At the retreat for USC’s Department of Stem Cell Biology and Regenerative Medicine, newly appointed leader Chuck Murry shared a vision as sweeping as the panoramic view of the Pacific Ocean from the top floor conference room of Ventura’s Crowne Plaza hotel.
Nearly 200 faculty, staff, students and trainees gathered from April 9–10 for the annual event, where Murry outlined his big-picture plans for expanding the department, strengthening interdisciplinary and clinical collaborations across USC and the LA Basin, and accelerating translational medicine.
“My goal is to have our first program in the clinic in three years, and to have three programs in the clinic within a decade,” said Murry, who is the Chair of the Department of Stem Cell Biology and Regenerative Medicine and Director of the Eli and Edythe Broad Center for Regenerative Medicine at USC. “I think this is something we can do.”
To support this goal, Juliane Glaeser, the Program Director for Translational and Collaborative Research at USC Stem Cell, gave a progress report about building a SPARK Global Program at USC. Founded in 2006 at Stanford University, SPARK Global brings together academic and industry partners to provide education and mentorship to advance research discoveries into new patient treatments.
Keynote speaker Aileen Anderson, Professor and Director of the Sue and Bill Gross Stem Cell Research Center at the University of California, Irvine, shared her complicated story of bringing a stem cell-based treatment for spinal cord injury to clinical trials, including many roadblocks and challenges.
In another talk focused on clinical translation, Justin Ichida, a department faculty member who serves as Vice President and Head of Neuroscience Research at BioMarin Pharmaceutical, shared thoughtful reflections about the future of gene and cell therapies. As an example, he talked about the successes and limitations of three approved gene therapies for Spinal Muscular Atropy, which is the leading genetic cause of infant mortality.
Addressing another key aspect of clinical translation, Mohamed Abou-el-Enein, Executive Director of the USC/CHLA Cell Therapy Program, provided updates about the university’s clinical-grade cell manufacturing facility, called the cGMP.
In addition, the retreat’s career panel highlighted USC scientists with experience in industry as well as academia. Moderated by PhD students Pedro Medina and Catherine Diadhiou, the panel included: Camilo Ansarah-Sobrinho, the Director of MESH Strategic Partnerships; Alix Vaissié, Associate Director of USC cGMP Operations; Professor Senta Georgia, Director of USC’s CIRM COMPASS Program; Professor Danny El-Nachef, who recently returned to academia from a biotech startup; Professor Nancy Pire-Smerkanich, Associate Director of Regulatory Knowledge and Support; and PhD student Una Stevic, Director of Partnerships at Nucleate.
The retreat also featured three sessions of faculty talks focusing on areas of major research strength for USC Stem Cell: the musculoskeletal system; the kidney; and neurogenesis and neurodegeneration.
Gage Crump kicked off the musculoskeletal session with a talk about his recent Nature study describing the mammalian outer ear’s evolutionary origin in the gills of fishes and marine invertebrates. Francesca Mariani shared her research about mammalian rib regeneration, which is coordinated by a special type of cartilage cell with properties of both cartilage and bone. Tom Lozito talked about his study in Nature Communications showing how to improve lizard tail regeneration and more recent experiments attempting to stimulate lizard limb regeneration. Albert Almada talked about Duchenne muscular dystrophy and the role of the gene FOS in muscle stem cell activation. Denis Evseenko discussed his progress in preparing for clinical trials of a stem cell-based surgical implant for focal cartilage lesions and of a new drug for osteoarthritis.
The kidney session began with talks from two recent recruits to USC’s Department of Medicine: Chair Susan Gurley and Assistant Professor Jonathan Nelson. Gurley introduced her research related to diabetic kidney disease and the roles of the hormone angiotensin and the enzyme ACE2 in blood pressure regulation and kidney injury. Nelson talked about different cell populations that contribute to kidney scarring or fibrosis, or how to prevent it. Laura Perin from Children’s Hospital Los Angeles (CHLA) discussed how fasting mimicking diets can reprogram cancer stem cells into normal progenitor cells in the most common type of pediatric kidney cancer, known as Wilm’s tumor. Janos Peti-Peterdi talked about how a salt-sensing kidney structure called the macula densa can orchestrate kidney remodeling. Zhongwei Li gave an update about his project to build a synthetic kidney using two distinct populations of stem cell-derived progenitor cells that can form filtration units called nephrons and urine drainage structures called collecting ducts. Nils Lindström explored how to connect the nephrons with the collecting ducts, as well as with the blood supply.
In the session focused on neurogenesis and neurodegeneration, Shafali Jeste from CHLA talked about challenges and successes involved in launching a clinical trial for children with a rare disease called 15Q Duplication Syndrome, a neurodevelopmental disorder that can cause developmental delays, motor impairment, intellectual disability, autism and epilepsy. Justin Ichida delved into his efforts to identify drugs that can treat many different forms of ALS, despite the divergent, complex and often unknown underlying genetic causes. To address the nearly universal problem of age-related cognitive decline, Michael Bonaguidi introduced a computational strategy that identified a drug that encourages neural stem cell proliferation and neuron formation in mice. Yulia Shwartz shared her discoveries about how nerves regulate hair follicle stem cells, and how males lose this innervation more than females with age, leading to hair loss, thin skin and impaired wound healing. Ksenia Gnedeva talked about gene regulation affecting the sensory hearing cells of the inner ear, which don’t naturally regenerate after hearing loss in mammals.
In addition, Kayla de la Haye and Mayla Boguslav from the Southern California Clinical and Translational Science Institute hosted a special interactive session about team science. They introduced simple strategies, such as taking formal turns during group discussions, that can strengthen the collective intelligence and psychological safety of scientific teams.
“This year’s retreat reflects our strong commitment to clinical translation,” said Murry. “We want to use stem cells and genes both as agents to discover new therapeutics, and to use stem cells and genes as medicines themselves. So I’m still bullish on regenerative medicine, and now is the time to make big progress in creating new and better options for patients.”