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Melanoma drug a seismic shift in cancer

Progress in cancer treatment usually creeps forward. While more people are living longer with more kinds of cancer than ever, the better treatments are often incremental improvements. But every five or 10 years a new medication arrives that totally shifts the paradigm, allowing thousands to live who would have otherwise died.

Researchers today set off such a seismic shift for the treatment of advanced melanoma, the often-deadly skin cancer for which few treatments worked. The results came out at annual meeting of the American Society of Clinical Oncology (ASCO) in Chicago — and were published simultaneously in the New England Journal of Medicine.

The new drug is known by the tongue-twisting name ipilimumab. Patients and doctors often call it “ipi” for short. Soon, Bristol-Myers Squibb — which bought the drug from a biotechnology company in 2009 — will give it an easy-to-pronounce trade name. The pharmaceutical giant will also apply for permission from the Food and Drug Administration to market it by the end of the year, perhaps even sooner.

In the double-blinded placebo-controlled trial (the highest standard of clinical research), 676 patients with highly advanced melanoma (stage 4) got either the drug or another treatment regarded as mostly ineffective. Those getting "ipi" lived on average for 10 months compared to six months for those not getting it.

A 67 percent survival improvement is significant, but that few months of additional survival mask what really happened with the drug. In 20 percent to 30 percent of patients getting the drug, the response was spectacular — the advanced cancer that had spread through their bodies either dramatically reduced in size, or simply disappeared. The other 70 percent to 80 percent of patients had no response at all. 

At this point, doctors are not able to predict which patients will respond. But even with those numbers, the drug should allow additional survival for up to 30 percent of the almost 9,000 Americans a year who are now dying of advanced melanoma.

More potent effect possible?

Ipilimumab is a laboratory-made protein called an antibody. It attaches to and stimulates certain white blood cells (called CD8 killer cells) which in turn destroy the melanoma when the drug works. “Ipi” does not cause the usual nausea and hair loss associated with chemotherapy.

However, it often has a side effect that can be much worse. Because it hyper-stimulates the immune system, it can set off autoimmune diseases, including rheumatoid arthritis and colitis. But doctors have found they can treat those conditions, usually with steroids, and it does not disrupt the anti-cancer action of the new drug.

What happens next with this drug? The people in this trial had very late-stage disease. New research is currently underway to see if the drug given soon after initial surgery can have a more potent effect, possibly elevating that 20 percent to 30 percent of patients who experience dramatic reduction in tumors. In addition, very early research indicates the drug might be useful against other cancers.

The drug has already shifted the way doctors and patients think about melanoma treatment, says Dr. Steven Day, lead researcher of the study and head of The Angeles Clinic and Research Institute in Santa Monica, Calif.

“Patients are energized, our waiting rooms are becoming fuller ... because patients are living longer," he said. "Melanoma has given cancer a bad name and has been the graveyard of many new drugs. So, this is producing tremendous excitement both for the clinical researchers who are in the battleground of this horrible disease, as well as the patients and their families.”

Until the drug wins FDA approval, Bristol-Myers Squibb is offering it on a compassionate access basis to people with advanced melanoma. More than 1,600 have gotten it. You can learn more about that program at the government's Clinical Trials web site.