When ‘Right to try’ isn’t Enough: Congress Wants a Single ALS Patient to get a Therapy Never Tested in Humans


By Nicholas Florko 
May 31, 2019

WASHINGTON — A family in Iowa believes the Food and Drug Administration will decide whether their only surviving daughter lives or dies, and they’ve been on a monthslong crusade to break through its bureaucracy. And they’re succeeding.

Just last week, the FDA gave Jaci Hermstad, a 25-year old Iowan who is dying from a rare form of ALS, an early sign that she will receive the first dose of an experimental drug never before tested in humans. The FDA’s move, which was confirmed to STAT by Jaci’s family and doctor, is a breakthrough for the Hermstads.

For months, the family has circulated petitions, tweeted at President Trump, and called on some of Washington’s most controversial and powerful politicians — including the speaker of the House and a Republican under fire for racist comments — all to convince the FDA to waive even the most basic of studies before the drug is injected into Jaci’s already fragile immune system.

The Hermstads’ progress reopens an ardent debate in Washington over the FDA’s role in determining when sick or dying patients can access experimental therapies — and what role career politicians should play in the nation’s decisions about how and when to let patients access untested treatments. It also underscores a fundamental question about the role of lawmakers: Is it fair for a member of Congress to intervene on behalf of a single constituent?

“Do you really want the FDA responding to one-by-one requests by families from congresspeople?” asked Arthur Caplan, the founding head of the New York University School of Medicine’s division of medical ethics.

The FDA is no stranger to this debate: The issue came to a head this time last year, when Washington deliberated over and ultimately passed a federal “right-to-try’’ law, which largely cuts the FDA out of the process for determining when dying patients can access experimental treatments being studied in clinical trials.

The Hermstad case is far more convoluted. The drug Jaci’s trying hasn’t gone through clinical trials, making it ineligible for the pathway outlined in the right-to-try law, which was supported by President Trump. And though Jaci’s family felt compelled to press Trump and lawmakers to help, they may not have needed to: The FDA had never denied Jaci’s request for access to the drug; regulators simply asked that toxicology testing to be done. In fact, Jaci’s own doctor has had nothing but kind words for the FDA throughout the process.

The chaotic backstory underscores how complicated it has become to access experimental therapies in the U.S. And it raises a host of questions about the role of the FDA as a gatekeeper, the propriety of Congress acting on behalf of a single patient, the role of crowdfunding to pay for basic research, and the most basic and fraught question of all: Who is to blame when a 25-year-old is facing virtually certain death and can’t get access to a drug she thinks might save her life?

“You’ve got a large ethical and policy quagmire here,” Caplan said.

When Jaci’s family heard about the drug in February, it felt like divine intervention. The call came just a day after Jaci was first diagnosed: Dr. Neil Shneider, the head of Columbia University’s ALS clinic, described a new therapy known as an antisense oligonucleotide that, at least in theory, could prevent the production of proteins suspected to kill motor neurons in ALS patients.

“It’s like God has paved this path for this to happen, and there’s a reason for it,” Lori Hermstad, Jaci’s mother, said.

Quickly, however, the Hermstads learned that the therapy was far too early in development for them to access it. It hadn’t even been vetted for basic safety and toxicity — which meant it would be a lengthy, uphill battle to get the drug through the FDA’s existing pathway for potentially lifesaving experimental treatments, known as its “expanded access” program. The Hermstads also couldn’t rely on the federal right-to-try law that Trump signed in May 2018, because the drug hadn’t gone through Phase 1 clinical trials.

But the Hermstads were determined not to give up. They contacted their local lawmakers, started a Facebook group called “Cowgirl Up for Jaci,” and launched an online petition to the FDA.

The Hermstads’ aggressive campaign eventually pulled in an unlikely band of lawmakers: Democratic Speaker of the House Nancy Pelosi (Calif.), Sen. Chuck Grassley, the powerful chairman of the Senate Finance Committee, and fellow Iowa Republican Rep. Steve King, who had been stripped of virtually all his power after he espoused racist views in a January interview with the New York Times.

King took the most brazen step.

