WASHINGTON — Next Wednesday, a cadre of ALS patients will gather for a protest outside the FDA’s headquarters in suburban Maryland with a clear message: “No More Excuses.”

The rally is being organized by a ragtag group of ALS patients who connected mostly through Facebook, and it’s far less a production than other efforts like the 2014 ice bucket challenge that swept around the world.

These protesters haven’t even established a formal organization or a website; some said they’ve never even been to a protest. There’s nary a poster board in sight, yet.

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But the band of patients is already catching the attention of regulators at the Food and Drug Administration, establishment advocates at the ALS Association, and top bioethicists around the country. Top Senate lawmakers, too, are beginning to advocate for their cause.

These protesters say, at least, that they plan to emulate the aggressive, even “radical” approaches and the take-no-prisoners strategy of the AIDS activists who protested the FDA’s slow work on that disease in the 1980s. Like the ACT UP activists before them, the ALS patients are coming armed with a list of specific therapies they want regulators to approve — and fast. But unlike their predecessors, they’re hoping not to be arrested next week.

The ALS patients are fed up: They had hoped that the $115 million raised from the viral ice bucket challenge would be poured into promising research. They’ve seen no results from their advocacy for the national “right-to-try” law that promised access to new cures. And they’ve felt left out as the FDA approved treatments for other similarly fatal neuromuscular diseases.

“We as ALS patients are just tired of no action being taken on our part,” said Mike Henson, one of the organizers. “I don’t know what’s worse, getting ALS 10 years ago and just knowing you’re going to die or getting it now and knowing that you could be saved if only people would take this seriously.”

Henson lives in Tulsa, Okla., and was diagnosed with ALS last year. He is a former logistics manager for a rock casino who speaks his mind, calling ALS “a murderous son of a bitch of a disease.’’ He’s made a name for himself in the ALS community by starting his own ALS talk show on YouTube, complete with a chyron and green screen.

Just Wednesday, the protesters adopted a name for their troupe: “Contagious for A Cure.”

And there’s a specific potential cure they’re seeking — or rather, for now, there are three. A trio of drug companies is in the middle of the development process for separate, still-investigational ALS drugs: BrainStorm’s stem cell treatment NurOwn, Collaborative Medicinal Development’s synthetic molecule CuATSM, and Dr. Stanley Appel’s cell therapy known as Tregs.

The drugs are all at quite different stages of clinical development: BrainStorm is currently enrolling a Phase 3 clinical trial, Collaborative Medicinal Development has only completed Phase 1 testing, and Appel’s Tregs has only been studied in three patients.

For those knowledgeable about ALS, it feels like deja vu: ALS patients have demanded access to BrainStorm’s NurOwn throughout the fight for a federal right-to-try law. So much so that the company’s CEO Chaim Lebovits promised that patients would be able to access the drug once right to try was law, only to largely go back on that promise and instead provide access to only one right-to-try activist, Matt Bellina. (BrainStorm declined to comment for this story.)

Henson and his fellow organizers want to know why these drugs aren’t being fast-tracked through the FDA’s approval process, especially while drugs for other neuromuscular diseases are being approved, seemingly left and right. He points to Zolgensma, Novartis’ gene therapy for spinal muscular atrophy, which was approved based on a trial of only 15 children — albeit with impressive results.

“That drug was approved very quickly with a very small trial,” Henson said. “So, we know this can be done, if we have the will to do this.”

The FDA said it “stands ready to help” the ALS community — including by helping patients and their doctors evaluate options like clinical trials or using the FDA’s expanded access program to get ahold of experimental, unapproved treatments. A spokeswoman also said the agency “is prepared to use all expedited development and approval programs at its disposal” to make new treatments available.

Two top officials in the agency’s drug approval centers, Janet Woodcock and Peter Marks, also offered to meet with advocates and drug makers, so long as the drug makers would also participate.

“We encourage you to reach out to each of the manufacturers to request a meeting with the agency that includes you and any other members of the ALS community that you would like to include. With their permission, we welcome the opportunity to have a transparency discussion about their investigational products,” Woodcock and Marks wrote in an email shared with STAT.

More and more, cries for more progress are coming from Capitol Hill as well. Republican Sens. Ted Cruz (Texas), Marco Rubio (Fla.), Mike Braun (Ind.) and Mike Lee (Utah) wrote to acting FDA Commissioner Ned Sharpless last month making a similar demand.

