SHORT ANSWER
A
Collaboration Is Key
The Cords That Bind
CARDIOLOGY
As one of just eight centers in the country participating in Mayo Clinic ’ s Hypoplastic Left Heart Consortium , Children ’ s Hospital Colorado allows pregnant women with a prenatal diagnosis to participate in groundbreaking clinical trials studying stem cell therapy via the collection of umbilical cord blood at birth . With 18 patients enrolled , 5 babies had stem cells
Openhearted
CARDIOLOGY injected during their second HLHS surgery .
The consortium ’ s multifaceted research approach includes imaging and outcomes , human genetics and regenerative medicine , plus a collaboration that facilitates ideation and cross-pollination between researchers from all over the country , decreasing the time from research and discovery to clinical application . Best of all , the multi-site design allows families from all over the country to participate , no matter where they live .•
Each year , medical professionals diagnose about 42,000 babies in the United States with a congenital heart defect . Yet despite the prevalence of the diagnosis , information on quality of care and surgical outcomes is scarce and confusing to interpret .
Together with Children ’ s Hospital of Wisconsin and Mott Children ’ s Hospital , Children ’ s Hospital Colorado is working with the Pediatric Congenital Heart Association on a website for program-specific pediatric and congenital cardiology data that adhere to uniform tenets like standardized variables and substantiated data .
The goal is not to rank or compare hospitals but to drive conversations between stakeholders . Honest reporting and dialogue are key to improving outcomes , says Jim Jaggers , MD , cardiothoracic surgeon and Medical Director of the Heart Institute at Children ’ s Colorado . “ When hospitals see other facilities with better outcomes ,” he says , “ it can help them improve their own .” •
Boston Children ’ s Hospital
FDA Approves First Drug for 1 Person
NEUROLOGY
9-year-old Mila had perplexing symptoms . Her parents took her to over 100 doctors across the U . S . before they arrived at
Children ’ s Hospital Colorado . Genetics specialist Austin Larson , MD , uncovered one of two rare mutations in Mila and diagnosed her with a type of Batten disease only 25 people in the world are known to have .
The location of Mila ’ s second mutation was a mystery until Timothy Yu , MD , PhD , an attending physician and researcher at Boston Children ’ s Hospital , found it hiding in noncoding DNA . With that , he developed a drug solely for Mila to fix her fragmented sequence . It ’ s the first time the FDA has approved a drug for just one person — remarkable progress for precision medicine .
“ The drug acts as a molecular patch , allowing the gene to be spliced back together in the proper way ,” Dr . Yu tells WBUR , Boston ’ s public radio station .
Mila ’ s treatments were initially in Boston , but a growing collaboration between Dr . Yu and Children ’ s Colorado genetic epilepsy specialist Scott Demarest , MD , is now allowing Mila to receive this incredible treatment closer to home . “ The fact that Milasen is even possible tells us we have something to aim for and that there ’ s a route we can take to make it accessible to more people ,” says Dr . Demarest . Using a new machine called Novaseq , Children ’ s Colorado plans to commence its whole-genome sequencing program later this year . •
8 | CHILDREN ’ S HOSPITAL COLORADO