Anti-CD33 Chimeric Antigen Receptors for Treatment of Human Acute Myeloid Leukemia

Acute Myeloid Leukemia (AML) is an aggressive malignancy that is treated using intensive cytotoxic chemotherapeutic regimens. There are limited alternative therapeutic options once the disease becomes refractory to chemotherapeutic treatment. CD33 is expressed on the surface of the vast majority of AML blasts and cells in chronic myeloid leukemia-blast crisis (CML-BC) – and is a well-validated immunotherapeutic target.  CD33 is also aberrantly expressed on a subset of T cell acute lymphoblastic leukemias (ALLs). Normal tissue expression is restricted to normal myeloid cells. Treatment of pre-B cell ALL and lymphoma using chimeric antigen receptors (CARs) targeting CD19 and CD22 in various clinical trials have demonstrated impressive clinical results in children and young adults with up to 70-90% complete remission rates. Researchers at the National Cancer Institute (NCI) have developed CD33 targeted CARs based on humanized anti-CD33 antibodies with potent activity against AML.

Potential Commercial Applications: Competitive Advantages:
  • Acute Lymphoblastic Leukemia (ALL)
  • Acute Myeloid Leukemia (AML)
  • Chronic Myelogenous Leukemia (CML)
  • General hematological cancers
  • Adoptive immunotherapy 
  • First report of a Chimeric Antigen Receptor with the current anti-CD33 antigen 
  • Immunotherapies targeting surface molecules expressed by leukemic cells are a promising tumor-specific approach
  • Adoptive T-cell immunotherapy may be particularly potent due to the longevity and strong cytocidal activity of transferred T cells, and the accessibility of ALL/AML/CML as blood cancers    

Development Stage:
Pre-clinical (in vivo)


Terry Fry (NCI)  ➽ more inventions...

Intellectual Property:
Application No. 62/643,015

Yang L, et al. Analysis of CAR 41-BB versus CD28 co-stimulatory domains exposes emergence of extramedullary disease in acute myeloid leukemia.  Abstract #1534

Collaboration Opportunity:

Licensing and research collaboration

Licensing Contact:
John Hewes, Ph.D.
Phone: 240-276-5515

OTT Reference No: E-097-2018
Updated: Aug 2, 2018