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5 Most Significant Discoveries of FY2020 from Cincinnati Children’s

Works chosen from more than 2,500 research publications shed light on organoids, blood cell development, stem cell therapy, sickle cell disease and HLH.

Monday, March 22, 2021

Fueled by more than $240 million in external grants and $26 million in philanthropic support for science, research teams at Cincinnati Children’s made numerous significant discoveries to improve child health in fiscal 2020 even as the institution shifted gears to take on COVID-19.

The latest Research Annual Report from Cincinnati Children’s reflects the wide range and tremendous depth of scientific exploration occurring at one of the world’s top pediatric academic medical centers. Nearly one third of the more than 16,000 employees at Cincinnati Children’s are dedicated to research, including physicians, scientists, research nurses, pharmacologists, informatics experts and more. Together, in collaboration with co-investigators around the world, they produced more than 2,500 peer-reviewed findings across more than 50 research divisions.

“We are especially grateful to the children, young adults, and families who place their trust in our compassionate scientists and clinicians. Many of the advancements featured in this report could not have occurred without their participation in our clinical research,” says Michael Fisher, president and CEO of Cincinnati Children’s. “By volunteering, they have contributed not only to the betterment of science but also humanity.”

The annual report features the most significant accomplishment of each of the medical center’s research divisions. From these, a committee of senior science leaders at Cincinnati Children’s selected five publications as the most significant. All the papers in the report were published during the fiscal year ended June 30, 2020. The five top works are:

World’s First Three-Organoid ‘System’ Opens Doors for Future Uses

Experts at Cincinnati Children’s have made headlines in recent years with several breakthroughs at creating tiny lab-grown organoids that mimic the functions of full-sized human organs. Now, for the first time, the researchers have succeeded in growing multiple organs at the same time so that they are connected to each other. A team led by Takanori Takebe, MD, PhD, reported their success at growing an interconnected liver, pancreas and biliary duct system on Sept. 25, 2019, in Nature.

Clinical Trial Success Caps Long Journey for HLH Treatment

The disease hemophagocytic lymphohistiocytosis (HLH) still has no cure. But now it has an approved treatment that appears to help most patients live long enough and well enough to receive crucial stem cell transplants. The benefits of the monoclonal antibody emapalumab (Gamifant) were detailed in a multi-center study co-authored by Michael Jordan, MD, a member of the Divisions of Immunobiology and Bone Marrow Transplantation and Immune Deficiency, and Alexei Grom, MD, Research Director, Division of Rheumatology. Results were published May 7, 2020, in The New England Journal of Medicine.

Dose Escalation Sharply Improves Hydroxyurea Benefit for Children with Sickle Cell Anemia

Globally, approximately 300,000 children are born with sickle cell each year, with about 80% located in sub-Saharan Africa where the disease kills many children before they reach age 5. In previous research, Russell Ware, MD, PhD, and colleagues demonstrated that a low-cost treatment called hydroxyurea is safe and effective for African children. In this latest advance, the research team reports that escalating doses to their individually tolerable levels vs using fixed dosing sharply improves outcomes. Their findings appeared June 25, 2020, in The New England Journal of Medicine.

Single-Cell Approach Reveals Impact of Disease-Causing Gene Mutations

By analyzing genetic changes in mouse blood cells at a cell-by-cell level as their bodies passed key stages of development, researchers now have deeper understanding of not just which gene mutations can lead to disease, but also how much it matters when the mutations occur. This groundbreaking work was led by H. Leighton Grimes, PhD, Division of Immunobiology, and Nathan Salomonis, PhD, Division of Biomedical Informatics. The paper was published in June 2020 issue of Nature.

A Reality Check for Cardiac Stem Cell Therapy

Stem cell therapy helps hearts partially recover from a heart attack by triggering an immune response that results in better scar formation and improved performance of surviving tissue—not by helping hearts grow new muscle. In fact, living stem cells are not actually needed to achieve this effect. Injecting dead cells produced the same reaction. This unexpected finding, led by Jeffery Molkentin, PhD, Director of Molecular Cardiovascular Biology at Cincinnati Children’s, helps clarify why a number of stem cell therapy trials have produced disappointing, but not entirely negative results. The findings were published Nov. 27, 2019, in Nature.