First-of-its-Kind Stem Cell and Gene Therapy Highlighted at Annual Stem Cell Meeting

Newswise — LOS ANGELES (June 9, 2022) -- Investigators from Cedars-Sinai will present the latest novel stem cell and regenerative medicine research at the International Society for Stem Cell Research (ISSCR) Annual Meeting, which is being held in person and virtually June 15-19 in San Francisco.

At this year’s scientific forum, Clive Svendsen, PhD, a renowned scientist and executive director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute, will assume the role as treasurer for the organization. In this position, he will be working with leading scientists, clinicians, business leaders, ethicists, and educators to pursue the common goal of advancing stem cell research and its translation to the clinic. 

Along with taking on this leadership role, Svendsen’s work on a combination stem cell-gene therapy for the treatment of amyotrophic lateral sclerosis, a fatal neurological disorder known as ALS or Lou Gehrig's disease, was selected as a “Breakthrough Clinical Advances” abstract and one of the year’s most compelling pieces of stem cell science. Svendsen will present data from the first spinal cord trial and a synopsis of the ongoing cortical trial and the potential impact this may have on this devastating disease.   

The breakthrough oral session, “A new trial transplanting neural progenitors modified to release GDNF into the motor cortex of patients with ALS,” takes place on Thursday, June 16, from 5:15 to 7 p.m. The presentation is part of the Biotech, Pharma and Academia–Bringing Stem Cells to Patients’ Clinical Applications track.

“Through this highly collaborative work, we hope to develop new therapeutic options for patients with such a debilitating and deadly disease,” said Svendsen, who is also the Kerry and Simone Vickar Family Foundation Distinguished Chair in Regenerative Medicine.

All abstracts are embargoed until the start of each individual presentation.

Additional noteworthy presentations featuring Cedars-Sinai investigators at ISSCR 2022 include:

•  Alexandra Moser, PhD, a postdoctoral fellow in the Svendsen Laboratory, will be presenting an oral abstract on how iPSC-derived immune cells improve cognition and neural health in aging mice, which is part of the Biotech, Pharma and Academia–Bringing Stem Cells to Patients’ Clinical Applications track.
•  Arun Sharma, PhD, principal investigator in the Regenerative Medicine Institute, Smidt Heart Institute, and Department of Biomedical Sciences, and Madelyn Arzt, research associate in the Sharma Laboratory, will discuss the use of hiPSC-derived cardiovascular cells to investigate the reduced cardiotoxicity of a novel protein-encapsulated doxorubicin variant. 
•  Paul Linesch, a researcher in the laboratory of Joshua Breunig, PhD, is presenting an update on the non-viral inducible transgenic system used for transcription factor based directed differentiation of neuros and glia.
•  Ruchi Shah, PhD, postdoctoral fellow, will be discussing the role of epigenetic regulation of WNT5A in diabetic corneal epithelial wound healing and limbal stem cell expression.
• Tyler M. Pierson, MD, PhD, assistant professor in the Department of Neurology and Pediatrics, has a poster on iPSC modeling of neurodevelopment and GATAD2B-associated neurodevelopmental disorder and the nurd complex.

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