“POSITIVE RESULTS”

It’s a problem that Merck and other companies developing new immunotherapy drugs will have to solve. The drugs, including Merck’s MK-3475 and Bristol-Myers’ nivolumab, help the immune system fight cancer cells by disabling a protein called “programmed death 1” or PD-1 that acts as a brake on the body’s ability to detect them.

Andrew Baum, an analyst at Citi, estimates treatments that coax the immune system to target cancer will become the backbone therapy for up to 60 percent of cancers over the next decade, generating $35 billion in annual sales.

Dr. Antoni Ribas at the University of California, Los Angeles says the immunotherapies are showing so much promise that they, like Zelboraf, raise doubts over whether randomized trials are needed. He believes they could be approved in the United States on the basis of a single-arm trial. Yet Merck has started enrolling patients in a study where patients will be randomized to get the new treatment or existing chemotherapy.

One patient who has already put himself forward for MK-3475 is Stew Scannell, 65, head of operations at global defense company Northrop Grumman in Oklahoma City. Scannell, who served a couple of tours in Vietnam and spent several years in various deserts testing helicopters, figures his melanoma may be the result of cumulative sun damage.

When his doctors were talking about buying him another couple of months, he decided to do his own research. He started MK-3475 shortly after his first meeting with Ribas, in April 2012.

Several of his tumors have disappeared. At his last scan in April, there was no sign of any tumor in his brain. In Merck’s trial, the most common side effects of the drug include fatigue, fevers, skin rash, loss of skin color and muscle weakness. But so far, Scannell has had none. “I really haven’t missed a step. I’ve continued working. The radiation was difficult. But the marvelous thing about the immunotherapy is no side effects. No lethargy. No loss of appetite. No anything.”

South African melanoma patient Christina Chrysostomou, 45, would be more than happy to see the end of randomized trials when a treatment has shown early promise.

After her cancer got worse on Bristol-Myers’ immunotherapy Yervoy, she and her husband and 8-year-old son headed for the United States in the hopes of trying one of the new anti-PD-1 drugs.

But when she arrived in late June, Merck’s Phase I trial had closed, and she was told she would have to take her chances in a randomized test. Luckily for her, a spot opened up in a non-randomized Phase I study and she is now getting MK-3475 - but she feels for others less fortunate.

“It’s really hard knowing there is something out there that could possibly help and having to go through a gamble and maybe not even get that,” she said.

(Edited by Sara Ledwith, Richard Woods and Simon Robinson)

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By Julie Steenhuysen and Ben Hirschler