The blood-brain barrier is a “gatekeeper” that prevents toxins and foreign matter in the bloodstream from traveling to the brain.
Check Orphan recently carried an article that had been published in the Cell Stem Cell journal describing this new discovery and what it means to patients with neurological disorders such as Parkinson’s, amyotrophic lateral sclerosis, and Huntington’s.
Collectively, these three diseases affect millions. Scientists have identified a link between these disorders and blood-brain barriers that are defective and block therapeutic drugs from entering the brain.
The defective barriers are known to shut out biomolecules that are part of the process that keeps organisms alive.
The newly recreated brain component functions just as it does in the person whose cells were used for the procedure. The creation offers an entirely new pathway for scientists to be able to hone in on drugs best suited for patients.
About The Study
Pluripotent stem cells that are capable of developing into any type of cell or tissue were generated by Cedars-Sinai investigators. These stem cells use an adult’s blood sample to produce cells.
Although scientists have previously created the barrier outside the human body, this new procedure is a significant advance in science. It uses pluripotent stem cells to generate a blood-brain barrier inside an Organ-Chip that replicates the defect in a patient’s disease.
The cells were used to make neurons, support cells, and linings in blood vessels which taken together create a blood-brain barrier.
The various cells were placed within Organ-Chips, recreating the microenvironment (molecules, cells, structures, and forces) experienced by cells in humans.
This newly-created barrier functioned outside the body similar to its functioning inside the body- i.e. it blocks certain drugs from reaching the brain.
It is noteworthy that when cells of patients with diseases such as Huntington’s disease were used to create a blood-brain barrier, it malfunctioned in a similar fashion just as it does within patients who carry the disease.
A Joint Effort
This research is a joint effort by Cedars-Sinai and Emulate, Inc.
Cedars’ investigators provided its innovative stem cell science. Emulate of Boston, Massachusetts provided its Organs-on-Chips technology.
Emulate’s system uses software apps, instrumentation, and Organ-Chips as small as AA batteries. Living human cells numbering in the tens of thousands line tiny channels within the batteries.
This extraordinary technology is unprecedented. It provides control of human biology as well as controlling disease mechanisms.
Practical Applications
Patients will be able to benefit from a myriad of clinical applications. The patient’s cells will be placed in Organ-Chips and exposed to a drug or drugs. Clinicians can then collect accurate data gauging the patient’s response to potential treatment.
This is especially important as it avoids the risk of treating the patient with a drug that could be harmful, ineffective or costly.
The potential for using Organ-Chips is extraordinary. Organ-Chips can be used by scientists to create a living model of Parkinson’s, amyotrophic lateral sclerosis or Crohn’s disease.
