Dad Pleading for Unapproved Cancer Drug Dies/FDA control out of control

[barbara]

By SYDNEY LUPKIN | Good Morning America

http://gma.yahoo.com/dad-pleading-unapproved-cancer-drug-dies-164352557--abc-news-topstories.html

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.

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."

 

[Jeannine]

Isn't that just horrible? However, the FDA has time to write threatening letters to 23andme.com.

 

[barbara]

For those not aware of the controversy about 23andme -


FDA Just Banned 23andMe's DNA Testing Kits, and Users Are Fighting Back
Posted: 11/26/2013

http://www.huffingtonpost.com/berin-szoka/fda-just-banned-23andmes-_b_4339182.html

Since 23andMe launched in 2007, over 500,000 Americans have had their saliva tested to learn more about their ancestry, health risks and genetic predisposition to a wide variety of factors. The price has plummeted ten-fold: just $99 for the home-test kit. The scrappy company was poised to take its product mass-market with marketing campaigns aimed at user numbers into the millions.

Perhaps that's why the Food and Drug Administration finally cracked down on the site. Today, the FDA shut down further sales of the saliva home-test kit, citing the "potential health consequences that could result from false positive or false negative assessments for high-risk indications... For instance, if the BRCA-related risk assessment for breast or ovarian cancer reports a false positive, it could lead a patient to undergo prophylactic surgery, chemoprevention, intensive screening, or other morbidity-inducing actions, while a false negative could result in a failure to recognize an actual risk that may exist." In other words, the FDA think that Americans can't be trusted with more information about their potential health risks because some people might make rash decisions with it. But banning personal genomics isn't the answer.

23andMe is simply doing what the Internet does best: forcing old dogs to learn new tricks. That's what the fight between Uber and taxicab commissions is about. Same for AirBnB and hotel regulators. The only profession slower to change how they do things than doctors is bureaucrats. So the FDA's reaction is understandable -- but misguided.

Those of us who have already used the site know the real problem is that doctors themselves are behind the curve. When 23andMe sent us our results, we followed their advice: we asked our doctor to talk about them. Most doctors didn't know where to begin. But the more of us ask about 23andMe, the more the medical profession is catching up. Slowly but surely, they're brushing up on genomics, taking the time to understand the site, and talking to us about our results and what, if anything, to do about them.

By prompting such dialogue, 23andMe has sparked a revolution in how the medical profession uses genetic information. Change like that rarely, if ever, comes from within an industry; it's almost always driven by people on the outside with new approaches and technologies that disrupt old paradigms -- like the idea that genomics aren't something "ordinary" doctors need to understand or that patients shouldn't be active, informed participants in their healthcare.

We're urging the FDA not to short-circuit this revolution, and have launched a Change.org petition to rally those who agree. Our message is simple: "Please trust us -- and our doctors -- to make responsible use of our own genetic information. Instead of banning new technologies, the FDA should focus on educating doctors and patients about the benefits, and limitations, of genetic testing."

If it takes the FDA as long to sign off on innovative services like 23andMe as it does for prescription drugs (around 8 years), the medical profession will continue dragging its feet, as if genomics and the Internet were only distant tools that might some day be incorporated into medicine. But they're here now, and they'll continue changing medicine for the better. Doctors and patients will both adapt to the new reality of medical care personalized for our genes -- but only if the FDA gets out of the way.

 

[barbara]

No DNA Testing For You, Thanks to the FDA
By Nick GillespieNovember 26th 2013

http://www.thedailybeast.com/articles/2013/11/26/no-dna-testing-for-you-thanks-to-the-fda.html

Personal genetic tests are safe, innovative, and the future of medicine. So why is the most transparent administration ever shutting down a cheap and popular service? Because it can.
In its infinite wisdom, the Food and Drug Administration (FDA) has forbidden the personal genetic testing service 23andMe from soliciting new customers, claiming the company hasn’t proven the validity of its product.

The real reason? Because when it comes to learning about your own goddamn genes, the FDA doesn’t think you can handle the truth. That means the FDA is now officially worse than Oedipus’s parents, Dr. Zaius, and the god of Genesis combined, telling us that there are things that us mere mortals just shouldn’t be allowed to know.

23andMe allows you to get rudimentary information about your genetic makeup, including where your ancestors came from and DNA markers for over 240 different hereditary diseases and conditions (not all of them bad, by the way). Think of it as the H&M version of the haute couture genetic mark-up that Angelina Jolie had done prior to having the proactive mastectomy that she revealed this year.

Good technology tends to win out over time, despite all the attempts by the old guard to stifle it.
23andMe is fun and it’s cool and, as my Reason colleague Ronald Bailey has shown, it is a small but meaningful step toward a glorious future of radically individualized medical interventions and a deeper understanding of the human condition on a fundamental level. Marlo Thomas had no idea just how right she was when she released her Free to Be...You and Me records.

