ALS advocates say criticism of new drugs misses bigger picture

A diagnosis of ALS has long been seen as a death sentence.

But in recent years, progress in the world of ALS research and drug development has come to embody a conundrum with far broader implications: The balance between moving aggressively on promising new cures and guarding against false hope.

With the Food and Drug Administration expected to decide later this year on the latest in a series of contentious ALS drug approvals, the stage is set for yet another debate about what drugs make sense for patients to try — and for taxpayers to fund. But Brian Wallach and Sandra Abrevaya, the founders of the advocacy group I Am ALS, don’t see it as a particularly difficult balancing act.

“We know that these treatments are not cures, but they are important steps forward that give people real hope, and give people the chance to live longer and better lives,” Wallach said on Thursday at the STAT Future Summit, speaking over Zoom from his home in suburban Chicago.

Since Wallach’s ALS diagnosis in 2017, the couple has spearheaded arguably the most successful patient advocacy campaign this century. Since founding I Am ALS the following year, the organization has helped push through a series of government investments in ALS research, culminating in a $500 million bill that President Biden signed in late 2021.

“Brian and Sandra are joining us today virtually — I say hi to you both — because they turned their pain into purpose,” Biden said during a bill signing ceremony in late 2021. “They were told that it’d be hard, and there’d be too many obstacles … but they never gave up.”

In the years since, Wallach and Abrevaya have also become closely engaged in advocacy on behalf of several promising new ALS therapies that offer hope to a patient population desperate for a cure, even if the evidence behind them is mixed.

The Food and Drug Administration is set to convene an advisory panel on Sept. 27 to consider the approval of NurOwn, a controversial bespoke cell therapy that the agency had previously refused to consider.

Late last year, the FDA approved Relyvrio, a medicine made by Amylyx Pharmaceuticals that was shown in a small trial to moderately slow the progression of ALS. And in April, the agency granted accelerated approval to tofersen, a Biogen drug that is the first to target a genetic root cause of the disease.

Wallach acknowledged, however, that while any disease space without existing treatment options is an opportunity for biomedical science to help save lives, it also risks inviting profiteers seeking to sell interventions that may not be safe or effective. But he and Abrevaya took pains to differentiate ALS from other areas where profiteering firms have sought to capitalize on patients’ desperation.

“The ALS community does not feel that they’re being taken advantage of,” Abrevaya said. “They’re so grateful for those small companies that have entered this space, because unfortunately, we haven’t seen many of the big players enter the space, and that’s actually been really hurtful to us.”

She added, too, that federal regulators themselves have expressly argued the need for regulatory flexibilities not seen in a typical drug development and clinical trial process.

“If we don’t embrace the flexibility that FDA themselves wrote is needed for this disease, then it’s possible that we’re going to be denying promising therapies to people,” Abrevaya said.

Abrevaya and Wallach’s impassioned but nuanced espousal of new ALS drugs working their way through government approvals is emblematic of arguably their strongest trait as patient advocates: their decade-plus in Washington.

Prior to being diagnosed with ALS, Wallach worked as President Barack Obama’s political director in New Hampshire before working as a White House lawyer and assistant U.S. attorney. Abrevaya, also a trained lawyer, worked as press secretary to education secretary Arne Duncan before taking on a role in the White House communications office.

Though the couple’s name is most closely associated with I Am ALS, in early 2022 they also announced the founding of a for-profit company, Synapticure, which helps connect patients newly diagnosed with ALS and other neurodegenerative diseases to health providers and other specialized resources.

For all their advocacy wins, Abrevaya was frank not just about the emotional toll that a disease diagnosis can exact on family members, but also about the profound financial toll that Wallach’s disease has taken.

“The experience of navigating these illnesses affects the entire family … it was given to Brian as a death sentence, and to me, frankly, it felt like imprisonment,” she said. “Brian’s caregiving costs upwards of $300,000 a year, out of pocket, no insurance coverage, and the only way we’ve been able to manage it is by friends and family pitching in.”