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Archive for the ‘Clinical Trials’ Category

The article can be found at Health and Age’s Website

The First Real Triumph for Gene Therapy – Parkinson’s

Robert W. Griffith, MD

Phase I clinical trials are intended to define the general safety profile of an experimental drug, to enable a dose schedule to be worked out for Phase II efficacy trials. Any indication of effectiveness from a Phase I study is a bonus. According to a report in the medical journal Lancet, the first study to use gene therapy to treat any neurodegenerative disorder – in this case, Parkinson’s disease – showed no toxic effects and 25% to 30% improvement in a specific rating scale after 1 year.

Twelve patients (11 women) with advanced Parkinson’s had the glutamic acid decarboxylase gene delivered directly into the cells of the subthalamic nucleus of the brain. Four patients each received a low, medium or high dose or the preparation. The gene was carried by a harmless adeno-associated virus; it produces gamma-aminobutyric acid (GABA), an inhibitory neurotransmitter that quiets excessive neuron firing. The injection was done on one side, and the major improvements were seen in the opposite side of the body.

This is indeed a ‘breakthrough’. But the authors believe it carries implications for gene therapy beyond Parkinson’s disease, into a whole realm of neurodegenerative conditions, such as Alzheimer’s, Huntingdon’s , and Lou Gehrig’s disease.

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I would like to extend a warm welcome to members of the North Florida Parkinsons Awareness Group and to those who found us while searching for Parkinsons related information and research. The NFPAG is headquartered in Tallahassee, Florida and serves North Florida, South Georgia and Southeastern Alabama.

This website is designed to act as a central point of information for the NFPAG. Members are encouraged to pass along information and share thoughts or ideas in order to make the website a valuable tool from which we can all benefit. The plan is to have an up to date site filled with information including,

  • Meeting minutes
  • Member Info and Feedback
  • Notification of NFPAG events
  • Info on the latest Clinical Trials and Medications
  • News of legislation with an impact on Parkinsons
  • Exercise Ideas
  • Recipes and Nutritional Planning

These are just a few areas that we can touch on. Member participation and knowledge sharing will be an important part of the NFPAG and this website will help us get our ideas out to all.

If you have any ideas or comments please send me email at nfpag1@gmail.com .

Thank you for your participation and help!

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