WASHINGTON — For Frank Mongiello, Jan. 30, 2018 was a day of hope. After dedicating the last few years of his life to the “right-to-try” movement, an effort to get dying patients access to experimental treatments, Mongiello heard President Trump use his first-ever State of the Union address to urge Congress to pass a federal law on the issue.

Just one year later, the law is in place. But Frank, who suffers from ALS — and for whom the national law is even named — still hasn’t gotten access to treatment.

“We had a lot of hope that if the right to try was passed it would give an incentive for the drug companies to make available the drugs. But now it doesn’t seem as though the drug companies are giving away their drugs either,” Marilyn Mongiello, Frank’s wife, told STAT. She speaks for him in interviews because he is only able to communicate via technology that turns his eye movements into text. In a brief email exchange, Frank confirmed to STAT that he has not found treatment but that he remains optimistic, despite his worsening condition.

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Patients and family members like Frank and Marilyn — a half dozen of whom spoke to STAT — described making dozens of unreturned calls to drug makers, outlining plans to pitch the companies on how right to try could be good for business, and even trolling Food and Drug Administration Twitter accounts hoping to drum up some help getting access. Their failures so far underscore just how many questions remain: Did the law change anything, or did it just give patients false hope? Were the detractors who made such critiques right all along?

“It’s confusing. It’s confusing for even people like me who do this day in and day out,” Alison Bateman-House, a bioethicist at New York University who opposed the federal right-to-try law on the grounds that it would give patients false hope and could potentially lead to patient harm. “So I think there’s probably going to be a little bit of churn as everyone tries to figure out what is happening. But I still maintain my conviction that in the long run this is not going to be a useful way to get patients access.”

Those same patients, though, remain hopeful — particularly in light of a recent albeit complicated decision to use the right-to-try pathway to help a California patient get access to a brain cancer treatment. Advocates for the law, like the libertarian Goldwater Institute, say it’s too early to judge the law’s success.

“There is a rush to conclusion here on the part of people who had opposed right to try,” Naomi Lopez Bauman, director of health care policy at the Goldwater Institute, told STAT. “I think you are going to see a lot more activity this first quarter, and it will, I think, accelerate from there.”

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Marilyn’s Facebook page is a living history of Frank’s ALS journey. It chronicles both hardships, like Frank’s 20-day stay in the hospital in October, and triumphs, like when Frank was able to go deer hunting with his father in October, thanks to special technology.

But one post in particular stands out.

“Please email and tweet the Brainstorm CEO. Chaim Lebovitz [sic],” the post, dated June 28, pleads. “Please for the sake of my 6 beautiful children help their dad.”

BrainStorm Cell Therapeutics is the biotech firm behind an as-yet-unapproved treatment for ALS known as NurOwn. It’s currently in Phase 3 clinical trials. Like Marilyn, thousands of ALS patients and family members around the country are banking on BrainStorm for hope. And in June, it seemed to grant their wishes: BrainStorm’s CEO, in an interview with Bloomberg, seemed to promise the company would let patients access its treatment under the federal right-to-try law, but hinted the treatment would cost roughly $300,000.

But less than a week later, BrainStorm reversed that decision. The company’s official reason: being unable to find “alternative funding solutions for patients who are unable to afford the high cost of regenerative medicine cell therapies.” The company agreed to provide just one course of treatment free of charge, for Matt Bellina, another leader of the right-to-try movement who also has ALS and for whom the federal law is also named.

Marilyn contacted BrainStorm at least 10 times pleading for the same treatment, to no avail.

“As Frank’s wife, I just didn’t understand,” Marilyn recalled. “It was devastating.”

While Marilyn said she is thrilled that Matt will get treatment, she has struggled with her husband being passed over, especially given his role in getting the law passed. And she has a point: Frank was seated stage left of Trump when the bill was signed into law, he’s talked health policy with more Washington lawmakers than some of D.C.’s highest paid lobbyists, and the law literally bears his name.

