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Mouse studies suggest that inhibiting PP2A could treat MECP2 duplication syndrome, an X-linked genetic disorder with neuromotor, developmental and other symptoms. In a mouse model of the syndrome, tool compounds that inhibit PP2A -- a negative regulator of MECP2 -- decreased MECP2 levels in brain tissue and motor deficits compared with vehicle. Next steps could include testing additional PP2A inhibitors in MECP2 duplication syndrome models.

Lixte Biotechnology Holdings Inc. has LB-100, a small molecule PP2A inhibitor, in Phase I testing for advanced solid tumors and preclinical testing for leukemia and depression...