Parkinson’s UK invests in clinical trial of a potential treatment that could protect brain cells

The Parkinson's Virtual Biotech, alongside The Michael J. Fox Foundation for Parkinson's Research (MJFF), is partnering with UK based biotech company Mission Therapeutics to see whether MTX325 is safe, gets into the brain and improves energy production in brain cells.

If this small, early stage clinical trial is successful then larger trials would be needed to continue to uncover the potential of MTX325 to protect the brain cells that are lost over time in Parkinson's.

The role of mitochondria in Parkinson's

Brain cells need energy to function properly. It is the mitochondria, the energy producing power houses of the cell, that are responsible for this.

In Parkinson's, mitochondria inside dopamine-producing brain cells stop working properly. These faulty mitochondria cannot produce enough energy and leak harmful chemicals, causing damage that affects cells' ability to make dopamine and contributing to the death of brain cells over time.

Testing a drug to help protect brain cells

MTX325 is a compound that has been designed to help get rid of faulty mitochondria. By removing 'problem mitochondria' the team hope to protect dopamine-producing brain cells and ultimately slow or even stop the progression of the condition.

It can be taken as a pill and has been shown in lab based research to reach the brain, to allow brain cells to remain healthy and prevent them from dying.

What this investment will support

MTX325 is currently being tested in healthy individuals to understand its safety. This is vital as it is the first time this compound is being given to people.

The investment from the Parkinson's Virtual Biotech will help to carry out the next stage of the research - investigating the safety and benefits of the potential drug in people with Parkinson's.

The trial plans to recruit 50 people with Parkinson's to take part in the study over 28 days and is currently being planned across multiple sites in the UK and other European countries.

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Vital UK based clinical trial

Arthur Roach, Virtual Biotech Director at Parkinson's UK, said:

"We are delighted to be working with Mission Therapeutics to help fund this vital, UK-based clinical trial. Disease-modifying treatments for Parkinson's are one of the great hopes of people with Parkinson's. We now know that mitochondria play a crucial role in the development of Parkinson's, so addressing mitochondrial problems could have far-reaching benefits for those living with the condition."

Dr Paul Thompson, PhD, Chief Scientific Officer, Mission Therapeutics, said:

"We have already made excellent progress in healthy volunteers with preliminary data from the ongoing clinical trial showing a good single dose safety profile for MTX325. We look forward to starting the Parkinson's patient part of the trial in the new year, which this generous funding is helping to support."