Children’s Hospital Los Angeles Awarded $9.5 Million by NIH for Sickle Cell Disease Research

Children’s Hospital Los Angeles Awarded $9.5 Million by NIH for Sickle Cell Disease Research

<0> Children's Hospital Los AngelesEllin KavanaghOffice: (323) 361-8505 </0>

Thomas Coates, MD, section head of Hematology in the division of Hematology Oncology at Children’s Hospital Los Angeles and principal investigator at The Saban Research Institute, along with four other co-principal investigators, recently received a five-year, $9.5 million grant from the National Heart, Lung, and Blood Institute of the National Institutes of Health. The award will fund research into the underlying physiology of sickle cell disease (SCD) and identification of biomarkers that will aid in the development of new treatment options.

“Sickle cell disease is a devastating disorder and progress toward developing new treatments for battling this disease has been limited by our inability to measure the basic physiology of the disorder in humans. Our unique approach involves sampling multiple physiological variables, using computational modeling to determine the underlying pathophysiology and to identify biomarkers of interest,” says Coates, who is also a professor of Pediatrics and Pathology at the Keck School of Medicine at USC. “By translating these biomarkers into standardized tools, we will provide the research community with a way to monitor disease status and measure the effectiveness of new therapies for patients with SCD.”

Sickle cell disease is a life-threatening genetic disorder of hemoglobin, the molecule in red blood cells that supplies oxygen to the body. Instead of rounded, flexible discs, the red blood cells in SCD patients are stiff and crescent-shaped. These sickled cells can become lodged in the blood vessels and cause painful episodes that deplete the body’s oxygen supply and lead to organ damage, especially in the lungs, kidneys and brain. Sickled cells also break down prematurely, leading to a low red blood cell count. Because of this defect, SCD patients have severe pain episodes, ongoing organ damage and premature death.

In previous studies, Coates and colleagues discovered that individuals with SCD exhibit physiologically-based biomarkers, such as an abnormally-regulated autonomic nervous system (ANS) and resultant constriction of blood vessels. They hypothesize that these biomarkers of blood flow and tissue oxygen delivery reflect the collective results of all molecular and cellular pathologies, aside from the initial gene mutation, in SCD patients. With this award from the NIH, Coates, and co-principal investigators Michael Khoo, PhD, and Herbert Meiselman, ScD, from the Biological Engineering and Physiology programs at USC, John Wood, MD, PhD, associate professor of Cardiology at Children’s Hospital Los Angeles, and Lonny Zeltzer, MD, professor of Pediatrics and director of UCLA’s Pain Program at the David Geffen School of Medicine, will study their hypothesis. This multidisciplinary team will focus on developing and calibrating biomarkers that reflect ANS imbalance, pain-triggered constriction of blood vessels, vessel function and the brain’s blood and oxygen levels.

“Dr. Coates and his colleagues are making significant discoveries that are advancing our knowledge of the causes and progression of complications caused by sickle cell disease,” says Brent Polk, MD, director of The Saban Research Institute. “We expect that this research will lead to new therapies and diminish the burden of such disorders on children and young adults and their communities world-wide.”

Sickle cell disease currently affects nearly 80,000 individuals in the United States, and millions across the world. Symptoms can appear by three months of age. While 95% of patients in the US survive to age 20, half of these die by age 40. Of the 240,000 children born in Africa with sickle cell disease annually, 80% die by their second birthday. By studying the biomarkers of devastating secondary afflictions, Coates and colleagues hope to provide clinicians with standardized assessment tools that will be useful in developing new therapies and will improve the quality and length of life of SCD patients.

Children's Hospital Los Angeles has been named the best children’s hospital in California and among the top five in the nation for clinical excellence with its selection to the prestigious US News & World Report Honor Roll. Children’s Hospital is home to The Saban Research Institute, one of the largest and most productive pediatric research facilities in the United States, is one of America's premier teaching hospitals and has been affiliated with the Keck School of Medicine of the University of Southern California since 1932.

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