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SAN FRANCISCO — Twenty years ago, CAR-T cell therapy was not quite “believed in,” said Michel Sadelain, director of the Center for Cellular Engineering at Memorial Sloan Kettering Cancer Center. It was hard to find patients to put in a trial, he said, because physicians were skeptical of the new technology. Then, when the first patients were successfully treated, there was “suddenly this turnabout, a tsunami as some would say, of young scientists and not-so-young scientists embracing this.”

The enthusiasm for CAR-T has not let up since the mid-2000s, agreed Carl June, a professor at the University of Pennsylvania and a CAR-T pioneer, speaking with Sadelain and Christi Shaw, co-founder of the More Moments More Memories Foundation, on a panel about CAR-T therapy at STAT’s Breakthrough Summit in San Francisco.  Autologous CAR-T cells, immune cells taken from a patient and engineered in a lab to recognize and destroy cancer cells, have cured patients of blood cancers once considered incurable. But the field is still being hampered by early regulations initially aimed at safeguarding against concerns about a fledgling technology that has since matured and proven its safety.


“When we started in 2010, we were appropriately concerned that genetic engineering would create so-called Frankenstein cells that would be uncontrollable,” June said. “There were lots of regulatory issues put in that has slowed research. But 11 to 12 years in, more than 20,000 people treated, and it’s never happened — the Frankenstein cell turned into a CAR-T tumor hasn’t happened. Autologous cells are safe, they don’t need to be regulated as in experimental, early stage by the FDA.”

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