With the retirement of Dr. Anthony Fauci after almost 40 years at the helm of the National Institute of Allergy and Infectious Diseases, many have remarked his preeminent role in the struggle against HIV/AIDS, H1N1 influenza, Ebola, Zika, Covid-19 and monkeypox, as researcher, administrator, and public figure. Far less is known, however, about Fauci’s role in helping to transform biomedical research by bringing people living with HIV/AIDS into the research process after ACT UP and other groups pressed him to do so in the late 1980s.

Research on HIV/AIDS is far better as a result. People living with the disease and their advocates have been constructive partners in all areas of investigation—particularly on clinical research. But the scope of community involvement has stretched from basic immunology and virology to drug development and beyond. Community leaders have sat on NIAID and NIH advisory bodies as full voting members alongside scientists from around the country. People living with HIV have a keen knowledge of what is happening in the real world—and sometimes a better perch from which to think about what the important research questions are or should be, how to answer them, and how best to engage patients in the process.

A case in point: In the early 1990s, we approached Fauci about what we were calling “long-term survivors” of AIDS—people infected with HIV who did not get sick. Fauci and the chief of epidemiology for HIV at NIAID, Sten Vermund—now my colleague at Yale—met with us, and once they realized there was a subset of patients with delayed progression to disease, this led to a whole new area of research on long-term non-progressors and elite controllers, opening up genetic, virological and immunological investigations into what influences HIV’s clinical course. This also led to the discovery of the co-receptor HIV needs to get in cells—all because of community engagement with NIAID. Current day work on Covid-19 and monkeypox from groups like PrEP4All are the fruit of similar collaborations, and many AIDS activists are active in work on these new diseases.

But this democratization of research—its enrichment by the voices of those living with the disease—did not happen across the board at the NIH. Some patient groups became professionalized charities, with staff drawing big salaries, despite little true scientific engagement with the NIH. Some cultivated close ties with drug companies, doing their bidding to push for quick approval for not-ready-for-prime-time medicines, as the Alzheimers Association did for Biogen’s Aduhelm several years ago—a drug which had so little evidence to support its use that members of the FDA’s advisory committee resigned in protest when the agency approved it for marketing nonetheless.

However, there are still inspiring stories that need telling. A few years ago, I met a group of people living with amyotrophic lateral sclerosis (ALS) who call themselves the ALS Problem Solvers—also known as the Cog Squad—led by the amazing Sandy Morris. I met Sandy through Cathy Collet, who heard me talk at a meeting at New York University on expanded access to experimental drugs for AIDS. Thirty years ago, AIDS activists were able to get drug companies to offer their experimental agents to patients who had no other options and were ineligible for the clinical trials of these new drugs. Tens of thousands of people got access to these medicines in this way.

When it came to ALS and other diseases, though, drug companies almost never offered expanded access before approval by the FDA. In fact, withholding expanded access was seen as a way for companies to get patient groups to push for approval at the agency—with the companies crying poverty all the way to the bank since they could charge an arm and a leg after FDA gave them the green light. Cathy wanted me to talk with Sandy and her colleagues about all this.

While you could say Sandy was the Larry Kramer of ALS—angry, determined, uncompromising, brilliant—this would be a disservice to her memory. Actually, she was nothing like Larry—except in her determination to do right by her people: those living with the disease. Though we began by talking about expanded access programs, we pivoted quickly to the NIH. Sandy realized that the problem with ALS wasn’t just that the companies wouldn’t offer expanded access programs and the FDA was loath to grant accelerated approval to ALS. It was that the research pipeline on ALS was dry—and that pipeline started at NIH, at the National Institute for Neurological Diseases and Stroke (NINDS).

I asked Fauci to introduce us to Dr. Walter Koroshetz, the NINDS director, and quietly in 2021, during the pandemic,  Koroshetz and his team met with Sandy and her group of ALS Problem Solvers. I was a fly on the wall at that meeting, and it felt like history as Sandy and her team laid out their five cogs—their core demands—which included quite basic requests, such as the establishment of a large, dedicated natural history study and clinical trials network for ALS. Over a period of months, NINDS staff would give Sandy and her group the runaround, making bureaucratic excuses—all things we’d seen some NIH staffers do during our own interactions with them over AIDS treatment 30 years ago. But Sandy kept her focus on figuring out how to break the logjams and get NINDS to act—how to “act up” to save people lives. It is clear to me that NINDS still doesn’t get it—doesn’t understand the central role of patients not as mere subjects of research but also active participants in the design and conduct of basic research—even though, on the same campus at NIAID this insight is old hat, just the way things work and have worked for 30+ years.

The road to better treatment—and eventually, a cure—for ALS doesn’t start at the FDA. That is the end of the pipeline which begins at NIH, where the formative research to develop these agents will emerge. Sandy Morris, like her comrades in the AIDS movement so many years ago, realized this—and was a pioneer in the crusade to open up NIH decision-making processes to people living with ALS. This kind of work is vital, not just for ALS but for all biomedical research. In fact, Sandy drafted the Morris ALS Principles,  modeled on the Denver Principles by people living with AIDS, which set out five key expectations for anyone engaging with the ALS community, but which was meant to be a broader call to action to all patients—no, scratch that, all people living with serious and life-threatening diseases.

Sandy, who was diagnosed with ALS on January 6, 2018, at the age of 51, died this week of the disease. Now it’s up to us to finish her work.