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New nano-vault delivery system can fight cancer

April 15, 2016

If successful, the vault delivery method would add a desperately needed weapon to the arsenal in the fight against lung cancer, which accounts for nearly one-third of all cancer deaths in the United States and kills one million people worldwide every year.

"It's crucial that we find new and more effective therapies to fight this deadly disease," Dubinett said. "Right now we don't have adequate options for therapies for advanced lung cancer."

The vault nanoparticles containing the CCL21 have been engineered to slowly release the protein into the tumor over time, producing an enduring immune response. Although the vaults protect the packed CCL21, they act like a time-release capsule, Rome said.

Rome, Dubinett and Sharma plan to test the vault delivery method in human studies within the next three years and hope the promising results found in the pre-clinical animal tumor models will be replicated. If such a study is approved, it would be the first time a vault nanoparticle is used in humans for a cancer immunotherapy.

The vault nanoparticle would require only a single injection into the tumor because of the slow-release design, and it eventually could be designed to be patient specific by adding the individual's tumor antigens into the vault, Dubinett said. The vaults may also be targeted by adding antibodies to their surface that recognize receptors on the tumor. The injection could then be delivered into the blood stream and the vault would navigate to the tumor, a less invasive process that would be easier on the patients. The vault could also seek out and target tumors and metastases too small to be detected with imaging.

Rome cautioned that the vault work is at a much earlier stage than Dubinett's dendritic cell research, but he is encouraged by the early results. The goal is to develop an "off-the-shelf" therapy using vaults.

"In animals, the vault nanoparticles have proven to be as effective, if not more effective, than the dendritic cell approach," he said. "Now we need to get the vault therapy approved by the FDA for use in humans."

Because a vault is naturally occurring particle, it causes no harm to the body and is potentially an ideal vehicle for use in delivery of personalized therapies, Rome said.

Source: California NanoSystems Institute (CNSI)