The basic premise
American titans of industry are approaching the business of medical philanthropy just as they do their day jobs: in a big way. It’s not unusual to hear of gifts in the hundreds of millions of dollars, if not the occasional one with close to yet another digit to the left. The inevitable institutes and research centers named for these benefactors grace the campuses of major universities or stand alone in our cities. But I’m not convinced this trend is wholly a good one.
My reason: some of these gifts come with a catch, although their founders would no doubt refer to it as a cause. The catch is that the research carried out in the new institute or center should focus on understanding—and, more important, helping to cure—cancer, or psychiatric disease, or diabetes, or autism, or some other specific affliction. To be clear, of course I believe in the idea of using the work of biologists to eliminate some scourge of modern existence: the promise of doing so underlies the commitment that society makes in funding research. And many scientists feel a moral obligation to use their findings to relieve suffering. But these institutes might not be the most efficient means to achieve this goal (with the possible exception of institutes that target global diseases with histories of woeful underfunding).
Maybe I’d feel better if John Q. Billionaire had watched in sadness as his pet zebrafish was moved into hospice care after her last cancer chemotherapy failed, or if he saw his family’s beloved nematode institutionalized upon making threats to harm himself and others, or if he became disheartened by the multiple daily injections his favorite yeast cell needed to maintain her blood sugar. For in these cases, John Q. might well endow an institute devoted to basic research on model organisms. And then this philanthropist’s billion-dollar commitment might result in even more good.
Multiple counterarguments could be made in support of targeting specific diseases. For one, adding the extra boost to biomedical research from philanthropy dollars doesn’t diminish the funding for basic research available from the NSF, NIH, and other agencies. For another, these new institutes generate important new data that may benefit the entire community. A third argument is that even an institute centered on a single disease spreads around some of its money to target more fundamental aims. Finally, these institutes provide employment for legions of biologists, support staff, vendors, and the like—jobs our community should be happy to see become available.
But I’m not persuaded.
There remains much we don’t understand—about how genes interact with each other and with the environment; how a brain is wired; how development can go awry; how cells commit to divide; how protein aggregates form and become toxic; and how lots of other biological stuff happens. We still don’t fully understand how even a simple repressor binds to DNA and responds to the presence of a sugar. The best way forward, then, is to figure out as much as we can first in the simplest creatures amenable to easy, cheap, rapid, comprehensive experimentation. And these creatures have sophisticated genetics and powerful technologies available for their analysis.
Just as the cell cycle became clear in yeast and cell death in C. elegans, just as DNA replication and repair were dissected in E. coli, and just as developmental mutants of Drosophila shed light on embryogenesis, many breakthroughs await discovery in existing and emerging model organisms. But these discoveries won’t happen if too many of our research dollars—whether public or private—are funneled into translational research. They won’t happen if our best grad students and postdocs choose positions in shiny new disease-focused institutes. They won’t happen if we devote enormous resources to sequencing more individuals, analyzing their transcriptomes, proteomes, metabolomes and other -omes, and generating mammalian cells with precise disease-causing mutations—at the cost of failing to acquire fundamental knowledge about exactly how cells work from research on tractable organisms.
Are there diseases understood well enough that aggressive translational research efforts on them are worthwhile? Certainly. Are there rare disorders for which targeted analyses are justified? Sure. But these do not constitute a compelling rationale to pour vast sums prematurely into research better done by pharmaceutical and biotechnology companies.
How do we convince John Q. and his fellow patrons that the greatest progress in medicine often ensues when they give money not to seek cures for a single disease but to fund studies relevant to all organisms? We must connect our goals as biologists to their goals as philanthropists. We need to find common values between scientists and non-scientists, including awareness that fundamental knowledge about cells and organisms benefits our search for cures for many diseases. We have to play the long game: while maybe antithetical to those impatient for a cure for their own afflictions, it may well be the fastest path to the therapies we seek.