Hope is most sustainable when it soars from a foundation of real progress. At the Gates Center for Regenerative Medicine, the progress we worked for in 2019 is the basis for new hope that the most promising concepts in our field will help transform medicine and bring treatment to patients who have long looked for answers and relief. We enter 2020 on the verge of human clinical trials initiated by Gates Center member researchers on the University of Colorado Anschutz Medical Campus. With colleagues throughout the campus, we have established a joint clinical and research center of excellence in a debilitating and scientifically challenging disease that begins to show symptoms in adolescence, greatly impacting patients and their families. We’ve also refined our strategy and honed our focus in a planning process we called Gates Center 3.0. And we were honored with expanded national funding for key projects and announced a groundbreaking sponsored research agreement with a private company in one of our areas of focus. These advances toward potential treatments encourage and motivate us as we spend more time in trial recruitment and clinical settings, along with families looking to regenerative medicine with their own hopes. For the first time ever we have decided to include cover photos of a patient along with some stories in this report, which remind us of the spirit of our namesake, philanthropist Charles C. Gates, and the Gates family leadership whose funding both launched and helps sustain the Gates Center. The family’s success in entrepreneurship, business and community building has never lost sight of making real improvements in human lives. Hope backed by progress was on display when the Epidermolysis Bullosa (EB) iPS Cell Consortium, a unique collaboration of research teams from the Gates Center, Stanford and Columbia universities, received an $800,000 grant from the National Institutes of Health 21st Century Cures Act. The award was immediately doubled by a required match from a group of private foundations united to back research into EB – a disease from which their founders’
4
Gates Center for Regenerative Medicine
children sadly suffer. Notably, the Consortium was the only hopeful in a group of eight research applicants funded in 2017 to be awarded a second round of funding by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) – one of twelve institutes within the NIH participating in the 21st Century Cures Act Regenerative Medicine Innovation Project. The growing endorsement of the Consortium’s research, evidenced by additional grant funding from a variety of sources, has already resulted in a 24-fold “return on investment,” with the initial $500,000 NIAMS grant in 2017 sparking more than $12 million in total research funding; even before the newest award. The EB Research Partnership in New York and the California-based EB Medical Research Foundation co-founded the Consortium in 2016 to create this multi-institutional partnership, and they along with the London-based Cure EB Charity have generously matched both NIAMS awards. We believe the continuing endorsement from the 21st Century Cures Act second round of funding underlines that what we’re doing here is leading-edge, and that we’re competitive with the best institutions in the country for acquiring federal funding. Our work in finding a cure for epidermolysis bullosa, from which 3½-year-old Brady Attar shown on our cover suffers, also opens doors for the kind of partnerships between universities and private enterprise that Charles C. Gates envisioned for his family’s philanthropy. The core science and expertise of academia can be infused with the urgency and organizational skills of entrepreneurs to more quickly advance discoveries toward human applications. In November, AVITA Medical, a regenerative medicine company with a technology platform positioned to address unmet medical needs in therapeutic skin restoration, and scientists at the Gates Center announced a preclinical research collaboration to establish proof-of-concept and explore further development of a spray-on treatment of genetically modified cells for EB patients. The technology holds promising potential applicability to other genetic skin disorders, as well.
