Breakthrough Drug Delivery System Crosses Blood-Brain Barrier

Scientists at Mount Sinai have developed a platform that safely delivers large therapeutic molecules to the brain, potentially transforming treatments for neurological diseases.

• The new Blood-Brain Barrier-Crossing Conjugate (BCC) system uses γ-secretase-mediated transcytosis to transport drugs into the brain via intravenous injection.
• In mouse models, the platform successfully reduced harmful genes associated with ALS and Alzheimer’s disease with minimal side effects.
• The treatment was also effective in human brain tissue samples, demonstrating its potential for clinical applications.
• Researchers emphasize the platform's ability to safely deliver large biomacromolecules, such as oligonucleotides and proteins, into the central nervous system.
• Further studies in larger animal models are planned to validate the system and explore its therapeutic potential for a wide range of brain diseases.

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