Dad Pleading for Unapproved Cancer Drug Dies

Nov. 25, 2013— -- Nick Auden, the 41-year-old Denver man who had been pleading with drug companies for one last chance to beat his terminal cancer, has died.

"He never stopped fighting and never stopped believing he would conquer this absolutely horrendous disease," Auden's wife, Amy Auden, said in a statement following his death on Nov. 22 at home.

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Auden, a father of three, was admitted into a clinical trial to get what he called a "wonder drug" to treat his stage 4 melanoma over the summer, but hours later, he suffered a complication and was immediately disqualified from the trial. What's more, the drug companies that make the drug wouldn't allow him to take it on his own.

So after lying awake at 3 a.m., Amy started "Save Locky's Dad," an online petition and campaign named for Auden's oldest son, 7-year-old Locky. The goal was to get the companies to him the drug under "compassionate use" or "expanded access" programs, which allow still unapproved drugs to be used outside clinical trials.

They gathered more than 520,000 Change.org signatures, but the drug companies wouldn't budge. Two drug companies -- Merck and Bristol-Myers Squibb -- make versions of the so-called anti-PD-1 drug, which teaches the immune system to attack cancer, but they both declined to provide it to Auden outside a clinical trial. Bristol-Myers Squibb cited safety concerns, and Merck said it just didn't have enough of the drug to give it to him.

Auden's story started in March 2010, when he had a cancerous mole removed. Although it put him at risk for more skin cancer, he continued to live an active life, running, biking and hiking.

But in September 2011, Auden said his doctors sat him down and told him the cancer had returned and had spread throughout his body. The official diagnosis was stage 4 melanoma.

"Some people survive, 90-odd percent don't," he said. "There's no doubt that was tough news. I had trouble not being emotional about it every time I thought of the concept of not being there to watch the kids grow up."

Auden's wife was pregnant with the couple's third child when doctors told her husband that his median life expectancy was between six and nine months. He survived more than two years with the help of radiation and other experimental treatments, but time was running out.

When he learned about the anti-PD-1 drugs and their ability to treat melanoma, he got excited. Studies of Merck's version of the drug found that 38 percent of participants in a clinical trial for patients with melanoma saw tumors shrink. Of those who took the highest doses of the drug, 52 percent experienced tumor shrinkage.

Dr. Jedd Wolchok, an oncologist who has not met or treated Auden but has corresponded with him through email, told ABCNews.com that immune cells typically don't attack cancer in a meaningful way because of a kind of natural brake function called PD-1. But the new anti-PD-1 drugs cancel out that brake and allow the immune cells to attack the cancer.

Although there are currently no anti-PD-1 drugs in "compassionate use" trials -- trials for individuals who don't qualify for clinical trials but still want the drug -- Wolchok said there was chance the drug could offer Auden long-term benefits.

"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 Auden 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, she said she thought.

"I could not sleep," Amy Auden told ABCNews.com. "I was lying awake at night thinking, I can't just lie here and do nothing."

"Save Locky's Dad" launched in September.

"I want my dad to get the PD-1 drug because then I can do the things I like to do with him all the time," Locky says in a video on the "Save Locky's Dad" website, flashing a smile that's missing two front teeth between shots of the two playing Frisbee.

But by October, the companies still hadn't granted Auden access to the drug, and he needed surgery to remove two tumors that had grown in his brain since the site's launch.

"His oncologist says he must have the drug now to survive," his wife, Amy Auden, told ABCNews.com in an email that week. "The tumors do not stop growing throughout this process."

The FDA gave Merck's drug "breakthrough therapy" status in April, allowing the pharmaceutical company to speed up clinical trials in the hopes of approving it, and making it more widely available, sooner, according to company filings.

But that meant it would need to increase its supply of the drug for the ramped-up clinical trials, which Merck spokesman Steven Cragle said was no easy feat. Because the drug is made from mammalian cells, it takes time to literally grow the supply in a lab. As such, Merck only has enough for the clinical trials.

"Nature is working against us," Cragle said. "It's hard to conceptualize why we can't just 'bake a bigger loaf of bread,' so to speak."

He added that Merck is working to make enough of the drug to develop an expanded access program -- which would benefit patients like Auden, who don't fit into clinical trials -- but there's no timeline for it yet.

"Not everyone has as short a window as I," Auden said in October. "Why can't they supply me now rather than me missing by a couple of months? Imagine Amy explaining that to the kids ..."

Although Auden was the highest profile patient seeking the anti-PD-1 drug outside of a clinical trial, he was not the only one, Cragle said.

In a statement on the "Save Locky's Dad" website following Auden's death, the family declared "this mission is not over."

"In the end, Nick's death beams a spotlight on a glaring need for change in compassionate access practices for life-saving drugs in late-stage investigational trials," they said in a statement. " More on this, when the time is right."