In a new study, researchers found a one-time treatment could generate new neurons in the brain and eliminate Parkinson’s disease.
They found a single treatment to inhibit protein PTB in mice converted native astrocytes, star-shaped support cells of the brain, into neurons that produce the neurotransmitter dopamine.
As a result, the mice’s Parkinson’s disease symptoms disappeared.
PTB is well known for binding RNA and influencing which genes are turned “on” or “off” in a cell.
The finding could one day be a new therapeutic approach for Parkinson’s disease and other neurodegenerative diseases.
The research was conducted by a team at the University of California San Diego School of Medicine.
In the study, the mice lose dopamine-producing neurons and develop symptoms similar to Parkinson’s disease, such as movement deficiencies.
The treatment works like this: The researchers developed a noninfectious virus that carries an antisense oligonucleotide sequence—an artificial piece of DNA designed to specifically bind the RNA coding for PTB, thus degrading it, preventing it from being translated into a functional protein and stimulating neuron development.
Antisense oligonucleotides, also known as designer DNA drugs, are a proven approach for neurodegenerative and neuromuscular diseases, and it now forms the basis for a Food and Drug Administration (FDA)-approved therapy for spinal muscular atrophy and several other therapies currently in clinical trials.
The researchers used the PTB antisense oligonucleotide treatment directly to the mouse’s midbrain, which is the part of the brain that typically loses dopamine-producing neurons in Parkinson’s disease.
In the treated mice, a small subset of astrocytes converted to neurons, increasing the number of neurons by approximately 30%. Dopamine levels were restored to a level comparable to that in normal mice.
What’s more, the neurons grew and sent their processes into other parts of the brain. There was no change in the control mice.
By two different measures of limb movement and response, the treated mice returned to normal within three months after a single treatment and remained completely free from symptoms of Parkinson’s disease for the rest of their lives.
In contrast, the control mice showed no improvement.
The team says this whole new strategy for treating neurodegeneration gives hope that it may be possible to help even those with advanced disease.
Next, the team plans to optimize their methods and test the approach in mouse models that mimic Parkinson’s disease through genetic changes.
They have also patented the PTB antisense oligonucleotide treatment in order to move forward toward testing in humans.
One author of the study is Xiang-Dong Fu, Ph.D., a Distinguished Professor in the Department of Cellular and Molecular Medicine at UC San Diego School of Medicine.
The study is published in Nature.
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