A vision for Children's: Become one of nation's top pediatric research centers

April 26, 2009 12:00 am
  • Dr. David Perlmutter, scientific director of Children's Hospital of Pittsburgh of UPMC, during a tour of the new facility in Lawrenceville. He is one of the world's top experts on the leading cause of liver disease in children.
    Dr. David Perlmutter, scientific director of Children's Hospital of Pittsburgh of UPMC, during a tour of the new facility in Lawrenceville. He is one of the world's top experts on the leading cause of liver disease in children.
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As he stands on the top floor of the new research center at the Children's Hospital of Pittsburgh of UPMC complex in Lawrenceville, Dr. David Perlmutter can see a sweeping panorama of Oakland, Downtown and the North Hills.

But the scientific director of Children's has really got his eye on more distant points: Boston, Cincinnati and Philadelphia -- the three pediatric research centers that he believes are at the top of the heap in the United States and whose company he has been pushing to join.

And there is solid evidence that Children's Hospital is fast on its way to getting there.

From fiscal year 2001 to fiscal year 2008, its research funding from the National Institutes of Health rose from $7 million to $26.7 million, a 280 percent increase and one of the fastest growth rates in the nation. In the last year alone, he said, Children's got an 11 percent increase in NIH funding at a time when the federal agency had cut funds to many other institutions.

While he is justifiably proud of the $120 million, 10-story John G. Rangos Sr. Research Center, he doesn't believe it's the main reason he has been able to recruit some of the top children's disease researchers to work in Pittsburgh.

When scientists are deciding whether to move to a new city, he said, their main consideration is "who else is there. Researchers want to move where there are other great researchers. That's what attracts them."

On the other hand, he said, not having the new research facility might have dissuaded many of the recruits.

"Every recruitment I've done," he said, "has been with the anticipation that there's going to be a new facility. If I'd have said we're going to have the same physical plant in Oakland that we used to have, I wouldn't have gotten any of them."

The old Children's had only half as much research space as the new building, which explains why the gleaming structure is still only half filled.

The colorful, user-friendly hospital next door is drawing the main public attention as Children's gets ready to complete its move Saturday to its new campus. But it is arguably the research building and what it represents that will elevate Children's Hospital of Pittsburgh into national prominence.

"I always thought the clinical programs here were outstanding," said Dr. Gary Silverman, who was recruited five years ago from Boston to become chief of newborn medicine research. "But the strong research component was lacking."

And while Pittsburgh may never reach the level of Boston -- the acknowledged mecca of pediatric research in America -- it could now join other top-tier institutions like Philadelphia, Cincinnati and San Diego, researchers here said.

The key to that is to lure top scientists focusing on children's disorders, Dr. Perlmutter said. To do that, he said, he hasn't concentrated on the particular research a scientist has been doing, but how outstanding his or her career has been.

Dr. Perlmutter himself is in that mold. He is one of the world's top experts on the leading cause of liver disease in children, a genetic condition known as alpha-1 antitrypsin deficiency, which allows tangled clumps of protein to accumulate in the organ.

A key part of his research is a primitive, 1-millimeter-long worm known as c. elegans. Because the tiny roundworm is relatively simple, scientists find it much easier and quicker to study the effect of different genetic changes and to test different drugs on the worm than on a human or even a mouse, he said.

And that points up another key feature of the Rangos center. None of the 330 researchers working there, who will double in number over the next five years, is doing experiments with human patients. They all are focusing on basic cellular and microbiology research, using cells grown in the lab and various animal models of diseases.

Cecilia Lo, the newly hired chief of developmental biology for the University of Pittsburgh School of Medicine, will move into the Rangos center this summer and bring with her the mice she has bred to study congenital heart defects in children.

Nearby will be Carlton Bates, who was recruited last year from Ohio State University to become chief of nephrology. He uses mice to study the genetic defects in kidneys that are the top causes of kidney disease in children.

It may seem hard to link such research to childhood diseases, but Dr. Bates said the leap from laboratory to bedside is often quicker than people might think. And he certainly feels the urgency of pushing forward with basic research.

"If your kidneys don't work right at birth, you have about a 22 percent chance you'll be dead by your first birthday," Dr. Bates said. "My bias is that if we understand some of the basic building blocks that make a kidney, we'll be able to apply that in time to children."

When he was being recruited, Dr. Bates said, the importance of the new Rangos center was that "it was clear evidence that Pittsburgh's commitment to research is not just talk, but that they are putting their money where their mouth is."

Besides its stunning views from the glass-faced western side of the building, the research center's design is calculated to accomplish two other goals.

First, by concentrating eating areas, conference rooms and offices in their own sections, the rest of the building is practically unbroken laboratory space -- fully 75 percent of its square footage. When scientists obtain federal research grants, they are allowed to charge overhead for indirect building costs, and maximizing the laboratory space increases those critical payments, Dr. Perlmutter said.

The second feature is that the laboratories are all open. Teams of researchers work in their own bays, but there are no walls separating the groups.

"It's almost impossible for someone to have a lab in this building without sharing information with a neighbor," he said. "The building is designed to foster interactions."

To increase the odds of scientists discussing their work with each other even further, the fifth- and sixth-floor labs and the seventh- and eighth-floor labs each feed into common dining areas in the building's sun-drenched west side.

Each lab floor also contains common procedure rooms for such work as growing tissue cultures, and each has a "core facility." One of those core labs, Dr. Perlmutter noted, is a highly secure biocontainment lab for working on a tuberculosis vaccine. Besides electronic locks, it has its own air supply and filtering system, and researchers must take showers and change before they leave the area.

The fact that the center is still only half filled is actually a hopeful symbol of the hospital's intention to keep building its research capability, he said.

"When people come in here, they say this is a big-time commitment to research," he said.

Scientific director plans to make hospital one of the nation's top pediatric research centers

Mark Roth can be reached at mroth@post-gazette.com or at 412-263-1130.
First Published April 26, 2009 12:00 am

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