Page 18 - Gates-AnnualReport-2018

COLLABORATING FOR EB PROGRESS AND REGENERATION KNOWLEDGE
Scientists from the Gates Center are part of a consortium awarded $3.8 million from the U.S. Department of Defense (DOD) to move discoveries in stem cell-created skin grafts into the manufacturing stage, bringing further hope to victims of debilitating inherited skin diseases.
The major grant for the Epidermolysis Bullosa (EB) iPS Cell Consortium, which includes research teams from CU Anschutz, Stanford University School of Medicine and Columbia University Medical Center, will move production of stem cells into the Gates Biomanufacturing Facility.
The grant follows recent awards for the same investigators by the 21st Century Cures Act and the California Institute of Regenerative Medicine, discussed in our 2017 Annual Report. The backing will boost research that could not only benefit EB sufferers, but also countless patients with severe chronic skin wounds.
‘CUTTING-EDGE SCIENCE’
In announcing the new grant, DOD reviewers issued one of the most emphatic research endorsements possible, saying, “This study is based on the strongest cutting-edge scientific rationale in the field of wound care and dermatology. It is also a collaborative effort among top physician-scientists, scientists, health care providers, epidermolysis bullosa patients, families, and charities across the United States.”
One evaluator wrote: “The proposed research has the highest probability of success of bringing gene-corrected tissue to patients in the hospital . . .” Roop often points out that EB may represent an ideal platform from which to test an iPSC-based therapy. It is an orphan disease and it can be devastating, sentencing patients to a life of severe pain and disability and even early death. Further, the skin is readily accessible, easy to monitor and to excise in the event of an adverse event. If deemed safe and effective, iPSC therapies could then be expanded to treat monogenetic diseases affecting internal organs, in which the difficulty of monitoring adverse effects would make them very unlikely first targets.
Zoobiquity Colorado’s panel on the Future of Regenerative Medicine (left to right): Dennis Roop, Ph.D.; Ryan Crisman, Ph.D.; Mike Perry, D.V.M., Ph.D; Sue VandeWoude, D.V.M. and CBS Medical Correspondent Max Gomez, Ph.D.
The DOD award will allow the EB research team to further investigate best manufacturing practices for larger-scale production of stem cell-created skin grafts, utilizing the unique RNA-based reprogramming and gene editing technology developed by Gates Center investigators and the state-of-the-art resources of the GBF. The goal now is to move the technology from the laboratory into clinical trials.
Roop has talked frequently about behind-the-scenes collaborations and networks that have furthered the EB consortium’s work, of which he is a key part. In 2016 the EB Charities, composed of foundations formed by families of children suffering from the disease*, challenged research teams from the University of Colorado and Stanford and Columbia Universities to collaborate rather than compete in finding a cure for EB. The teams’ willingness to collaborate within the EB iPS Cell Consortium has been rewarded by EB Charities’ initial and continued funding, leading to public support outlined above and fervent hope for future success.
Three years ago, Roop was presenting data at Harvard and seeking ways to improve homing of stem cells into injured tissue. Robert Sackstein, M.D., a Harvard professor of dermatology, offered to solve the problem and sent reviews and papers over the following months.
*EB Charities (the EB Research Partnership (EBRP), the Los Angeles- based EB Medical Research Foundation, and the London-based Cure EB Charity—formerly SOHANA Research Fund)
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