factor that the body produces to help increase the number and function of white blood cells. It is commonly used to boost white blood cell counts after chemotherapy or stem cell transplantation.

This study included 245 patients with metastatic melanoma (melanoma that has spread to other parts of the body) who had received no more than one other treatment before the study began. As part of the study, the volunteers received either ipilimumab plus GM-CSF or ipilimumab alone.

Researchers found more than two-thirds of patients (69%) who received the drug combination were alive after one year versus half (53%) of those treated with ipilimumab alone, a significant advance for metastatic melanoma. Interestingly, adding GM-CSF also decreased some of the serious side effects of ipilimumab, especially those involving the lungs and gastrointestinal tract.

What this means for patients

“This study provides another important sign that immunotherapy can be important for patients with advanced melanoma,” said lead author F. Stephen Hodi, MD, Associate Professor of Medicine at Dana-Farber Cancer Institute in Boston, Massachusetts. Currently, ipilimumab is a standard treatment option for advanced melanoma in many countries; however, this study used a higher dose of ipilimumab than is currently approved by the U.S. Food and Drug Administration (FDA). Additionally, GM-CSF is not currently approved by the FDA to treat patients with melanoma. As a result, this drug combination is only available through clinical trials at this time. If you are interested in participating in a clinical trial, talk with your doctor for more information.

Dr. Hodi was a recipient of a Conquer Cancer Foundation of ASCO Young Investigator Award in 1998.

Questions to Ask Your Doctor

  • What stage of melanoma do I have? What does this mean?
  • What are my treatment options?
  • What clinical trials are open to me?

For More Information

Guide to Melanoma

Understanding Immunotherapy