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Monday, August 22, 2016

New Parkinson’s Hope - Preliminary ONLY

I don’t normally cover medical developments at this stage but with zero useful long term treatments for Parkinson’s Disease available, I feel that this is worth reporting.

The standard of treatment (essentially the ONLY treatment) for this nerve disease which affects about 700,000 Americans and upwards of 10 million worldwide has, for thirty years, been the chemical levodopa drug which initially gives very good relief from the disease which causes tremors and progressive paralysis along with a number of other symptoms.

Problems with L-dopa

Levodopa or L-dopa works great for most patients when initially given to Parkinson’s and other nerve damaged patients but the effectiveness wears off either gradually for Parkinson’s or dramatically for some other diseases.

Now a Cambridge, MA company, Voyager Therapeutics is conducting AADC gene therapy trials which shows promise in extending the period when L-dopa works.

This was shown in the 1990 Robert De Niro/Robin Williams movie Awakenings which chronicle the discovery by Dr. Oliver Sacks who produced dramatic awakening of catatonic patients who had suffered encephalitis infections.

Clinical Trial

The following is a direct quote from the clinical trial information sheet.

Parkinson's disease is a neurodegenerative disorder involving loss of neurons that release dopamine in the striatum. To compensate for the loss of dopamine, patients are typically prescribed levodopa medication which is converted to dopamine by the enzyme Aromatic L-Amino Acid Decarboxylase (AADC). As Parkinson's disease progresses, levodopa therapy becomes less effective and is associated with motor fluctuations, involuntary movements and other complications.

This study will primarily investigate the safety of increasing AADC levels in the striatum via AADC gene delivery. The hAADC gene is packaged into a gene transfer vector derived from a common, non-pathogenic virus (AAV2) to which >90% of humans have been exposed. This investigational drug, termed AAV2-hAADC, will be injected directly into the striatum during a neurosurgical procedure that is performed with real-time MRI imaging to monitor delivery.
Subjects will continue to take Parkinson's disease medications, including levodopa.
The safety and potential clinical responses to AAV2-hAADC will be assessed by repeated clinical evaluations of Parkinson's disease, cognitive tests, laboratory blood tests and neuroimaging. Clinical evaluations will be performed over a 3 year follow-up period. A test to specifically assess the clinical response to levodopa will be performed once before AADC gene delivery and approximately 6 months after.”

Find information on the clinical trial here:

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