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Hedgehog Pathway Inhibitors: A New Therapeutic Class for the Treatment of Acute Myeloid Leukemia

  • Catriona Jamieson(corresponding author)
    ,
  • Giovanni Martinelli
    ,
  • Cristina Papayannidis
    ,
  • Jorge E. Cortes
*Corresponding author for this work
  • University of California at San Diego
    ,
  • Sanford Stem Cell Clinical Center
    ,
  • IRCCS Istituto scientifico romagnolo per lo studio e la cura dei tumori - Meldola (FC)
    ,
  • Azienda Ospedaliero-Universitaria di Bologna Policlinico S. Orsola – Malpighi
    ,
  • Medicine
Scholary Output:
Contribution to journal
Review article
 Peer-review

Open access

Abstract

Targeting Hedgehog (Hh) pathway components, such as Smoothened (SMO), is a developing strategy for the treatment of acute myeloid leukemia (AML) and for overcoming relapsed/refractory forms of this disease. Several SMO inhibitors are in clinical development for the treatment of various tumor types and the results from some clinical trials in AML have been reported. This review will discuss the role of Hh signaling in AML pathogenesis, describe the preclinical and clinical development of Hh pathway inhibitors for the treatment of AML, and examine the current evidence on Hh pathway inhibitor resistance and the implications for treatment selection in AML.

Publication Information

Output type

Scholary Output:
Contribution to journal
Review article
 Peer-review

Original language

English (US)

Pages from-to (Number of pages)

Pages 134-145 (12 pages)

Journal (Volume, Issue Number)

Blood cancer discovery (Volume 1, Issue 2)

Publication milestones

  • Published - 09/01/2020

Publication status

Published - 09/01/2020

ISSN

2643-3230

Publication IDs

  • Scopus: 85101317840
  • PubMed: 34661144

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Citation count
69
Captures
68
Mentions
3

Funding Details

Pfizer provided a formal review of the publication, including for medical accuracy, but the authors had final authority. The experts were not compensated for manuscript preparation. Medical writing support was provided by Anne Marie McGonigal, PhD, of Engage Scientific Solutions, and funded by Pfizer.