Signs of the toll amyotrophic lateral sclerosis has taken on Kelli Johnsen’s body are scattered throughout the living room of her Emporia, Kansas, home.
A wheelchair in one corner. A lift in another. A walker near the television.
Next to her chair there’s an Eyegaze system — a screen that tracks her eye movement and blinks — that she uses to control the TV, lights and other devices. She can still move her hands, but not much.
Before she got sick, Johnsen, 45, was a critical care emergency veterinary technician and had a paper on tracheal trauma published in a veterinary trade magazine. Then she began struggling to hoist 60-pound animals onto operating tables. Then she started slurring her speech. Seven years ago a neurologist explained why, diagnosing her with ALS, also known as Lou Gehrig’s disease.
Though Johnsen now struggles to move and talk, her mind remains sharp. She spends six to eight hours a day using the Eyegaze to scour the Internet while researching medical breakthroughs that could save or at least prolong her life.
That’s how she heard about Eric Valor, a man with late-stage ALS who is in a trial for a drug made by a company called Genervon. Her face lights up when she talks about the improvement he reports in his breathing and swallowing.
“Last I heard, he had held on to the gains,” Johnsen says.
Johnsen would like to try the drug too, but it’s in the middle of U.S. Food and Drug Administration clinical trials. In fact, so many ALS survivors and their caregivers are interested in the medication that Genervon issued a news release in March updating them on its status.
The company has applied to fast-track the drug through an accelerated approval FDA program. But if that’s not granted and it remains in Phase 3 clinical trials, it could be three more years before the drug hits the open market.
Johnsen doesn’t believe she can wait that long. People with ALS, statistically, are expected to live two to five years after their diagnosis.
Kansas lawmakers are promoting bills at the state and federal level intended to streamline the FDA approval process or let people like Johnsen bypass portions of it and access experimental drugs sooner.
But some fear those efforts could allow profit-seeking drug companies and medical device manufacturers to rush products to market before anyone knows the potential risks or side effects.
Right to try
Don Hill grabs a chair from the kitchen and pulls it across the hardwood floor to where Johnsen and her mother, Vicki Triemer, are sitting.
Hill is a pharmacist. He’s also Johnsen’s representative in the Kansas House.
He’s considered a moderate Republican, but last session he joined with three of his more Libertarian-leaning colleagues to promote a "Right to Try" bill on Johnsen's behalf.
House Bill 2004 seeks to give Kansans with terminal illnesses access to drugs in the early phases of FDA clinical trials. It’s based on model legislation from the Goldwater Institute, a small-government advocacy think tank.
The bill did not get a House vote last year. But Kansas is in the middle of a two-year legislative cycle, Hill explains, which means the bill remains eligible for a vote after the Legislature reconvenes in January.
Johnsen wonders what the holdup is. She’s watched as other states, enact aid-in-dying laws and wonders why some Americans with terminal illness are being given the right to die while she’s not allowed the right to try a potentially lifesaving medicine.
“I have a big problem with that,” Johnsen says.
Hill — one of the calmest, most measured speakers in the Kansas Legislature — slowly and carefully explains the political landscape.
The “Right to Try” bill in Kansas is crafted for a narrow set of very sick people, he says. But the fear is that other legislators will try to broaden it with amendments if it comes up for a House vote.
For example, Hill says Rep. Mario Goico, a Wichita Republican, would like to extend access to experimental drugs to patients like his wife, who has late-stage cancer.
Hill says he understands Goico’s position, but some of his House colleagues already were concerned about the medical ethics of the bill. Opening untested drugs to a wider patient population would have increased those concerns.
“It would have no doubt drawn more opposition and controversy,” Hill says. “The bottom line is the speaker — and I didn’t necessarily disagree — decided to hold it.”
‘Protect the public’
In a phone interview a few days later, Goico says he has decided to introduce his bill as a standalone measure next session, rather than as an amendment.
He says he believes the current version of Right to Try proposed in Kansas is too broad because it allows any medical provider with prescribing power to approve their patients’ access to drugs early in the FDA testing process.
He would like a bill that allows only those working at the University of Kansas Cancer Center to prescribe drugs that are well into clinical trials. That would provide more protection for patients like his wife, he says, who is going through her second bout with cancer.
“It seems to me that we need to protect the public,” Goico says. “By being in the last stage of testing, you already know it will work. You know there won’t be any negative consequences.”
Back at Johnsen’s house, Hill says medical marijuana is another issue “playing in the background” of the Right to Try bill. He’s not sure medical cannabis advocates could shoehorn a germane amendment into the legislation, but he’s guessing they could try. That’s an issue with enough political baggage to sink the whole thing.
By the end of the conversation, it’s clear there are varied opinions at the state level about who should be able to access treatments not approved by the FDA — and when.
Johnsen is disappointed but not deterred.
She will keep pushing for Right to Try, but it’s only part of the battle. Even if the bill passes, it still requires a drug company to agree to provide her experimental drugs against the wishes of the FDA.
She’s read about patients still unable to access unapproved medications in other states that have passed similar legislation.
Hill says he’ll keep working on it anyway. More than 20 states, including Missouri, have passed some version of Right to Try. The more states that push back on the FDA, he says, the more pressure there will be for federal changes.
