Targeting of the Hedgehog pathway in myeloid malignancies: still a worthy chase?

Abstract:

:Deregulated Hedgehog (Hh) signalling activity may be associated with a broad range of cancer types and hence has become an attractive target for therapeutic intervention. Although initial haematological interest focused on the therapeutic targeting of this pathway in chronic myeloid leukaemia), small molecule inhibitors targeting the Hh pathway are now being tested in a range of other myeloid disorders, including myelofibrosis, myelodysplasia and acute myeloid leukaemia. In this review we will evaluate the rationale for targeting of the Hh pathway in myeloid diseases and discuss the novel agents that have entered the clinical arena. We will discuss pre-clinical models, emerging clinical trial data, and suggest how these targeted therapies may address current unmet medical needs. Finally, we will explore potential limitations of these therapies due to the emergence of secondary resistance mechanisms and speculate on future developments within this arena.

journal_name

Br J Haematol

authors

Khan AA,Harrison CN,McLornan DP

doi

10.1111/bjh.13426

subject

Has Abstract

pub_date

2015-08-01 00:00:00

pages

323-35

issue

3

eissn

0007-1048

issn

1365-2141

journal_volume

170

pub_type

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