FRAXA, Marvel Biosciences team up to test therapy in fragile X animal models
MB204 blocks adenosine A2A receptors, which are potential treatment targets
The nonprofit FRAXA Research Foundation has teamed up with Marvel Biosciences to test MB204, a drug that blocks adenosine A2A receptors, in a preclinical model of fragile X syndrome.
As part of the collaboration, FRAXA will run the preclinical studies at its drug validation facility in Chile, a lab dedicated to testing promising drug candidates for fragile X.
“We are very interested in testing MB204 in our independent laboratories,” Mike Tranfaglia, MD, the medical director and co-founder of FRAXA, said in a press release from Marvel.
Fragile X syndrome is a genetic condition caused by mutations in the FMR1 gene that leads to cognitive impairments, learning difficulties, and behavioral challenges. It’s one of the most common causes of inherited intellectual disability, affecting about 1 in 4,000 males and 1 in 8,000 females.
There are no approved treatments for fragile X syndrome, making it a challenging condition to manage. Adenosine receptors, specifically A2A receptors, have been identified as potential targets for new treatments.
What is MB204?
Marvel Biosciences’ MB204 is a modified version of istradefylline, which is approved under the name Nourianz for treating Parkinson’s disease in the U.S. Its two molecules work by blocking A2A receptors in the brain, which should help with conditions other than Parkinson’s, such as depression, Alzheimer’s disease, autism spectrum disorders, and fragile X. Istradefylline has shown promise in animal models of autism carrying mutations in the FMR1 gene, the gene that causes fragile X.
A2A receptors are found on the surface of brain cells. When activated with adenosine, a natural brain chemical, they slow movements and make people feel tired. It’s been consistently shown that adenosine blockers such as caffeine can increase alertness, boost cognitive function, and reduce fatigue.
Previous FRAXA-funded work from researchers in Italy showed A2A receptors can impact many different signaling pathways involved in fragile X and inhibiting them can normalize the abnormal signals in animal models of the disease.
“MB204 has completed its preclinical toxicology studies and [manufacturing processes] and is … ready for clinical testing,” Tranfaglia said.
“We are very grateful to be able to collaborate with FRAXA to test MB204 in their mouse models of Fragile X,” Mark Williams, Marvel’s chief scientific officer, said.
About the Author
