Top 9 Research Breakthroughs of FY17 from Cincinnati Children’s Highlighted in Research Annual Report

Record Year for Funding Produces Numerous Discoveries, Including Lab-Grown Stomach Organoids, Progress Against Disfiguring Nerve Tumors

Tuesday, December 19, 2017

Success at growing tiny human stomachs in a lab dish. Discovering a way to shrink painful, disfiguring nerve tumors. Developing a faster method of detecting kidney injury that could save lives when babies need intensive care.

These are just some of the many innovations and discoveries made in fiscal 2017 (ended June 30) by the research faculty of Cincinnati Children’s—one of the nation’s largest centers for pediatric medical science. The center’s 2017 Research Annual Report, features the most significant findings of the year from more than 50 research divisions.

The papers featured in the report were chosen from approximately 2,000 findings published in FY17 by Cincinnati Children’s experts in numerous peer-reviewed medical and science journals.

This powerful flow of progress against childhood disease was fueled by a record-high level of research funding for Cincinnati Children’s, which totaled $218.9 million in FY17. The largest source of funding was the National Institutes of Health, but the funding figure also reflects grants from other federal and state agencies, non-profit foundations, and industry.

“This growth has been possible in part because our faculty remains dedicated to team science within our walls and to developing partnerships with colleagues worldwide,” says Margaret Hostetter, MD, Director of the Cincinnati Children’s Research Foundation. “Our research spans the spectrum of discovery from basic exploration of biological processes, to translation of new knowledge to clinical practice, to improving population health in our local community and around the world.”

In this year’s report, research leaders at Cincinnati Children’s also named nine discoveries as the center’s most significant breakthroughs of the year:

Biomedical Informatics: AERSMine Software Tool Helps Experts Dig Into Massive Drug Database

This tool, revealed in Nature Biotechnology, connects more than 9 million de-identified patient reports to a massive database of drugs approved by the Food and Drug Administration. This information helps experts analyze medication outcomes to identify unexpected side effects, unpredicted benefits, and alternative treatment choices.

Developmental Biology: Stomach Organoid Success Advances Work to Build a Complete GI Tract on a Chip

This paper, published in the journal Nature, reports success at producing fully functional human stomach organoids capable of producing digestive acid and enzymes. Now, experts at our new Center for Stem Cell and Organoid Medicine (CuSTOM) are working to translate this and other recent organoid innovations into practical test platforms that could transform drug research, while continuing to pursue personalized tissue growth methods that may someday reshape clinical care.

Experimental Hematology: A Protein Connected to Leukemia Risk Also May Influence Other Disorders

This discovery, published in Nature Immunology, sheds new light on how blood stem cells function. Eventually, this knowledge could lead to better treatments for blood cancers like acute myeloid leukemia (AML). Beyond cancer, this discovery reveals another dimension to how cells respond to infection, which has implications for a variety of autoimmune and inflammatory conditions.

Human Genetics: New Therapy for Gaucher Disease May be Less Risky and Less Costly

Gaucher disease is one of about 50 rare lysomal storage diseases that wreak havoc with how cells within our bodies clear themselves of waste particles. This discovery, published in Nature, shows that an experimental treatment that blocks a molecule known to drive inflammation and organ damage in Gaucher disease may be safer and less expensive than current enzyme replacement therapies.

Immunobiology: Bold Findings About Blood Cell Formation Stir the Scientific Pot

This project used new single-cell RNA sequencing technology to delve deep into the earliest stages of blood cell formation. The team’s groundbreaking paper, published in Nature, reports that the process does not work the way most scientists have thought. This basic science discovery may help other researchers refocus how to attack a variety of hard-to-treat blood and immune system disorders.

Nephrology and Hypertension: Better AWAREness of Acute Kidney Injury Could Transform Intensive Care

A powerful set of data from a study that involved 32 medical centers across four continents may spark a new way of thinking about preventing an often-overlooked cause of death for children receiving intensive care. The study, published in The New England Journal of Medicine, reports that improved use of low-cost urine tests can save lives by detecting signs of kidney damage much earlier than traditional testing methods.

Neurology & Behavioral Medicine and Clinical Psychology: Migraine Study Challenges Medication-Driven Approach to Care

To the surprise of many physicians, a 31-center clinical trial led by experts at Cincinnati Children’s determined that the two most commonly prescribed medications for preventing migraines in teens are no more effective than placebo. The results, published in The New England Journal of Medicine, suggest that controlling the life-disrupting pain of migraines should go beyond medications to include effective cognitive behavior therapy.

Oncology: Inhibiting MEK Protein Shrinks NF1 Plexiform Tumors

Breakthrough research at Cincinnati Children’s led to the first promising human clinical trial results for a drug to control the growth of disfiguring and sometimes deadly tumors that grow along nerve fibers throughout the body. The encouraging findings about the drug selumetinib were published in The New England Journal of Medicine.

Pediatric General & Thoracic Surgery: Scientists Tissue-Engineer Human Intestines with Functioning Nerves

In 2010, experts at Cincinnati Children’s made headlines by reporting the first successful production of tiny organoids of functional human intestinal tissue, grown from scratch in the lab using stem cells. This year, the research team achieved another major advance by successfully coaxing such tissue to grow a nerve system. This discovery, published in Nature Medicine, moves the field a big step closer to producing personalized intestinal tissue for transplantation.

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Jim Feuer