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Children's Long-term Health Gets Lift With New Cincinnati Children's Program

Wednesday, October 26, 2005

CINCINNATI - Doctors are finding that a small investment in the future of children's health can quickly reap benefits, according to an evaluation of a new initiative at Cincinnati Children's Hospital Medical Center designed to boost the transfer of medical knowledge to patients.

"Our understanding of the disease process has grown tremendously in the last 30 years, thanks largely to the substantial investment made in basic research," said David Williams, MD, professor of pediatrics and associate chairman for translational research. "Unfortunately, translating those findings into clinical practice has often proved daunting, particularly for diseases that affect mostly children. We wanted to try to speed the translation of basic research to improve the health of children facing serious, but potentially treatable, medical conditions."

The result was a five-year, $5.8 million plan begun in 2001 to support doctors at Cincinnati Children's who proposed new ideas that had the potential to significantly improve patients' health. The preliminary results of the program, published in the November 2005 issue of the journal Academic Medicine, demonstrate that a small investment can substantially increase the rate of movement of research from the laboratory to the patient's bedside.

"Our results are particularly encouraging in light of recent studies that shows a particular need for research on diseases that affect children," said Timothy Cripe, MD, PhD, associate professor of pediatrics and lead author of the study. "We hope these results can serve as a model for other medical centers that are struggling with how to find funding to support translational research. Our experience has been that creating an infrastructure supportive of translational research and encouraging faculty to use it can make a big difference."

Indeed, the Institute of Medicine issued a report in May 2005 showing "a near absence of research in pediatric cancer drug discovery," and concluding that the current research climate is not sufficient to produce new drugs needed for children with cancer. The report recommended new models for research and development in diseases that affect children.

The Cincinnati Children's program demonstrated that a relatively modest $700,000 per year investment by the institution had stimulated 26 new patient-centered studies, including 15 new drug investigations, three new gene therapy trials and led to more than $19 million in funding from other sources to continue the work. The program also supported three new repositories dedicated to patient-centered research: the Human Milk Bank, the Normal Human Blood Stem Cell Repository and the Fanconi Anemia Cell Repository.

The repository is part of a larger Fanconi Anemia Comprehensive Care Center at Cincinnati Children's. Fanconi Anemia is caused by a defect in one of several genes that are critical for forming normal blood cells. Doctors treating Fanconi Anemia patients collect and store the blood cell samples from patients with the aim of correcting the defect using gene therapy and returning "normal" blood cells to their bodies. The repositories also serve as a hospital-wide resource for future research.

In addition, the program also has funded the creation of an institution-wide Translational Research Trials Office that provides help for doctors in preparing the research proposals and regulatory documents required to conduct patient research.

"Many scientists aren't trained in the intricacies of federal regulatory and reporting requirements that are essential to conducting translational research," said Dr. Cripe. "By providing an office staffed with people experienced in regulatory affairs, we are relieving some of the burden of moving clinical trials forward." The result has been four new drug investigations initiated in the last three years, where before there had been none.

Finally, to train the next generation of physician-researchers, the program conducts a series of educational sessions that encourage physicians to mingle with basic research scientists and to educate them in how the federal regulatory process works.

Some of the projects funded by the program include:

• The Rare Lung Disease consortium
• Eight projects studying the role of human milk components in human health
• A phase I gene therapy clinical trial that will attempt to correct the defective gene in Fanconi Anemia patients using their own blood stem cells
• A phase I gene therapy trial for brain tumors

"While we can't yet say that we have made a significant impact for patients yet with this new program, we can say that we now have put into place the crucial pieces that will allow us to speed promising new treatments to patients much faster than we have been able to in the past," said Dr. Cripe.

The findings come as the National Institutes of Health announced a major new initiative to fund translational research. In announcing the initiative, NIH Director Elias Zerhouni wrote in a New England Journal of Medicine editorial accompanying the announcement,"… in addition to the much-needed improvements in the policies and practices of health care delivery as we know it today, medical and public health practices in this nation will have to undergo a profound transformation in the coming decades if we are to succeed in providing access to care for all Americans at reasonable costs."

Cincinnati Children's is a 423-bed institution devoted to bringing the world the joy of healthier kids. Cincinnati Children's is dedicated to transforming the way health care is delivered by providing care that is timely, efficient, effective, family-centered, equitable and safe. It ranks third nationally among all pediatric centers in research grants from the National Institutes of Health. The Cincinnati Children's vision is to be the leader in improving child health.

For more information:

Making Better Drugs for Children with Cancer. National Academies Press, April 2005. Available online at http://www.nap.edu/catalog.php?record_id=11259.

Zerhouni, E: Translational and Clinical Science — Time for a New Vision. New England Journal of Medicine, October 13, 2005.