"We don't see patients, and there are no physicians in white lab coats with stethoscopes around their necks walking our hallways. But, if we don't do what we do, there will be no physicians in white lab coats with stethoscopes around their necks, doing clinical trials. Because there's nothing to do clinical trials on."—Richard Woychik, director, The Jackson Laboratory

BAR HARBOR — You don’t need a doctorate in molecular biology to understand the impact that research at The Jackson Laboratory is having worldwide on the quality of medical care.

“You take a look at Lipitor, or take a look at Tylenol, or any tablet, and you wonder ‘What is it?’,” he said. “You swallow it, and what happens? The simple answer is, in most places [within your body], nothing happens. In fact, that’s what drug companies want to do: they want it to be highly specific.

“That tablet contains a highly purified and really small molecular entity that circulates throughout the body. In a select number of tissues and in a select number of cells in certain places, that molecule will bind to a protein, where it basically does one of two things: it turns it on or it turns it off. That’s how drugs work.”

Why does it cost $800 million, on average, to create a new drug?

“Because biology is complicated,” says Woychik, who should know. A world-renowned geneticist, he’s also the author of more than 80 scientific papers grounded in molecular genetics.

“You have to ask which one molecule in the body, that when you turn it on or off, is likely to produce a therapeutic effect for Type 1 diabetes or Alzheimer’s or Parkinson’s. That’s what we do here. The problem is there are 25,000 different genes that produce lots and lots of different types of proteins.”

Beyond developing drugs, insights into the molecular mechanics of the human genome can help identify gene sequences that are predictors of diseases such as Alzheimer’s and disease risk factors such as obesity.

“You can know if you have a disease susceptibility, which is a big part of this now,” Woychik said. “You may have a sequence variation within a gene that we know, that in our mouse model, will predispose you to the loss of brain cells at an older age. That’s now an indicator. Using a robust and relatively inexpensive technology, you can be geno-typed and you can change your lifestyle. If you have gene sequence variations in certain genes associated with obesity, don’t gain weight. It’s all part of the health focus we have at The Laboratory now.”

In addition to exploring the roles of individual genes in affecting human health, Woychik said Jackson Lab research teams are now working to identify networks of genes that can either trigger or suppress disease.

“We’re looking to the future in a way that we take a leading role in helping to guide the international community in how to use what we call ‘systems genetics,’ where it’s not about a single gene, but you look at disease as a genetic system.”

Beyond in-house research efforts, the 2.4 million laboratory mice that The Jackson Laboratory shipped in fiscal year 2007 to 16,000 investigators in 60 countries extend the scope and reach of the Lab’s impact worldwide, as does its extensive list of seminars, courses and workshops.

“We know a lot about using mice and the molecular genetics of the mouse to impact the way research is done,” Woychik said. “We have an awesome set of courses, conferences and workshops, where we bring in some of the world’s experts. We bring world-class people together, and they schmooze and interact with each other.

The Jackson Lab traditionally has been heavily reliant upon federal research grants provided through the National Institutes of Health. Such grants are not as abundant now as federal dollars for medical research are being diverted to deal with the economic fallout of Hurricane Katrina and long and very costly wars in Iraq and Afghanistan. Woychik feels the Lab needs to do more to get its story in front of potential private-sector donors.

“We’re going to be much more actively raising money philanthropically,” he said. “I think we have got a great mission, one I believe in and one that will engage the people who have the means to support what we do here in a way that we have never done before. Everything we do is about partnerships. We can’t do the work that we do without partnerships and interaction with others, who are not scientists.

“I’m trying to get people to understand why it’s important to do the work we do. We don’t see patients, and there are no physicians in white lab coats with stethoscopes around their necks walking our hallways. But, if we don’t do what we do, there will be no physicians in white lab coats with stethoscopes around their necks, doing clinical trials. Because there’s nothing to do clinical trials on. For the first time in the history of the Lab, we have now a tagline: ‘Leading the Search for Tomorrow’s Cures.’ It’s a concise way of describing what we are doing.

“We’re at the front end, leading this process that ultimately ends up at the bedside and beyond. It ultimately ends up in pharmaceutical companies and in pharmacies and as part of the prescription repertoire of a typical physician who sees patients and prescribes medicines. We’re the place that makes that stuff happens.”

Woychik wants people in Hancock County and throughout Maine to share his deep sense of pride in the Laboratory.

“It’s important to me that people in Bar Harbor recognize what we do and say, ‘Damn, I’m really proud that The Jackson Laboratory is here, and I really feel good about what they do because it’s a partnership between me, living in Bar Harbor, and The Jackson Laboratory.’

“I want to be able to go out and to tell the world about this great institution that contributes to the scientific knowledge of the world in helping us understand what diseases are all about and that it’s a great place that brings in terrific people.”