Parkinson’s Treatment Boosted by Gene Therapy

Parkinson’s disease drug benefits may be boosted by gene therapy, thereby opening a new path for late-stage Parkinson’s therapy as per a study at Northwestern University, to be published in the journal Nature. Parkinson's disease (PD) is a progressive neurodegenerative disorder that primarily affects movement due to the loss of nerve cells – neurons that produce a chemical messenger (neurotransmitter) in the brain called dopamine (black substance).

Levodopa is the standard treatment of care in Parkinson’s. In late-stage Parkinson’s disease, the drug levodopa becomes less effective due to inexorable loss of dopamine-releasing neurons.

Gene therapy and Parkinson's Disease

However the new study shows that gene therapy targets a small brain region (substantia nigra) to boost the levodopa’s effects.

It helps restore the ability of neurons in the substantia nigra to convert levodopa to dopamine. The study thereby aims to identify people who will develop Parkinson’s in five or 10 years.

The team also show that damage to the powerplants inside dopamine-releasing neurons (mitochondria) is sufficient to trigger a sequence of events that faithfully recapitulates what happens to brain circuits in Parkinson’s disease – loss of dopamine-releasing neurons using advanced genetic tools in mice.

“The development of effective therapies to slow or stop Parkinson’s disease progression requires scientists know what causes it. 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,” says lead study author D. James Surmeier, chair of neuroscience at Northwestern University Feinberg School of Medicine.

These findings may help identify humans in the earliest stages of Parkinson’s disease, and further develop therapies to slow disease progression and treat late-stage disease.



Source-Medindia