31 May Congress wants a single ALS patient to get a therapy never …
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…