Mesothelin-directed CAR T Cells for the Treatment of Acute Myeloid Leukemia

CAR T cells targeting mesothelin to treat Leukemia

  • Stage: Preclinical in vivo work completed
  • Type: Cell Therapy
  • Categories: Biomarker, Target, Vectors

Technology Overview

Despite maximally intensive therapy, approximately 40% of patients with acute myeloid leukemia (AML) will relapse. An effective targeted therapy is still needed to save the lives of AML patients. Through computational interrogation of the transcriptome data, Dr. Soheil Meshinchi’s group discovered and validated mesothelin (MSLN) to be highly expressed on the cell surface of AML blasts and leukemic stem cells in a subset of AML patients. Specifically, MSLN is expressed in 36% of pediatric and adult AML patients and is virtually absent in normal marrow. Dr. Meshinchi’s group has developed MSLN-directed CAR T cells and preclinical in vivo experiments further establish that MSLN CAR T cells are highly effective in eliminating MSLN-positive AML cells in cell line and patient-derived xenograft models. Importantly, MSLN CAR T cells can target and eradicate MSNL-positive cells without impacting the viability of normal hematopoietic stem cells. These finding demonstrate that MSLN-directed CAR (T or NK cell) immunotherapy could be potential therapeutic candidates for MSLN-positive AML.


  • Treatment of MSLN-positive acute myeloid leukemia


  • Preclinical proof of concept of MSLN CAR T cells in vivo is target specific

Patent Information


Investigator Overview

  • Soheil Meshinchi, M.D., Ph.D. - Professor, Clinical Research Division
Tech ID: 21-103
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