Patients with chemotherapy-resistant acute myeloid leukaemia are rarely cur
ed by non-allogeneic transplant therapies. Multiple new investigational age
nts have become available for treatment of these patients and there are few
tools to permit rational drug and clinical trial selection. In this review
, we describe the chemical and biological properties of some of these agent
s and some of their initial clinical activity to date. The selected agents
react with either cell surface molecules or signal pathway intermediates an
d include antibody and antibody conjugates to CD33 and CD45, a fusion prote
in directed to the granulocyte-macrophage colony-stimulating factor recepto
r, an anti-sense oligonucleotide to Bc12, a farnesyl transferase inhibitor,
and a protein kinase C agonist/inhibitor. The challenge for the next decad
e will be how to select patients for particular molecularly targeted therap
eutics and how to combine these agents.