For years, one of the most powerful weapons against certain blood cancers, called CAR-T therapy, has required an elaborate process: Doctors extract a patient’s immune cells, ship them to a specialized facility where they’re genetically reprogrammed to fight cancer, then ship them back for infusion into the patient’s bloodstream. This has revolutionized cancer treatment, but the time and expense place it out of reach for thousands of patients.
Now, scientists at UC San Francisco have developed a method to precisely reprogram these cancer-fighting cells directly inside the body, potentially eliminating the barriers that have kept this life-saving therapy out of reach for many patients around the world.
It is the first time that scientists have integrated a large sequence of DNA at a specific site in human T cells without removing them from the body. This targeted approach, which did better than the standard method, is a breakthrough that goes beyond CAR T to advance the fields of cell and gene therapy overall.
In experiments using mice with humanized immune systems, described March 18 in Nature, the researchers used the method to successfully treat aggressive leukemia, multiple myeloma, and even a solid tumor. Scientists hope the new method will lead to an off-the-shelf therapy, like a vaccine, that could one day be given to anyone with the same condition.
“I think this is just the beginning of a big wave of new therapies that will be truly transformational and save a lot of lives,” said Justin Eyquem, PhD, an associate professor of medicine at UCSF, member of the Weill Cancer Hub West, and the senior author of the new paper. “I’m incredibly excited to be part of it.”