BrainStorm did not respond to multiple requests for comment. The company’s website currently states that it “will not make NurOwn® available under the Right to Try Pathway, at this time.”

There are scant details available about whether Bellina has undergone treatment and how he is responding. He also did not respond to multiple requests for comment.

Opponents of the law say this was all to be expected. The National Organization for Rare Disorders, the nation’s largest rare disease group, vociferously opposed law, largely out of concern that it would only give patients false hope. Prior to the law, companies, if they so chose, could already provide experimental drugs to dying patients via a so-called expanded access pathway run by the Food and Drug Administration. A new law cutting the FDA out of the process, opponents like NORD argued, wasn’t going to suddenly make companies more eager to share their drugs.

“It seems as if it is playing out, in as sense, as we would expect,” Paul Melmeyer, director of federal policy for NORD, told STAT. “To us, it’s not a surprise that companies would not find [right to try] any more appealing compared to the expanded access pathway.”

Frank Mongiello
Frank Mongiello (center) communicates with his wife Marilyn Mongiello and his son during a news conference following the passage of the Right to Try Act in Washington. Manuel Balce Ceneta/AP

The right-to-try movement began back in 2012 in a boardroom of the Arizona-based Goldwater Institute. Advocates argued the FDA’s expanded access program was too slow and burdensome, and that FDA had no business deciding who should and shouldn’t get access to experimental drugs. The idea was politically popular, and by 2017, 33 states had their own right-to-try laws. But nothing catapulted the idea into the mainstream like Trump’s prime-time call for the policy during his first State of the Union address, one year ago this week.

Eight months later the idea was federal law — a rare feat during a hyperpartisan year that claimed the lives of many of Trump’s other legislative priorities.

Advocates for the law, like Sen. Ron Johnson (R-Wis.), who spearheaded it in the Senate, say it’s too early to judge the law’s success.

“I never tried to oversell right to try,” Johnson told STAT last week. “I can’t predict how it’s going to be used, but I think it’s actually really performing the way I expected it to. No dramatic results, but it will save people’s lives and it certainly gives people a little more freedom, a little more hope.”

But Frank Mongiello’s story isn’t an anomaly.

Mike Cimbura was also on stage with Frank during the right-to-try signing. Like Frank, Mike has ALS. He, too, is still waiting for treatment.

Mike’s wife, Nicole Cimbura, called BrainStorm the day after Trump made right to try the law of the land. Like Marilyn, she had no luck. Nicole has called six companies since seeking a treatment for Mike — everything from established biotechs like Biogen to startups finishing up Phase 1 clinical trials. Some companies flat out reject her, others don’t even return the call.

Nicole believes guidance from FDA will give companies more comfort with right to try.

She’s frustrated that the guidance has taken so long. She now frequently tweets at the White House, Trump, and Johnson asking from an update.

“Can you please ask the FDA to get a framework out for Right to Try!  It’s been almost 6 months since the bill was signed into law,” Cimbura tweeted to Trump in November. “You asked Azar & Gottlieb to get in this ASAP, but this dying patients are still waiting.  What is the hold up?”

Sometimes she replies to Gottlieb when he’s tweeting about something unrelated.

“Sometimes I can be a little snarky to try to get Gottlieb’s attention,” she told STAT, recalling when she replied to a tweet about youth vaping — and suggested perhaps kids were trying e-cigarettes “to handle the stress of ALS on their family.”

Others, like Sandy Morris, a 52-year old mother of three living with ALS, think there just needs to be someone leading the charge and explaining why right to try is necessary.

“I don’t blame anyone personally for any of this,” Morris, who is able to speak without the help of technology, said. “It’s certainly not the FDA’s fault and it’s not the drug manufacturers’ fault. It’s going to take education, communication and tenacity. It’s going to take somebody at the top to make the law the law.”

She and five other patients are banding together with the hope of meeting with companies and convincing them that it makes business sense to give out drugs under right to try.

There is, however,  a glimmer of hope, one that all of the patients and advocates were quick to mention in interviews with STAT.

