For the Institute for Stem Cell and Regenerative Medicine (ISCRM), 2024 was another groundbreaking year of collaboration, innovation, discovery, and progress toward treatments for patients. It was also a year of transition. In August, Chuck Murry began a new role leading stem-cell research at the University of Southern California’s Keck School of Medicine. During his time as ISCRM Director, Chuck secured crucial support from university leaders, private donors, and the Washington State legislature, which now provides $2.625 million in annual funding, and recruited more than 20 rising star faculty members to ISCRM while pursuing his lifelong goal of regenerating the human heart. Interim Director Jen Davis is now leading ISCRM, alongside Co-Interim Directors Nate Sniadecki and Hannele Ruohola-Baker.
In this report, you’ll read about just a few of the exciting discoveries made by ISCRM researchers in 2024. Each one of these breakthroughs was powered in one way or another by contributions from private donors who share our commitment to improving well-being for people everywhere. Thank you for your investment in the future of medicine!
Jennifer Davis, Interim Director, ISCRM
Breakthroughs and Other Big News
David Baker Wins the Nobel Prize
In a milestone for the UW research community, Institute for Protein Design (IPD) Director and ISCRM faculty member David Baker received the 2024 Nobel Prize in Chemistry for computational protein design. He shares the award with Demis Hassabis and John M. Jumper of DeepMind, who were honored for protein structure prediction. The award, announced on October 9th by the Royal Swedish Academy of Sciences, was presented in a ceremony on December 10th. Among many achievements, the Baker Lab has created new proteins that neutralize viruses, target cancer cells, and even serve as catalysts for chemical reactions. His research contributed to the development of the world’s first computationally designed protein medicine, a vaccine for COVID-19 pioneered by colleagues at UW Medicine.
Stem Cells and Designed Proteins
The Baker Lab and other IPD teams have partnered with the Ruohola-Baker Lab, Freedman Lab, Mathieu Lab to advance research in cancer, dentistry, other health challenges by using stem cells to model diseases and tissues and to test the efficacy and safety of designed proteins. Here are two examples from the past year.
- UW researchers have used computer-designed proteins to direct human stem cells to form new blood vessels in the lab, offering new hope for repairing damaged organs. Read More.
- Scientists from ISCRM, the UW School of Dentistry, the Institute for Protein Design, and the Brotman Baty Institute are changing the future of dental care by harnessing the regenerative capacity of stem cells. Read More.
Advances in Kidney Research
In March, research led by ISCRM faculty member Beno Freedman revealed insights about the genetic mechanisms of polycystic kidney disease (PKD). In the investigation, the Freedman Lab used CRISPR base editing to show in organoid models that having one healthy copy of a gene associated with PKD was sufficient to prevent cysts formation while the presence of two defective copies was not – an encouraging sign. The team then demonstrated that drugs known to be safe in humans could have therapeutic benefits for patients with PKD.
Additional Kidney Research News
A $7.3 million NIH grant will allow Ed Kelly and Beno Freedman to lead the effort to advance kidney-on-a-chip and organoid testing technology and submit a 3D engineered platform for FDA approval. Read More.
New Gene Therapy for Muscular Dystrophy Offers Hope
Jeff Chamberlain, an ISCRM faculty member and Director of the Wellstone Muscular Dystrophy Research Center, is the senior author of a paper that describes a transformational method for treating Duchenne muscular dystrophy (DMD), one of the most common human genetic disorders. The first author of the study, which appeared in Nature, is Hichem Tasfaout, an acting assistant professor who was a postdoctoral fellow in the Chamberlain Lab. Building on three decades of pioneering DMD work and gene therapy performed in the Chamberlain Lab, the researchers used a series of shuttle vectors (known as AAVs) to deliver parts of replacement dystrophin to muscles, along with embedded instructions to begin assembling the necessary genetic fix in the body. The new approach has been shown to halt the disease and could be a stepping stone to reversing muscle damage in patients. The next step for the therapy is human trials, which should begin in approximately two years.
