Let’s start with a little history: In the middle of the 20th century, the National Institutes of Health (NIH) was born, coincident with the dawn of the molecular approach to understanding medicine and human physiology. The combination spawned a nationwide explosion of grant-based research by a new breed of “clinician-scientists”.
At Massachusetts General Hospital, a combination of NIH funding, operational hospital support and philanthropy has since made ours the largest hospital-based research enterprise in the U.S. In fact, we devote more than a half-billion dollars annually — more than any U.S. hospital — to the work of more than 2,100 scientists in biomedical research. It is a massive investment that comprises more than a quarter of the hospital’s annual operating budget. We’ve reaped vast rewards as a result: untold numbers of laboratory discoveries have translated into a better understanding of how the human body functions — and malfunctions. That knowledge, in turn, translates into new and improved ways to advance patient care.
Our scientists have led the way in defining how research is conducted. Until recently, it was organized around specific organs or diseases, so we had cardiology research, kidney research, skin research and so on; and individual chiefs directed research in each of their respective areas, largely informed by their clinical work with patients. But in the last 20 years, all that has been upended.
Now, the laboratory informs the clinic like never before. We are using chemistry, biochemistry and protein chemistry to develop diagnostics to understand how cells misbehave in disease. Then we translate that new information into specific, practical clinical tools like drugs and devices. Thus the value of a basic research enterprise inside a hospital, as we have here at MGH, is incalculable. It was a Mass General scientist named James Gusella, for example, who traced the gene for Huntington’s disease to a particular chromosomal region — the first time a link was made between a disease and its genetic underpinnings — sparking the Human Genome Project, which in turn continues to inform medicine and science. The whole movement towards personalized medicine — tailoring the use of drugs to a patient’s unique genetic makeup — is an outgrowth of basic lab investigations of how our genes function and malfunction. And those are only a few examples.
Thanks to other discoveries at MGH, surgeons can transplant human organs without the need for immunosuppressive drugs. Our clinicians can better manage the treatment of cancer patients and use a simple test to monitor the presence of tumor cells in the blood. Advances in imaging have made diagnoses quicker, easier and more accurate.
Then, just in the last decade, we’ve not only witnessed the evolution into multi-disciplinary science — we’ve been major actors in it. In what we like to call Big Science, teams across departments and disease areas work together to tackle wide-sweeping scientific challenges whose solutions hold tremendous promise for a spectrum of diseases and conditions. That trend signaled a major transition away from “siloed” organ- and disease-based research. We established the five Thematic Centers, focused on regenerative medicine, computational and integrative biology, systems biology, photomedicine and genetics. This cross-fertilization approach exists throughout the hospital and in fruitful partnerships with places like the Harvard Stem Cell Institute and MIT.
Our point is this: the translation of lab discoveries into practical applications can only be done in an environment in which scientists have the ear of clinicians, and vice versa. That two-way street is absolutely essential to advancing human health. Without our research enterprise, we would be a hospital offering the standard of care — yet, unfortunately, the standard of care often isn’t good enough. Encompassing research within the clinical setting creates the opportunity for bench research to be translated effectively and rapidly to patient care.
The world’s best research scientists have a home at MGH and put their brain power to work in the realm of medicine. Indeed, two of the last three Lasker Prizes, commonly known as the ‘American Nobel’, have gone to Mass General researchers Jack Szostak, PhD, and Gary Ruvkun, PhD (see stories). This fall, Dr. Szostak received the Nobel Prize in Physiology or Medicine for his discovery of chemical “caps” on the ends of chromosomes that are key players in aging and the growth of cancer.
But one of our biggest challenges is financial. The existing funding framework for scientists is failing our best and brightest young investigators and preventing innovative ideas from being leveraged in the lab. Most researchers fully depend on NIH grants to sustain their salaries, and they spend more than half their time applying for these grants, also known as ‘soft money’. Young investigators need to be relieved of this aspect of their work so that they can focus on the science, but that can only happen if they have a platform of ‘hard money’ to stand on. Monies devoted to helping young people explore new areas in an unencumbered manner not only generates great discoveries, but, in turn, a huge payback in future funding from NIH.
That’s why philanthropy must play a more central role in the MGH research enterprise moving forward.
Let us belabor the point just a bit more. NIH is conservative, tending to fund scientists with a track record and only those projects with a proof-of-concept — not the riskier, outside-of-the box ones. Yet the most innovative time in a scientist’s career is typically in his or her 30’s, when the scientist hasn’t yet built a large research funding base. As a result, these ideas, and the scientists behind them, aren’t getting the infusion of funds they need to even achieve that initial proof-of-concept. In addition, even innovative ideas from scientists with proven track records are overlooked.
Yet if you look back on the biggest breakthroughs in science, they all emerged from what was originally a speculative, utterly risky idea. That is why we need to provide unrestricted seed money to our scientists — so funds can be shuttled quickly to the most urgent projects. This will assist in bridging the gap and help them get their ideas off the ground. For donors, I couldn’t imagine a more exciting opportunity.