Dr. Jeffrey Cummings of the Cleveland Clinic (LV) has reported early positive results for the oral agent rasagiline, in mild-moderate Alzheimer Disease (AD). The Phase II “proof of concept” trial was started four years ago, but here’s one news report: https://www.nj.com/healthfit/2019/10/heres-a-much-needed-dose-of-good-news-in-the-quest-to-cure-alzheimers.html  

A second news item, from the Alzheimer Drug Discovery Foundation, a non-profit that helped fund the work, confirms that there were positive changes in measures of both cognition and function, along with neuroimaging improvements.  Combined, those results might meet the basic efficacy requirements that the FDA has set out for the approval of AD drugs.    

While one would love to see the peer-reviewed data, including the methods and measures used, this is amazing news, if for no other reason that this follows years of negative studies of powerful amyloid and tau targeted immunotherapies.  Still, it’s a preliminary report of a small trial, listed as randomized, double blind and placebo controlled.

This is what I tweeted: “Dr. Cummings has been forward-thinking and pragmatic for years, writing about repurposing with colleagues (search PubMed). I’ve always thought he should be named ADRD Czar!! Now for some organization to take up leadership in sponsoring comprehensive repurposing trials…”  

Although the NIA Alzheimer Clinical Trials Consortium (ACTC), the Alzheimer Disease Research Centers (ADRC) and the Alzheimer Disease Cooperative Study Group (ADCS) already exist, I wonder if any of the leaders or institutions can gear up their existing infrastructures to rapidly mount a confirming, combination or even contrasting trial. Maybe they’re already working on it, one would hope.  

Methylphenidate (Ritalin), seemed to show some cognitive improvements in a VA study of dementia apathy, so it might be an interesting repurposing combination candidate.  

Rasagiline was FDA approved (in 2006) to help with symptoms like fatigue, in Parkinson Disease.  It does have side effects.  It’s in the MAO Inhibitor category (remember those?), used for anxiety and depression back in the day, which suggests there are other FDA-approved MAOI that could be tested.  Mechanism of action in AD?  Well, if it works, can’t we get back to that later?  

Maybe the goal of having a disease-modifying therapy by 2025, established by Obama’s National Alzheimer Project Act, can be achieved!  To perform the trials necessary for approval, though, leadership will still be needed.  I posted about my fantasy clinical leader, and about repurposing clinical trials last year:

 If rasagiline has a positive effect, it will be interesting to see if it was predicted by all the work done by the AMP-AD and folks doing in silico (computer) modeling.  At this point though, let’s hope for confirmation studies, and soon!