On Thursday, March 6, the Institute for Stem Cell and Regenerative Medicine (ISCRM) hosted Stem Cells in Clinical Trials: A Panel Discussion on Progress in the Field of Regenerative Medicine. The event was hosted by ISCRM Interim Co-Director Dr. Hannele Ruohola-Baker and featured nine UW researchers using stem cells to study and stop a wide range of chronic and inherited diseases.
We are pleased to share a summary of the presentations. You can also access the full recording of the brief talks and the Q&A below.
Tom Reh, PhD and Jennifer Chao, MD, PhD – Vision Disorders
At the University of Washington, the Reh Lab uses retinal organoids grown from stem cells in an attempt to replace photoreceptors that are lost in multiple vision disorders. The Chao Lab differentiates stem cells into retinal pigment epithelium, which acts as support cells for the photoceptors, and is investigating how to transplant these cells into the eye. Beyond UW, clinical trials are planned or are underway for people with Usher syndrome, retinitis pigmentosa, and other eye diseases.
Vincenzo Cirulli, MD, PhD – Type 1 Diabetes
Researchers pursuing stem cell therapies for type 1 diabetes face several key challenges. They must find ways to protect cell grafts from immune-mediated rejection, using biomaterials or gene edits; determine the optimal site where to transplant the cells; minimize contamination from unwanted cell types; and scale up production of insulin-secreting cells. Human clinical trials at Vertex have produced encouraging results in patients and are expanding, while Sana Biotechnology has shown that gene-edited stem cells prevent rejection in non-human primates.
Brandon Hadland, MD, PhD and Sergei Doulatov, PhD – Blood Disorders
Advances in gene editing technologies and lentiviral methods, paired with hematopoietic stem cells, have contributed to the first FDA-approved treatment for sickle cell disease and to new CRISPR approaches to treat Beta thalassemia. While limitations and risks remain, other gene therapies to treat a wide range of blood, immune, and metabolic disorders are also in clinical trials. Finally, interventions derived from cord blood are being evaluated as off-the-shelf treatments for blood cancers, solid tumors, and autoimmune disorders.
Erica Jonlin, PhD – Regulatory
FDA-required preclinical studies include safety, toxicology, and efficacy assessments in cells and animals; recently, stem cell-derived organoids and microphysiological systems are also being used to acquire safety and efficacy data. If the intervention appears safe and the data is acceptable to FDA, and there is enough money, the next steps are a phase one clinical trial to look for safety and appropriate dose. This may be followed by a larger phase two controlled trial with affected patients. Throughout the process, questions about who goes first and misconceptions about the purpose of the trials can cause confusion and tension with the public.
Ryan McCarthy, PhD – Liver Disease
Currently, transplantation is the only cure for cirrhosis of the liver, but lack of supply and immune complications are challenges. Work on new approaches involves three strategies: support (ensuring patients are healthy enough to receive an organ), repair (healing scarring caused by cirrhosis), and replace (generating healthy replacement tissues). Clinical trials for cell therapies are underway at Penn, at the University of Edinburgh, and in Japan and China, and emerging gene-editing approaches are also promising.
Jessica Young, PhD – Neurodegenerative Diseases
Neuronal cells, which are important for memory and movement, and glia, which mediate neuroinflammation, both play a role in diseases like Alzheimer’s, Parkinson’s, and ALS. The impervious nature of our skulls and blood brain barriers, and inefficient differentiation into proper cell types, are challenges in the use of stem cells as treatments. One promising therapy for Parkinson’s is now in clinical trials led by stem cell biologist Dr. Lorenz Studer and neurosurgeon Dr. Viviane Tabar.
Learn more about stem cells and regenerative medicine in the ISCRM Learning Center.