Good! I was hoping someone would set me straight... I am a newbie and at this point am relying on you all to help me keep to the right path. Thanks (and also to Laura)!

From original reference abstract:
"Moreover, treatments targeting this malfunction in preclinical models delayed or reversed disease symptoms."

Anyone able to determine what these treatments are?

I noted the secretiveness too. I know a PWP in a Facebook group whose doctor was in on the study. Here is what she posted:

My doc says the study is promising (and supports studies about the protein alpha synuclein)--that they can see that there is impaired efficiency in the transport of cargo proteins in people with PD. In animal models, targeting this impairment seems to slow/stop progression. The study had 12 participants with PD, and now they are putting together a second study with 45 people here in San Fran. I'm thinking of participating. My doc emailed me the paper on the study

Interesting...looks like treatment was with nocodazole:

Nocodazole is an anti-neoplastic agent which exerts its effect in cells by interfering with the polymerization of microtubules. Microtubules are one type of fibre which constitutes the cytoskeleton, and the dynamic microtubule network has several important roles in the cell, including vesicular transport, forming the mitotic spindle and in cytokinesis.

If you would like a copy. please PM me.

Laura

Hi Laura,
Thank you so much for posting this info - it's so interesting.
By any chance did you FB friend mention where the study is (UCSF?). I live in San Francisco too and would be interested in participating, if there is room left.

Thanks!
Ana

Ana,

Yes! It is UCSF. I can get her doctor's name and forward it with article.

Laura

Thank you so much!

Ana,

I tried to PM you but could not. I need your private e-mail to share the full text article

Hi Laura,
I tried to post a PM for you too and could not. I must be doing something wrong! I'm working on updating my profile and trying to figure out how to use this site.

Progression biomarkers are essential for development of disease modifying drugs. Without them, most drug makers won't bother.

MJFF has been leading to search for biomarkers since 2002 and has funded probably close to $50M by now with at least another $50M still needed.

The study finding posted has encouraging results at an early stage. Very exciting. It looks to distinguish PD from non PD in a small population. We've already committed funds to test it further (can read outs distinguish "stages" of PD?--a step toward seeing if it illuminates progression). It has just been supplemented so it might be a couple of years (depends on patient recruitment among other things -- slow recruitment will slow results). If it holds in the supplement, then it would likely be a candidate to use PPMI samples.

That is the beauty of PPMI...it enables us to evaluate candidates that have already shown promise in the two stages mentioned above and now need to be "verified" in the larger PPMI population (as the subjects are recruited we get more data about the current candidates). Ultimately, reserves of the data and biosamples collected in PPMI will stand ready (banked for future use). So let's say this particular new idea gets positive results in its supplement, then the PPMI infrastructure will enable a fast turnaround in the critical verification stage. Future verifications can happen relatively quickly and at relatively low cost since the PPMI resources will have the needed elements standing ready. I hope this makes sense.

I guess my final point is that this new finding is indeed exciting (and we are already moving it forward) but there are some candidates that are even further along in this process.

Essential progress coming from a very deliberate, very expensive but highly orchestrated strategy that MJFF has been driving for the past decade. Because this is what it takes. It is clear that in the absence of us driving (the work and with leadership/high risk funds) this kind of achievement would not be possible.

Debi

PS: Lots more information on biomarkers on our website in the "priority areas"
here https://www.michaeljfox.org/research...php?biomarkers