Backed by Immunotherapy, New Approach to Treating Rare Cancer Shown to be Effective for Some Patients

A national multicenter phase II trial has found that a combination of nivolumab and ipilimumab is safe and effective for some sarcoma patients

Jun 1, 2017

New York


An immunotherapy drug used in combination with a traditional cancer drug could be an effective approach to treating a rare cancer known as sarcoma, according to a new study. Co-authored by Dr. Gary Schwartz, chief of hematology/oncology at NewYork-Presbyterian/Columbia University Medical Center, the study found that a combination of nivolumab, a common cancer drug, and ipilimumab, an immunotherapy drug, is safe and effective for sarcoma patients who have tried all other options.

Sarcoma is a deadly and complex form of cancer, with more than 60 different subtypes. While traditional treatments are often effective if the cancer is detected early, five-year survival rates plummet if the cancer spreads throughout the body (metastasizes).

“This is the first time ever that immunotherapy has been shown to work in sarcoma, a rare cancer that is a major focus of basic and clinical research at our institution,” said Dr. Schwartz. “Before this trial, we had little hope that immunotherapy would ever work in this cancer, but the new data looks extraordinary for some patients who had failed all treatment and for whom no other therapies were available. We’ve seen some dramatic clinical responses to this approach.”

The phase II, multicenter trial came out of the ALLIANCE for Clinical Trials in Oncology rare cancer group, a national committee chaired by Dr. Schwartz. Researchers compared a group of 38 patients treated with nivolumab with a group of 38 patients treated with a combination of nivolumab and ipilimumab – all patients in the study had exhausted all other treatment options. The group who received the combination therapy demonstrated a greater response rate than the group treated with just the traditional drug, as well as trends favoring progression-free response rate and overall survival rate. While a number of patients in the combination therapy group dropped out due to treatment-related adverse events, the combination therapy was found to be safe.

Dr. Schwartz believes that more research is needed to understand why the new treatment was so effective for some patients, and if there are certain types of sarcomas that respond better to the immunotherapy-backed treatment. NewYork-Presbyterian/Columbia is home to one of the few laboratories in the country that focuses on new drug development with immunotherapy in this cancer. The results of this trial will likely inform the institutions approach to developing future therapies.

Dr. Schwartz will discuss the study at the 2017 American Society of Clinical Oncology meeting in Chicago. For more information about immunotherapy at NewYork-Presbyterian, visit

Media Contact:

Office of Public Affairs