Preclinical Testing of Promising Drug Leads (Neuron and Frog Models)

MAODF continues to make important progress in identifying potential treatments for MAO Deficiency. Following an AI-driven screening of approximately 40,000 compounds conducted by Unravel Biosciences, 14 promising drug candidates were selected for further testing. These compounds are currently being evaluated in Unravel’s Xenopus (frog) model of MAO Deficiency.

At the same time, MAODF is collaborating with University of Radbound in the Netherlands to test both Unravel’s lead candidates and seven known neurotransmitter-related compounds in patient-derived nerve cell models. These studies examine whether the compounds can improve the abnormal nerve cell activity associated with MAO Deficiency and restore cellular function toward normal levels.

Early results have been very encouraging. Three of the seven neurotransmitter-related compounds tested so far, L-pCPA, Ketanserin and Tetrabenazine significantly improved disease-related abnormalities in patient-derived neurons, bringing their activity closer to that seen in cells from an unaffected parent.

While additional testing is still underway, these findings represent an exciting step forward and provide hope that effective treatments for MAO deficiency can be identified. We look forward to sharing further updates as results from the remaining compounds become available.