eJHaem (Aug 2022)

CB‐103: A novel CSL‐NICD inhibitor for the treatment of NOTCH‐driven T‐cell acute lymphoblastic leukemia: A case report of complete clinical response in a patient with relapsed and refractory T‐ALL

  • Michael Medinger,
  • Till Junker,
  • Dominik Heim,
  • Alexandar Tzankov,
  • Philip M. Jermann,
  • Maria Bobadilla,
  • Michele Vigolo,
  • Rajwinder Lehal,
  • Florian D. Vogl,
  • Michael Bauer,
  • Jakob Passweg

DOI
https://doi.org/10.1002/jha2.510
Journal volume & issue
Vol. 3, no. 3
pp. 1009 – 1012

Abstract

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Abstract Relapsed T cell acute lymphoblastic leukaemia (T‐ALL) has a very poor prognosis. A 24‐year‐old patient with relapsed high‐risk T‐ALL (PTEN gene deletion; NOTCH1 mutation), was treated with the NOTCH inhibitor CB‐103. Within 1 week of starting CB‐103, the bone marrow was free of T‐ALL blast infiltration (MRD+) and successfully underwent allogeneic hematopoietic stem cell transplantation (allo‐HSCT). Sequential samples of ctDNA to monitor the disease after allo‐HSCT showed a decrease of circulating Notch1 and PTEN alterations. This is the first T‐ALL patient treated with CB‐103. The observed clinical response encourages further exploration of CB‐103 in ALL.

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