Table 1.

CAR T cell clinical trials and case reports on AML (ALT: alanine aminotransferase; AST: aspartate aminotransferase; CLL-1: C-type lectin-like molecule-1; CR: complete response; CRS: cytokine release syndrome; Cy: cyclophosphamide; Flu: fludarabine; ICANS: immune effector cell-associated neurotoxicity syndrome; MRD: minimal residual disease; PR: partial response; SD: stable disease).

Study	Target	CAR T Cell/Lymphodepletion	Number of Patients	Response	Safety	Reference
Phase I/II	CLL-1	0.35−1 × 106 kg/anti-CLL-1-CD8-41BB/Cy+Flu	8	4/8 morphological leukemia-free, MRD (−) 1 morphological leukemia-free, MRD+, 1 CR with incomplete hematologic recovery MRD(+), 1 PR, 1 SD	CRS: 5 grade 1, 3 grade 2	[7]
Phase I	CD123	Dose escalation 50 × 106−200 × 106/anti-CD123-IgG4-CD28/Cy+Flu	6	4/6 CR 2 reduced blasts	CRS: 4 grade 1, 1 grade 2; 1 adenoviral pneumonia requiring intubation; and 1 grade 3 rash due to drug hypersensitivity	[8]
Phase I	CD33	CD33-4-1BB/0.3 × 106/kg	3	0/3 response	2 CRS; 1 ICANS. A grade 3 tumor lysis syndrome-acute kidney injury, grade 2 mucositis, grade 1 tachycardia for 1 patient; and a second patient experienced grade 2 intermittent orthostatic hypotension, grade 2 increased bilirubin and grade 3 increased ALT and AST	[9]
Case report	CD33	CD33-4-1BB/1.12 × 109	1	Disease progression at week 9	Grade 4 chills and a high fever, pancytopenia	[10]
Phase I	CD38	NA	6	66.7% of patients (4/6) CR (including 1 with CR and 3 with CR with incomplete count recovery (CRi)) and full donor chimerism)	Five patients presented mild CRS (Grade I–II), and only one experienced grade III hepatotoxicity with elevated serum transaminase and bilirubin levels	[11]
Phase I	LeY	Anti-LeY-CD28/Flu-Cy	4	In the patient with active leukemia, a temporary reduction in peripheral blood blast cells was observed. One other patient achieved a cytogenetic remission, while the other two patients had SD	One patient (patient 2) had a transient grade 2 neutropenia	[12]