to the Vanguard
By Edward D. Miller, M.D.
One of the hardest things in this job is staying focused on the future: Where is medical science going? What new developments should be riveting our attention? I was reminded of this by recent publicity surrounding the 50th anniversary of Crick and Watson's discovery of the double helix-the structure of DNA. It was a seminal event, and yet unraveling the mysteries of the human genome might still be in its infancy without the work of two Hopkins giants, Victor McKusick and Daniel Nathans. It's not surprising that when we created an Institute of Genetic Medicine to consolidate Hopkins' diverse activities in this exploding field, we named it after Victor and Dan.
Twenty-five years ago, Dan Nathans, along with colleague Hamilton Smith, won a Nobel Prize for using restriction enzymes as biochemical "scissors" that could cleave DNA at predictable sites. Out of their basic laboratory research came an indispensable tool for molecular biologists that made possible the Human Genome Project and the biotechnology industry.
Victor McKusick, meanwhile, had been paving the way at the bedside for the genetic revolution. His work over four decades with Marfan syndrome, dwarfism, inherited diseases among the Amish and the first reference book of inherited disorders (he continues to update "Online Mendelian Inheritance in Man") explains why he is regarded as "the father of medical genetics."
Nathans and McKusick were way ahead of the times. Genetics did not blossom into a driving force of medicine until computers made possible the identification of all 30,000 genes and the sequencing of 3 billion chemical-base pairs that make up human DNA. Think what this could mean in coming to grips with major illnesses, such as chronic obstructive pulmonary disease, hypertension, heart disease and cancer.
Elias Zerhouni, now the National Institutes of Health director, cites a statistic showing that if we could find ways to delay the onset of Alzheimer's disease by just five years, we'd save enough to fund all this country's research-not to mention the improved quality of life for millions of people. This gets me back to my original point-how to stay on the cutting edge of research. I believe that beyond attracting and retaining world-class talent, we need to focus on three areas:
Core resource centers
Science is becoming collaborative rather than solitary. It's no longer practical for one researcher, working with a couple of technicians, to possess the resources to do it all. The individual investigator may have a great idea, but you can't test a hypothesis without sophisticated equipment that is too large and expensive to place in every lab.
Our solution is to create core resource facilities. You might compare them to central copying centers, where you can run off thousands of duplicates of a thick document with complex charts and graphs.
Similarly, in the McKusick-Nathans Institute for Genetic Medicine (IGM), we've invested in superb resources for investigators to share-a microarray facility, a diagnostic lab, a new proteomics center, a fragment analysis facility, a DNA analysis facility and a cell center.
Research space for better science
The $140 million Broadway Research Building that will open this year is an example of what I'm convinced needs to be done. A few years ago, this project wasn't on anyone's radar screen. Now we've also broken ground for a second cancer research building, expected to cost $80 million.
Much thought has gone into designing these structures to "work" for investigators across departmental lines, not just this year but in 2010, 2020 or 2030. When the IGM moves into its permanent home in the Broadway Research Building, laboratories won't be arranged by departments but by grouping investigators working on similar scientific problems.
Programs nurturing original thinkers
Most Nobel laureates publish their award-winning work early in their careers. We need to find ways to identify young researchers with brilliant ideas and give them resources, freedom and training to soar. Our Young Investigator Awards serve a vital role in doing this and are an important psychological boost, but we need to go further.
How about duplicating the MacArthur Foundation's approach with its "genius awards?" Why not seek young researchers with really novel concepts and give them funds, unencumbered, for several years so they can test their hypotheses? Their salaries, their laboratory, would be fully supported. At the end of five years, we might even renew the grant. Let's see if one of them can discover the next double helix.
That's one suggestion we're evaluating. Looking down the road to where we should be heading requires innovation and priority-setting. These choices aren't easy: everybody wants resources and there's natural resistance to change. I believe, though, that by constantly re-examining our approach to basic research, this institution can continue the Nathans-McKusick tradition and make a profound impact on both medical science and our patients.