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We believe it takes the courage to ask difficult questions, the creativity to develop ideas, the intellect and talent of our faculty and staff, and the discipline and dedication to continue our history of scientific discoveries. This is how we have produced the breakthroughs that have benefited children over the 85 years since the founding of our Cincinnati Children’s Research Foundation (CCRF). This year’s research report summarizes the work of our exceptional research divisions. More detail is available online. In selected stories, we profile the efforts of a few extraordinary scientists and clinicians who have joined forces to tackle some of the most perplexing childhood diseases.
Read the print report.
scroll down for the full division reports.
A clinical trial will soon begin for a potentially groundbreaking treatment for sickle cell disease developed at Cincinnati Children’s. Punam Malik, MD, and colleagues have engineered a fetal hemoglobin gene that stays “on” and prevents red blood cells from sickling. They hope it will replace the defective hemoglobin found in patients with sickle cell.
Cystic fibrosis expert John Clancy, MD, played a large role in developing a drug called Kalydeco, which can dramatically improve lung function for about 10 percent of CF patients. Now he leads efforts to combine the drug with other compounds to benefit a wider range of people with CF.
Researchers at Cincinnati Children’s are pushing organ transplantation ahead on several fronts. We are using heart assist devices in new ways. We are helping kids stay healthier after kidney transplant. And we are conducting research to make liver transplants a thing of the past.
From discovering a way to rejuvenate aging stem cells to developing a drug that controls cancer-causing inflammation, scientists at Cincinnati Children’s attack cancer at its genetic and molecular roots.
Developing and perfecting the medium by which gene therapy is given in a clinical trial is not for the faint of heart – it takes months of preparation and testing at a level that few facilities can provide. Cincinnati Children’s core laboratories offer this expertise, from selecting and purifying the vector to testing before, during and after therapy.
Our researchers have a proud history of discovery. Josef Warkany’s work helped link toxic exposures to birth defects. Leland Clark invented a heart-lung bypass machine. Albert Sabin’s oral vaccine revolutionized polio vaccination. And a rotavirus vaccine developed by Richard Ward and David Bernstein has saved hundreds of thousands of lives worldwide.
As Airway Therapeutics pursues development of a major improvement in artificial surfactant, the start-up company offers a valuable lesson in what it takes to cross the drug development “funding desert.” The new company’s product is based on research by Jeff Whitsett, MD.
The anti-fibrosis drug eplerenone appears to slow the heart damage that kills most boys with Duchenne muscular dystrophy, according to research led by Kan Hor, MD. Now a larger clinical trial will evaluate whether early detection combined with early drug therapy might add years, perhaps decades, to patients’ lives.
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