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Vault Nanoparticles Engineered at UCLA Show Promise for Cancer Treatment
Posted Date: 8/22/2014 11:00 AM

A multidisciplinary team of scientists from UCLA and Stanford University has used a naturally occurring nanoparticle called a vault to create a novel drug delivery system that could lead to cancer treatments that are more effective with smaller doses.

The research team was led by UCLA Jonsson Comprehensive Cancer Center members Dr. Leonard Rome, associate director of UCLA’s California NanoSystems Institute, and Dr. Jerome Zack, co-director of the UCLA AIDS Institute.

Rome and his then-postdoctoral student Nancy Kedersha discovered vaults in the 1980s. The naturally occurring nanoparticles number in the thousands inside each of our cells. A nanoparticle is a tiny substance, in this case constructed of proteins, that is measured in nanometers (1 nanometer is equal to 1 billionth of a meter).

Over the years, Rome and his collaborators discovered how to create vaults in the laboratory using the proteins that they consist of. Naturally occurring vaults contained other elements, but Rome’s team built empty ones, which eventually enabled them to pursue the idea of inserting drug molecules inside so they could be injected into a patient and directed to specific cells, where they would release the drugs.

Rome’s next goal was to improve upon existing chemotherapy drugs, which kill cancer cells, but also cause side effects because they are toxic to healthy tissue as well. Rome and his team theorized that using vaults to deliver drugs directly to the cancer cells would eliminate the drugs’ contact with healthy cells and should greatly reduce the treatment’s side effects. This concept has yet to be proven in humans, but the researchers are now closer to clinical trials of vault delivery technology.

“These experiments demonstrate the novel ability of these vaults to encapsulate therapeutic compounds up to more than 2,000 molecules per single vault,” said Rome, also a UCLA professor of biochemistry. “And these particular vaults can completely internalize their cargo, adding an extra layer of protection for healthy cells.”

The paper is the cover story of the Aug. 26 print edition of the journal ACSNano, and it was recently published online.

UCLA's Jonsson Comprehensive Cancer Center has more than 240 researchers and clinicians engaged in disease research, prevention, detection, control, treatment and education. One of the nation's largest comprehensive cancer centers, the Jonsson center is dedicated to promoting research and translating basic science into leading-edge clinical studies. In July 2014, the Jonsson Cancer Center was named among the top 10 cancer centers nationwide by U.S. News & World Report, a ranking it has held for 14 years.

For more information on the Jonsson Cancer Center, visit our website at


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