Interim data from Voyager Therapeutics Inc. suggests that a combination of gene-therapy with the drug levodopa can improve the Unified Parkinson’s disease rating scale (UPDRS) of a patient by up to 55%.
Levodopa is usually prescribed to patients in the early stages of Parkinson’s disease. The medication is converted to dopamine by the enzyme Aromatic L-Amino Acid Decarboxylase (AADC) however as the disease progresses the drug loses its effectiveness. This is because in advanced Parkinson's disease, levels of dopamine and AADC decrease in the putamen, an area of the brain that regulates movement. Increasing AADC levels in the putamen, to produce more dopamine could mean that patients in the advanced stages of the disease might better manage their movement-related symptoms.
Massachusetts based Voyager Therapeutics neurosurgically injected its trial gene therapy, VY-AADC01, into the striatum (which contains the putamen) using MRI guided navigation. Interim results from cohorts 1 and 2 of the study show improvement in AADC activity and reduction of Parkinson’s symptoms. Cohort 2, saw a 9-point or 55% increase in UPDRS III on medication, and ‘very importantly, these improvements in motor function occurred with a substantial 34% reduction in daily doses of oral levodopa… at six months… that was maintained at 12 months’.
Although the study is still at an early stage, and not due to be completed until 2018, the results have led to a 35% percent jump in Voyager Therapeutics share price. Dosing of a third cohort of patients is currently ongoing, and expected to be completed by 2017. The company expects to be able to post longer-term data from Cohorts 1 and 2 at a medical conference next year.
http://www.phgfoundation.org/news/17537/
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