Up until a few days ago, for $99, you could spit in a test tube and send it to 23andMe’s Los Angeles-based labs and get a picture of who you were on a genetic level. The company keeps a constantly growing online database of new studies and information about genetics and illnesses; it also hosts a thriving (and totally voluntary) community of people who can talk among themselves. Unlike your doctor, who is likely to ***** and moan and drag his heels when you ask for a copy of your latest blood test, 23andMe lets you easily access and even download your personal genetic service (PGS) readout. (Some folks have even used the info to make the ultimate statement in personal music.)

Not no more. While the company can keep serving its current clients (full disclosure: I’m one of them, though with an asterisk I’ll explain in a second), it can’t grow its customer base until it jumps through more hoops as laid out by the FDA.. Although the company says it will do what it takes, the smart money is always on government bureaucrats shutting down whatever they want. The ultimate beef, says the government, is that 23andMe is giving away too much information to the unwashed masses without scratching the right bureaucratic backs first:

Your company’s website at www.23andme.com/health (most recently viewed on November 6, 2013) markets the PGS for providing “health reports on 254 diseases and conditions,” including categories such as “carrier status,” “health risks,” and “drug response,” and specifically as a “first step in prevention” that enables users to “take steps toward mitigating serious diseases” such as diabetes, coronary heart disease, and breast cancer. Most of the intended uses for PGS listed on your website, a list that has grown over time, are medical device uses under section 201(h) of the FD&C Act. Most of these uses have not been classified and thus require premarket approval or de novo classification, as FDA has explained to you on numerous occasions.

The FDA is especially worried that folks like you and me will get some news that will literally cause us to hack off our own breasts, remove our own testicles, or become so genetically arrogant that we’ll stop seeing the doctor altogether:

If the BRCA-related risk assessment for breast or ovarian cancer reports a false positive, it could lead a patient to undergo prophylactic surgery, chemoprevention, intensive screening, or other morbidity-inducing actions, while a false negative could result in a failure to recognize an actual risk that may exist.

You got that? The FDA is seriously claiming that you might learn you have a possibility of developing breast cancer and then insist on having the potentially cancerous body part lopped off. And that you'll be able to find a doctor or hospital or taxidermist who will do any or all of that without doing further tests and followups. They don’t provide a single instance of this happening nor do they specify any possible medical world in which this might happen, but that’s enough to shut down 23andMe for the foreseeable future. (The company has issued a short statement about how they will work to meet the FDA’s demands.)

The FDA is now apparently taking policy cues from The End, a 1978 comedy starring Burt Reynolds and Dom DeLuise. In that groaner, Burt is convinced he is going to die soon and sets about offing himself. Spoiler alert: Hilarity doesn’t ensue. It’s a Burt Reynolds-Dom DeLuise showcase after all.

I’m still waiting on the results of my second spit test from 23andMe, which I sent in a couple of weeks ago. I was informed that the saliva I provided in my first sample was so lame that it couldn’t provide a legitimate reading of my DNA—a curious and dispiriting result that made me angry without triggering any action in me other than wanting to drink some water. If and when I learn something potentially dangerous—or potentially uplifting (like most kids, I went through a phase in which I hoped against hope that I would turn out to be adopted rather than the obvious and incontrovertible product of my dear, departed parents)—it will be the start of a new conversation with my doctors and friends.

And regardless of the limits of a $99 DNA test, it will be a more informed, not a more-hysterical conversation revolving around what body parts I need to lose immediately.

But don’t expect the FDA to understand any of that. Peter Huber of the Manhattan Institute, a conservative think tank, has an important new book out called The Cure in the Code: How 20th Century Law is Undermining 21st Century Medicine.. Huber writes that whatever sense current drug-approval procedures once might have had, their day is done. Not only does the incredible amount of time and money – 12 years and $350 million at a minimum – slow down innovation, it’s based on the clearly wrong idea that all humans are the same and will respond the same way to the same drugs.

Given what we already know about small but hugely important variations in individual body chemistry, the FDA’s whole mental map needs to be redrawn. "The search for one-dimensional, very simple correlations - one drug, one clinical effect in all patients - is horrendously obsolete," Huber told me in a recent interview. And the FDA's latest action needs to be understood in that context - it's just one more way in which a government which now not only says we must buy insurance but plans whose contours are dictated by bureaucrats who arbitrarily decide what is best for all of us.

There’s no doubt that over time outfits such as 23andMe will flourish. Individualization and personalization have already arrived everywhere in our lives, from the infinite numbers of coffee drinks we can get in the morning to online experiences we tailor without a sense of wonder that ability once inspired to so many other aspects of our lives. Good technology tends to win out over time, despite all the attempts by the old guard to stifle it.

But there’s no question that as part of what Wired co-founder Louis Rossetto, who knows a bit or two about innovation and prognostication himself, has recently called “the death throes of the mega-state,” the FDA and the federal government more generally will do everything it can to hold back the future like Canute with the waves.

My advice for those of you who want to sign up with 23andMe but can’t spit in their test tubes for at least a while? Hock your lougies in the FDA’s general direction instead.