Accidental Scientific Discovery Brings Cure to Mice with Parkinson’s Disease

Generating neurons is only one of many goals that have so far escaped scientists worldwide. According to an article in Yahoo, the lead author of a new study in the UK described their work with a protein called PTB.

He explained that they were originally hoping to generate neurons (units of nerve tissue) for study purposes or to replace lost neurons in neurodegenerative diseases. But instead, they were “surprised” at the number of neurons they were actually able to generate with very little effort.

About Parkinson’s Disease

In 2018 an estimated 145,000 people in the United Kingdom were known to have Parkinson’s disease. The total in the United States is approximately one million people with the disease.

Parkinson’s disease, a neurological and progressive disease, is characterized by slow movement, rigidity, shuffling, and tremors.

Working with PTB

Polypyrimidine-tract-binding protein (PTB) is an RNA-binding protein that regulates genes by switching them on and off.

PTB was primarily known for the role it played in splicing. Currently, it is recognized for its function in many cellular processes.

The scientists began the study by investigating PTB’s influence in cell function. They proceeded by silencing PTB in fibroblasts which are the primary active cells in connective tissue.

The next step was to check for any changes by growing fibroblasts in Petri dishes.

Within a few weeks, they discovered that the majority of fibroblasts were gone and were replaced with neurons.

An article subsequently featured in “Nature” described the team’s experience with another discovery. The scientists discovered that astrocytes (brain cells) also transformed into neurons following the silencing of PTBs.

Astrocytes are specialized cells that are five times more prevalent than neurons. Astrocytes “tile” the entire area of the central nervous system.

Focusing on Parkinson’s Disease

The team was encouraged and decided to focus on Parkinson’s disease. A chemical that poisons the neurons that produce dopamine was used on animal models. This created Parkinson’s symptoms in rodents.

As a result of PTB being silenced, thirty percent of the rodent’s astrocytes became nerve cells. Their nerve cells rose to similar levels found in untreated rodents.

Not only did the neurons grow normally, but they also sent signals to other areas in the brain.

Lasting a Lifetime

As far as symptoms after PTB was turned off, normal movement returned, and the single treatment the mice received lasted throughout their lifetime.

About a New Approach

Co-author of the study, William Mobley M.D., said he was ”stunned.”

David Dexter, professor at Parkinson’s UK, explained that scientists have been trying to replace lost cells. The problems they have encountered are the new cells’ inability to function properly in the brain.

He further explained that their new technique overcame this hurdle. Dr. Dexter said that this is a new and exciting approach to treatment that may eventually reverse Parkinson’s.

The new approach will, of course, require extensive analysis before it can begin human trials. In the future, scientists intend to silence the PTB in animal models using genetic changes instead of dopamine poisoning.

Additional research must be developed for a better understanding of potential side effects to determine if it is even feasible to use the technique in the human brain.


What are your thoughts about the possibility of a cure for Parkinson’s disease? Share your stories, thoughts, and hopes with the Patient Worthy community!

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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