Newly-developed approach enhance drug delivery for brain tumors in children

March 28, 2023 |

Researchers from Mount Sinai Hospital devised a technique for enhanced delivery of anti-cancer drugs to specific locations of brain tumors while avoiding normal brain regions. This approach resulted in greater effectiveness of lower doses and reduced bone toxicities of anticancer drugs as seen in younger patients.

The most common malignant pediatric brain tumor, medulloblastoma, accounts for over 20% of all brain tumors in children and is considered incurable in almost 30% of patients. Highly aggressive, children who are said to be cured still experience long-term disabilities and health issues as a consequence of radiation and chemotherapy. Site-directed drug delivery is hindered by a blood brain barrier that protects the brain from infections and harmful substances. 

The researchers utilized the homing mechanism, an immune system mechanism used in directing white blood cells to sites of infection, inflammation or injury. The team applied this feature in targeting their drug-loaded nanoparticles composed of sugar molecules to the specific site of the disease while bypassing normal areas of the brain. At the sites of inflammation, certain proteins appear on blood vessels that help the diffusion of white blood cells and nanoparticles also target them to speed the process.

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The new drug delivery system was performed in a relevant mouse model of medulloblastoma and they were able to improve the drug’s efficacy which could potentially be used on a subset of medulloblastoma patients. However, the applicability is limited due to the bone toxicity it secondarily creates in children.

Dr. Raju, associate professor from the said institution, said that the drug delivery approach can be enhanced with a low-dose radiation. Furthermore, the approach can also aid drug delivery for other pediatric brain tumors and localized diseases in the brain for both children and adults. This includes focal epilepsy, multiple sclerosis, stroke and other neurodegenerative disorders. 

Continued development of this method and other techniques can be used to treat brain cancers as well as other inflammation-related diseases of the central nervous system.

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