UCSF scientists have developed a “molecular GPS” to guide immune cells into the brain and kill tumors without harming healthy tissue.
It is the first living cell therapy that can navigate through the body to a specific organ, addressing what has been a major limitation of CAR-T cancer therapies until now. The technology worked in mice and the researchers expect it to be tested in a clinical trial next year.
Working in mice, the scientists showed how the immune cells could eliminate a deadly brain tumor called glioblastoma – and prevent recurrences. They also used the cells to tamp down inflammation in a mouse model of multiple sclerosis.
“Living cells, especially immune cells, are adapted to move around the body, sense where they are and find their targets,” said Wendell Lim, PhD, UCSF professor of cellular and molecular pharmacology and co-senior author of the paper, which appears in Science on Dec. 5.
Navigating to the source of disease
Nearly 300,000 patients are diagnosed with brain cancers each year in the U.S., and it is the leading cause of cancer mortality in children.
Because of their location, brain cancers are among the hardest cancers to treat. Surgery and chemotherapy are risky, and drugs can’t always get into the brain.
To get around these problems, the scientists developed a “molecular GPS” for immune cells that guided them with a “zip code” for the brain and a “street address” for the tumor.