Researchers have made an exciting discovery that could lead to new treatments for Parkinson’s disease.
A team from the UK Dementia Research Institute at the University of Cambridge found that blocking a specific enzyme called ACLY can restore healthy brain cell function in animals and lab-grown human cells.
Their study was recently published in the journal Neuron.
Parkinson’s disease is a serious condition that affects the brain and causes movement problems like shaking, stiffness, and trouble with balance.
One of the main features of Parkinson’s is the build-up of a protein called alpha-synuclein inside brain cells.
These clumps, known as Lewy bodies, stop the cells from working properly and eventually cause them to die. As more brain cells die, the symptoms of Parkinson’s get worse.
Normally, our bodies have a way to clear out damaged proteins and other waste through a process called autophagy. Autophagy acts like a recycling system, breaking down and removing harmful materials inside cells. But in Parkinson’s, this system does not work well, allowing toxic proteins like alpha-synuclein to build up and damage the brain.
In the new study, Dr. Sung Min Son and colleagues found that alpha-synuclein triggers the over-activation of an enzyme called ACLY. They used human brain cells, mini-brains grown in the lab (called organoids), zebrafish, and mice to study the effects.
They discovered that when ACLY becomes too active, it disrupts the autophagy process. This leads to more alpha-synuclein build-up and more damage to the brain cells.
Importantly, the researchers showed that when they blocked ACLY, they were able to fix the broken recycling system. As a result, the levels of toxic alpha-synuclein dropped, and the brain cells started to function more normally.
They achieved this by using drugs that block ACLY. These treatments worked not only in human cells and mini-brains but also in animal models like zebrafish and mice.
In mice that carried a gene mutation known to cause Parkinson’s in humans, blocking ACLY led to improved removal of the toxic protein and less disease-related damage. This suggests that targeting ACLY could directly address one of the root causes of brain cell death in Parkinson’s.
There are already some known compounds that can block ACLY. One example is hydroxycitrate, a chemical often found in weight-loss supplements, although its use has been controversial. Other ACLY-blocking drugs have been studied for cancer treatment.
However, the problem with current ACLY inhibitors is that they cannot easily cross the blood-brain barrier—a protective shield that keeps most substances in the blood from reaching the brain.
Because of this, the next big step for the research team is to develop a version of an ACLY inhibitor that can get into the brain. If they succeed, it could lead to a completely new type of treatment for Parkinson’s, one that does more than just treat symptoms. It could actually slow down or even stop the disease from getting worse.
Professor David Rubinsztein, the lead author of the study, explained that ACLY seems to act like a switch inside brain cells. When it gets switched on too much, it causes a chain reaction that harms the cells.
Blocking ACLY flips the switch back off, helping the cells heal. He also said that their research shows that the problems in Parkinson’s disease are not only caused by the alpha-synuclein protein itself but also by how it messes up important cell processes like recycling waste.
The discovery that ACLY could be a powerful drug target brings new hope for developing treatments that could halt or even reverse Parkinson’s disease, offering new possibilities for millions of people around the world who are living with the condition.
If you care about Parkinson’s disease, please read studies that Vitamin B may slow down cognitive decline, and Mediterranean diet could help lower risk of Parkinson’s.
For more health information, please see recent studies about how wheat gluten might be influencing our brain health, and Olive oil: a daily dose for better brain health..
The research findings can be found in Neuron.
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