At first glance, Jordan McLinn of Indianapolis appears to be a healthy, vibrant seven-year-old boy. In this case, however, looks are deceiving.
Watch him interact with his peers for just a few minutes, and it becomes apparent there’s something different about Jordan. He’s a bit slower and a little weaker than the other kids. And this probably isn’t going to change.
Jordan suffers from Duchenne muscular dystrophy, a rare condition that will cause his muscles to gradually degenerate. Though he can walk and play much the same as most children his age now, and even puts in time as a junior firefighter at his local firehouse every Thursday, by this time next year, he could require the use of a wheelchair. He might survive to the age of 20.
“Quality is way more important than quantity,” Jordan’s mother, Laura, tells Citizen. “If my son can live his life to the fullest for the 10 or 20 years or whatever God decides is his time here on Earth, that’s okay.”
The McLinns are relying on their faith in Christ as they navigate this trial with Jordan. They’re also pursuing any possible means of saving or improving his life, including experimental medicine. In fact, they’ve had their eye on a promising treatment for some time, but their attempts to get Jordan into clinical trials have regularly fallen short. He hasn’t qualified because of factors like his age and his walking speed.
Jordan isn’t alone. The McLinns know several other families whose sons also are failing to qualify for the tightly controlled trials.
So the McLinns have turned their frustration into action and become one of the most prominent advocates of the country’s burgeoning Right to Try movement, which gives terminally ill people the right to try investigational medicines that have not been approved by the U.S. Food and Drug Administration.
Sweeping Through The States
Since 2014, 33 states, including Indiana, have passed Right to Try legislation.
“People have a fundamental right to try to save their own lives, especially when they’re dying (and) they’ve exhausted all government-approved options,” says Christina Sandefur, executive director of the Arizona-based Goldwater Institute, a libertarian public policy think tank which has spearheaded the recent wave of state-level Right to Try laws.
Sandefur describes Right to Try as a grassroots movement. The Goldwater Institute got involved with writing model legislation about four years ago after being approached by several patients and physicians about the need for it.
Sandefur and her team were told that while the FDA has an expanded-access program allowing the terminally ill to try experimental treatments, there was so much red tape involved in applying that few physicians—from whom the requests needed to come—had the time for it. Plus, patients needed to have the green light from the company producing the drug in question in order to qualify, which was rarely easy to obtain. The upshot was that while the FDA could accurately say it granted most expanded-access requests, most terminally ill patients were not in a position to complete the application.
The Goldwater Institute staff found the advocates’ rationale compelling (“These are people who just want one last hope, even if it’s a long shot,” Sandefur says) and helped Colorado pass the nation’s first Right to Try law in 2014. Since then, the legislation the institute helped craft has become the template for the dozens of other states where lawmakers have passed Right to Try.
A Federal Case
In an era when hyper-partisan bickering tends to dominate America’s political headlines, Right to Try is transcending the bounds of ideology and party affiliation. Wherever it has become law, it’s done so with bipartisan support, and the movement is exploding.
Right to Try legislation has now been introduced in every state except New Mexico. It has been vetoed only twice, in Hawaii and California—and the governor of the Golden State, Jerry Brown, ended up reversing his 2015 veto and signed the bill into law last year.
Now the momentum is rapidly bubbling up to the federal level, where Sen. Ron Johnson (R-Wis.) is leading the charge. Johnson first introduced a federal Right to Try Act modeled on—and designed to protect—the state laws in mid-2016 and even held a rally on Capitol Hill to garner support. “Federal legislation is required to ensure the FDA does not interfere with those state laws,” Johnson said at the time. “This isn’t a Republican or Democrat issue. It is a human rights issue, and I hope all of my colleagues will join me in supporting this critical bill.”
Johnson is making progress. Within weeks of its introduction, his 2016 bill had garnered 24 co-sponsors (all Republicans) and resulted in a Senate hearing, though lawmakers did not pass it. This January, Johnson reintroduced the bill with the support of 43 co-sponsors, including two Democrats and one Independent. A bipartisan effort to pass the legislation is also afoot in the U.S. House.
Most heartening to many advocates, however, were the words of President Donald Trump earlier this year.
“One thing that’s always disturbed me: They come up with a new drug for a patient who’s terminal, and the FDA says we can’t … approve the drug, because we don’t want to hurt the patient,” Trump told journalists. “But the patient is not going to live more than four weeks (anyway). So, we’re going to be changing a lot of the rules.”
“I’m thrilled to know it’s important to him because it’s important to a lot of people,” Laura McLinn tells Citizen. The president’s comments and the efforts in the works in Congress strongly suggest that, one way or another, Right to Try may well soon be federal policy. “It’s going to happen this session,” she confidently predicts.
But just how it will happen is unclear. Trump’s comments seem to suggest there will be a change in FDA rules. Lawmakers like Johnson persist in seeking a change in federal law. Whichever avenue ends up being the chosen route, advocates feel the White House is committed to making sure Right to Try ultimately prevails at the federal level.
