Could This Breakthrough Unlock the Blood-Brain Barrier and Transform Brain Disease Treatment?

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Healthcare (Commonwealth Union) – Scientists at the Icahn School of Medicine at Mount Sinai have unveiled a groundbreaking method for delivering treatments directly to the brain. Tested on mouse models and human brain tissue, this innovative technique holds promise for addressing a variety of neurological and psychiatric disorders.

Published in the November 25 online edition of Nature Biotechnology, the research presents a novel blood-brain barrier-crossing conjugate (BCC) system. This system is designed to bypass the brain’s natural defense mechanism, which typically prevents large biomolecules from entering the central nervous system (CNS).

The blood-brain barrier serves as a vital shield, protecting the brain from harmful substances. However, this barrier also obstructs the delivery of critical therapies, posing a significant obstacle in treating diseases such as Brain and addiction, among other CNS-related conditions.

The BCC platform leverages a unique biological process known as γ-secretase-mediated transcytosis to transport large therapeutic molecules—such as oligonucleotides and proteins—into the brain through a straightforward intravenous injection.

Dr. Yizhou Dong, co-senior author of the study, Professor of Immunology and Immunotherapy, and a researcher at both the Icahn Genomics Institute and the Marc and Jennifer Lipschultz Precision Immunology Institute at Icahn Mount Sinai indicated that the blood-brain barrier is crucial for protecting the brain but remains a major hurdle for drug delivery.

Dr. Dong further indicated that their BCC system offers a safe and effective way to deliver biomacromolecules, including oligonucleotides, directly to the CNS.

The research revealed that injecting a compound called BCC10, combined with specialized genetic tools known as antisense oligonucleotides, into mice effectively reduced the activity of harmful brain genes. In a transgenic mouse model of ALS—a neurodegenerative motor neuron disease—the treatment significantly decreased the levels of the Sod1 gene and its associated protein, which are linked to the disease. Likewise, another antisense oligonucleotide paired with BCC10 substantially lowered the Mapt gene, responsible for producing the tau protein, a key target in therapies for Alzheimer’s disease and other forms of dementia.

BCC10 demonstrated remarkable efficiency in delivering these genetic tools to the brain, enhancing their capacity to suppress harmful genes in various models, including studies on excised human brain tissue in the lab. Furthermore, the treatment was well-tolerated in mice, with minimal or no damage to major organs at the doses tested, according to the researchers.

Researchers of the study indicated that while advancements have been made in this field, there remains a critical need for technologies that can cross the blood-brain barrier and enable systemic delivery of biomacromolecule-based therapies to the central nervous system.

“Our platform could potentially solve one of the biggest hurdles in brain research—getting large therapeutic molecules past the blood-brain barrier safely and efficiently,” added co-corresponding senior author Eric J. Nestler, MD, PhD, Nash Family Professor of Neuroscience, Director of The Friedman Brain Institute, as well as Dean for Academic Affairs of Icahn Mount Sinai, and Chief Scientific Officer of the Mount Sinai Health System. “This development has the potential to advance treatments for a broad range of brain diseases.”

Subsequently, the researchers intend to carry out additional experiments on sizable animal subjects to confirm the effectiveness of the system and explore its potential for treatment applications.

As the global population ages and the prevalence of devastating neurodegenerative disorders continues to rise, the need for innovative solutions has never been more urgent. By harnessing the power of science, technology, and personalized medicine, researchers can make significant strides in the battle against dementia as in the case of this study. It is through collaboration, investment, and unwavering commitment that we can unlock the potential for transformative treatments and offer a brighter future for those affected by Alzheimer’s and related conditions.

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