One patient suffering from glioblastoma, among the most aggressive forms of brain cancer, was treated in November under right to try with ERC1671, a drug currently in a Phase 2 clinical trial.

The news puzzled policy wonks. The decision to provide the drug was approved by an institutional review board and the FDA was notified of the move well in advance — two steps that aren’t mandated under the federal right-to-try law and that make this case look a lot more like the FDA’s existing expanded access program than the one set up by the federal right to try.

The doctor responsible for the treatment, Dr. Daniela Bota of the University of California, Irvine, declined an interview with STAT.

“The patient’s family requested access to the treatment through federal and California Right to Try laws,” Bota said in a statement. “I agreed and sought to implement this access on their behalf. It was believed that RTT offered a more expedited path to treatment, which began after meeting regulatory and compliance requirements.”

But those details don’t matter to folks like Frank and Marilyn Mongiello, and Mike and Nicole Cimbura, or Sandy Morris. For them it’s a sign that the cogs in the right-to-try machine are starting to turn, albeit slowly.

“You have to start somewhere, right?” Nicole Cimbura said, adding that she hopes the California case will increase other companies’ comfort with the right-to-try law. “I truly hope that other companies will quickly come on board offering more drugs and therapies. Again, they’re dying. So why not try?”

In October, Frank underwent intubation and a tracheostomy, a life-changing procedure that now means Frank relies on a ventilator the majority of the time to breathe. His personality is intact and he can still communicate using eye-tracking software, but he can no longer keep his head up and he hardly has power in any of his limbs. His physical condition is a far cry from the man who, just three  years earlier, gave an impassioned speech at a congressional press conference about the need for a right-to-try law.

Despite all those setbacks, the Mongiellos still hope their luck will change. “We are taking care of Frank to the best of our ability, we are keeping him germ free, and we are just waiting,” Marilyn said. “We just have hope in our hearts that eventually there’s going to be a cure.”

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  • My son Frank P Mongiello was one of the original signers of The Right to Try. No pharmaceutical company expressed any interest in helping him although he has rapid progression als. So sad. I guess it’s who you know or how much money you can donate. There are thousands in the als community hanging between life and death waiting for cure. Why can’t they be a part of clinical trials? Thru all my readings American pharmaceutical companies are governed by GREED. They’re looking at the easy fix. Erectile dysfunction, sick dick is more important than saving someone’s life with als. American pharmaceutical companies lack the intelligence to find cures for complicated diseases. See the research going on in other countries. It’s no wonder why Americans are leaving their own country to go to a foreign country to receive treatment for their illnesses. Shame, shame American pharmaceutical companies and our outdated FDA.

  • Update: Mike Cimbura has passed away. Matt Bellina remains the only person with ALS granted treatment by BrainStorm under Right To Try; he being granted a chance at longer life because of his personal connection with the CEO.

  • As I said below , RTT helped bring attention to the small, slow, broken ” Compassionate ” Use access to pre- approval drugs. RTT helped reform the FDA . We patient activists have always known that Pharma is a big part of the lack of access and needs reform . Some of the co.’s get it , most do not. Their lobbyists gutted the Andrea Sloan Compassionate Use Act a few yrs. ago. But our pressure is not going away – the 21st century drugs are getting better and better . Just like Nick Auden’s family questioned why he couldn’t get a breakthrough drug like Keytruda ( ” Save Locky’s Dad ” ) and Andrea Sloan’s family asked for Olaparib, now ALS pt.’s are speaking up . Pharma needs to step up and do the right thing morally. They are not making gadgets , they stepped into the field to produce life – saving drugs. I have met many top FDA and Pharma execs . They are not bad people – their geeks are making amazing drugs like no where else in the world. Back in 2010 when the FDA denied my wife a chance at Kadcyla , I was enraged . Couldn’t sleep . I realized that it wasn’t personal , they didn’t even know my wife. It was IMPERSONAL to them . I got many to know my wife and put a personal stamp on it. By the way , my wife , dx. stage iv at dx. in 2006, who should have died in 2011, today is cancer free since 2011, on no tx. Thousands of others could have lived from her drug alone, our friends Jay, Ann, Tracy’s mom … we speak for them .

