Related Experiment Video

Updated: Jan 24, 2026

A Real-time Potency Assay for Chimeric Antigen Receptor T Cells Targeting Solid and Hematological Cancer Cells
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A Real-time Potency Assay for Chimeric Antigen Receptor T Cells Targeting Solid and Hematological Cancer Cells

Published on: November 12, 2019

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Hedgehog signaling inhibitors in solid and hematological cancers

Jorge E Cortes1, Ralf Gutzmer2, Mark W Kieran3

  • 1Department of Leukemia, MD Anderson Cancer Center, 1515 Holcombe Blvd. #428, Houston, TX 77030, USA.

Cancer Treatment Reviews
|May 25, 2019
PubMed
Abstract

Insights

Hedgehog inhibitors (HHIs) show promise in treating various cancers beyond skin cancer. Further clinical trials will determine their role in combination therapies to overcome drug resistance.

Area of Science:

  • Oncology
  • Molecular Biology
  • Pharmacology

Background:

  • The hedgehog signaling pathway is crucial in development but its aberrant activation drives cancer.
  • Mutations in hedgehog pathway components can lead to uncontrolled cell growth.
  • Hedgehog inhibitors (HHIs) are emerging as targeted therapies for various cancers.

Purpose of the Study:

  • To review known hedgehog inhibitors (HHIs).
  • To explore the potential of HHIs in treating hematological cancers and solid tumors beyond basal cell carcinoma (BCC).

Main Methods:

  • Literature search for preclinical and clinical studies on HHIs in non-cutaneous cancers.
  • Inclusion of relevant clinical trial data from clinicaltrials.gov.
  • Inclusion of pharmacological information on HHIs.

Main Results:

  • HHIs demonstrate efficacy in preclinical models of pancreatic cancer, rhabdomyosarcoma, breast cancer, and acute myeloid leukemia (AML).
  • Clinical studies show HHI activity in medulloblastoma, prostate cancer, pancreatic cancer, and hematological malignancies.
  • Ongoing clinical trials are evaluating HHIs for prostate, pancreatic, breast cancers, multiple myeloma, and AML.

Conclusions:

  • Clinical trial outcomes will identify additional tumor types responsive to HHI monotherapy or combination therapy.
  • Combination strategies may prove effective in delaying or overcoming acquired drug resistance.
  • HHIs represent a promising therapeutic avenue for a range of difficult-to-treat cancers.
Keywords:
Hedgehog inhibitorsHedgehog signalingItraconazoleSonidegibVismodegib

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