General AML

The FDA grants UCART123 Investigational New Drug approval for treatment of AML

This month, the U.S. Food and Drug Administration (FDA) has granted Investigational New Drug (IND) status to UCART123 for the treatment of patients with Acute Myeloid Leukemia (AML).1

UCART123 is an allogenic gene-edited Chimeric Antigen Receptor (CAR) T-cell therapy targeting CD123.1 CD123, the Interleukin 3 α-Chain Receptor (IL3RA), is differentially and significantly overexpressed in the majority of patients with AML. Additionally, CD123 has been identified as a marker for Leukemic Stem Cells (LSCs),2 which are a small population of stem cells that have properties including differentiation, self-renewal, and homeostatic control, and they contribute to the maintenance and propagation of AML. In AML, the LSC reservoir can lead to disease resistance, relapse, and often death in patients.3

Currently, UCART123 is at the preclinical stage of development.4 The IND approval granted by the FDA allows UCART123 to be evaluated in phase I clinical trials in AML patients. Trials are anticipated to start in the first half of this year.1

  1. ASH Clinical News: FDA Grants IND Application for CAR T-Cell Therapy. 2017 Feb 27. [Accessed 2017 Feb 27].
  2. Al-Hussaini al. Targeting CD123 in acute myeloid leukemia using a T-cell–directed dual-affinity retargeting platform. Blood. 2016 Jan 7: 127(1): 122–131. DOI: 1182/blood-2014-05-575704. Epub 2015 Nov 3.
  3. Pollyea D. A. et al. Targeting acute myeloid leukemia stem cells: a review and principles for the development of clinical trials. Haematologica . 2014 Aug; 99(8): 1277–1284. DOI: 3324/haematol.2013.085209.
  4. Cellectis: Universal Chimeric Antigene Receptors. [Accessed 2017 Feb 27].
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