Access Still Not Guaranteed
Right to Try is clearly popular and apparently on the verge of completing a stunningly swift national victory. But is it good policy?
The FDA says it has taken no position on any Right to Try laws.
Pharmaceutical Research and Manufacturers of America (PhRMA), a leading trade group representing the nation’s drug companies, advises a cautious approach.
“Any legislation should protect the integrity of clinical trials and the FDA oversight of expanded access to maintain the best interests of patients,” says spokesperson Caitlin Carroll.
Right to Try advocates say they’re not opposing FDA oversight, noting that under the state laws that have been passed, terminally ill patients are only allowed to take treatments that have cleared Phase 1 (which supporters call “the basic safety phase”) of the FDA’s three-phase clinical trial process—and only if they have exhausted all already-approved therapies. “Even if we don’t know whether the drug will work or what the side effects are, patients and doctors ought to make the decision about whether they want to try this option while it’s still making its way through clinical trials,” says Sandefur.
Johnson—who named his federal bill after Trickett Wendler, a Wisconsin woman who died of Lou Gehrig’s disease in 2015, agrees.
“These patients don’t have the time to wait—often years and years—for the FDA to grant final approval,” he says.
Yet for all of the emotionally compelling arguments fueling the rise of Right to Try, some say that as a practical matter, it will accomplish little.
“While every Right to Try law says that terminally ill patients have the right to ask for access to experimental medications, the laws do not, and in fact cannot, require anyone or any entity—including drug companies—to acknowledge this ‘right’ by mandating that they provide any medications,” researcher Lisa Kearns and bioethicist Beth Roxland wrote in the digital edition of The Hill last June.
Right to Try supporters concede there’s some validity to that concern. Sandefur acknowledges that many drug companies, having spent a fortune seeking FDA approval, will probably choose to play it safe while awaiting the agency’s official green light for their treatments. She understands how pharmaceutical companies could be concerned that adverse events from expanded access could be used against them by the FDA while positive outcomes could potentially carry little to no weight in their favor. She would like to see clarification on that matter at the federal level.
PhRMA declined to comment on whether the potential impact of adverse events affects the pharmaceutical industry’s approach to expanded access.
For its part, the FDA says only twice in the last decade have adverse events caused it to hold up approval of an experimental drug being used outside of clinical trials. In each case, the issues that caused concern were addressed and the drug was eventually approved. Still, some believe such cases can make drug companies jittery.
“People want a perfect plan, and that’s just not how life is,” says Laura McLinn, who insists that in spite of Right to Try’s limitations, some physicians and drug companies are willing to grant expanded access outside of the FDA’s formal structure.
One is Dr. Ebrahim Delpassand of Houston, who says he has now successfully treated more than 100 patients for neuroendocrine cancer under Texas’s Right to Try law, passed in 2015.
Delpassand spent five years administering clinical trials of the drug LU-177 to 150 patients. The drug, already available in Europe, had completed every phase of its U.S. clinical trials. Patients, says Delpassand, were benefiting. But then the FDA told him he had to stop administering the therapy until the agency gave it final approval.
Delpassand, who would not make himself or any of his patients available to Citizen but did speak to the Goldwater Institute, was devastated. He knew it could be another year or two before the FDA granted the drug final approval. In the meantime, many of his patients would die.
Months later, however, Texas passed its Right to Try law, giving Delpassand the permission he felt he needed to continue the therapy.
“Many of these patients were only given three to six months to live,” he told the Goldwater Institute. “Now, a year later, many of them are still alive.”
Delpassand is such a believer in Right to Try, he testified for its passage at the federal level during last year’s Senate hearing.
“If it saves or betters one life, and we know in fact it’s been many more than that, then it’s done its job,” Sandefur insists.
Laura McLinn agrees. “Even if one person or a small group of people can benefit from it, then it’s worth having the law.”
Laura’s son Jordan—without having to invoke Right to Try, as it turned out—did finally qualify for the treatment his family had long been seeking. Yet they made the agonizing decision to pull him out of the clinical trial when they learned one-third of the patients would be given only a placebo for two years.
Now, Jordan is starting a new clinical trial with another company. Laura believes a day is coming in the not-too-distant future when doctors will apply a cocktail-of-drugs approach to Duchenne, lengthening the lives of boys like Jordan and enhancing their quality of life. Her hope is that this experimental drug will slow the progression of his disease enough that Jordan will live to see that day.
“Every night when he says his prayers before going to bed, he says, ‘Thank you, Jesus, for my miracle,’ ” Laura says of her son, who sees his weakness as a gift—one he can use first and foremost to draw people to Christ, but also to help carry Right to Try across the proverbial goal line. “We feel like he is touching so many lives.”
For More Information:
To learn more about Right to Try legislation, visit www.righttotry.org. More information about the Goldwater Institute can be found at goldwaterinstitute.org. Further specifics about Duchenne muscular dystrophy are available at www.mda.org/disease/duchenne-muscular-dystrophy.
Originally published in the April 2017 issue of Citizen magazine.