Gene Therapy Shows Early Signs of Parkinson's Improvement

[Farooqji]

An investigative adenovirus-delivered gene therapy yielded encouraging improvement in symptoms of Parkinson's disease for patients whose condition was refractory to standard treatment. The therapy was well tolerated, and the effects were maintained through 36 months, preliminary results indicate

medscape.com/viewarticle/90...

[reedboat2]

Very interesting study but how do they get the genetically engineered viral infusion into the brain? Drill a hole in the skull and directly inject? I guess this is the brave new world we're looking at...

[Farooqji in reply to reedboat2]

Simon of SOPD has explained this as follows

""Voyager Therapeutics also shared the early findings from a Phase I study evaluating a different route of administration of their treatment VY-AADC. Rather than injecting the treatment from the top of the skull (or transfrontal), this new study was injecting from the posterior (or back) of the head.

The purpose of this change was an attempt to improve the spread of infection of the virus in the region of the brain being injected (the putamen). The company wanted to increase the area being infected with VY-AADC, which will hopefully increase the area of the putamen that will now produce the AADC protein."

[lempa_nik in reply to Farooqji]

Thanks Iqbaliqbal. For those who want to read more, here is is the link to what I believe is Simon's latest update (July 21, 2018) on the Voyager trial:

scienceofparkinsons.com/201...

An earlier report (March 9, 2018) reviewing the Phase 1b trial is given here:

scienceofparkinsons.com/201...

[Farooqji]

Simon comments on the subject triall

"The VY-AADC product will only allow the brain to produce more dopamine and alleviate the motor features of Parkinson’s. While this treatment may allow individuals to reduce the number of tablets they need to take each day, it will not slow the progression of Parkinson’s. Having said that, VY-AADC may provide a significant improvement in quality of life to those with more advanced Parkinson’s, and in the absence of a curative treatment, an addition treatment options is certainly better than nothing."