The mice were “cured” of Parkinson’s disease after a scientific study took an unexpected turn.
Scientists at the University of California (UC) at San Diego set out to better understand the role of proteins in connective cells, only to discover a way to transform many different types of cells into neurons.
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Parkinson’s occurs when the neurons (nerve cells) that make the messenger chemical dopamine, which regulates movement, die. Patients often experience tremors, slow movements, and loss of balance when 80% of dopamine is lost.
Scientists used this discovery to develop a unique treatment that eliminated Parkinson’s symptoms in mice, raising hopes for a cure in the future.
Existing treatments aim to alleviate the signs of the disease and improve the patient’s quality of life, such as through physical therapy.
‘I was surprised by what I saw’
More than 145,000 people over the age of 20 in the UK are believed to have lived with Parkinson’s in 2018. In the United States, nearly 1 million people have the disease.
UC scientists were studying a protein called PTB that turns genes on or off within cells.
In an attempt to better understand how PTB influences cell function, they silenced the protein in fibroblasts from connective tissue cells.
Fibroblasts were cultured in Petri dishes to verify any changes.
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A couple of weeks later, the scientists were surprised to discover that there were very few fibroblasts left, which had been largely replaced by neurons.
In a later experiment, published in the journal Nature, the team found that brain cells called astrocytes also “turned” into neurons when PTB was silenced.
“Researchers around the world have tried many ways to generate neurons in the laboratory, using stem cells and other means, so that we can better study them, as well as use them to replace lost neurons in neurodegenerative diseases,” said the lead author, Dr. Xiang-Dong Fu.
“The fact that we could produce so many neurons in a relatively easy way was a big surprise.”
Then the scientists turned their attention to Parkinson’s.
The rodents were exposed to a chemical that poisons dopamine-producing neurons, creating symptoms of the disease.
After silencing the PTB, 30% of the mice astrocytes converted to nerve cells, reaching levels comparable to those of normal rodents.
These new neurons seemed to grow normally and even sent connections to other parts of the brain.
As for symptoms, deactivation of PTB completely restored normal movement in the mice, with a single treatment that lasted a lifetime.
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“I was surprised by what I saw,” said co-author Dr. William Mobley.
“This completely new strategy for treating neurodegeneration gives hope that it will be possible to help even people with advanced disease.”
Parkinson’s Professor David Dexter from the UK added: “Cell transplants have long been aimed at replacing lost cells in Parkinson’s, but their effectiveness has been limited as they struggle to integrate and function effectively within the brain.
“This new technique has overcome this major hurdle in mice and opens the door to an exciting new treatment approach, which can reverse Parkinson’s in people in the future.”
‘Critical progress is at stake’ due to coronavirus
Although promising, much more rigorous testing is required before the approach can be tested in humans.
Scientists plan to silence PTB in mice with genetic changes that cause Parkinson’s-like symptoms, rather than the disease being caused by dopamine poisoning.
“Advances in technologies like this are vital and this is promising and well-conducted research at an early stage, but it is in mice and it is not yet clear if this approach could be used in people,” said Dr. Sara Imarisio of Alzheimer’s Research UK.
Neurons are also destroyed in Alzheimer’s disease.
“Further research will need to develop a better understanding of the possible adverse effects of converting these cells in this way before we can know if this technique is even possible in a human brain,” said Dr. Imarisio.
Professor Tara Spiers-Jones from the University of Edinburgh added: “While the principle of this study is remarkable and promising, it is important to note that it was conducted in mice with groups of three to eight and there is a long way to go to translate this into a treatment for people. “
However, the UC team has patented its treatment in hopes that it may one day help patients.
“It is my dream to see this through clinical trials, to test this approach as a treatment for Parkinson’s disease, but also for many other diseases where neurons are lost, such as Alzheimer’s and Huntington’s disease, and stroke. “said Dr. Fu.
“Dreaming even more, what if we could target PTB to correct defects in other parts of the brain, to treat things like hereditary brain defects?
“I intend to spend the rest of my career answering these questions.”
While everything looks promising, medical research charities in the UK have warned that “patients will suffer the consequences” unless they receive government support amid the coronavirus outbreak.
“Results like this highlight the potential of medical research, but critical progress is at stake and it is essential that dementia research is backed by the government throughout the COVID-19 crisis,” said Dr. Imarisio.
COVID-19 is the respiratory disease that can be triggered by the coronavirus.
“Research is the only way we can end fear, anguish, and the damage that diseases like Parkinson’s cause,” added Dr. Imarisio.
Before the pandemic, Parkinson’s UK “knew” a breakthrough and the cure for the disease was “close.”
Instead, the organization has been forced to “fight for fair treatment and better services” for patients who are most at risk of complications from the coronavirus.
To maintain this increased support, Parkinson UK must raise £ 95,000 ($ 118,872) per week for the next three months.
