Clinical Research Directory

Functionally Optimized CD33 CAR-T Cell Therapy Targeting Recurrent/Refractory Acute Myeloid Leukemia

Relapsed/refractory acute myeloid leukemia (AML) currently lacks effective CAR-T therapy products due to the absence of specific target antigens. Most AML antigens are frequently expressed in normal hematopoietic stem/progenitor cells (HSPCs) or healthy organ tissues, thereby increasing the risks of target toxicity and non-neoplastic toxicity. CD33 is expressed in leukemia cells from over 80% of AML patients. Compared to other targets such as CLL-1 and CD123, CD33 typically exhibits higher expression levels across various AML subtypes, reducing the risk of treatment failure and relapse caused by tumor antigen escape, making it an ideal therapeutic target for AML. However, conventional CD33 CAR-T therapies have demonstrated suboptimal efficacy in clinical trials and face challenges such as toxicity and insufficient expansion. To further investigate the safety and efficacy of CAR-T therapy for AML, our center has initiated a clinical study on functionally optimized CD33 CAR-T (FO33 CAR-T) cell therapy for refractory/refractory AML.