Her healthcare marketing career cut short by a diagnosis of ALS, Lisa Stockman-Mauriello suddenly found herself running a campaign to save her own life. Leveraging professional experience, she went on to challenge biotech’s decades-old approach to barring access to experimental drugs. 

And thanks to high-profile advocacy waged on her behalf, Stockman-Mauriello won one last advocacy battle before ultimately succumbing to the disease on Wednesday. She was 52.

“Her story leveraged a well-oiled PR machine to execute a very effective campaign to educate people about lack of access,” said a patient advocate from the ALS community, which has seen other big publicity efforts, like 2014’s ALS Ice Bucket Challenge. “I’ve not really seen much else like it in such a short amount of time.”

Those in pharmaceutical marketing knew Stockman-Mauriello as a highly respected communicator. Starting in the mid-1990s, she worked her way up the career ladder in the world of healthcare PR, first with Burson-Marsteller. At 25, she became the first employee at Chandler Chicco Agency, one of the earliest boutique health firms. She went on to helm CCA’s Biosector 2 shop, along the way launching big drug brands and spearheading multimillion-dollar campaigns. 

Eventually CCA became part of the Syneos Health network. Stockman-Mauriello rose to president, diversified communications services, with responsibilities spanning 

PR, medical communications and branding services. All of that came to a screeching halt earlier this year. 

After noticing some speech difficulty, she was diagnosed in January with bulbar ALS, a particularly fast-moving form of the fatal neurodegenerative illness. It affects about 25% of the estimated 14,000 to 15,000 Americans living with ALS, according to the Johns Hopkins School of Medicine.

There were no approved disease-modifying therapies, but drugmaker Biogen happened to be developing a targeted gene therapy that had helped some people with the bulbar form – also known as SOD1, after the genetic mutation carried by these patients. A late-stage clinical trial was about to begin. 

There was just one problem: Biogen had stopped accepting people for its Phase III study a week prior to Stockman-Mauriello’s diagnosis. 

The family requested Biogen open a compassionate use program for those who didn’t qualify for the trial. Meanwhile, Stockman-Mauriello was trapped in a nebulous window, between the shuttered gates of the clinical trial and some unknown point in the future when the drugmaker might see fit to open the doors again for her and others. 

“It’s the worst window to be in,” her husband Bob Mauriello told MM+M at the time. 

Thus began a high-profile advocacy campaign to gain access to the Biogen drug, tofersen, which Stockman-Mauriello and her physician felt could help stave off the disease’s rapid progression and extend her life. Starting on social media, grassroots efforts resulted in more than 100,000 people signing her Change.org petition

The family staged live demonstrations that attracted local news coverage. Momentum spread, and major outlets such as People, The Boston Globe, Bloomberg and CNBC soon picked up the story. Her plight garnered congressional and senatorial support from such lawmakers as Senator Cory Booker (Dem.-NJ) and New Jersey Congressman Tom Malinowsky. 

“If this were a PR campaign from Lisa’s former life, we would have spent six figures from all the pro bono,” her husband mused. “We keep thinking this happened to Lisa because she really is uniquely positioned to get this message out thanks to the relationships she developed over the past 25 to 30 years.”

But while the campaign gained traction, convincing Biogen was another matter. The company at first dug in its heels, saying that offering tofersen outside the trial “would risk failing to complete the study and risk failing to obtain access for all SOD1-ALS patients,” according to company R&D chief Al Sandrock. CEO Michel Vounatsos wrote that Biogen was “exploring all options.”

Meanwhile, Stockman-Mauriello’s disease was progressing rapidly relative to other ALS patients. In February, she was seen on video dancing with her sons. By April, the disease robbed her of her ability to talk, walk and type. Stockman-Mauriello could only communicate via signs and, with the aid of assistive technology, by using her eyes.

