NIH Director Addresses Gathering of Experts on Autoimmune Diseases

NEW YORK—Having the director of the National Institutes of Health (NIH) speak to a room full of systemic lupus erythematosus (SLE) experts about autoimmune disease is preaching to the choir. Having the director of the NIH care enough about autoimmune diseases that he talks about them to other groups, too? Well, that’s conducting a national choir.

“Having an advocate at that [national] level for lupus is invaluable,” says Lupus Research Institute (LRI) President Margaret “Peggy” Dowd. “We know that if we want to move biomedical research in this country, we have to be active. We have to be advocates. We have to have our folks on [Capitol] Hill, which we do every year. And we have to speak with a louder voice.”

NIH Director Francis Collins

It doesn’t get much louder than when NIH Director Francis Collins, MD, PhD, spoke Oct. 19 at Forum for Discovery: The 15th Annual Scientific Conference in Midtown Manhattan.

Dr. Collins, known to most for leading the international Human Genome Project, has likely gained popularity in lupus circles for including SLE in a partnership between the NIH, the U.S. Food and Drug Administration (FDA), 10 biopharmaceutical companies and multiple nonprofit organizations. The program’s goal is to develop “new diagnostics and treatments by jointly identifying and validating promising biological targets for therapeutics,” according to the NIH.

“United we stand, divided we fall,” Dr. Collins said at the event. “I want to stand here this evening and say, NIH wants to be part of that ‘united-we-stand’ circumstance.”

According to Dr. Collins, progress in biomedical research is on a precipice that has never been seen before, thanks to scientific and technology breakthroughs in recent years. But, dichotomously, the resources to push progress forward—“moving faster,” he likes to say—are anemic.

“We are riding an exponential curve of knowledge acquisition right now,” he added. “The paradox of my experience every day is having the sense that science has never been at a more exciting juncture than right now. And yet, at the same time, biomedical research—in the U.S. anyway—has never been in a more stressful circumstance because your chance, if you send a grant to us, of actually getting [your research] funded is the lowest in history.”

As a new way to fund biomedical research for SLE and rheumatoid arthritis (RA), Dr. Collins and his NIH staff teamed with the biggest pharmaceutical developers and nonprofit organizations, such as LRI and the Alliance for Lupus Research (ALR), which are merging, to create the Accelerating Medicines Partnership (AMP). The two autoimmune diseases were in the first cohort targeted by the AMP, along with Type 2 diabetes and Alzheimer’s disease. The funding available for SLE and RA research is $41.6 million, split between NIH and six pharmaceutical companies: Sanofi, AbbVie, Pfizer, Merck, Takeda and Bristol-Myers Squibb.

Kenneth Farber, ALR president, says that Dr. Collins’ support of research for autoimmune diseases—and SLE, in particular—is encouraging because it helps address the three main frustrations of the lupus community.

“Frustration No. 1 is the disease is misunderstood,” he says. “Frustration No. 2 is the disease is so heterogeneous that it becomes very difficult for pharma to begin to develop therapies to redress it. Frustration No. 3, as compared with other autoimmune or inflammatory diseases that impact a similar number of patients, [is] the federal funding and the private funding for lupus is significantly less.”

Enter AMP and its $40 million-plus investment over the next three to five years.

“We were thrilled, delighted, excited and just pumped when we learned that he had decided to include lupus as one of the diseases that AMP would pursue,” Mr. Farber says. “The bottom line is [that] anytime you can tell your story to a person whose opinion really matters—and Francis Collins’ opinion really matters—you’ve done something important.”

For his part, Dr. Collins said it’s important that SLE and RA were taken together in AMP because “recognizing there is some considerable interest in the overlaps between those [diseases], and by working on them together, we would probably make interesting progress.”

He also noted that unlike more abstract academic pursuits, AMP funding is looking for fast results.

“This is not like the typical academic effort, where you give a grant and say, ‘Hope you do something nice; talk to you in three years,’” Dr. Collins joked. “This is very much milestone driven.”

AMP’s RA and SLE research will focus on genes, proteins, chemical pathways and networks involved at a single-cell level, announced the NIH when the program was unveiled last year. Aside from work at the cellular or small-group level, funding from AMP will collect tissue samples from people with RA and SLE for molecular analysis, develop computational tools to integrate data types so molecular pathways can be understood and make the data available to the broad research community for further analysis, The Rheumatologist reported.

“Wouldn’t it be interesting if we could look at what’s going on in lupus at the unit that biology actually uses to carry out things that biology cares about?” he said. “Which is the cell—the single cell. That seems almost unimaginable to somebody like me who, 10 years ago, thought we would be doing really well to look at thousands of cells. But things have changed.”

Dr. Collins said now that the technology exists to see which “genes are on, which genes are off,” lupus researchers have the opportunity to look at the kidneys in ways barely envisioned when his career began. And RA experts can similarly glean insights into the synovium.

“This is a window into what’s going on in the immune system of a kind of precision that has not previously been possible,” he said. “But that means we are inventing and improving the technology day by day as we go along.”

That takes money. More specifically, money the NIH hasn’t had in recent years. Dr. Collins said that over the past 12 years, the NIH has lost 22% of its purchasing power for biomedical research. And although the budget approved in November by Congress and President Obama calls for an increase in NIH funding, the battle for funding never ends, he added.

Although the head of NIH can’t lobby Congress—he has to wait to be invited to testify before lawmakers—he noted that the restriction applies only to him. He exhorted lupus professionals and other physicians to make known their needs for resources, funding and support.

“We all have to figure out how we can use our voices to raise the consciousness of the decision makers about just how important this is,” Dr. Collins said. “All of us should … get over our shyness or our sense of ‘it’s not really appropriate’ and argue positively for what we are about in terms of biomedical research. This is a unique moment in history, whether it’s lupus that you care about or whatever disease. We could be going faster.”

Richard Quinn is a freelance writer in New Jersey.