The senators are calling for the FDA to bring back the so-called parallel track that the agency unveiled for AIDS patients in the 1990s. Under that pathway, which was never extended beyond HIV and AIDS patients, those ineligible for clinical trials could gain access to certain drugs still being studied. It’s estimated that thousands of AIDS patients got access to one such drug, Stavudine, under the parallel track in the 1990s.

“It is time for the FDA to create and encourage an environment that promotes patient choice, patient access, and patient affordability,” they wrote. “Expansion of the Parallel Track is a proven solution to do just that, and we strongly encourage the FDA to act immediately on this issue.”

It’s a perplexing request. In the years since it created the parallel track, the FDA has stood up several more concrete pathways for patients to access experimental drugs.

The FDA isn’t the only target of these protesters. The group also plans to protest the ALSA. They want answers as to why the $115 million raised by the ice bucket challenge hasn’t gone to fund development of their three favorite potential therapies. ALSA has technically funded two of the therapies, although the protestors insist their funding was not nearly enough.

“What we just can’t figure out is why ALSA is not vigorously pursuing getting any of these drugs into our bodies immediately!” the organizers of next week’s protest wrote to ALSA.

They’re so frustrated with ALSA that they’re calling for the resignation of the group’s President and CEO Calaneet Balas.

In a statement to STAT, ALSA said it was “working urgently to end ALS,” which includes funding “many early stage clinical trials and work[ing] with the FDA and scientists to help trials run faster.” An ALSA-sponsored report released Tuesday also found that the ice bucket challenge resulted in ALSA increasing its research funding by 187%. Among the achievements from that research: discovery of five new genes potentially tied to ALS.

But the protestors want more specific commitments from ALSA to work with BrainStorm, Appel, and Collaborative Medicinal Development “with the ultimate goal of having any of these viable therapies available to the ALS community within the next 6 months maximum.”

Henson is convinced these three treatments — either alone or in some combination — are the cure to curing ALS.

“They’ve been proven,” he insisted.

But experts aren’t so sure.

“These people are desperate, I can see that. I see them every day, it’s a horrible disease,” said Dr. Jonathan Glass, the director of Emory University’s ALS Center. “But fast-tracking things that don’t work, or may not work, or might even be harmful is not the way to go.”

Glass argues some of the treatments these patients have latched onto show promise. He had particularly kind words for Appel’s theory, but he insisted it was far too early to allow access to any of these drugs. He also strongly disagreed that any parallels could be made to Novartis and subsidiary AveXis’s Zolgesma, or even the more controversial Biogen drug Spinraza.

“This is not what we are seeing with spinal muscular atrophy,” Glass said. “The reason AveXis and Biogen can get their drugs approved is you take a universally fatal disease and you actually not only stop it from being fatal, but you watch these kids get better. If we had a drug like that for ALS, great, let’s approve it. But we don’t, and none of those drugs are going to do that.”

And Glass isn’t the only one increasingly uncomfortable with next week’s protest.

To the organizers of next week’s protest, former ACT UP members like Gregg Gonsalves, are celebrities.

“They are definitely our role models,” said Annie Swartz, another organizer of next week’s protest, referring to ACT UP. “They showed us how to kick ass, and how to get treatment, and how to speak for those that might not speak for themselves.”

But that’s an uncomfortable comparison for Gonsalves, who is now a professor at Yale and a MacArthur Genius Fellow. Now 30 years removed from the day ACT UP stormed the FDA’s campus, Gonsalves maintains that the group’s early anger toward the FDA was misdirected.

He laments the fact that the group’s message has been co-opted — bastardized, as he put it — by groups urging deregulation at the FDA. He fears that pushing the FDA to allow access to largely unproven treatments will stifle the research that could eventually lead to a cure for ALS and countless other fatal diseases.

But Gonsalves also understands the anger, pain, and most importantly the relative deprivation of seeing friends and family die from a disease that seems to be altogether forgotten by Washington bureaucrats. He, too, understands the desire of patients to examine how the organization purporting to represent them is spending its money. He’s squeamish about telling patients not to organize.

In his words: “They should be angry, they should be acting up.”

He hopes that the protestors read a bit more of ACT UP’s story —  the parts where they shift their focus away from the FDA toward other targets like the National Institutes of Health. And he maintains there’s plenty of potential targets for the ALS patients’ anger, including insurance companies that deny necessary care or members of Congress who balk at funding basic research.

“I’d do exactly what we did in HIV, I just wouldn’t make the same mistakes we made with 30 years of hindsight,” Gonslaves said. “As we dug deeper, our targets changed. … Hopefully people living with ALS can learn those lessons quickly.”

