In a first-of-its-kind clinical trial, UCLA scientists have shown it’s possible to reprogram a patient’s blood-forming stem cells to generate a continuous supply of functional T cells, the immune system’s most powerful cancer-killing agents. This approach suggests a new way to deliver immunotherapy, acting as an internal factory that produces tumor-targeting immune cells over time and potentially offering longer-lasting protection. 

This early-stage study, published in Nature Communications and led by physician-scientist Dr. Theodore Scott Nowicki in collaboration with Dr. Antoni Ribas, Dr. Owen Witte, Dr. Donald Kohn, Dr. Lili Yang of UCLA and Dr. David Baltimore from the California Institute of Technology, represents a novel strategy for treating hard-to-treat cancers, especially solid tumors that have proved to be more resistant to conventional T cell therapies.

Nowicki, assistant professor-in-residence of pediatric hematology/oncology and microbiology, immunology, and molecular genetics at the David Geffen School of Medicine at UCLA, and Ribas, professor of medicine and director of the tumor immunology program at the UCLA Health Jonsson Comprehensive Cancer Center, discussed the promise of this approach and its potential to transform cancer treatment and beyond. 

What’s the most important thing people should take away from this study?

Nowicki: We’ve shown that it’s possible to reprogram a patient’s own stem cells to create a renewable immune defense against cancer. That’s never been done in humans before. It’s not a cure yet, and it’s not ready for widespread use, but it points to a future where we don’t just treat cancer—we prevent it from coming back. 

Ribas: It took a team of more than 30 dedicated academic investigators and over a decade to bring to patients the concept of genetically programming the human immune system to result in a renewable source of cancer-targeted immune cells. The basic science principles had been established in preclinical models, and this study demonstrated that it is feasible to test them in patients with cancer. 

Read the full Q&A on the UCLA Health website.