Table 1.

Representative single agent activity of targeted therapies in AML.

Treatment	Complete response %	Partial response %	Blast response %	Comments	Ref.
FLT3 inhibitors
Sorafenib n = 50; n = 39 with FLT3 ITD or D835 point mutation or both	10% (13% FLT3 mutation)		34% (all with FLT ITD)	Phase I, relapsed/refractory AML	[24]
Midostaurin n = 95; n = 35 FLT mutant	0	1.6%	71% FLT3 ITD 42% FLT3 WT	Phase II, relapsed/refractory MDS and AML	[25]
Lestaurtinib n = 14	0%	0%	29%	Phase II, relapsed/refractory AML with FLT3 ITD	[26]
Lestaurtinib n = 5 with FLT 3 mutation n = 22 WT	0	0	60% (FLT3 mutated) 23% (WT)	Phase II, newly diagnosed AML in the elderly	[27]
AC220 n = 76; n = 47 with FLT3 mutant	12% (22% flt3 mutated; 6% WT; 18%unk)	18% (33% flt3 mutated; 13% WT; 18%unk)		Phase I, relapsed refractory AML unselected for FLT3 ITD	[28]
Epigentic modulation
5-azacytidine n = 113	18% (vs. 16% in BSC)			Subanalysis of patients with low blast count in phase III trial	[29]
Decitabine n = 485	17.8% (vs. 7.8% conventional care)			Phase III trial compared to conventional care (TC) AML	[30]
mRNA processing and translation
Ribavirin n = 11	7%	13%	27%	AML FAB M4 and M5 M4 and M5 subtypes, phase II, refractory, relapsed or newly diagnosed, unfit for induction chemotherapy	[31]
PI3K/Akt/mTOR
Deformolus (Rapamycin analogue) n = 22	0%	0%	0%	Phase II	[32]
Protein recycling
Tosedostat n = 73	12%	10%		Phase II, patients aged 60 and over with relapsed, refractory disease	[33]
CD33
Gemtuzumab ozogamycin n = 142	29%		17%	Phase II; relapsed AML; complete response includes patients with incomplete platelet recovery	[34]
Farnesylation and RAS targeting
Farnesyl Transferase Inhibitor R115777 n = 34	6%	24%		Phase II; relapsed/refractory AML	[35]

WT = wild type; unk = unknown mutational status; BSC = best supportive care.