Brain Health
In a recently published study, Jessica Young and her lab used stem cell-derived neurons to test the effects of medications people take as they age to understand how these medications could be linked to increased risk for dementia. The research confirmed that stem cells can complement epidemiological studies and help determine how certain drugs can adversely affect brain health, which could lead to important information for clinicians considering which drugs to prescribe.
Heart Research
How Small Molecules Can Have a Big Impact on the Heart
The muscle contractions that allow the heart to beat are generated by interactions between actin and myosin, proteins that enable movement at the molecular level by converting energy-rich molecules of ATP into muscle power. This force is diminished in patients with heart failure. In recent years, the Regnier Lab has shown that even small infusions of dATP as medicine can supercharge the contractile force of myosin. In October a study conducted in partnership with scientists at UC San Diego revealed insights about why this happens that will help the researchers design new cell therapies that could help doctors actually reverse reduced heart function. Read More.
Protein-based Biomaterial Protects Injected Cells, Improves Engraftment
Scientists hoping to treat heart disease, liver disease, and other conditions by injecting healthy cells have faced a challenge: the vast majority of cells don’t survive the turbulence of the injection. In March, Cole DeForest, Chuck Murry, and Kelly Stevens showed that when injected cells are embedded in protein-based hydrogels designed by the DeForest Research Group, they are more likely to engraft with the host tissue or organ. This would mean, for example, that stem cell-derived cardiomyocytes would have a better chance of helping to revascularize an injured heart.
Collaboration and Community
Three ISCRM Faculty Members Receive Washington Research Foundation Grants
This summer, Jen Davis, Shelly Sakiyama-Elbert, and Kelly Stevens were awarded major grant funding by the Washington Research Foundation (WRF) to support leadership in modeling aging and disease, direction of the Washington Initiative for Neuroscience (WIN) at UW, and the oversight of the Volumetric Imaging and Spatial Tissue Analysis (VISTA) at UW. Read more.
Kelly Stevens to Co-Lead NIH Funded Biomaterials Center to Foster New Ideas and Fight Inequity
ISCRM faculty member Kelly Stevens and Lola Eniola-Adefeso (University of Illinois Chicago) are co-leaders The Humanity Unlocking Biomaterials (HUB), which builds on an ongoing effort to correct the funding disparity faced by Black biomedical researchers. Supported by a $10.5 million NIH grant, the HUB will foster solutions that have potential in medical treatments by bringing together traditional, nontraditional, and historically excluded biomaterials researchers.
Inclusive Leadership Workshop
In April ISCRM hosted the second annual Inclusive Leadership Workshop in partnership with the Office of Research and Graduate Education (RGE). We were honored to showcase ISCRM as a possible next research home for a talented cohort of postdoctoral scientists who are exploring academic career paths in regenerative medicine and who are committed to inclusive teaching, mentoring, and lab leadership.
Summer Program
This year, ISCRM held its third ISCRM annual Undergraduate Program, a ten-week experience that provides a combined cohort of Bothell Fellows, ISCRMU Fellows, and ISCRM REU Fellows with paid summer jobs in labs, weekly training in professional skills related to publishing, presenting, and communications, and an opportunity to connect with other undergraduate researchers from around the world.
ISCRM Team Award Supports Diabetes Research
Five ISCRM researchers turned a $100,000 state-funded team award into more than $1,300,000 in additional grants from the
NIH and BreakthroughT1D to support the development of new cell transplant treatment for type 1 diabetes. Congratulations to Vincenzo Cirulli, Hannele Ruohola-Baker, Laura Crisa, Julie Mathieu, and Shiri Levy.
Thank you!
We are grateful to the generous supporters who have helped ISCRM become an engine of discovery in the field of regenerative medicine. If you’d like to learn more about how philanthropic investments fuel our work, please contact Elizabeth Damore, director for philanthropy, at edamore@uw.edu.