New collaboration will fight childhood diseases

ST. LOUIS -- A hospital and medical school ranked among the nation's best are launching the Children's Discovery Institute to try to speed up cures for childhood illnesses using findings from the Humane Genome Project, the program to identify all the genes in human DNA.

St. Louis Children's Hospital and Washington University's School of Medicine will announce a $355 million endeavor on Wednesday to accelerate cures for childhood diseases in four areas -- congenital heart disease, which is heart disease existing since before birth; cancer; lung and respiratory disorders; and muscle and skeletal diseases.

Some things remain constant: "We have a hospital full of sick children who need help," noted Dr. Jonathan Gitlin, director of genetics at the hospital and pediatrics professor at the university, where the medical school is ranked third in the nation by U.S. News & World Report.

But other factors are changing dramatically: "We are clearly in the middle of a scientific revolution," he said.

Washington University in St. Louis served as one of four centers on the Human Genome Project, a 13-year effort through the U.S. Department of Energy and National Institutes of Health to identify the 20,000 to 25,000 genes in human DNA.

Mapping that blueprint was a tremendous accomplishment, but new understandings continue, with advances in the biology of the genetic sequencing, improved computers to analyze the information and developments in nanotechnology, the manufacture and manipulation of materials at their smallest levels.

"We can take these advances and focus them directly at the bedsides of children," Gitlin said.

They could lead to plenty of future possibilities. If a child has brain cancer, his genes one day could be compared to 500 cancer-free children to identify a mutation or combination of mutations in the ill child. That knowledge might allow for new therapies to fight the cancer. And through nanotechnology, tiny devices then might be able to monitor the bloodstream for cancer cells or used to fight the cancer in the body.

While it's important not to give patients false hope and new treatments could be at least a decade away, Gitlin said the new institute will be about dreaming big and combining minds across disciplines to find solutions. He said 60 to 80 people may work on institute research.

"We have to say we can do it, or we won't get there," he said.

Jessi Nienke, 24, of St. Louis had a double lung transplant at Children's Hospital when she was 11 years old to fight her cystic fibrosis, a disease where a defective gene causes the body to produce mucus that clogs the lungs and obstructs the pancreas.

She was close to death before the transplant and said she would consider adopting children one day, in part to spare future generations of her family from the disease.

Nienke said she'll be on hand Wednesday to support the new institute. "You always want it to be better than you had it when you were a child," she said. She encouraged donations to a $125 million campaign at the hospital -- ranked among the 10 best children's hospitals in the country by Child magazine last year -- to support the Children's Discovery Institute.

"It's going to benefit them tremendously," she said of the new effort.

"I only see them growing from this."