2019 was a momentous year for the Institute for Stem Cell and Regenerative Medicine (ISCRM). In this brief report, you’ll learn about five noteworthy achievements and meet some of the incredible people behind them. Indeed, none of this would be possible without the faculty, staff, and trainees who make ISCRM such a dynamic research community. Please also join me in saluting my friends and co-founders Randall T. Moon and Tony Blau. We share these points of pride with them, and with all of you who have been relentless and generous champions of stem cell and regenerative medicine.
Last summer, ISCRM and the Heart Regeneration Program (HRP) demonstrated that human stem cells could be used to regrow heart muscle tissue in non-human primates. That landmark study put ISCRM, HRP, and UW Medicine in a position to begin charting a course to human trials in 2021. As supporters of ISCRM, each of you played an instrumental role in that achievement. This fall, our journey reached a new milestone that brings us one giant leap closer to the clinic and, eventually, to the most effective treatment yet for the world’s leading cause of death.
Our heart regeneration work is now set to become the lead clinical program for Sana Biotechnology, a company launched by Steve Harr and Hans Bishop, respectively the former CFO and CEO of Juno Therapeutics. Steve, of course, is also a member of ISCRM’s Campaign Committee. I firmly believe this partnership is the best opportunity to successfully move heart regeneration through the clinical trial process and to reward your early vision with the ultimate ROI: hope and healing for billions of people worldwide.
While I am not leaving ISCRM or the UW, I do plan to play an active role in the leadership of Sana. My focus, as a part-time employee, will be to build Sana’s cardiac biology program, secure FDA approval for our first clinical trial and, ultimately, oversee the trial. We expect the process to take about a year, after which I’ll once again increase my focus at the UW. Throughout this period, I will continue my direct involvement in state legislative relations, faculty recruitment, and strategic planning for ISCRM.
Over the past four years, ISCRM has recruited 14 new faculty to join a cohort of rising stars who are becoming globally recognized leaders in their fields. Within ISCRM, this group is part of the Junior Leadership Team, a council of assistant and associate professors who represent the future of the institute. Combined, they have drawn $4.7 million in federal research to the UW in the last year alone. Here are four of the junior faculty members pioneering new technologies and treatments.
Ron Kwon, PhD, Associate Professor of Orthopaedics and Sports Medicine
Ron joined ISCRM two years ago and is already establishing himself as a leader in the field of bone biology and an expert in the mechanisms of regeneration. Fueled in part by a John H. Tietze Stem Cell Scientist Award, the Kwon Lab is on a mission to understand the genetics of osteoporosis, an investigation that will include one of the most comprehensive screens of osteoporosis-linked genes ever conducted. NIH funding will enable Ron to study the genetic risk factors for osteoporosis, opening the door to possible new treatments for bone diseases.
Ying Zheng, PhD, Associate Professor of Bioengineering
Imagine a house, beautifully constructed, with four walls and a roof, but no pipes, vents, wiring, or doorways. That’s roughly what regeneration would look like without proper vascularization. Right now, few labs anywhere can rival Ying Zheng’s ability to engineer blood vessels, the conduits that carry blood, oxygen, and nutrients throughout our bodies. In fact, the Zheng Lab recently became the first team in the world to successfully engineer capillaries, the tiniest and most abundant vessels. This is a huge breakthrough for tissue printing, organoid technology, and disease modeling.
Jennifer Davis, PhD, Assistant Professor of Pathology and Bioengineering
Jen may be the ultimate ISCRM collaborator — a molecular biologist teaming up with chemical engineers, mechanical engineers, and bioengineers to solve some of the biggest challenges in medicine. For the last decade, Jen has been exploring profoundly important questions about the role scarring plays in heart disease and in wound healing throughout the body. Jen and her team are now revealing ways to control scarring, a breakthrough that could have major therapeutic implications for patients with damaged hearts, lungs, and livers. Fittingly, Jen was recently named the new director of the UW Center for Cardiovascular Biology, an important ISCRM research partner.
Benjamin “Beno” Freedman, PhD, Assistant Professor of Medicine and Nephrology
Beno Freedman’s name is almost synonymous with kidney organoids, the stem cell-derived tools that enable his lab to study kidney function, understand the onset of kidney diseases, test potential drugs quickly and humanely, advance the field of tissue regeneration (from a patient’s own cells) and, soon, predict the effects of gene therapies. In one breakthrough, the Freedman Lab recently identified several molecules that reduced cyst formation in organoids that carry the mutation for polycystic kidney disease. The ultimate goal is to develop faster, safer, and more permanent alternatives to current interventions like dialysis and organ transplants.
The progress we have seen in muscle research over the last year deserves a special report of its own. First, ISCRM welcomed Jeff Chamberlain, whose gene therapy approaches for Duchenne muscular dystrophy (DMD), the most common form of muscular dystrophy, are showing promising results in clinical trials. Jeff is the director of the NIH-funded Paul D. Wellstone Muscular Dystrophy Research Center, now in its 11th year, which has provided more than $17 million in funding. The Wellstone Center is focused on developing therapies for various muscular dystrophies, including DMD and facioscapulohumeral muscular dystrophy (FSHD). At the same time, a gene therapy developed in part by ISCRM faculty member David Mack is helping young boys live longer and more active lives.
Finally, this fall we celebrated the opening of the Center for Translational Muscle Research (CTMR) at UW Medicine, a new NIH-funded facility led by ISCRM faculty member Mike Regnier, who is also a member of the Wellstone Center. This new center complements the activities of the Wellstone Center and will galvanize researchers across campus and around the region, making the UW an international leader in muscle research and accelerating urgently needed treatments for ALS, heart disease, and other muscle disorders.
Over the last year, ISCRM investigators have been featured in media outlets around the world, from local television in Seattle to newspapers in Europe and Asia. As always, big stories start with great science. In May, research co-led by our own Kelly Stevens, Assistant Professor, Bioengineering and Pathology, appeared on the cover of Science, one of the most prestigious and widely read scientific journals. The article detailed groundbreaking 3D tissue printing advances made by Stevens and her collaborators at Rice University — breakthroughs that have exhilarating implications for everything from disease modeling to organ printing.
Community engagement is a core part of our mission at ISCRM, and it comes in many forms. In 2019, we welcomed student groups and elected officials for tours of our labs, drew an overflow crowd for our January public forum on neurodegenerative diseases, offered four evening programs in Seattle’s South Lake Union neighborhood, and helped hundreds of high school and middle school students learn about cell biology, bioengineering, organ printing, and gene editing. We also spoke out about the predatory clinics offering fraudulent, dangerous treatments to vulnerable patients. It’s rewarding and exciting to see how these efforts reciprocate the public support we receive, build bridges between ISCRM and the world outside our labs, foster community within the institute, spark a sense of possibility for future scientists, and allow us to be a voice for safe, responsible science.
The advances ISCRM made in 2019 are vital to the future of medicine, and all were made possible because of the visionary support of supporters like you. We are forever grateful to you for being our champions — and we look forward to sharing more with you in the months and years ahead.
Best wishes for a safe and happy start to 2020 from your friends at ISCRM!