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Department of Pediatrics : Research : News & Events : DCRI bridging the gap

MUSC hopes institute will bridge gap between research, treatment

by Holly Auer
Of the Post and Courier Staff

Children are storehouses of knowledge for medical scientists. Inside their tiny bodies are the secrets behind some of adulthood's cruelest diseases and the keys to attacking those that strike kids themselves.

Today marks the opening and dedication of the Medical University of South Carolina's Charles P. Darby Children's Research Institute, a facility poised to link some the nation's best science with its sickest children.

It's a new era for medicine in South Carolina, hoped to generate better treatments and cures for elusive genetic diseases such as cystic fibrosis and sickle cell anemia. Though doctors have dramatically slashed the death rates for many childhood cancers and leukemia, help for other disorders remains out of reach.

Doctors believe the answers lie at the place where basic science and clinical treatment meet, a junction that hasn't been mined very well until recently.

"We understand the molecular basis of almost all of these diseases, yet we have not developed very much effective therapy," said Dr. David Nathan, president emeritus of Boston's Dana Farber Cancer Institute, who will speak at today's CRI dedication ceremony, which begins at 4 p.m. "We're going to have to chase these diseases by really understanding them at the molecular level and using drugs that go straight to the target."

A huge part of that quest will involve training people who can slide seamlessly between the lab bench and the bedside, speaking the language of complex biology as well as soothing families' fears and delivering top-notch care.

CRI leaders plan to provide those opportunities through new programs for pre-medical school clinical research assistants and fellowships that allow doctors to train in the lab for two years before beginning their practice.

"There's a huge chasm between those two worlds, which has gotten deeper and deeper in recent years," said Dr. Bernard Maria, the CRI's executive director. "What we're doing is all about creating those bridges across the two worlds."

Recipients of the work the "translational clinical investigators" seek to do are untold in number, ranging from toddlers with rare bone diseases to teens battling brain tumors. Adults with heart defects and autoimmune diseases such as lupus also are expected to benefit from the knowledge gleaned about such problems.

Waiting on the sidelines are the families who already have had a brush with devastating illness.

"I was no different than any average Joe, then all of a sudden the doctor calls and changes your life," said Dave Kreber, who serves on the board of the Children's Hospital Fund and will speak at today's events. "In our case, he called twice."

First, in 1996, Kreber's then-7-year-old daughter, Monica, was diagnosed with acute lymphocytic leukemia. Kreber and his wife, Sally, spent more than 300 nights with their daughter in the hospital over the next three years as the little girl fought a disease that strikes 4,000 people, most younger than 10, each year.

Just as Monica inched into remission, the Mount Pleasant family got another jolt: Their son, Zach, had juvenile diabetes, a vexing endocrine disease that consigned him to a lifetime of blood sugar testing, insulin dependence and sitting out the cake and ice cream part of every birthday party.

Zach's 10 now, and he's beginning to understand that the key to getting rid of the insulin pump he's tethered to 24 hours a day most likely lies inside a laboratory like the ones at the CRI.

Zach, Monica and their older brother, Drew, who witnessed a lifetime of family medical drama in just a few years, will all speak at today's ceremony.

Although Monica, now 15, remains healthy, the Krebers have seen firsthand that fate doesn't get doled out equally. In the spring of 2003, Monica's disease struck Ashley Everman, the daughter of Dave Kreber's bookkeeper, Cathy.

She died just eight months later, prey to a disease doctors still don't fully understand. "I've been a parent whose kid survived, but I've also watched the parents whose kid didn't," Kreber said.

Inside the new labs at the CRI, in incubators, on slides and in test tubes, he believes, are the seeds of knowledge that someday will obliterate that cruel dichotomy.