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  • Excellent article. Thank you, Nick Florko. I hope you come to the protests. We will have signs. We have studied Act-Up. Read the Book: And the Band Played On. We’ve even gotten counsel from former Act-Up Members. What I do not understand is Gonsalves’ comments about the FDA. Act-Up’s activism against the FDA led to the approval of AZT- Still controversial today. However, it got drugs in the bodies of people dying from HIV/AIDS. Moreover, larger policy changes were made at the FDA including PDUFA- Prescription Drug Users Fee Act, Expanded Access and Parallel tracking. I am hoping that Golsalves just does not understand the plight of ALS with the FDA. We’ve asked for an ALS guidance document to develop drugs for ALS for over 5 years. We met with them in February 2019 and were promised it would be delivered in April 2019. We are still waiting. 2 of the 10 members at the meeting in February have died. The FDA requires placebo-controlled trials, for other diseases placebos, are considered unethical but NOT for ALS. My friend participated in the Nurown BrainStorm stem cell trial, her ALS progression STOPPED and she’s not the only one there are dozens of others( Dr. Glass has no idea what he’s talking about. He epitomizes what we are up against – many arrogant doctors with God-Like complexes who have failed at finding a solution to ALS so they say it can’t be done. ) My friend can no longer get access to Nurown and 6 months after her last dose, her progression has started again. We asked BrainStorm to continue to treat her as it is unethical to not treat someone who responds. Brainstorm says they do not have the funds to do so. That is unacceptable. Sorry Brainstorm, you are letting those in your trial die and progress when you know they are responders. In oncology, responders always continue on the therapy, why is this not the case in ALS? The FDA, the doctors, the ALS Association and Brainstorm are all failing us. Please join our fight. Help us!

  • As a cancer pt. activist we have walked this path. We protested outside the FDA and where FDA officials spoke . We understand the frustration of having s fatal disease and seeing treatments right there but out of reach . Know that others don’t get it – press , govt and yes pharma execs , doctors even . Even ACT UP a activists who have got theirs and forgotten where they came from . Fight On . Remember it’s not personal they weren’t trying to kill my wife years ago. They didn’t even know her . But their policies , views were killing her , so we Spoke Up . She got her experimental drug and is alive . But so many never got the chance. We are Dying and know it , all we ask is for that Chance

  • Those of us, in the USA, who suffer from one of the neuro-degenerative diseases, such as Amyotrophic Lateral Sclerosis, or Multiple System Atrophy, or Lewy Body Dementia, Alzheimer’s Disease, or others, know, only too well, that the urgency of needed research, and a breakthrough in treatment, that can slow, stop, or cure neuronal death/degenereration, would be advantageous, because, what’s at stake, is a person’s life span. It’s shattering to a person, when they are told by a neurologist, that they have an incurable disease, and the prognosis is in the single digits, many times. Patients need to hang on to hope! Hope, that a treatment will be discovered soon, that researchers in this great country, are funded, so that, perhaps, a cure could be discovered! Clinical trials of experimental, newly discovered treatments, need to be funded! Government allocated funds, for research, need to go, not only to studies on cancer, diabetes, heart disease, and many other ailments, but, also, to research into incurable neuro-degenerative diseases!

  • What is unfair is having ALS and not even given a chance at a potential therapy. What is unfair is that the FDA has the same standards for ALS as they do for developing a drug for other disease states. What is unfair is that Dr. Glass and others refuse to support the ALS community and challenge the status quo. Shame on you and Dr. Glass. Come walk( or should I say ride in my wheelchair) shoes.

  • There is a big difference developing therapies when you know the exact cause of the disease and when you just don’t. The comparison this Group is making to other neuromuscular diseases is unfair and misdirected. Great Quote from Dr. Jonathon Glass.

  • It’s time to do more. It’s been decades with little progress in halting or slowing down this disease.
    Please go and support this protest and cause if you can. Writing emails is another way to participate.

  • Nurown by Brainstorm has shown to halt and improve ALS. The person mentioned in this article, Matthew Bellina, got the treatment under “right to try”
    and has publicly shown remarkable improvement in strength and muscle function.

  • EXCITING Article but could someone take a moment to explain what the letters ALS stand for? Some folks do not speak you code. A lmost L ike S cience? A lways L ove S ugar??

    • Amyotrophic Lateral Sclerosis (also referred to as Lou Gehrig’s disease, a famous baseball player who was diagnosed with ALS)

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