Jul 17, 2019 | Updated: 11:17 AM EDT

Scientists Can Now Recreate Blood-Brain Barrier Defect Outside the Body

Jun 06, 2019 02:41 PM EDT

Scientists Can Now Recreate Blood-Brain Barrier Defect Outside the Body
(Photo : Cedars-Sinai/Cell Stem Cell) High-magnification image shows a blood-brain barrier Organ-Chip, with brain macrovascular endothelial-like cells in magenta on the blood side and support cells known as astrocytes in green on the brain side.

It is not possible for scientists to make a living copy of your brain outside your body. According to new research, scientists recreated a critical brain component, the blood-brain barrier, that functioned as it would in the individual who provided the cells to make it.

The scientists detailed their achievement in a study published in the peer-reviewed journal Cell Stem Cell, and it provides a new way to make discoveries about brain disorders and potentially predict which drugs will work best for an individual patient.

As a gatekeeper, the blood-brain barrier blocks toxins and other foreign substances in the bloodstream from entering brain tissue and damaging it. Also, it can prevent potential therapeutic drugs from reaching the brain.

Some of the neurological disorders including amyotrophic lateral sclerosis (Lou Gehrig's disease), Parkinson's disease, and Huntington's disease, which collectively affect millions of people, have been connected to defective blood-brain barriers that keep out biomolecules needed for healthy brain activity.

In this research, a team led by Cedars-Sinai investigators generated stem cells known as induced pluripotent stem cells, which can produce any cell, using an individual adult's blood samples.

The researchers used these specialized cells to make neurons, blood-vessel linings, and support cells that together make up the blood-brain barrier. Then, they placed the various types of cells inside Organ-Chips, which recreated the body's microenvironment with the natural physiology and mechanical forces that cells experience within the human body.

Soon, the living cells formed a functioning unit of a blood-brain barrier that functions as it does in the body, including blocking entry of certain drugs. Essentially, when this blood-brain barrier was derived from cells of patients with Huntington's disease or Allan-Herndon-Dudley syndrome, a rare congenital neurological disorder, the barrier malfunctioned in the same way that it does in patients with these diseases.

Even though scientists have created blood-brain barriers outside the body before, this study further advanced the science by using induced pluripotent stem cells to generate a functioning blood-brain barrier inside an Organ-Chip that displayed a characteristic defect of the individual patient's disease.

The senior author of the study and director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute, Clive Svendsen, Ph.D., said that the findings of the study open a promising pathway for precision medicine. The possibility of using a patient-specific multicellular model of a blood-brain barrier on a chip represents a new standard for developing predictive, personalized medicine.

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