HOW UCLA STEM CELL RESEARCH IS TRANSFORMING MEDICINE

Director's Message | Summer 2026

Aug 04, 2026 Center News

Twenty years ago, a short paper appeared in the journal Cell that would quietly rewrite the rules of biology. There was no press conference, no fanfare to match the scale of what had been accomplished; there was only a deceptively simple finding: that an ordinary skin cell, treated with just four genes, could be coaxed backward through developmental time until it became, once again, a cell capable of becoming almost any cell in the body. Reading that paper today, 20 years on, what stands out is not just the result but the audacity of the question behind it.

The scientist behind the discovery, Shinya Yamanaka, had an unusual path to the bench. He trained first as an orthopedic surgeon, by his own admission not a particularly gifted one, and has said that watching colleagues operate with far greater skill than his own helped redirect him toward research. That redirection led him, with his student Kazutoshi Takahashi, to screen two dozen candidate genes down to a final four (Oct4, Sox2, Klf4, and c-Myc) that together could reprogram a mature cell into what we now call an induced pluripotent stem cell, or iPSC. The achievement was honored with remarkable speed: in 2012, just six years after publication, Yamanaka shared the Nobel Prize in Physiology or Medicine with John Gurdon, whose earlier work had first shown that cellular identity was not a one-way street.

UCLA's connection to this story runs closer to home than many may realize. Barely a year after Yamanaka's original report, our own Kathrin Plath, William Lowry, Amander Clark, and April Pyle were among the first researchers in the world to successfully reprogram human cells into iPSCs. Their work arrived almost in step with the founding of the UCLA Broad Stem Cell Research Center, which had launched just two years earlier. In a sense, the iPSC field and our Center grew up together, and that early alignment shaped the scientific identity we carry today.

Two decades on, the technology has moved from a remarkable laboratory trick to a genuine therapeutic pathway. Our Translational Cell Therapy Lab has spent nearly 10 years developing iPSC-derived retinal pigment epithelial cells as a potential treatment for macular degeneration, one of the leading causes of blindness. Last spring, that work reached a milestone years in the making: the submission of an Investigational New Drug application to the FDA, a pivotal step toward testing the therapy in patients. It is no coincidence that our newly opened UCLA Health Center for Advanced Biotherapies, which I described in the spring issue of this newsletter, was designed from the outset with iPSC-derived therapies explicitly in mind. The infrastructure and the science have, once again, grown up together.

What strikes me most, looking back across these 20 years, is how thoroughly a discovery once met with healthy scientific skepticism has become a shared language across our Center's more than 250 members. iPSCs now sit at the foundation of work ranging from autism and Alzheimer's research to heart disease and rare genetic disorders, each project a small extension of that original 2006 paper. Anniversaries like this one are useful not because they mark an ending, but because they remind us how far a single, well-asked question can take us.

I am grateful, as always, for the dedication of our faculty, trainees, and staff who carry this work forward every day. As we celebrate this anniversary, we do so not simply to honor a remarkable discovery, but to reaffirm the mission it continues to inspire: to pursue bold questions, translate discovery into therapies, and improve the lives of patients through rigorous, collaborative science.

 

Thomas Rando, MD, PhD
Director, UCLA Broad Stem Cell Research Center
Professor of Neurology and Molecular, Cell and Developmental Biology