Scientists Crack The Code To Deliver Medicines Directly To Brain
The blood-brain barrier is a protective membrane that shields the brain from harmful toxins and pathogens. However, it also makes it difficult for medications and therapies to reach the brain. Recently, researchers at Mount Sinai have discovered a potential solution to this challenge. They successfully delivered genetic therapies into the brains of mice by utilizing a natural cellular transport process, according to a study published in Nature Biotechnology. This breakthrough enabled the reduction of harmful gene activity in the brains of lab mice related to conditions like ALS, Alzheimer’s, and dementia.
Yizhou Dong, a senior researcher and professor at Icahn School of Medicine, explained that while the blood-brain barrier plays a crucial protective role, it also poses a significant obstacle for drug delivery. The new method, known as the blood-brain barrier-crossing conjugate (BCC) system, allows drugs to bypass this barrier safely and effectively. The blood-brain barrier itself consists of tightly packed cells that prevent larger molecules from entering the brain, safeguarding it from harmful substances but also limiting the delivery of therapeutic treatments.
The technique centers on a process called transcytosis, where substances are transported through cells. By linking the gene-based therapies to a compound called BCC10, the researchers were able to use transcytosis to prompt cells in the blood-brain barrier to carry the drugs across into the brain. This method successfully introduced genetic treatments into the bloodstream of mice, with minimal impact on major organs and good tolerance observed in the animals.
This new platform could represent a major advancement in brain research, as it addresses one of the biggest hurdles in treating brain diseases: delivering large therapeutic molecules through the blood-brain barrier. According to Dr. Eric Nestler, another senior researcher, this development holds promise for improving treatments for a variety of neurological conditions.
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