On May 20, the congressman introduced an almost unheard of piece of legislation: a one-sentence bill directing an entire federal agency to make an exception to its rules for a single, named individual: Jaci Hermstad. It runs just 40 words.

The bill never was formally considered by Congress, but it got the attention of some of Washington’s top lawmakers. King had “multiple productive discussions” on the issue with Pelosi, who eventually contacted the FDA directly to talk about the Hermstad case, according to a press release from King. (Neither King nor Pelosi responded to requests for comment.)

Jaci’s senator, Republican Sen. Chuck Grassley, one of Washington’s most influential health policy legislators, also “has urged the FDA and the White House to give Jaci’s request every consideration,” according to Michael Zona, a Grassley spokesman.

Less than a week after King’s bill and Pelosi’s intervention, Jaci’s family got the first sign that the FDA was backing down from its previous demands. While the FDA hasn’t formally accepted or denied Jaci’s expanded access petition, Columbia’s Shneider told STAT Wednesday that “there was some bending” at the FDA. He said the agency seemed willing to back off its earlier requests that the drug company conduct significant toxicology studies before Jaci’s first infusion.

“I think maybe that we were lucky in that we hit them at a moment when there was a lot of external pressure and internal discussion about this, and we were a test case,” Shneider said.

(Shneider emphasized that Jaci’s first planned infusion on June 10 still won’t be finalized until he formally hears back from the FDA regarding the expanded access application, which he formally filed Wednesday.)

“You’ve got a large policy and ethical quagmire here.”


The rare personal interventions from some of the nation’s highest ranking public officials — and the FDA’s exceedingly early exception to its own policies — raise questions about the role the federal government plays in the lives of dying patients. Such questions roiled the agency when Washington first began to debate a federal right-to-try law: The FDA’s main job is to determine which drugs are safe and effective for Americans. But are there times, perhaps when life is on the line, that it shouldn’t carry out that mission?

“What right to try has done, and what one-patient bills like this will do, is put us back into a position where we have to justify FDA’s existence to society,” said Holly Fernandez Lynch, a bioethicist at the University of Pennsylvania, who has raised concerns with the Hermstad case and with the federal right-to-try law. “For this particular patient it seems like an individual decision, but it has long term impact on whether we allow FDA to do its function.”

Congress does have the power to pen so-called private legislation — bills which only apply to a select group of people, rather than to the public writ large. These private bills have been written to grant citizenship, relieve debts, and pay out those harmed by government wrongdoing. Sen. Hubert Humphrey Jr. even penned a piece of private legislation to give special benefits to former employees of the Senate restaurant.

But experts raised concerns with the precedent set by a bill intended to help one single patient.

“What does that mean for everyone else who is similarly in a situation that is very dire but who maybe [doesn’t] have the same resources to be able to communicate with legislators, and the media, and with the larger public through social media?” Kelly McBride Folkers, a research associate at NYU’s division of medical ethics, asked.

In half a dozen interviews by STAT with experts on both sides of the debate over the FDA’s expanded access policies, no expert could point to another piece of legislation that so clearly attempted to force the FDA’s hand regarding an individual’s treatment.

Even Shneider, Jaci’s doctor, was left feeling uneasy about the level of political pressure being put on the FDA. In an interview with STAT, he said the FDA has been “really responsive” and “really responsible” throughout the process of trying to get Jaci treatment.

“I did not encourage it, frankly,” said Shneider, referring to the congressional pressure put on the FDA. Shneider even described the federal interventions as people putting “their foot [on] the neck of the FDA.”

He also acknowledged that Jaci’s case likely will provide a road map for doctors hoping to get preclinical treatments to their patients, just as his team at Columbia relied heavily on the experience of Dr. Timothy Yu, who pioneered a similar process to get an experimental drug for Batten disease to a patient last year.

But nonetheless, he hopes the congressional pressure isn’t an enduring lesson of his endeavor.

“I don’t know how sensitive the FDA is to congressional pressure, I would hope frankly that they’re not too sensitive to it. They have a job to do,” Shneider said, adding that he wasn’t made aware of King’s bill before it was introduced.

The ethical quagmire of Jaci’s case is all the more complicated by the fact that the Hermstads are trying to raise $700,000 to pay for the treatment.

