At one point, Shinya Yamanaka, M.D., Ph.D., was so down and discouraged that he almost gave up his research into embryonic cells and their powerful potential to replace diseased cells. He was finding little interest among colleagues; funding was hard to come by; and research journals rejected his studies.

Then, discovery of human embryonic cell generation at the University of Wisconsin–Madison renewed his hope and inspiration. Dr. Yamanaka persisted in his goal to harness the power of embryonic stems and their healing potential. His groundbreaking discovery of induced pluripotent stem cells (iPS cells) — reprogramming cells to differentiate into any type of cell in the body — earned him the Nobel Prize in Physiology or Medicine in 2012.

Dr. Yamanaka and his team succeeded in generating iPS cells in mouse models in 2006. Then, in 2007, Dr. Yamanaka discovered how to generate iPS cells, which have the potential to become any type of cell, including brain, heart and nerve cells, from small patches of human skin, known as fibroblasts.

"The discovery of iPS cells has ushered in a new era of cell therapy. In Japan, clinical trials using iPS cell-derived target cells and tissues are already underway," says Dr. Yamanaka, director emeritus and professor of the Center for iPS Cell Research and Application. "Disease models using patient iPS cells have created an entirely new drug discovery process, which is now being applied to intractable diseases."

The Nobel laureate will be a keynote speaker at The Mayo Clinic Symposium on Regenerative Medicine & Surgery 2023 that will be held April 3–5 at the Westin Kierland Resort and Spa in Scottsdale, Arizona. 

"In the presentation, I would like to introduce recent progress and future prospects of medical applications using iPS cells in Japan, as well as challenges we are facing," says Dr. Yamanaka. "Regenerative medicine using iPS cells is expected to bring to reality innovative medical treatments that go beyond conventional symptomatic therapies to actually help patients regenerate lost functions. On the other hand, drug discovery using patient iPS cells is expected to help deliver drugs that are truly safe and effective."

Transforming research

Dr. Yamanaka's discovery of induced pluripotent stem cells has shaped research into complex disorders at Mayo Clinic. Examples of reprogrammed cells in Mayo research include:

• Reprogramming bile duct cells to study progression and possible treatments for liver disease.
• Growing brain organoids — so called "mini brains" — to create a humanlike model for studying Alzheimer's disease.
• Creating sensory neurons to investigate and track progression of peripheral neuropathy.
• Recreating disease-in-a-dish models of multiple sclerosis and genetic heart disease to understand progression and test new therapies.
• Engineering retinal pigment epithelial cells to understand and treat macular degeneration.
• Studying reprogrammed heart cells in a clinical trial to determine if they could safely repair heart tissue and heal congenital heart defects.

"Understanding induced pluripotent stem cells is vitally important for the current and next generation of physicians and scientists," says Richard Hayden, M.D., course director for the Mayo Clinic Symposium on Regenerative Medicine & Surgery 2023. "Induced pluripotent stem cells are tools for generating a vast supply of human tissue vital to understanding disease. Researchers and clinicians who are trained to use this tool may be equipped to provide new regenerative solutions for complex diseases, particularly those with no therapeutic options."

Mayo Clinic's Center for Regenerative Biotherapeutics is sponsoring the Symposium on Regenerative Medicine & Surgery 2023 as part of its objective of training the future workforce to deliver new regenerative therapies.

The symposium will address topics such as biologics — medicines derived from human sources, such as cells, blood, enzymes, tissues, genes or genetically engineered cells. It also will feature the latest in regenerative immunotherapies, oncology, regulatory issues, cell-free therapies, tissue engineering and 3D printing. Conference attendees may earn 9.25 continuing medical education credits.

Additional keynote speakers include:

• Peter Marks, M.D., Ph.D., director of the Center for Biologics Evaluation and Research at the Food and Drug Administration.
• Bruce Levine, Ph.D., immediate past president of the International Society for Cell & Gene Therapy. Dr. Levine also is founding director of the Clinical Cell and Vaccine Production Facility in the Department of Pathology and Laboratory Medicine and the Abramson Cancer Center, Perelman School of Medicine, University of Pennsylvania.
• Raghu Kalluri, M.D., Ph.D.,professor and chairman of the Department of Cancer Biology and the director of the Metastasis Research Center at MD Anderson Cancer Center.
• Harald Ott, M.D., associate professor of surgery at Harvard Medical School and a thoracic surgeon at Massachusetts General Hospital.

New sessions will be added to the agenda as the conference planning continues.

Register now for the Mayo Clinic Symposium on Regenerative Medicine & Surgery 2023.

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*Photo courtesy: Center for iPS Cell Research and Application, Kyoto University

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