There are still many questions about degenerative diseases such as ALS or Alzheimer’s. The scientific community continues to work with the aim of discovering an effective treatment for these ailments, although it is not an easy undertaking. Our most complex organ, the brain, is still unknown to us in many aspects.
Despite this, small victories are being achieved, like the one we are dealing with in this article. And a group of researchers from Mount Sinai Hospital (New York), with the collaboration of some external colleagues, has managed to successfully implant certain drugs related to some of the most complex diseases into the brains of model mice.
A novel way to directly deliver drugs to the brain
Today we tell you about the discovery of a process that allows drugs to be administered directly into the brain, thus crossing the main “obstacle”, the blood-brain barrier.
What is the blood-brain barrier and why is it a “problem”?
In reality, this element is essential for our survival. And the so-called “blood-brain barrier” is a protective shield for the brain that prevents toxic and harmful particles from entering it.
The blood-brain barrier is made up of endothelial cells that line the capillaries that supply blood to the brain organ. It is a unique and amazing defense system, which protects the brain from the potential toxicity of certain molecules.
This protective function of the blood-brain barrier can become a problem, however. And, in the same way that it does not allow harmful particles to enter the central nervous organ, it also does not allow in those drugs that could have very beneficial effects on the nervous system and, specifically, to combat diseases such as Alzheimer’s or ALS ( Amyotrophic Lateral Sclerosis).
Mount Sinai research and its positive results
In this sense, the research and positive results of the Mount Sinai team is crucial. The scientists collaborating on the study have discovered a way to “get around” the blood-brain barrier and allow drugs to penetrate directly into the brain.
This new method takes advantage of a biological process called γ-secretase-mediated transcytosis to safely and efficiently transport the corresponding drugs to the central nervous system. The drug is administered through an intravenous injection and, once it manages to cross the natural protective barrier of the brain, it successfully penetrates the brain tissue.
At least, this has happened with the study’s model mice, genetically treated to develop ALS. After the injection, there was a significant decrease in the harmful genes causing the disease, with no harmful side effects observed in the animals.
In the same way, a reduction of the gene was also observed Maptresponsible for encoding the TAU protein and which is behind diseases such as Alzheimer’s and other dementias.
A new horizon for Medicine
Yizhou Dong, professor of Immunology and Immunotherapy, member of the Icanh Genomics Institute and one of the authors of the experiment, was really satisfied with the results. As he himself states, the blood-brain barrier is an invaluable natural defense, but it can sometimes represent a challenge when it comes to administering drugs to the brain.
The new procedure breaks this barrier and thus allows the entry of biomacromolecules and oligonucleotides into the nervous system, effectively and safely. Eric J. Nestler, director of The Friedman Brain Institute and co-author of the work, states that this finding can, at the moment potentially, solve one of the most important questions in the treatment of brain-related diseases. All of this opens a new horizon full of hope towards the cure of complex diseases such as ALS, Alzheimer’s and other dementias.