Shneider said the money is being used to “do the very basics” needed to get Jaci treatment — things like paying a contract research organization for the remaining testing needed for the drug, as well as the costs of the infusions, hospitalizations, and Jaci’s travel to Columbia.

“I don’t have, nor should I, use the resources of my center for one patient like this, not that it doesn’t have implications for others, and so I didn’t have the money to pay for this and so the burden of this financially is largely going to be on the family and the community,” Shneider explained.

While a drug maker was involved in developing the drug, none of the money will go to that company, which “has nothing to do with this, at this point,” according to Shneider. Shneider declined to name the company involved, which appears to be Ionis Pharmaceuticals.

“When contacted by scientists and physicians looking for a potential treatment for Jaci, we said yes, providing some of our early research into oligonucleotides. … We also continue to advise them on antisense technology and its use in the treatment of ALS,” Frank Bennett, senior vice president of research and franchise leader for neurological programs at Ionis, told STAT in a statement.

Even if the money being collected by the family isn’t going to line the pockets of a drug maker, the University of Pennsylvania’s Fernandez Lynch said the funding for this treatment “raises a ton of ethical concerns.”

“What about other patients who can’t afford this kind of access and don’t have this kind of political clout? Should people be contributing to a GoFundMe that is extremely unlikely to lead to benefit? What are we doing to patients when we advance this spirit of fighting disease at all costs?” she asked.

For the Hermstads, Jaci’s path to treatment is far less a philosophical debate about the separation of powers or health care financing and far more about the importance of continuing to try.

Their story with ALS starts back in 2005, long before right to try was a national slogan or Jaci was even diagnosed with the disease, when Jaci’s twin sister, Alex, was diagnosed with the same form of ALS that now is forcing Jaci to fight for her life, a rare mutation known as FUS P525L.

The fear that Jaci, too, might have the mutation was always in the back of the Hermstads’ heads, but those fears didn’t reach their height until last November, when Jaci started to experience symptoms like back pain and trouble walking long distances.

Then came Valentine’s Day 2019. A blizzard had blanketed the interstate in Iowa, turning a tense three-hour drive to Jaci’s doctor into a precarious six-hour trek. And it only got worse when Jaci was given the news she had feared all along: She, too, had the same rare and almost certainly fatal form of ALS that had killed her twin sister.

Lori Hermstad, Jaci’s mother, describes it as the worst day of her life, second only to Valentine’s Day 2011: The day she buried Alex.

“You can’t explain the emotional pain,” Lori Hermstad said. “It’s the worst kind of heartache anybody can describe.”

Since then, the ALS has continued to zap away Jaci’s nerve cells.

“I do this for a living, and I’m telling you, it’s striking, the case of her decline,” Shneider said.

Within months of her diagnosis, she could no longer ride her beloved horse, Bud, and she had to seriously consider whether to be put on a ventilator.

“To know that you have your last remaining child going through it, and there’s actually a possibility that it could be a breakthrough, and that it might be too late is another kind of torture that you can’t even describe,” Lori Hermstad told STAT.

The Hermstads met Rep. King at a benefit for Jaci held in March. The two hit it off, even making a plan to work together in opening a horse arena when Jaci is feeling herself again.

And after they met, reaching out to him was like reaching out to any other friend or family member. They were, and are, doing everything they can to fight for Jaci.

“He’s been an amazing support and advocate and we are just so thankful that he’s done all he has done to help Jaci,” Lori Hermstad said.

And Lori, at least, is emphatic that any family should be allowed to do the same — no matter what the political environment.

“There should be absolutely nothing, no bureaucracy, nothing, to stop the FDA from allowing somebody who is going to die anyway to not try a drug. Period,” she said.

For the most part, the debate isn’t worrying the Hermstads. They’re busy dealing with the daily struggles associated with advanced ALS, like Jaci’s ever worsening breathing troubles. And they’re not done pushing. Their new campaign is closer to home: They’re hoping Jaci can get her treatment pushed up from her scheduled appointment on June 10.

“Until it actually happens, I think that’s when we can really celebrate, because a lot can happen between now and two weeks,” Lori Hermstad said. “It’s still scary.” [read more]