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AbledCauses Post Banner shows Amy Auden, the widow of Nick Auden leaving a memorial service for her husband at St. Andrews Church in Brighton, Australia. She has long brown hair and his wearing a shamrock green coat and is walking between two unidentified women. She is looking down, her face full of grief and her eyes full of tears. The headline reads: AbledCauses: The Legacy of 'Save Locky's Dad': 'Right To Try' Laws To Access Unapproved Drugs.

It was one of the bravest and heartbreaking fights – and at the same time – one of the most unfair. While Nick Auden didn’t live long enough to see it, the legacy of the ‘Save Locky’s Dad‘ campaign is galvanizing state governments across the United States into passing ‘Right To Try‘ laws to allow terminally-ill patients, in consultation with their doctors, to gain access to experimental drugs not yet approved by the FDA.

But it’s not an achievement without controversy. Read more about the laws and the issues they are raising by clicking on the additional tabs in this section. 

In mid-May, 2014 in the Auden family’s adopted state of Colorado, Governor John Hickenlooper signed into law the first ‘Right To Try’ law in the United States to allow terminally-ill patients to gain access to experimental drugs without federal approval.

The ‘Right To Try’ For The Terminally Ill

by Michael Ollove | Stateline

Photo by Justin Ferland Photography shows 15 year old Austin Leclaire sitting in an electric wheelchair with his left arm around the shoulder of his 12 year-old brother Max in the backyard of their home in Pembroke, Massachussetts. Both show the physical effects of Duchenne muscular dystrophy but they are less pronounced in Max who is receiving experimental drugs for the disease. Both boys are smiling at the camera and you can see fallen autumn leaves and a woodpile in the background.

Fifteen-year old Austin Leclaire and his brother Max, 12, of Pembroke, Massachusetts, share the deadly degenerative disease Duchenne muscular dystrophy. But over the course of the past 144 weeks, the boys’ physical conditions have progressed in opposite directions.

Max has regained functions – he has abandoned his wheelchair and can now run, tote his own backpack to school and even ride a bike. Austin’s capabilities are diminishing. He cannot walk at all, and he’s lost the ability to brush his teeth or hold a water bottle. He expects that soon he won’t be able to play drums anymore, one of his few remaining physical pleasures.

The difference in their  trajectories is that Max has been in a clinical trial for nearly three years, during which he has been given weekly infusions of the experimental drug eteplirsen, manufactured by Sarepta Therapeutics Inc. Austin was deemed unqualified to get the drug because of his more advanced condition.

Withholding experimental drugs from the most gravely ill has fueled several states to pass so-called “right-to-try” legislation that would make these drugs available without Food and Drug Administration approval to terminally ill patients with no other options. 

Some critics of the years-long FDA drug approval process, with its requirement for multiple clinical trials, contend that it is much longer than it should be, thereby keeping some promising drugs from those who might benefit, particularly those, like Austin, with time running out. The Tufts Center for the Study of Drug Development says it normally takes 5½ to 10½ years for a drug to receive FDA approval.

Because of that, Colorado, Missouri and Louisiana all passed right-to-try legislation this year that would enable those with terminal illnesses and no other treatment options to secure investigational drugs before final FDA approval if the drug manufacturer is willing to provide them. Arizona voters will decide a similar measure in a referendum in November.

The legislation passed overwhelmingly and with bipartisan support, often after lawmakers heard anguished testimony from terminally ill patients or their families.

The laws would cut the FDA out of the process, at least in terms of a single, terminally ill patient. The decision would be left entirely in the hands of the patient, the doctor and the drug company.

Whether these laws would withstand a court challenge is an open question. Typically, state laws cannot supersede federal statutes. Even if the laws don’t withstand a challenge, advocates say, they may serve a valuable service in spotlighting the issue.

