Genetic Research Discovery Leads to Human Brain Repair

In the June 25, 2020 edition of the science journal, Nature, genetic researchers at the University of California, San Diego, announced a procedure that ends Parkinson’s disease in lab mice. The lead researcher, Dr. Xian-Dong Fu, PhD.,  stated the lab’s intention to, “….test this approach as a treatment for  Parkinson’s disease, but also many other diseases where neurons (key cells in the brain) are lost such as Alzheimer’s, Huntington’s disease, and stroke. “

There are many steps before this procedure will become a Federal Drug Agency (FDA) approved treatment for human use, but this is the first approach that does not rely on controversial stem cell replacement or genetic manipulation of brain neurons.  Dr. Fu’s lab at UC San Diego has patented their discovery so that human trials can be moved along faster.  They are aiming for human medical use before the end of 2021.  Even though the process has only been tested in lab mice, these mice were genetically engineered to have a disease immune system mimicking that of humans.  Over 150 lab mice were genetically “infected” with human Parkinson’s disease, with half not given any treatment, while the other half were.  Within 3 months the mice receiving the treatment exhibited reduction (and eventually total elimination) of muscle tremors and spasms, while the untreated “control” group continued to worsen.  At that point, the mice were dissected, and researchers found that in the treated mice there was NEW brain cell growth and little brain scarring due to the disease. The untreated mice showed no improvement and exhibited even worse neuron brain cell loss.  In effect, the treated mice were cured of Parkinson’s disease, with the new brain cells functioning exactly as they should.

In the human brain, there are billions of neuron cells that transmit electrical pulses for muscle coordination.  Supported by star-shaped astrocyte cells, healthy neurons produce dopamine which is essential for bodily movement, pain processing, learning and concentration, sleep, blood vessel function, healthy organ function, a sense of well-being, and is a key neurotransmitter.  As a neurotransmitter, dopamine acts like lubricating oil in a car’s engine, keeping electrical pulses in the brain moving freely between neurons.  In brain diseases, like Parkinson’s, neuron death and the loss of dopamine over time leads to a loss of mental and physical stability, and eventually death. 

Most brain cells are in place at birth and are meant to last a lifetime.  Unlike other cells, they are not constantly being replaced, even when they are damaged or die off. The UC San Diego researchers discovered injecting a lab created  “designer” gene into the region of the brain responsible for muscle coordination, resulted in astrocytes developing into NEW dopamine-producing neurons, replacing those lost to disease.  This led to a healthy brain free from Parkinson’s disease, or potentially any brain disorder where the loss of neurons occurs.

For UC San Diego’s press release see the web site, https://ucsdnews.ucsd.edu/pressrelease/one-time-treatment-generates-new-neurons-eliminates-parkinsons-disease-in-mice/.