Parkinson's AADC Gene Therapy Shows Promise as Maintenance Treatment, Voyager Says

I was at Voyager's presentation yesterday at the MJFF Therapeutics Research conference. It was quite impressive. On the other hand, the stock got wacked today when Sanofi dropped their option to their US marketing rights (which were maintained by Voyager). Seems possible, yet still very suprising that a major big pharma would walk away from potentially ground breaking drug and technology. I guess we'll know more as future results come in........


There were a number of other very interesting studies, including more evidence of the involvement with the gut; evidence of immune response and T cell involvement (although not autoimmune); and a great debate on whether or not PD is a prion-like disease (the researcher showed why he believes it is NOT a prion-like disease).

How does this proposed therapy compare with simply reducing the amount of carbidopa taken by PwP?

AADC is found in both the body and in the brain, so I suspect, but don't know, that it crosses the BBB.

Sinemet contains both levodopa and carbidopa, usually in a ratio by weight of 4:1, but a 10:1 variant also exists. Carbidopa is an AADC inhibitor. It does not cross the BBB.

Reducing the ADDC inhibitor is likely, therefore, to increase ADDC levels in the brain. This is the intent of the new therapy also.

It seems to me that a better approach is to find, for each PwP, what ratio of carbidopa best suits them individually.

John