Speeding Stem Cells from Lab to Clinic Cedars-Sinai Skip to content Close Select your preferred language English عربى 简体中文 繁體中文 فارسي עִברִית 日本語 한국어 Русский Español Tagalog English English عربى 简体中文 繁體中文 فارسي עִברִית 日本語 한국어 Русский Español Tagalog Translation is unavailable for Internet Explorer Cedars-Sinai Home 1-800-CEDARS-1 1-800-CEDARS-1 Close Find a Doctor Locations Programs & Services Health Library Patient & Visitors Community My CS-Link RESEARCH clear Go Close Navigation Links Academics Faculty Development Community Engagement Calendar Research Research Areas Research Labs Departments & Institutes Find Clinical Trials Research Cores Research Administration Basic Science Research Clinical & Translational Research Center (CTRC) Technology & Innovations News & Breakthroughs Education Graduate Medical Education Continuing Medical Education Graduate School of Biomedical Sciences Professional Training Programs Medical Students Campus Life Office of the Dean Simulation Center Medical Library Program in the History of Medicine About Us All Education Programs Departments & Institutes Faculty Directory 2019 Research News Back to 2019 Research News Speeding Stem Cells From Lab to Clinic Twelve leading experts discussed the potential clinical applications of stem cells at a Cedars-Sinai workshop held in association with the annual meeting of the International Society for Stem Cell Research, a leading professional organization of more than 4,000 scientists worldwide. Spinal motor neuron cells (blue) and capillary cells (red) were generated using induced pluripotent stem cells for a 2018 study of brain development led by Cedars-Sinai investigators. "The workshop was very exciting and stimulating," said speaker Jun Takahashi, MD, PhD, of Kyoto University in Japan, where he works at the renowned Center for iPS Cell Research and Application directed by Shinya Yamanaka, MD, PhD. Yamanaka shared the 2012 Nobel Prize in Physiology or Medicine for his stem cell discoveries. The event, sponsored by the Cedars-Sinai Board of Governors Regenerative Medicine Institute, drew a sold-out audience of 300 to the campus in June. Besides Takahashi, investigators from the institute, the University of California system, Stanford University, Cincinnati Children's Hospital and Lund University in Sweden presented their latest research projects. Speakers from Origent Data Sciences in Vienna, Virginia, and the U.S. Food and Drug Administration (FDA) added insights from industry and regulators. Most of the investigators' studies involved induced pluripotent stem cells (iPSCs), which scientists create by genetically altering ordinary adult skin or blood cells so that they can make any tissue cell. These cells can be used to study how diseases develop, to test drug treatments and to generate healthy cells that potentially can replace diseased cells. "The FDA is committed to advancing the clinical development of cell-based therapies," said Peter Marks, PhD, MD, director of the Center for Biologics Evaluation and Research at the FDA, which has launched programs to expedite evaluation of these therapies. But so far the clinical effect of such treatments have been modest outside of hematologic disorders, which affect blood and blood-forming organs, he explained. The workshop's presenters are trying to change that picture. Among the diseases they are tackling: Amyotrophic lateral sclerosis (ALS): Clive Svendsen, PhD, director of the Board of Governors Regenerative Medicine Institute and professor of Biomedical Sciences, is conducting a clinical trial that combines stem cell and gene therapies. The goal of this line of research is to eventually stall progression of this devastating neurological disorder. Parkinson's disease: Takahashi is on a Kyoto University team that implanted progenitor cells of dopaminergic neurons, which make dopamine, into the brain of a Parkinson's patient in October 2018. The goal of this clinical trial is to develop a therapy for this disorder, which involves progressive impairment or death of these neurons. The progenitors were derived from iPSCs. Samuel Sances, PhD, a project scientist at the Board of Governors Regenerative Medicine Institute, is using iPSCs to create dopaminergic neurons on tissue chips to learn how genes may drive Parkinson's. Age-related macular degeneration: Dennis Clegg, PhD, from the University of California, Santa Barbara, is using IPSCs to make retinal pigment epithelial cells, which support the eye's photoreceptor cells. Loss of pigment epithelial cell function causes photoreceptor cells to die in this disorder, resulting in loss of vision. The ultimate goal is to replace these cells in patients and "stop macular degeneration in in its tracks," Clegg said. Wound-healing disorder: Anthony Oro, MD, PhD, from the Stanford University School of Medicine, is conducting preclinical studies on a rare, gene-based disorder that impairs healing of wounds. His lab is using iPSCs to generate sheets of skin cells that potentially might be grafted onto patients to repair wounds. The FDA's Marks said a major challenge for the stem cell field is that the public may find it difficult to distinguish science-based clinical trials, such as those discussed at the Cedars-Sinai workshop, from what he called "gray market" regimens offered by various companies to patients. In an interview, Marks said patients should be wary of a clinical trial offering if they are asked to pay money to participate, if they are not asked to sign an informed consent form or if the trial is not being conducted under an Investigational New Drug Application process involving the FDA. "The International Society for Stem Cell Research is really important because they are trying to develop stem cell therapies in a scientific and ethical manner," Marks said. Please ensure Javascript is enabled for purposes of website accessibility