FRAXA Research Foundation is working together with Marvel Biosciences Corporation in an exciting new preclinical study examining the effects of Marvel’s MB204 drug. FRAXA’s collaboration with Marvel Biosciences exemplifies FRAXA's strategy of feeding new treatment ideas into the development pipeline. By funding high-quality academic studies with a therapeutic focus and partnering with innovative companies like Marvel, FRAXA accelerates the development of promising new treatments for Fragile X syndrome.
MB204: Exploring New Possibilities for Fragile X Syndrome
This new study is testing Marvel’s lead compound “MB204” in a mouse model for Fragile X syndrome at FRAXA’s drug validation facility in Chile (FRAXA-DVI). MB204 is an enhanced version of istradefylline, an approved treatment for Parkinson’s disease which has also been studied in mouse models of autism. Istradefylline and MB204 increase levels of dopamine in the brain by blocking adenosine A2A receptors. Marvel is exploring the potential of MB204 beyond Parkinson’s, targeting conditions like depression, Alzheimer’s disease, autism spectrum disorders, and now Fragile X syndrome.
Building on a Foundation of Adenosine Receptor Research
Prior FRAXA-funded research conducted by Alberto Martire and Antonella Borreca in Italy set the stage for these drug therapies based on adenosine receptors, which are critical in the regulation of neurotransmitters. Their study, Coffee, Tea, and Chocolate: Adenosine Receptors in Fragile X, investigated the potential of adenosine receptor blockers like caffeine to treat Fragile X. They found that blocking A2A receptors could normalize aberrant signaling pathways involved in Fragile X. Their results highlighted new potential Fragile X therapy links to Parkinson’s and laid the groundwork for testing new compounds like MB204 in Fragile X models.
FRAXA is dedicated to continuing to bring increased education and awareness surrounding Fragile X and therapy-based treatments, and these funded research projects are an exciting step towards the future.
