MRI Guided Gene Therapy Trial About To Begin at Massachusetts General

[jimcaster]

To qualify as a candidate for this trial, a PwP must have been diagnosed at least three years ago and no longer be receptive to medication.

Massachusetts General Hospital researchers are joining a phase II clinical trial to test a promising gene therapy for Parkinson's disease. The team is using a state-of-the-art delivery technique that allows them to view an infusion as it enters the brain and adjust as needed in real time. The approach represents an evolution in neurosurgical gene therapy and will begin a new era of direct drug delivery in Parkinson's disease and other neurologic disorders.

advances.massgeneral.org/ne...

[park_bear]

Very interesting. Does require brain surgery though.

[pdpatient in reply to park_bear]

Quite correct, Parkbear. I don't think I am ready for the surgical options yet and may never be.

My diabetes just went back out of control and that's a no no and no go. Thanks to the quarantine, I have been mindlessly eating and sleeping and not getting outside even for a walk and that's taken a toll.

If I can not guarantee a consistent A1C, any brain surgery options are out. Just wanted to let fellow Parkinson's sufferers here know in they're in the same boat.

[park_bear in reply to pdpatient]

Sorry to hear that. Getting outside for walks is important. Is there any way you can do that?

[pdpatient in reply to park_bear]

Yes indeed if I can OBEY my wife and summon the motivation. Frankly, I think that I need to stop using the cover of Parkinson's apathy and the fear of the pandemic as an excuse!! There's no one else on the road all day due to the Bay area crisis and impending lockdown. I really don't have an excuse.

Thanks for the encouragement. I am immediately going for a walk but and dedicating my walk to you.

And make the wife happy😊

[lenamm]

I would worry that it might end up like the GDNF trials were the drug helped many but then the folks could not get the drug post trial. Plus as is mentioned brain surgery which might disqualify you from future FUS.

[pdpatient in reply to lenamm]

Exactly. We're not guinea pigs 😅

[MarionP]

Did I miss something, or was there no description of the underlying genetic treatment? Is this a study of a delivery system or a treatment? Is this treatment known to be efficacious and effective, so that the true variable of study has to do with the delivery method and only the delivery method?

[jrg54321 in reply to MarionP]

in a phase II clinical trial, which will administer AADC using the novel delivery method.

Dr. Richardson has helped pioneer one such therapeutic infusion: aromatic L-amino acid decarboxylase (AADC), an enzyme reduced in Parkinson's disease. "If you give the brain back AADC, it can make more dopamine in the place where it's needed," he says.

[MarionP]

This one looks like it might be rather good, perhaps even very very good, been reading about it some amount and it seems to hold some real promise. Worth digging into and following.

Makes me also wonder whether the process might be helped along with some sort of concurrent application of something to stimulate or provide some targeted (if targeted is possible or even the right sort of idea) neural growth factor, NGF. Wonder if NGF might be something right in SharonCrayn's wheelhouse, it's mechanism is by controlling on-growth and off-growth tyrosine kinase switches...one turns on NGF to grow cells, one turns off NGF to kill cells...looked at in alzheimers realm and aging but wonder if something in there could apply to cancer too.

I don't know if eating those certain mushrooms that seem to have some sort of ngf in them would help or cause problems, or just get broken apart by stomach acid.