Gene Therapy Boosts Parkinson’s Disease Drug (Levodopa) Benefits in Mouse Model

In late-stage Parkinson’s disease, the drug levodopa becomes less effective in treating symptoms because of the inexorable loss of dopamine-releasing neurons. But a new Northwestern Medicine preclinical study, published online on November 3, 2021 in Nature, shows that a gene therapy targeting the small brain region where these neurons reside, the substantia nigra, substantially boosts the benefits of levodopa. The gene therapy restored the ability of neurons in the substantia nigra to convert levodopa to dopamine. In essence, this allowed levodopa to recreate the environment found in the healthy brain and eliminated the aberrant brain activity responsible for difficulty in moving. The Nature article is titled “Disruption of Mitochondrial Complex I Induces Progressive Parkinsonism.”