“(A lawsuit) wouldn’t be all bad news because it would further elevate this issue in the public arena and put pressure on Congress and the FDA to make this change and literally save thousands of lives every year,” said Frank Burroughs, head of the Abigail Alliance for Better Access to Developmental Drugs, which he founded 13 years ago after his 21-year-old daughter died of cancer.

So far, only Colorado’s law has taken effect, a little over a month ago. The Goldwater Institute, a conservative nonprofit that defends states’ rights,  created the model legislation for the new laws.  While it has received word of interest, no patients have yet formally tried to receive an experimental drug under the new law, the institute said.

FDA’s Slow Process

Under FDA rules for drug testing, Phase One, the first studies to use human subjects, is quite limited in the number of patient participants (typically 20 to 80, according to theFDA website on the drug approval process). This first step is intended to determine the drug’s most frequent side effects and how the drug is metabolized and excreted.

If Phase One doesn’t reveal unacceptable toxicity, the drug can be approved for Phase Two trials, where the emphasis is on testing the effectiveness of the drug. This stage can involve a few dozen to 300 patients. If the drug passes muster, it goes onto Phase Three, which gathers additional evidence on safety and effectiveness and involves from several hundred to 3,000 subjects.

In the Leclaire brothers’ case, those selected for the trial had to still be ambulatory most of the time, but with the expectation that their walking days would end within a year, said Jenn McNary, the boys’ mother. Max fell down occasionally, but he still didn’t need the wheelchair most of the time. Austin no longer qualified.

So even as Austin has watched Max get stronger from the drug treatments, he wonders what motor skill he will lose next. And while he hopes s one day to earn degrees in mechanical engineering and zoology, he is aware that those with his condition rarely survive their twenties.

“I’m glad that Max has (the treatments), and he’s proving that it does work, and I should have it, too,” Austin said by phone from home. “But it’s also unfair…. I’m just really upset that the FDA won’t approve the drug, and it’s their fault that someone might die.”

The FDA has taken no official position on the new laws. “While the agency is supportive of patients’ access to experimental new treatments, the FDA believes that the drug approval process represents the best way to assure the development of safe and effective new medicines for patients,” an FDA statement said.

Although there are already provisions in place that allow for expedited approval of drugs and to make certain experimental drugs available to individuals prior to final approval, the Goldwater Institute insists that even those procedures take too long for a person whose time is running out. “A lot of patients don’t have five or six months” that those procedures take, said Victor Riches, a Goldwater spokesman.

The FDA  said it receives about 550 applications a year from drug companies to supply drugs to individuals before final approval and agrees to nearly all of them.

Rare Diseases

But that doesn’t account for patients who have been turned down by manufacturers, such as Holly Singh, a Texas second grade teacher whose 14-month-old daughter Harper has spinal muscular dystrophy, which leaves her incapable of picking her head up, crawling or rolling over. Without help, her life expectancy is quite limited.

Singh applied to Isis Pharmaceuticals, developer of the promising drug, ISIS-SMN-rx, which is soon to enter Phase Three clinical testing, for early access to the drug. In turning down her request, Isis chairman Stan Crooke raised the possibility that granting it could have prolonged the overall testing program and delayed the drug’s approval for other patients. He also said that all the safety concerns had not been fully addressed.

Singh’s correspondence with Crooke demonstrates her desperation.  She wrote, “What kind of mother would I be if I just told my child, ‘I’m sorry that there’s a drug, but unfortunately you can’t have it.  You just need to suffer a little while longer.’”

While sympathetic to the situation of individual patients, the pharmaceutical industry has been far from enthusiastic about the right-to-try laws.

“We have serious concerns with any approach to make investigational medicines available that seeks to bypass the oversight of the Food and Drug Administration and clinical trial process, which is not in the best interest of patients and public health,” said Sascha Haverfield, vice president of scientific and regulatory affairs for thePharmaceutical Research and Manufacturers of America. He suggested that medical providers, the pharmaceutical industry and FDA should seek ways to improve access to clinical trials.