  • I tried to get a prostate cancer vaccine during phase 3 trials under Expanded access, also called “compassionate use,” before this law passed and the vaccine manufacturer refused to release any. Can they still refuse to supply under the new U.S. law? Pricing not yet decided on; the President of the company (based in the EU) was initially saying his goal was to be under 30 Euros. They do research in California.

    • Manufacturers aren’t obligated to supply drug, but they can if they are so inclined. Unfortunately, many will fear that the FDA won’t look kindly on them working directly with patients and fear regulatory retaliation. I worked in the industry for two decades and so am very aware of these fears, real or imagined. A similar initiative, called Free to Choose Medicine, is being promoted by the Heartland Foundation and establishes a parallel track of development separate from FDA oversight once a drug has met certain safety considerations. A version of it is being used in Japan. You can learn more about it at https://www.heartland.org/search-results.html?q=free+to+choose+medicine.

  • als the ” I’m sorry ” disease. For to long people were told to get their things in order. Than people began to take control of their lives and fight for a cure. Unlike the past people opt for feeding tubes, trachs and ventilators. Their fighting to stay alive. Well why shouldn’t they? It’s not a old man’s disease as it was considered many years ago. People in their 20’s thru their 50’s are getting the disease and now young children are being diagnosed with als. It’s downright shameful and unAmerican that big Pharma has control over life and death. It’s shameful to see a big Pharma CEO make $22,000,000. Have they lost all compassion for another human being? Wait til it happens to them or one of their family members. I can tell you it’s heartbreaking to watch your child decline as the disease progresses. Please don’t let my son Frankie and all those who fought tiredlessly to get THE RIGHT to TRY passed go in vane. My sincere gratitude to Dr. Daniela Bota for her willingness to step out of the box to advocate for her patients to get possibly a life saving treatment. I wish more doctors had her spirit to advocate for their patients. Sincerely, Frankie’s mom, Carol. P.S. the debate over THE RIGHT to TRY is over. IT’S A LAW!

  • ALS is caused by a bacterial infection, Borrelia and at times co-infections and is a curable disease. go to http://www.winningthefight.org.
    For the past 10 years I have been receiving reports from ALS patients who have had stem cell treatments all over the world. They get better but then their disease progresses b/c the Borrelia attacks the new cells also.
    We are also finding that other neurodegenerative diseases are testing positive for infections usually the genus Borrelia but other infections also related to periodontal disease in AD. http://advances.sciencemag.org/content/5/1/eaau3333
    Commercial labs do not have the necessary antigens to test for these diseases.
    There are many clinical trials now underway to treat ND but they will ultimately fail b/c they fail to treat the underlying cause, bacterial infection.
    This is a travesty b/c it will establish a false result dooming stem cell therapy in these cases.

  • Nicholas,
    How much is the high cost of these treatments, like the $300,000 for NurOwn, hampering RTT and, possibly, the FDA’s expedited pathway? Is having a “right to try” moot if the cost is prohibitive and insurance won’t cover it? That’s what I got out of BrainStorm’s statement. What are your thoughts?

  • My wife was the first patient to testify for RTT . Lorraine Heidke McCartin . Before any als Pts had taken up the cause . She was denied a chance at a cure by the activist FDA of pres obamas 1 st term . They denied early approval for kadcyla , shocking the research medical community . Not the only obstruction of that time . See wsj ” FDA flexes its regulatory muscles ” 2011. She got her drug by traveling 450 miles each way from Boston to DC to get one of the few slots in expanded access . RTT brought light to the FDA’s paperwork delays and power with adverse effects data etc. RTT. RTT changed the FDA. Today they are more patient friendly. And flexible . Some companies are opening up more preapproval access . Janssen with myeloma drug Pfizer with Ibrance and more companies . There is much more work to be done with the companies . They can allot much more $ to early access while the FDA must remain flexible and all must listen to us the patient in the fire

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