Page 17 - Gates-AnnualReport-2019

On any given day, Eileen might tell Dr. Bruckner that Brady’s EB leaves him longing to taste ice cream, but unable on some days to even swallow water. Hours later, Eileen and Chris might sit at a table for an update with Gates Center Ph.D.s working assiduously toward EB cures, and explain how EB lesions close up the throat.
“We are so thankful,” Eileen said of the research meeting, “that they keep us in their brains while they do what they are doing.”
The close integration and interaction of basic scientific research, treatment development and production, and world- leading clinical knowledge are what has come to distinguish the Gates Center and the Anschutz Medical Campus from many other academic hubs in the nation. In rare and highly specialized disciplines such as EB, only a few locations can offer families and researchers the combination of hands-on treatment and exploration of the underlying causes.
Three Gates Center researchers are at the forefront of EB exploration, including Gates Center Director Dennis Roop, Ph.D.; Ganna Bilousova, Ph.D., assistant professor of Dermatology at the School of Medicine; and Igor Kogut, Ph.D., also an assistant professor of Dermatology at the School of Medicine. The Gates Center is one anchor of an EB iPS Stem Cell Consortium along with Stanford University and Columbia University, a consortium that has received multiple rounds of funding from the National Institutes of Health 21st Century Cures Act. They also receive generous matching private funds (a requirement of this funding mechanism) from the EB Research Partnership in New York and the California- based EB Medical Research Foundation, which co-founded the Consortium in 2016 to create the multi-institutional partnership, and the London-based Cure EB Charity. The Colorado Team has additionally received funding from the U.S. Department of Defense, the Dystrophic Epidermolysis Bullosa Research Association (DEBRA) International, the King Baudouin Foundation’s Vlinderkindje Fund and the Gates Frontiers Fund.
Elsewhere in the Annual Report, you can read about how the integration of basic research discoveries, on-campus production of treatment materials, and clinical capacity for on-site clinical trials in CAR-T cell cancer therapy will advance this model in 2020. This is the kind of real-world academic and clinical progress accelerating discovery from the lab to therapies and cures envisioned by Charles C. Gates and other
generous funders when the Gates Center was originally founded.
The reason Eileen Attar gave birth to Brady in a room where few people knew anything about EB is that it impacts only about 20 in 1 million live births. Researchers estimate about 25,000 to 50,000 families in the U.S. are living with EB patients.
With the Attar family now in Denver, after moving north from Houston to find Brady the best possible care, researchers can learn from Brady and dozens of other families at the Children’s specialty clinic just how the rare disease shows up every day. Eileen would welcome the company – she absorbs information, encouragement and support from every possible source.
“When I think about it too much, it’s too daunting,” she said. “How is this sustainable? That’s when it really hit me. It’s bigger than me.”
EB is an inherited skin and soft-tissue disorder that causes blisters and peeled skin with even light touches. It also can create sores and scar tissue in the mouth, esophagus, lungs, muscles, eyes, nails and teeth. There are multiple forms of EB, and Brady has the most severe kind, dystrophic EB.
Straight from the womb, Brady was born with sores and abraded lips from kicking in-utero and from descending the birth canal. Children with severe cases need hours of bandaging and wound care each day, like severe burn victims, and everyday movement such as crawling or walking can cause pain and more damage.
Brady often has throat wounds and scarring that make him not want to swallow, and nearly all his feeding is done through a GI portal in his stomach. Eileen and Chris carry around a worn notebook full of reminders about his nutrition, his wound care, his medications and appointments. Kristina Byrne is a full-time RN who also accompanies Brady to school, and has become so embedded in the family she’s often told she can never quit.
Kristina touches Brady affectionately on the leg, and tells his Children’s Hospital medical team, “I hope his wife likes me,” waiting for them to get her joke about her serious commitment.
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