Critics of the new laws contend that Phase One trials are too limited to assure that a drug can be safely used. “Safety (in Phase One) is tested in only the crudest way you can imagine, making sure it doesn’t kill people,” said David Gorski, an associate professor of surgery at Wayne State University and the editor of the Science-Based Medicine blog, which has been critical of right-to-try laws. Even for terminally ill patients, Gorski argues, the risks are too great based solely on Phase One trials.

“They say it couldn’t get any worse.” Gorski said. “Well, yes, it can get worse. If there’s anything worse than dying of a terminal disease, it’s added suffering and potentially cutting the time you have left.”

According to a January article in the journal Nature Biotechnology, only 16 percent of investigational drugs that complete Phase One clinical trials ultimately gain FDA approval.

Still, patients and families say they feel that they should be free to take the chance given that the alternative is death. “I should be able to take the risk of whether or not it would help,” said Austin Leclaire. “It’s my life.”

Proposed laws on experimental drugs stir debate

BY STEPHEN FEE PBS Newshour Weekend

This May, Colorado’s governor signed the nation’s first ”right to try” bill, which allows terminally ill patients to try unapproved — and potentially dangerous — drugs outside of clinical trials and without approval from federal regulators.

Missouri and Louisiana have passed similar statutes, and Arizona voters will vote on their own version this fall.

For the NewsHour Weekend broadcast, we profiled the Missouri State Representative Jim Neely who introduced that state’s bill and his daughter, who is fighting a deadly form of cancer.

Neely is backed by Phoenix-based Goldwater Institute and by patients and families who have lost relatives to diseases, but the proposed measure is not without controversy. While FDA remains neutral in the legislation debate, there are opponents to these type of laws.

To get more context about the debate surrounding “right to try,” I spoke with Julie Rovner of Kaiser Health News.

AbledCauses Update Banner

March 13, 2014 : A Bittersweet Victory

UPDATE FROM JULIA MEDEW, Health Editor – Sydney Morning Herald

 

AbledCauses- photograph of the Auden family  sitting on the step of a wooden shed painted with pastel light blue and  yellow vertical stripes. From left to right they are father Nick Auden, 7 year-old son Locky, 5 year-old daughter Hayley, while mother Amy holds their year old son Evan. The photo is by Catherine Sutherland.

 

In cruel timing, Merck announced this week that it was launching an “expanded access program” for its PD-1 drug Lambrolizumab (or MK-3475) in the US for people “who have serious or immediately life-threatening illnesses for which no comparable or satisfactory alternate therapies are available.”

A spokeswoman for the company said it planned to make the scheme available to Australian patients “at the earliest possible time” but did not say when that could be. At the moment, only 175 patients with melanoma are receiving the drug in clinical trials.

Mr Auden’s bereaved wife Amy Auden welcomed the program but said the timing was “absolutely devastating”.

“Words cannot express the emotion I feel… The people at Merck said that they did not want to ‘play God’ by giving the drug to Nick (they were concerned about not having enough of the drug for everyone with stage 4 melanoma), however by denying Nick the drug, they in effect did just that,” she wrote in an email.

“Every minute of every day I am reminded that Nick is not here and I am widowed at 38 with 3 young kids. Right now, however, I am trying to focus on the fact that were it not for the campaign, a compassionate use program for the new melanoma breakthrough drug would not be a reality for others.”


Read more at the Sydney Morning Herald
 

AbledCauses Update Banner

November 22, 2013 : THE SADDEST UPDATE

 

The man known around the world as ‘Locky’s Dad’ has lost his battle with cancer. Nick Auden died on November 22 at the age of 40.

 

Photo of Nick Auden. He is sitting in front of a window in a tall office building dressed in a suit and dress shirt with an open collar. He has sandy blonde hair cut neatly and is smiling at the camera.

 

 

He leaves behind his wife,  Amy and children 7 year-old Laughlin (‘Locky’), 5 year-old Hayley, and 1 year-old Evan.

