Portland, Ore.

Doernbecher received the highest score of recent applicants and is one of 20 children's hospitals in the country to receive this award

Doernbecher Children's Hospital pediatric metabolic disease pediatrician Robert Steiner, M.D., cares for many children who suffer from rare and complex medical disorders that are difficult to treat. It was his frustration in treating these ailments that prompted him to become a researcher.

"A lot of disorders I treat do not have good therapies, so I wanted to do something to improve the quality of life for these children," said Steiner, associate professor of pediatrics, and molecular and medical genetics in Oregon Health & Science University's (OHSU) School of Medicine. "I think doing research makes you a better physician because you know what's involved in developing new therapies."

Steiner's wish was fulfilled thanks to the Oregon Child Health Research Center (OCHRC) at OHSU's Doernbecher Children's Hospital. The center just received its second Child Health Research Career Development Award from the National Institute of Child Health & Human Development (NICHD) to fund research by junior faculty who also are pediatricians, like Steiner. Many pediatricians don't have the financial support or experience to get significant research grants. The purpose of the $2 million award is to develop ways to speed up the use of information gained through basic science in the laboratory to clinical applications that will improve children's health. Doernbecher is one of 20 children's hospitals in the country to receive this award, which will support the research, salaries and core laboratory for 10 pediatric physician-scientists. Other children's hospitals that have received this grant include Yale, Stanford and Johns Hopkins University. The NICHD funds only six programs each funding cycle. This is the second consecutive cycle Doernbecher has received this five-year grant award. This round Doenbecher received the highest score of all the institutions that applied. The OCHRC was created five years ago when Doernbecher received its first Child Health Research Center Development Award.

"This award validates that we are one of this country's preeminent pediatric institutions," said De-Ann Pillers, M.D., Ph.D., director of Doernbecher's OCHRC and associate professor of pediatrics, and molecular and medical genetics in OHSU's School of Medicine. "This award allows Doernbecher to invest in our faculty." Often pediatricians don't have the financial ability to take time away from their clinics to spend in the lab, according to Pillers. This new grant will help support their salaries, so they can afford to be in the lab more often. "Doernbecher has a good track record for training junior faculty and a great commitment to its pediatric research. We're hoping other centers will look to them as a model," said Karen Winer, M.D., pediatric endocrinologist and program officer of the NICHD's Child Health Research Career Development Award.

"The grant allowed me to hire research associates, which really helped because I can't be in the lab full-time due to my clinical work," said Steiner.

Early in Steiner's medical training he worked with two severely affected patients who suffered from Smith-Lemli-Opitz syndrome (SLOS). SLOS is a birth defect affecting one in about 20,000 children nationwide, making it one of the most common recessive disorders. SLOS patients suffer from mental retardation, physical malformations, failure to grow, difficulty eating and behavioral problems. Steiner's two patients passed away as a result of the disorder, however, research involving similar patients has helped scientists to discover the cause of the disorder, a defect in the body's production of cholesterol. The OCHRC funded Steiner's recent work that looked at whether providing SLOS patients with supplemental cholesterol from egg yolks would treat the problems created by the low cholesterol levels. The study printed in the April 2000 American Journal of Medical Genetics, found that the supplements increase cholesterol levels and decreased potentially toxic cholesterol precursors. Cholesterol supplementation also improved the patients' growth, feeding, overall well-being and behavior. However, it did not improve the children's developmental deficiencies.

This research project gained Steiner and his OCHRC mentor William Connor, M.D., professor of medicine (endocrinology, diabetes and clinical nutrition) in OHSU's School of Medicine, national recognition when they were honored with the Clinical Associate Physician/Investigator Award. This prestigious five-year grant will fund Steiner's and Connor's continued research on the biochemical and molecular genetic basis of SLOS. Currently Steiner runs the only active NIH-funded clinical research trial for SLOS in the country. Patients from Michigan, Alaska, California, Montana, Washington and Ohio travel to Doernbecher to participate in these trials.

"The OCHRC has been instrumental in advancing the research career of Steiner in his pursuit of the causes and treatment of the very devastating SLOS. With the aid of that grant, he was able to acquire initial data, which then allowed him to win other research awards, all of which helped establish him as one of the foremost investigators in this disorder of disturbance in cholesterol synthesis. Steiner has become a national figure in the genetic field. I know that many other young investigators have been helped and will be helped in the future by the center's grant," said Connor.

There are four Doernbecher faculty members currently funded by the OCHRC. Their areas of pediatric research include brain injuries, understanding the body's response to stress using a mathematical model and heart disease.

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