Scientists Develop In-Body Cancer-Fighting Immune Cells

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Researchers at the University of California, San Francisco (UCSF) have developed a groundbreaking new approach to CAR-T cell therapy that can reprogram cancer-fighting T cells directly inside the patient's body. This innovative method, which utilizes CRISPR gene-editing tools, has shown success in treating aggressive blood cancers and even solid tumors in mice. By eliminating the need for the complex and expensive external manufacturing process, this new in-body reprogramming technique could dramatically expand access to this potentially life-saving cancer treatment.

Why it matters

Current CAR-T cell therapy is an effective but inaccessible treatment, costing hundreds of thousands of dollars and requiring a lengthy manufacturing process that can take weeks. The UCSF team's breakthrough could make this personalized cancer immunotherapy more widely available, empowering community hospitals to offer the treatment rather than just specialized cancer centers.

The details

The UCSF researchers have developed a dual-particle system that delivers CRISPR gene-editing tools to T cells, precisely inserting new DNA that encodes cancer-fighting chimeric antigen receptors (CARs). This targeted approach has demonstrated success in treating aggressive leukemia, multiple myeloma, and even solid tumors in mice with humanized immune systems. Interestingly, the T cells engineered inside the body appear to outperform those manufactured in a lab, likely because they maintain more of their 'stemness' and proliferative capacity.

• The UCSF researchers have founded a company, Azalea Therapeutics, to advance this platform through clinical development.
• Seven CAR-T cell therapies are currently approved by the U.S. Food and Drug Administration for use in blood cancers.

What’s next

Clinical trials are necessary to confirm the safety and efficacy of this approach in humans.