FOLR1-directed CAR T Cell Therapy

CAR T cells to treat osteosarcoma, ovarian cancer, and high-risk pediatric leukemia

  • Stage: IND enabling work completed
  • Type: Cell Therapy
  • Categories: Immuno Oncology, Target, Vectors

Technology Overview

The folate receptors FOLR1 and FOLR2 are overexpressed in multiple cancers. The overexpression of FOLR1 is often associated with increased cancer progression and poor patient prognosis in osteosarcoma, ovarian cancer, pediatric AML, etc. A cryptic CBF/GLIS fusion found only in infants drives a rare but highly aggressive type of acute myeloid leukemia (AML). Dr. Soheil Meshinchi’s group at Fred Hutch Cancer Center found that FOLR1, which encodes for folate receptor alpha, is highly and uniquely expressed in CBF/GLIS AML but is entirely absent in normal hematopoietic cells. Additionally, FOLR1 surface expression is shown to be causally linked to CBF/GLIS-induced malignant transformation, thus making it an attractive antigen for targeted therapies against CBF/GLIS AML cells. Dr. Meshinchi’s group has now developed FOLR1-directed CAR T cells for treating CBF/GLIS AML. In vivo experiments show that FOLR1-directed CAR T effectively eradicates CBF/GLIS AML cells without compromising normal HSPCs, providing a promising approach for the treatment of high-risk CBF/GLIS AML.


  • Treatment of osteosarcoma, ovarian cancer, etc.
  • Treatment of CBF/GLIS acute myeloid leukemia


  • Preclinical proof of concept of FOLR1 CAR T cells in vivo is target specific
  • Both CD8 and CD4 FOLR1 CAR T cells produced higher levels of IL-2, IFN-γ, and TNF-α than unmodified T cells

Patent Information

Patent pending 63/274,914

Investigator Overview

  • Soheil Meshinchi, M.D., Ph.D. - Professor, Clinical Research Division
Tech ID: 22-084
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