GM1 is definitely on our radar but there are some challenges with moving GM1 forward to a larger community that explain why it is not being pursued more by pharma at this time. In order to administer GM1, it needs to be purified from brains. The brains used thus far are bovine – cow. So, while the early information *may* be providing some proof of concept, we have a real practical challenge to use as a therapy.

The concern is that although there may be neuroprotection in preclinical models, and possible symptomatic benefit in PD patients, are you ultimately subjecting the study population to potentially developing another disease in the future. Given that bovine spongiform encephalopathy (ie. mad cow disease, a prion disease) can be transmitted between species, there would always be the possibility that along with the GM1 one purifies, some prion protein comes along with it. Alternatives for this source have been proposed, including sheep – but sheep can develop scrapie, another prion disorder so again, there is cause for concern.

Given the potential for GM1, we are currently supporting 2 efforts around this:

Jay Scheider is being supported our on our Rapid Response program to examine an enzyme upstream in the pathway that ultimatately promotes the generation of GM1 to see if altering this, provides neuroprotection in a preclinical MPTP model (see more information on our website: https://www.michaeljfox.org/foundati...?grant_id=1010).

A small biotech is about to be supported through our Rapid Response program to evaluate a human source of GM1 to provide neuroprotection in a preclinical MPTP model. It will be compared to the ‘gold standard’ of bovine-derived GM1. If efficacious, one could potentially move this forward rapidly (abstract not online yet because contracting is not yet complete).

Debi

PS. I know it is frustratingly hard to get all scientific questions answered--we do our best to put all our work on our website to mitigate that challenge. But, our program team could spend all their time explaining details around every target we are investigating as well as defending every decision they make regarding which grants to fund and which not to. We review over 800 proposals a year and our portfolio has more than 350 active grants at any time. This requires an extraordinary amount of work and dedication on the part of the team...please realize we are aggressively pursuing many avenues even if we don't pause to provide all the details in emails or on our site.