Preclinical pharmacokinetic and pharmacodynamic data defined the optimal blood drug levels for therapeutic efficacy in humans (12 mg/m2 every other week) and early clinical results demonstrated very promising activity in ovarian cancer patients with FRα positive tumours (46% of the patients showed a GCIG CA125 response and 70% displayed a Recist response). The gene discussed is FOLR1; the disease is neoplasm.