An Exploratory Single Blind Study of Ergoloid Mesylates, 5-Hydroxytryptophan, and the Combination in Adult Males with Fragile X

Elizabeth Berry-Kravis MD, PhD
Principal Investigator

Rush University Medical Center
Chicago, IL

2021 Grant Funding: $106,600

Total Funding to date: $766,973

Summary

Can a combination of drugs make a meaningful difference for people with Fragile X? A new clinical trial is going to find out. 15-20 adult men with Fragile X will be included in this upcoming trial, which will test the effects of an available drug and a nutritional supplement taken together.

The Science

By Holly Roos

The Future is Now

We are excited to announce that another FRAXA-funded research project has received IRB approval and is ready to start clinical trials. Thanks to FRAXA’s biotech partner Purposeful’s advanced AI (artificial intelligence), and FRAXA-DVI, this trial was launched in record time. 

AI is no longer a thing of the future, it is now and making a huge impact in the world of research. AI provides researchers with the opportunity to create a combination of available drugs to make a meaningful difference to people with Fragile X.

Purposeful and FRAXA-DVI

Purposeful, a drug repurposing company based in Athens, Greece, used their advanced artificial intelligence and machine learning platform to generate predictions of the best treatments for Fragile X. FRAXA then arranged to test combinations from this list of candidates at FRAXA-DVI, based on the assumption that a combination of drugs would be needed to make a meaningful difference. At FRAXA-DVI, the available drug ergoloid mesylates and the nutritional supplement 5-hydroxytryptophan (5-HTP), when taken together, completely reversed all symptoms in Fragile X mice. This combination is now ready for human trials.

Georgios Drakakis

I still remember the feeling after our first call with Mike Tranfaglia: The work that FRAXA does and has done is beyond belief – the efficiency, the speed, the ability to fund so many research projects, the sound scientific advice, wow! Our first preclinical work was funded by FRAXA at the FRAXA Drug Validation Initiative, and now just over a year later, this work is going to trials with Elizabeth Berry-Kravis and a FRAXA grant!

Everyone at Purposeful is super excited to start this small phase 2 trial – we believe that the combination we have suggested could have an effect on both behavioral and cognitive phenotypes. We feel extremely fortunate to have worked with FRAXA. Without them, this work could not have been carried out – it is as simple as that.

Dr. Georgios Drakakis
CEO and Co-Founder
Purposeful

Global Leader in Fragile X Research

FRAXA-funded researchers around the world are leading the way towards effective treatments and ultimately a cure.

Explore Current Research Grants
Help Fund the Cure

Global Leader in Fragile X Research

FRAXA-funded researchers around the world are leading the way towards effective treatments and ultimately a cure.

Explore Current Research Grants
Help Fund the Cure