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Not anymore.
Thanks to significant philanthropic support from The Sprout Foundation, a Denver-area foundation funded by Suzanne and Bob Fanch, and gifts from others including Wag and Annalee Schorr, the Ehlers-Danlos Syndrome Center of Excellence was launched in 2019. The goal of the center is to eventually develop a cure for EDS at the Gates Center for Regenerative Medicine, while better addressing the clinical needs of patients today through specialty care at Children’s Hospital Colorado. The clinical components of this new center address the critical need for patient-centered, coordinated EDS care where physicians come together to agree on the treatment plan, rather than leaving the patient and their family to determine the course of action.
Simultaneously, the Gates Center for Regenerative Medicine scientists are conducting leading-edge research with the ultimate goal of finding a cure. Today, this research is aimed at discovering the genetic underpinnings of hypermobile EDS and leveraging this information to develop future therapies for patients like Calla.
The Fanches said, “Sprout Foundation has funded research and the outstanding staff at the Gates Center for Regenerative Medicine to accelerate the cure for this life-changing disorder and also for clinical care to patients through the EDS Center of Excellence.”
Joining in this effort are Calla’s own grandparents, Wag Schorr, an accomplished nephrologist and 1963 CU School of Medicine alumnus, and his wife, Annalee.
An essential component of the EDS Center of Excellence is a translational research program, which leverages existing campus resources and partnerships, including the Gates Center for Regenerative Medicine and the Colorado Center for Personalized Medicine.
The research program at the Gates Center is led by Dennis Roop, Ph.D., director of the Gates Center, in partnership with Ganna Bilousova, Ph.D., and Igor Kogut, Ph.D. The program brings EDS patients’ genetic information from clinical visits to the Gates Center where researchers are working on future treatments for the condition. In this virtuous cycle, patients inform future therapies in the lab that, in turn, could ultimately change lives back in the clinic.
Calla is one of those patients. She is motivated by the possibility of improving EDS research.
In preclinical models, scientists are collecting stem cells from Calla and other patients that indicate a possible mutation. These studies, using multiple patients, allow for a more accurate portrait of the errors in DNA. Early findings at the Gates Center suggest that a possible mutation for the hypermobile form of EDS may have been identified. The hope is that this research will lead to a potential treatment in the coming years.
Scientists are growing skin cells in the lab using Calla’s stem cells, with the EDS gene mutations removed. If successful, the modified stem cells will hook onto sites of inflammation and grow new cells – restoring function to damaged tissues and organs. It sounds like science fiction, but it could be a reality at the CU Anschutz Medical Campus in the years to come.
The research advances taking place at the Gates Center will ultimately mean incredible hope and healing for people like Calla, and others with rare genetic disorders, who are eager to regain their health and their independence.
A sense of anticipation captures the Schorr family.
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