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ShopGuy

Starting Phase 1 Clinical Trial Next Week

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ShopGuy    88

Hello all,

It's been a while since I've posted. This coming Monday, I start the inpatient segment of a Phase 1 double blind clinical trial, testing a new immunotherapy treatment for PD. Details of the trial are here:

https://clinicaltrials.gov/ct2/show/NCT02459886

The trial is fairly invasive, including IV administration of the study drug (or placebo), five days of inpatient stay with 24 hour monitoring, multiple lumbar punctures (spinal taps), and MRIs. To qualify, I've had to drop all meds except Azilect (people on stable doses of Sinemet also qualify, I believe), and undergo a series of health screens, including DaTScan. Reasonably good health (apart from PD) and a positive DaTScan are required.

Since most Phase 1 trials don't pan out, and I have a 1/3 chance of getting the placebo, I'm not expecting personal benefit. But the science is very cool--the hope is the compound (engineered antibodies) will clear misfolded alpha-synuclien proteins from the brain and dramatically slow PD progression. Immunotherapy treatments from other companies have already made it past Phase 1, and are starting Phase 2, so even if this particular trial doesn't produce good results, it seems the general approach has a lot of promise.

My MDS is one of the study doctors, so I feel like I'm in good hands, and compensation for my time and travel is quite reasonable. Certainly there are risks involved, but I believe they are relatively small and manageable.

I'll post more as the experience continues.

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Linda Garren    748

David, thank you on behalf of all Parkies for volunteering for this trial.  It will be exciting to hear of the results.  If positive, it would be a major, major step forward to help in several diseases that have the mis-folded protein (prion) issue.  Major, major, major advancement.

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ShopGuy    88

I think a lot of credit has to go to the 40 people without PD who went thru the trial first, to establish safety before the PWP cohort. They may have had many different reasons for doing so, but I appreciate that they were willing to take that kind of risk.

And I agree completely--if targeting mis-folded proteins works, it will be huge for quite a number of diseases much worse than PD. Even if this turns out to be a dead end, at least we'll have learned what not to spend time and money researching. That's important, too.

Not looking forward to the spinal taps, however...

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Lonnise    60

Best of luck David and thanks for having the courage and curiosity to do this.  Spinal taps aren't "fun" but I'm sure they've progress in the last 35 years (that's the last time I had one).  All the best.

Lonnise

 

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stump    463

Yes, thank you for volunteering for this study!  I have participated in a couple studies, though nothing like as invasive as you are talking about.  That is something that, at my current phase of life, would not be possible to participate in.  The risks to my kids if something bad happened are too high, plus with my career still very active the time required to be away from work would be a big problem.  Though, as of a couple minutes ago, I'm officially old enough for that trial ...

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stump    463
1 hour ago, miracleseeker said:

Hey Stump.  Happy Birthday.  One year older and 50 years wiser. :D

Hey now!  I'm only 40.  Let's not get premature. :)

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ShopGuy    88

Happy birthday, Stump!

I'm home now from the inpatient portion of the trial, none the worse for wear.

I'll post later today or tomorrow with some details of the experience--it was thoroughly worthwhile.

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miracleseeker    675
21 hours ago, stump said:

Hey now!  I'm only 40.  Let's not get premature. :)

What I mean is even though you are just a year older you have knowledge way beyond your years. It's a complement. 

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