The story of his battle with Stage IV melanoma went viral after his wife Amy started a petition at change.org to garner support and put pressure on pharmaceutical giants Bristol Meyers Squibb and Merck to give Nick special access to a couple of experimental ‘PD-1’ drugs that were showing great promise that were in clinical trials. Over 525 thousand people signed the petition.

And even though the U.S. Food and Drug Administration (FDA) said Nick could use the drugs if one of the companies gave him access but they refused saying they were only available to people in clinical trials.

Despite the PD-1 drugs lambrolizumab and nivolumab offering groundbreaking results, a series of small brain tumours prevented Mr Auden from initially qualifying for clinical trials of the wonderdrugs. 

 

This past July, Nick was finally accepted for a trial only to suffer a partial blockage in his bowel, which again ruled him out.

 

Following a funeral in the United States, Amy and the children returned to Australia for a memorial service there. She told the Herald Sun News in Melbourne, that, returning home without her soulmate was the toughest thing she had ever done.

“It’s really difficult coming back to Melbourne because I come here without Nick. We met here, and it is just so hard for me to come to terms with. Locky is absolutely devastated. When he asks me the really difficult questions, like who is going to teach me football … I am hoping that with our friends and family together we can show Lachlan what Nick would have suggested and how he would have guided him. Hayley is too young and she is just asking me ‘when is daddy coming back’.”

 

Nick Auden with his son Lachlan in combine harvester in happier times.

 

While Evan will be too young to remember his father, Amy has thousands of messages from people inspired by their campaign that she will treasure to show her children what a great man their father was.

With her children settled in the United States,  Amy said it was too early to make a decision about whether her family would return to Australia in the long-term. But she has promised to continue her husband’s legacy and pressure pharmaceutical companies to improve compassionate access for desperate patients.

All of us at Abled.com are heartbroken by the news and offer our heartfelt condolences to Amy and her children. We will keep this post current on our site as a tribute to Nick’s courage and perseverance and will join the fight to lobby for compassionate access to experimental drug treatments.

The Original Post

AbledCauses story banner shows Nick and Amy Auden of Lone Tree, Colorado and their three young kids with the headline: Save Locky's Dad: Dying Dad Pleads For Unapproved Drug.

 

 Disqualified from a clinical trial, Nick Auden fights for ‘compassionate’ use of PD-1

 

“When you’ve been given a terminal diagnosis, you’re prepared to accept a drug that’s 50 percent effective. Safety concerns don’t really figure in the same way.” Those words, spoken by Nick Auden in an interview with ABCNews.com, sum-up his fight to get access to what’s being called a new ‘wonder drug’.

The 40 year-old father of three was diagnosed with Stage 4  melanoma in September of 2011. On July 2nd of this year he was admitted into a clinic trial for a drug called PD-1 that helps a patient’s own immune system shrink cancer tumors for good. But a blocked bowel the very same day sent him to the ER and out of the trial.

Now, he and his wife Amy are fighting for him to become an individual case study under compassionate-use rules which can give people access to experimental drugs that haven’t passed FDA approval outside of a clinical trial, and they’ve launched a petition campaign at Change.org to lobby pharmaceutical giants Merck and Bristol-Meyers-Squibb to provide the treatment.

The Audens’ 7 year-old son Lachlan, nicknamed “Locky,” is included in a video on the “Save Locky’s Dad” website asking for people to support his dad. The “Save Locky’s Dad” campaign has skyrocketed since launching two weeks ago with the goal of reaching 150,000 signatures. Once it attained that goal, the Audens raised it to 200,000. 

Now with more than 258,000 signatures, the goal is 300,000 and as more media outlets share the story, it’s quite likely they’ll reach and even surpass that goal very soon.

Watch the ‘Save Locky’s Dad’ video below:

 

 

Read more of the interview with ABCNews.com |  SIGN THE PETITION HERE

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