Mayo Clinic identifies potential multiple myeloma drug to shrink tumors

Mayo Clinic researchers have found that an experimental drug, LCL161, stimulates the immune system, leading to tumor shrinkage in multiple myeloma patients. The findings are published in Nature Medicine.

“The drug LCL161 was developed to promote tumor death,” says Marta Chesi, Ph.D., Department of Health Sciences Research and lead author of the study. “However, we found that the drug does not kill tumor cells directly. Rather, it makes them more visible to the immune system, which recognizes them as foreign invaders and eliminates them.”

Mayo Clinic researchers will conduct a follow-up clinical trial of LCL161 in combination with an inhibitor of immune checkpoints that has been widely used in many cancer treatments to evaluate if LCL161 could present a potential new treatment option.

This research highlights the importance of studying the effects of drugs not only on the tumor cells in a culture plate but also on the interaction of the tumor cells with their own microenvironment. The finding that LCL161 is active against multiple myeloma suggests that similar drugs may have broader clinical activity than previously thought.