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Parkinson's Med News and views on the treatment of Parkinson's Disease Tuesday, 23 September 2008 GDNF Gene Therapy Takes a Major Step Forward Pharmaceutical Business Review published an interesting article this week indicating that Amsterdam Molecular Therapeutics has obtained a license from Amgen to use their GDNF gene for the development of a gene therapy treatment for Parkinson's disease. According to the article "The combination of this gene with Amsterdam Molecular Therapeutics's (AMT) proprietary adeno-associated virus gene therapy platform could potentially allow the development of an effective, long-term treatment for this progressive and crippling disease". This news is especially for patients with Parkinson's for three reasons: Amsterdam Molecular Therapeutics although a small and young company has a very strong management team with proven drug development experience. GDNF appears a particularly interesting growth factor which arguably has potential to protect against progression and perhaps reverse early Parkinson's disease (see previous posts on this blog). AAV may be the best vector for delivering gene therapy to the striatum of patients with Parkinson's disease. It offers significant advantages over competing vectors such as lentivectors. It appears easier to produce. Though AAV vectors produce long-lasting (effectively permanent) change to the cells they transduce they do not insert the genes they carry into the existing DNA of the cell. This may result in less risk of dangerous mutations or abnormal activation of existing genes. Finally because the AAV is smaller than competing vectors it may be easier to distribute the vector evenly throughout the putamen which may be important for optimal safety and efficacy. The Pharmaceutical Business Review article is available here. at 03:49 Rayilyn Brown Director AZNPF Arizona Chapter National Parkinson Foundation [log in to unmask] ---------------------------------------------------------------------- To sign-off Parkinsn send a message to: mailto:[log in to unmask] In the body of the message put: signoff parkinsn