Then in late April, Biogen relented somewhat, agreeing to provide tofersen to SOD1 patients under an expanded access program. But again, there was a catch: The drug wouldn’t be available until July, when the company’s late-stage study of tofersen was set to wrap up and trial subjects would be switched from placebo to the active drug. 

It was more a compromise than actually giving the family what it needed, which was access months earlier. But it was still significant, advocates said. 

“Nothing like this has ever happened in ALS,” wrote Nicole Cimbura, an ALS advocate who lost her husband to the disease, of the decision, which would provide expanded access for SOD1 patients worldwide. “It’s a watershed moment that needs to be recognized. The answer has always been ‘no’ for various reasons.”

The move might have contributed to some people with ALS receiving tofersen faster than they would have otherwise. But the delay was deeply frustrating to those patients who didn’t have the luxury of waiting. 

“For us, this is a death sentence because we will likely not make it to mid-July,” Stockman-Mauriello’s family wrote at the time. “And even if we do, we will be in such bad shape that we may not see any benefit.”

Stockman-Mauriello did make it to the expanded access point, finally receiving her first infusion of the drug in mid-July once the Phase III trial wrapped up — a mere week after the expanded use program began. But as predicted, by then there wasn’t much functionality left to lose. Stockman-Mauriello required a ventilator to breathe and had lost movement in all but one leg. 

Several weeks later, less than a month after her 52nd birthday, she succumbed to the disease. Patient advocates credit her with shining a spotlight on the inequity of ethical discussions surrounding access to experimental therapies, which remain the only hope for these patients.

“Lisa’s story and her effort elevated and magnified our efforts to educate people about expanded access and the fact that there are hardly any such programs in ALS,” said Phil Green, who co-chairs the clinical trials team at I Am ALS, a patient-led advocacy group. 

In addition, by increasing visibility around the drugmaker’s refusal to grant patients with the SOD1 gene mutation earlier access to the drug, Stockman-Mauriello “gave us a really compelling story about why we need expanded access and why we need to make sure that any trials moving forward have a parallel expanded access program,” Green added.

Drugmakers often view requests like this one as a threat to retaining patients in a trial. If a patient knows they can get access through compassionate use, they argue, it might affect a volunteer’s willingness to sign up for a study or — for those already enrolled — to stay enrolled. Some might demand to know whether they’re in the placebo arm.

But patients have argued that this is not the case. Stockman-Mauriello, for instance, was willing to be randomized in a compassionate use study, giving her the same odds as anyone in the trial to receive the medicine or the placebo. This, she said, allowed Biogen to avoid ethical questions, plus at least some patients with the rare form of ALS might benefit. 

I Am ALS, Green noted, is working to ensure that such considerations are addressed in trial consent forms moving forward. This would minimize the chance a biopharma company would refuse access to a treatment on ethical grounds while someone on the trial is receiving placebo.

Among Stockman-Mauriello’s career accolades, she was inducted into the MM+M and PRWeek Halls of Femme. She also helped create the curriculum for a health communication certificate program at her alma mater, the University of North Carolina at Chapel Hill, due to launch in 2022.

When people think of Stockman-Mauriello’s contributions to advocacy, ratcheting up the pressure on Biogen and compelling it to accelerate compassionate access to people with the more severe form of ALS is a memory that will endure.

“This was an unprecedented step for ALS patients, so I don’t want to downplay the achievement made and Biogen’s concession. We’re thankful for that,” Bob Mauriello acknowledged.

However, the clinical trials system is broken in the sense that it remains easier for someone to schedule a doctor-assisted suicide than to receive access to experimental drugs that could improve quality of life, he pointed out. “When you can do that but not get access to drugs like tofersen, there seems to be something wrong.”

Her legacy, Green summed up, will be in “not backing down from the biopharma giants, forcing their hand and creating enough public support and pressure for them to rethink their policy.”

In addition to her husband, Stockman-Mauriello is survived by sons Scott (22), Luke (19) and Dean (15);  a sister, Julie Stockman; and her father, Dennis Stockman.