New gene therapy discovered for late-stage Parkinson’s disease treatment

Written by Harriet Wall

gene therapy

In a new study from Northwestern University Feinberg School of Medicine (IL, USA), researchers have developed a new gene therapy to improve the effect of levodopa, a dopamine replacement agent for the treatment of Parkinson’s disease. 

As Parkinson’s disease progresses, levodopa becomes less effective due to the slow, progressive loss of dopamine-releasing neurons in the brain, which are responsible for converting levodopa into dopamine – thus recreating a healthy brain environment. Up until now, scientists have not fully understood what causes this depletion in dopamine-releasing neurons. 

In their study, recently published in Nature, the team have suggested that damage to mitochondria, the powerhouse within these neurons, is the reason behind their demise.  

“The development of effective therapies to slow or stop Parkinson’s disease progression requires scientists know what causes it,” commented lead study author James Surmeier, chair of neuroscience at Northwestern University Feinberg School of Medicine. “This is the first time there has been definitive evidence that injury to mitochondria in dopamine-releasing neurons is enough to cause a human-like parkinsonism in a mouse.” 

“Whether mitochondrial damage was a cause or consequence of the disease has long been debated. Now that this issue is resolved, we can focus our attention on developing therapies to preserve their function and slow the loss of these neurons.” 

You might also like:

The team have developed a novel gene therapy that targets the area of the brain, called the substantia nigra, that contains these neurons. Once the damaged cells are restored, levodopa can once again successfully replenish the dopamine levels within the brain.  

This discovery has provided a new avenue of therapy for Parkinson’s disease treatments – even before clinical symptoms appear. 

“This new ‘human-like’ model may help us develop tests that would identify people who are on their way to being diagnosed with Parkinson’s disease in five or 10 years,” Surmeier concluded. “Doing so would allow us to get them started early on therapies that could alter disease progression.”