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Novel Drug Delivery Approach to Treat Brain Tumors in Children

Researchers at Mount Sinai Health System and Memorial Sloan Kettering Cancer Centre have developed a new medication delivery technique that uses nanoparticles to deliver anti-cancer medicines to children with brain tumors in a more effective and focused manner.

The technology allows anti-cancer drugs to be administered to specific brain tumor sites while protecting normal brain regions. According to their findings, which were published in Nature Materials on March 2, 2023, the consequence is greater anti-cancer treatment efficacy and fewer toxicities.

The most common malignant pediatric brain tumor, accounting for roughly 20% of all pediatric brain tumors, is medulloblastoma. It is exceedingly aggressive and difficult to treat, with about 30% of cases judged incurable. Because of the terrible side effects of radiation and chemotherapy, even "cured" children have serious long-term problems and health risks.

The blood-brain barrier, known for its unique characteristics and strict regulation, serves as a protective shield for the brain, preventing infections or harmful substances from accessing the damaged brain tissue and impeding targeted drug delivery.

In this study, the researchers utilized a well-known strategy employed by the immune system to transport white blood cells to areas of infection, inflammation, or tissue damage. Instead of haphazardly distributing immune cells throughout the body, these cells employ a targeted mechanism on active blood vessels to navigate toward the required locations. Leveraging this specific homing ability, which is also present in blood vessels within brain tumors, the researchers directed drug-carrying nanoparticles to the diseased site rather than non-affected regions of the brain.

By employing the novel drug delivery technology in a mouse model of medulloblastoma with a genetic correlation, the researchers successfully enhanced the effectiveness of anti-cancer therapy. This breakthrough has the potential to benefit a specific subgroup of medulloblastoma patients who are currently hindered by bone toxicity, particularly among children.

According to the researchers, continued research and development of this technology aimed at enhancing the transportation of substances across the blood-brain barrier and other targeted locations could significantly enhance the efficacy of various approved and experimental therapies. This pharmaceutical delivery platform holds the potential to be employed in the treatment of brain tumors, as well as other types of cancer, along with inflammation-related conditions affecting the central nervous system and other parts of the body.

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