Hi everyone. Thank you for being here! It’s so great to see this crowd standing up for science. Today, I’m going to tell you what fruit flies and a remote Island in the Pacific Ocean have to do with breakthroughs in health research.
Let me start by telling you a little bit about myself. I do a little of everything. I am an oncologist at the Brigham and Women’s Hospital and Dana-Farber Cancer Institute. I direct a cancer research laboratory at the Brigham. I teach and mentor medical students and fellows. I’m also the faculty director of research at the Brigham, and president-elect of the Harvard Medical Alumni Council. So, I think about the future of science and medicine pretty much all the time.
Today, I’m going to tell you about how science has changed the lives of women with a rare lung disease. But first, in case you have a short attention span, as I do, let me tell you my most important take-home message. Here it is: It’s not possible to predict exactly which research will lead to a medical breakthrough. Let me say it again: We can’t predict what type of research will lead to a breakthrough.
So, back to the rare lung disease, which is called lymphangioleiomyomatosis, or LAM. This disease destroys the lungs of young, otherwise healthy, nonsmoking women. It affects only women, and it often gets worse during pregnancy. Ultimately, many women with LAM need oxygen to walk from one room to another, in their own home. Imagine trying to breathe through a small straw, all the time. Imagine breathing through a straw while commuting to your job, or caring for your children, or attending this rally.
So how did we find a treatment for LAM? It’s a truly miraculous story. First, we discovered the genetic cause of LAM, with NIH funding. Next, scientists at Yale, also sponsored by the NIH, discovered the function of the gene that is mutated in LAM cells. They weren’t studying LAM at all—they were studying genes that regulate the size of fruit fly eyes! The final part of this story began in the 1960s, with studies of molecules in the soil on Easter Island, in the middle of the Pacific Ocean. A drug was discovered that inhibits the exact defect we had discovered in LAM cells, also made possible by NIH funding. But we made the miraculous connection between the Easter Island drug, rapamycin, and LAM only because of the fruit fly data. Two years ago, thanks to a clinical trial supported in part by the NIH, we had the first FDA-approved treatment for LAM, allowing women with this devastating disease to breathe more easily.
So back to the take-home message: it’s impossible to predict which research will lead to breakthroughs. Improving human health requires a broad vision, a vision that is shared by Harvard Medical School, the Harvard hospitals and the NIH. For LAM, we needed scientists studying soil samples and fruit fly eyes. Who knows what is needed for the next breakthrough in asthma, or diabetes, or leukemia?
I’ll end with a small but very critical request. Tell seven people, this week, just one each day, about the NIH. Make sure they know that the N is for National—it’s for everyone—and the H is for Health, for each of us! Make sure they know how proud we are of the medical advances made possible by the NIH. Make sure they know that the next breakthrough in their health, or their child’s health, might just emerge from a fruit fly study sponsored by the NIH.
Thank you. Enjoy this very special Earth Day, where we celebrate science!
Adapted from a speech delivered by Lisa Henske, HMS professor of medicine at Brigham and Women’s Hospital, at the HMS March for Science rally on April 22, 2017.
