Several years ago, scientists learned how to harness the power of stem cells; these cellular blank slates that can turn into any cell type can now be created with adult cells using genetic reprogramming. They are called induced pluripotent stem cells (iPSCs). Stem cell therapies have been in development ever since.
Earlier this year, researchers at the University of California (UC) San Diego treated a cancer patient with a type of immune cell, called natural killer (NK) cells, that had been derived from stem cells. NK cells are able to detect abnormal cells without any prior knowledge of them and are highly effective at eliminating tumor cells. Tumor cells can employ strategies to get around them, including using compounds that stymie the effects of NK cells in the tumor microenvironment. Cancer researchers have been able to genetically modify NK cells to make them better at fighting cancer, however.
At UC San Diego, the scientists knew that massive amounts of NK cells could be created using stem cells, and can be used without having to match them specifically to a patient. Their clinical trial aimed to treat 64 patients with solid tumors that had grown to an advanced stage using FT500, an 'off-the-shelf' cell product.
"This is a landmark accomplishment for the field of stem cell-based medicine and cancer immunotherapy. This clinical trial represents the first use of cells produced from human induced pluripotent stem cells to better treat and fight cancer," said Dan Kaufman, M.D., Ph.D., professor of medicine in the Division of Regenerative Medicine and director of cell therapy at University of California San Diego School of Medicine.
"This is a culmination of 15 years of work. My lab was the first to produce natural killer cells from human pluripotent stem cells. Together with Fate Therapeutics, we've been able to show in preclinical research that this new strategy to produce pluripotent stem cell-derived natural killer cells can effectively kill cancer cells in cell culture and in mouse models," he added.
Collaborators at the University of Minnesota have also now begun a clinical trial of their own, which is sponsored by Fate Therapeutics.
"We potentially have an unlimited source of very similar, reproducible cancer fighters," said Claudio Brunstein, M.D., Ph.D., who is a professor of Medicine at the University of Minnesota Medical School. "This is opening a whole new door in cellular therapy. With increased modifications to these NK cells, we can elevate their ability to attack tumors. As we add more functionality to NK cells, we have the potential to bring together multiple anti-tumor mechanisms and more effectively target and kill cancer."
"FT516 is the first-ever cell therapy derived from a genetically engineered iPSC cleared for clinical testing in the world," said Scott Wolchko, president and CEO of Fate Therapeutics. "Our use of a master engineered iPSC line uniquely supports a new treatment paradigm, where engineered cell products are available off-the-shelf and multiple doses can be readily administered to a patient, with the goal of driving deeper and more durable responses."