Steven A. Rosenberg

In 1985, immunotherapy joined the pantheon of mainstream therapies—surgery, radiation and chemotherapy—that doctors can use to successfully treat patients with cancer.

This unique approach was the brainchild of Dr. Steven Rosenberg, a surgeon and researcher who has spent 40 years as chief of surgery at the National Cancer Institute (NCI) developing treatments that seek to harness the power of the immune system to fight cancer.

Immunotherapy uses the body’s own properties or treatments made in a laboratory to get the immune system to attack cancer cells, eliminating them or by stopping or slowing their growth.

Since Rosenberg introduced the first immunotherapy treatment, interleukin 2 (IL-2), and demonstrated its effectiveness at reducing tumors, millions of patients have lived longer and experienced an improved quality of life. He and hundreds of researchers—many of them graduates of Rosenberg’s program at the NCI—have gone on to discover variations of immunotherapies to fight previously incurable cancers.

“He is a national treasure,” said Dr. Michael Atkins, deputy director of the Georgetown-Lombardi Comprehensive Cancer Center in Washington, D.C. “He is fundamental to the success of immunotherapy. Without him, this program would not have happened.”

“He’s viewed as the leader in this field,” said Dr. Harold Varmus, who until recently led the NCI. Noting that Rosenberg began work on immunotherapy in the 1970s when few believed in the field’s prospects, Varmus added, “What really deserves credit here is his willingness to stick with a difficult kind of therapy and keep people aware that he had occasional successes, and that with new approaches there could be even more successes.”

Rosenberg began his professional life as a surgeon who operated to remove cancers. But if the cancer spread, surgery was not effective. “That got me very interested in whether or not the body’s own wisdom, the body’s own immune system, could treat cancer better than any outside force could,” Rosenberg said.

It took many years of research and clinical trials with varying levels of success before he was able to develop the first effective immune-based treatment, a result that was first published in 1985 in the New England Journal of Medicine.

Rosenberg continued leading research and clinical trials, and in 1988 developed adoptive cell therapy, which involves identifying and removing a cancer patient’s lymphocytes with anti-tumor activity, expanding and growing these cells in the lab, and then infusing them back into the patient.

He and his team at NCI, part of the National Institutes of Health, were the first to genetically modify lymphocytes, the normal immune cells in the body, and convert them into cells that can fight the cancer.

“That was the first effective immunotherapy using gene therapy,” Rosenberg said. “That is becoming a common treatment for patients with lymphomas because we can put in receptors that can recognize the lymphoma. We are in a period now of enormous change.”

According to Rosenberg, about 20 to 25 percent of patients with metastatic melanoma cancers that spread from the place where it first started to another place in the body can be cured with these immunotherapies. His work was highlighted in the PBS program Emperor of Maladies, the History of Cancer, shown on PBS this past spring, and in 2015 he was awarded the Medal of Honor by the American Cancer Society

“Rosenberg has been an indefatigable advocate for the potential of immunotherapy and has led a team of probably 50-100 investigators, research nurses and support staff at the NCI to focus explicitly on what immunotherapy can do,” said Atkins. “Every step along the way he has had his hands on their work, whether it’s treating patients, selecting patients to be treated, or identifying scientific experiments.”

As part of his continuing research, Rosenberg has “focused on unraveling the way that the immune system actually works, leading to a lot of novel approaches,” Atkins said.

Dr. David Sachs, professor of surgery and immunology at Harvard Medical School, said Rosenberg is “an inspiring teacher and gets people excited about this research.”

“Dr. Rosenberg is a true scientist. He shares his research freely with others so that they can advance as well,” said Sachs.

Rosenberg, his team of researchers at NCI and partners around the world are currently working on checkpoint inhibitors that wake up the immune system. Tumors have the ability to put the immune system to sleep after a while. Checkpoint inhibitors wake the immune system up so that it can work again and attack the cancer and destroy it.

“More than any individual, Steven Rosenberg has been the pioneer in the development of cancer immunotherapy,” said Dr. Nicholas Restifo, a senior investigator at NCI. While he “has recognized the need to compassionately care for patients with metastatic cancer,” Restifo added, “he is clear eyed about the inadequacies of current treatments. He knows more needs to be done and, as in decades past, he is continuing to search for breakthroughs that will prolong and save lives.”