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Translational Research in CHORI's Center for Sickle Cell Disease & Thalassemia

Through the thorough integration of the highest standards of excellence in clinical research with innovative and nationally recognized basic research laboratories, CHORI’s Center for Sickle Cell Disease and Thalassemia (CST) is at the forefront of translational research in the United States.

Under the direction of Elliot Vichinsky, MD, the CST hosts the Northern California Comprehensive Sickle Cell Center and the Thalassemia Clinical Research Network, both of which are involved in several key pilot and national clinical research projects. These NIH-supported, multi-center trials provide the invaluable opportunity for collaborative research between clinicians and basic researchers on which the best translational research is based.

Clinical investigators such as Lori Styles, MD, and her colleague, basic researcher and senior scientist, Frans Kuypers, PhD represent a key example of the unparalleled success of SCT’s translational research. Drs. Styles and Kuypers identified an easily measurable indicator for acute chest syndrome (ACS) that not only provides clinicians with a predictive tool with which to preemptively treat patients before a potentially fatal ACS crisis, but also provides a novel target for drug therapy.
In addition, Claudia R. Morris, MD, and Dr. Kuypers have been investigating the potential of arginine therapy in sickle cell disease. Through their bench to bedside collaboration, Dr. Morris discovered a new arginase mechanism associated with the development of pulmonary hypertension and mortality in sickle cell disease that also provides researchers with a new treatment target.

Part of the success of the CST’s translational research platform is that it also moves beyond laboratory and clinical integration to bring the results of research to the community at large. Marsha J. Treadwell, PhD, directs the HRSA-

sponsored “Talking Drums Project,” which is specifically designed to educate the community about sickle cell disease. One aspect of Dr. Treadwell’s innovative program teaches patients and family members self-advocacy tools, with the long-term goal of improving their emergency care.

These are only some examples of CST’s ground-breaking work in translational research. Explore the Investigator links in the sidebar menu to discover that CHORI’s SCT has to offer.

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