Credit: Mount Sinai Health System
Key points:
- New technology allows for the enhanced delivery of anti-cancer drugs to the specific locations of brain tumors while sparing normal brain regions.
- The method is based on nanoparticles that can slip past the blood-brain barrier.
- The drug delivery platform is much less toxic than the alternatives.
Researchers have developed a new drug delivery approach that uses nanoparticles to enable more effective and targeted delivery of anti-cancer drugs to treat brain tumors in children. The technology delivers anti-cancer drugs to the specific locations of brain tumors while sparing normal brain regions.
In the study, the researchers piggy-backed off a mechanism that the immune system uses to traffic white blood cells to sites of infection, inflammation or tissue injury. They used this unique homing feature, which is also found within brain tumor blood vessels, to target their drug-loaded nanoparticles to the site of the disease and not the normal brain regions.
“Certain proteins appear on blood vessels at sites of inflammation that help white blood cells exit the bloodstream. They work like police officers at the site of a car accident, who let in emergency personnel to help," explains Daniel Heller, Head of the Cancer Nanomedicine Laboratory at Memorial Sloan Kettering Cancer Center, and senior author on the study. “We sent in our own emergency personnel, in the form of drug-loaded nanoparticles, composed of certain sugar molecules that can target these same proteins.”
When Heller and colleagues tested the method in a genetically relevant mouse model of medulloblastoma, they were able to enhance the efficacy of an anti-cancer drug that could potentially be useful for a subset of medulloblastoma patients, but which is currently limited by the bone toxicity it secondarily creates in children.
Medulloblastoma is the most common malignant pediatric brain tumor, accounting for about 20 percent of all brain tumors in children. It is highly aggressive and difficult to treat, and is considered incurable in nearly 30 percent of patients. Even children who are “cured” experience severe long-term disabilities and health issues, primarily due to the adverse side effects of radiation and chemotherapy.
The researchers say continued investigation and development of this method will be instrumental for improving the efficacy of several classes of approved and experimental therapeutics. Additionally, the drug delivery platform can be used to treat cancers in the brain and other sites of the body, as well as other inflammation-related diseases in the central nervous system and elsewhere.
Information provided by Mount Sinai Hospital.