“The action on the federal level, that would be the best fix,” Hill says.
21st Century Cures Act
Earlier this year, Kansas Congressman Kevin Yoder made national headlines as a relatively conservative Republican pushing for more government spending.
Yoder was quick to point out he’s advocating not for an overall spending increase but for a funding bump with a specific purpose: medical research.
He has pushed to double the annual budget of the National Institutes of Health, a federal research agency that has doled out millions in grants to KU medical facilities in his Kansas City-area district.
Increased funding for research that could cure terrible diseases is an economic and moral imperative for the country, Yoder says.
“We know every year 600,000 people will die of cancer,” he said in a floor speech this summer. “We know each year in the United States 700,000 people will die of Alzheimer’s. These are real people, real families that are in anguish over these and many other diseases.”
The legislation Yoder is co-sponsoring, the 21st Century Cures Act, would put more money into research for new treatments and smooth the regulatory pathway for those treatments to get to market.
It would infuse the FDA with about $550 million in cash while requiring the agency to make changes that would speed the approval of new antibiotics and medical devices. The bill passed the U.S. House 344-77 and awaits action in the U.S. Senate.
Supporters of the legislation say it will modernize a ponderous FDA approval process, get treatments to patients sooner and encourage companies to invest in more research and development.
Some have hailed it as a rare beacon of bipartisanship in an increasingly polarized Congress.
But others have asked whether it has bipartisan support not because it’s good legislation but because both political parties have been bought through campaign donations by drug companies and medical device manufacturers who would benefit from it.
The House Energy and Commerce Committee, which originally passed the bill, is chaired by U.S. Rep. Fred Upton, a Michigan Republican who is the House’s top recipient of campaign contributions from the pharmaceutical and health care products industries.
Provisions in the bill that allow the FDA to approve drugs and devices based on evidence outside of randomized, controlled clinical trials drew concern from physicians in an op-ed published in June in the New England Journal of Medicine.
A former FDA medical officer, an orthopedic surgeon and a professor of clinical medicine teamed on a separate op-ed in September arguing that, if anything, the FDA’s current approval process is too lenient and already allows too many potentially dangerous products to market.
John Parisi sits in his downtown Kansas City, Mo., law office and scrolls through a long list of pending multidistrict litigation.
These cases combine similar lawsuits filed across several legal jurisdictions. Some are related to things like airplane crashes or asbestos exposure, but many are related to pharmaceutical drugs and medical devices.
Parisi is a personal injury attorney who, over the years, has represented many plaintiffs who claim they or their loved ones were injured or even killed by prescription drugs or devices.
The current list of pending multidistrict defendants includes the makers of Prempro, an estrogen supplement; the Zimmer NexGen knee implant; Pradaxa, a blood thinner; NuvaRing, a birth control device; and the Biomet M2a Magnum hip implant. More than 1,000 lawsuits have been filed related to each of these products.
Others — like the birth control pill Yasmin, the anti-inflammatory drug Vioxx and several pelvic repair products — have more than 10,000 suits filed against them.
“This is a list of unintended consequences, if you will,” Parisi says.
In some cases Parisi says those consequences were not known prior to the product’s FDA approval, but in other cases the companies involved shaded their research reports to hide evidence of potentially harmful side effects and federal regulators didn’t do enough independent checking.
Either way, patients end up being harmed, he says.
“Once you get the drug onto the market and once you find out it has devastating consequences that weren’t researched, it’s hard to put the genie back in the bottle,” Parisi says.
Drug and device companies are under pressure to get their products to market quickly to maximize profits before patents expire, he says. And the “revolving door” of people going from working for drug and device companies to working for the FDA has created a cozy regulatory relationship.
Parisi has not read the 21st Century Cures Act in detail. But in general he says he believes more FDA funding is a good thing, although quicker drug and device approvals might not be.
“I think we need more scrutiny, not less,” Parisi says. “That would be the bottom line.”
Parisi says he understands the position of people like Johnsen. If he or anyone in his family had ALS, he would want access to new treatments too.
The question is how to create a regulatory environment that provides access to drugs for people like Johnsen who are seriously ill but also provides rigorous oversight.
“It’s a balance, right?” Parisi says. “Where is the fulcrum on that seesaw?”
Back at Johnsen’s house, her meeting with Hill is wrapping up.
Triemer is talking about some of the other people in town who have ALS. The illness seems to be more prevalent in Emporia than elsewhere — or maybe she and Johnsen just hear from more families who are dealing with it than most people.
Johnsen’s willingness to be open and upfront about her illness has made her a magnet for other ALS sufferers in need of support and information about what to expect as the disease progresses.
It’s a role she embraces, with a strength and courage that impresses Hill and others.
Before the visit ends, Johnsen is asked if she has anything more she would like to say about the FDA. She nods. Her mother gets her a drink of water, so she can speak as clearly as possible.
“As far as the FDA goes,” Johnsen says, “I would like to see a little more flexibility and (that) they be more willing to listen to patients and understand that we are able to make an informed choice when we have all the information.”
Andy Marso is a reporter for KHI News Service in Topeka, a partner in the Heartland Health Monitor team. You can reach him on Twitter @andymarso