At the inaugural California Institute for Regenerative Medicine (CIRM) Trainee Network Conference, over 400 students and postdocs from across California gathered for three days of learning, networking, and career development on USC’s University Park Campus. The conference also hosted representatives from CIRM, the International Society for Stem Cell Research (ISSCR), the National Marrow Donor Program, and the cell manufacturing facilities at USC, Cedars Sinai, and Stanford University. The conference was organized by USC Stem Cell and funded by a grant from CIRM with additional sponsorship from ThermoFisher Scientific and Amgen.
The conference opened with welcoming remarks from Carolyn Meltzer, Dean of the Keck School of Medicine of USC, and from conference organizers and faculty members Francesca Mariani and Louise Menendez from USC’s Department of Stem Cell Biology and Regenerative Medicine.
During the three-day conference, attendees enjoyed presentations from 85 speakers, as well as 44 moderators and panelists.
Three patient advocates shared their remarkable stories. Jake Javier talked about his paralyzing spinal cord injury and participation in Phase 2 of an Asterias Biotherapeutics clinical trial, which injected an experimental treatment made from stem cells directly into his spine. Kristin Macdonald shared her experience living with a blinding condition called Retinitis Pigmentosa, and her participation in a Phase 1 clinical trial of a stem cell-based therapy developed by Henry Klassen from UC Irvine and the biotech company jCyte. Kim Cade, Director of Sister’s Hope Foundation, explained her advocacy work as someone who has two brothers with a rare terminal brain disorder known as adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP), which causes symptoms similar to ALS.
Attendees also enjoyed keynote addresses from prominent scientists.
Justin Ichida, who is the John Douglas French Alzheimer’s Foundation Associate Professor of Stem Cell Biology and Regenerative Medicine at USC and the Vice President for Neuroscience Research at BioMarin, talked about his research to develop new therapies for patients with ALS. He highlighted the importance of collaborations between academia and industry partners including Takeda, which will soon advance his research to the clinical trial stage.
Aileen Anderson, a Professor of Physical Medicine and Rehabilitation and Director of the Sue and Bill Gross Stem Cell Research Center at the University of California (UC), Irvine, discussed how her research supported a phase I/II clinical trial for using human neural stem cells to treat spinal cord injury.
Donald B. Kohn, a Distinguished Professor of Microbiology, Immunology, Molecular Genetics, Pediatrics, Hematology/Oncology, and Molecular and Medical Pharmacology at UCLA, recounted his success in using a lifesaving gene therapy involving blood stem cells to cure more than 50 babies who were born without an immune system due to a condition called ADA-SCID or “bubble baby disease.” He’s now applying the same approach in clinical trials for other genetic disorders including sickle cell disease and various immunodeficiencies.
Additional faculty presenters included Kenneth S. Kosik from UC Santa Barbara discussing the physiology of human brain organoids; Ritchie Ho from Cedars Sinai talking about CRISPR-based gene therapy; and Paula Cannon from USC sharing her work in reprogramming human immune cells. Bryce Carey from Vertex also presented about a promising stem cell-derived therapy for Type 1 Diabetes.
Mini-breakout sessions covered research topics related to various organ systems, early embryonic development, cell therapy, organoids, immunotherapy, genomics, gene editing and gene therapy, and other stem cell-related topics.
Wei Yan, editor at the scientific journal eLife, gave a thought-provoking presentation about the evolution of publishing. He delved into some of the pros and cons of the traditional publishing process at scientific journals such as Cell, Nature, and Science, as well as the rise of the preprint server bioRxiv. He also shared how eLife started as a free journal that eliminated the anonymity of peer review, and how it will now do away with binary “accept or reject” decisions.
Students and postdocs enjoyed presenting their own research during short talks by Sushma Kalmodia from Children’s Hospital Los Angeles, Marcella Birtele from USC, Sydney Prange from UC Irvine, Franco Felix from Stanford University, Robert Lu from UC Berkeley, and Zahir Shah from City of Hope. There were a total of 76 trainee speakers, and more than 250 poster presenters. Three of the best presentations won prizes.
The conference also offered opportunities for professional development, with a career panel featuring professionals working in a variety of biotech careers from research to policy, communication, outreach, education, and patient advocacy. Breakout sessions included presentations ranging from how to apply to graduate school to how to find biotech industry positions, and additional sessions focused on networking.
One of the keynotes speakers was Robert Klein, II, who authored the California ballot propositions that funded CIRM, the state agency charged with distributing public funding to support stem cell research and education. Klein gave a detailed history of how California voters decided to allocate state funding for stem cell research and what this has meant for advancing the future of medicine.
“You are at the right place in the right time in history for breakthroughs that have never been imaginable that will change human history and human suffering, so that your family, your brothers and sisters, may not suffer the chronic diseases of millennia that all of our families coming before you have,” said Klein, who also serves as CIRM’s Founding Chair Emeritus, Chairman of the nonprofit Americans for Cures, and a member of the Sanford Stem Cell Institute’s Executive Council.
CIRM President and CEO Jonathan Thomas talked about how the stem cell agency supports not only research, but also education and DEI in the stem cell field.
“Everyone’s job is to get the word out about this wonderful work,” he said.
Mariani, who secured the conference funding and organized the conference, added: “This conference provided an invaluable platform for our trainees to engage directly with patient advocates, leading scientists, and industry experts. It was an opportunity for them to not only showcase their research, but also to build the networks and skills that will help shape the future of regenerative medicine.”