A team of UCLA scientists will take an innovative lung cancer treatment through the final steps toward its first clinical trial with the benefit of a $1M grant from the National Cancer Institute’s Small Business Innovation Research program.
This pioneering treatment combines immunotherapy with leading-edge nanotechnology to deliver a drug therapy to cancer patients in a way that better preserves healthy tissue and lessens toxic side effects compared to traditional radiation and chemotherapy.
UCLA Jonsson Comprehensive Cancer Center members Dr. Leonard Rome, associate director of the California NanoSystems Institute and Dr. Steven Dubinett, director of the UCLA Clinical Translational Science Institute, developed the groundbreaking technique that uses biological particles called vaults to deliver a cancer immunotherapy agent directly to the tumor cells.
Vaults are made from proteins and exist within all human cells, and were discovered in Dr. Rome’s laboratory in the 1980s. He and colleagues then developed a way to construct the vaults from their original protein building blocks, but with hollow centers that could contain and gradually release drug molecules.
Inside these vaults is placed CCL21, a type of protein (called a chemokine) that normally resides in human lymph nodes. There it acts as a beacon to T cells and dendritic cells (often called the “foot soldiers of the immune system”) that find and destroy early indicators of disease such as bacteria and viruses.
Under normal circumstances, these foot soldiers do not recognize tumors as an enemy. But when the CCL21-vaults reach the cancer cells, the chemokines alert the immune cells to enter the cancer and eliminate the foreign cells. Thus, tumors are reduced without affecting surrounding healthy tissue.
Drs. Rome and Dubinett are next working toward a Phase I clinical trial, in hopes that cancer patients will be able to benefit from the therapy.
“Fifteen years ago, our discovery of how to reconstruct vaults as therapeutic nanocapsules completely changed the direction of my laboratory” Rome said. “Since then we have been working tirelessly to bring vault drug delivery technology to medical practice. We have many vault nanoparticle therapeutics in development, but the CCL21-vault is the first medicine that will reach clinical trials.”
Dr. Rome, co-principal investigator of the research, received the Small Business Innovation Research II (SBIR II) grant from the National Cancer Institute to complete the preclinical safety evaluation of the new technology. The SBIR is part of the National Cancer Institute’s program to develop and commercialize novel technologies to prevent, diagnose and treat cancer. It is one of the largest sources of early stage technology financing in the United States.