Dying Dad Pleads for Unapproved Cancer Drug

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"This kind of medicine is not the kind of medicine that if it works, it works for a few weeks and stops working," said Wolchok, a melanoma specialist at Memorial Sloan-Kettering Cancer Center in New York. "If medications like this work, they tend to benefit people for months or years. Some people might even be 'cured.'"

Auden said he was told that in order to be accepted into a clinical trial for the anti-PD-1 drug, he would need to have either no brain tumors or brain tumors that were at least no longer growing.

Wolchok explained that the drug had the potential to cause brain swelling in people with existing brain disease.

Since Auden had brain tumors, he and his oncologist, who declined to be interviewed for this story, worked to stabilize the tumors using a combination of drugs and other therapies. After months of monitoring his brain scans, Auden got good news in July: His tumors had not grown, and he qualified for the Merck trial.

They high-fived in the doctor's office.

But hours later, Auden experienced abdominal pain and sent his doctor a message. The doctor said to go straight to an emergency room, because there was a possibility he had a perforated intestine.

"Sure enough, I did," Auden said. "That instantly disqualifies you for the trial."

Auden's wife, of course, had other plans, hatched during those sleepless nights at 3 a.m.

Maybe he could become an individual case study under compassionate use rules, which give people access to experimental drugs even if they don't fit into clinical trials, the Audens thought.

That's when the "Save Locky's Dad" campaign took hold, launching two weeks ago with the goal of reaching 150,000 signatures. Once it attained that goal, the Audens upped it to 200,000.

"It's really surpassed anything that we could have imagined," Amy Auden said.

Emails poured into the Audens' inbox from people wishing them luck and sharing stories of how their relatives used the drugs and had positive results.

Bristol-Myers Squibb told ABCNews.com it couldn't comment on Auden's case but couldn't yet allow the drug to be used outside clinical trials, citing safety considerations.

"When you've been given a terminal diagnosis, you're prepared to accept a drug that's 50 percent effective," Auden said. "Safety concerns don't really figure in the same way."

In a statement to ABCNews.com, Merck said it was working to make the drug available to patients outside clinical trials, but its supply was limited. All available supplies, it said, were being used in clinical trials.

"Merck cares deeply about helping patients who face significant treatment challenges, and we are dedicated to making our medicines available to all patients as soon as possible," a Merck spokesman said in the statement. "That is why we have moved our PD-1 program forward faster than any other research program in nearly 20 years, and we have accelerated production."

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