JCI: Breakthrough of the blood-brain barrier allows delivery of drugs to treat encephalopathy and cancer

In a recent study published in the Journal of Clinical Investigation, Cornell researchers found a new way to penetrate the blood-brain barrier (BBB), which may soon allow the delivery of drugs directly into the brain to treat diseases, such as Alzheimer's disease and chemotherapy-resistant cancer.

The blood-brain barrier is a layer of endothelial cells that selectively allow molecules required for brain function to enter (the brain), such as amino acids, oxygen, glucose, and water, while excluding other substances.

Cornell University researchers report that an FDA-approved drug called Lexiscan activates adenosine receptors. Adenosine receptors are expressed in blood-brain barrier cells.

"We can open the blood-brain barrier in a short time window that is long enough to deliver the therapy to the brain, but not too long to harm the brain. We hope that this discovery can be used to treat multiple types of the future." "Senior author of the study," said Margaret Bynoe, associate professor of microbiology and immunology at Cornell University's College of Veterinary Medicine.

Bynoe's team is also able to deliver chemotherapeutic drugs to the brain of mice, as well as to deliver macromolecules, such as antibodies that bind to Alzheimer's disease plaques.

The researchers also used a human brain brain cell to establish a blood-brain barrier model. They found that in this model, Lexiscan was able to open the blood-brain barrier in a similar way to mice.

Because Lexiscan is an FDA-approved drug, potential breakthroughs in drug delivery systems for diseases such as Alzheimer's disease, Parkinson's disease, autism, brain tumors, and chemotherapy against cancer are not far off," says Bynoe.

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