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Panobinostat in lymphoid and myeloid malignancies

Amit Khot1, Michael Dickinson, H Miles Prince

  • 1Department of Cancer Medicine, Peter MacCallum Cancer Centre, Melbourne, Victoria, Australia. amit.khot@petermac.org

Expert Opinion on Investigational Drugs
|July 6, 2013
PubMed
Abstract

Insights

Panobinostat, a histone deacetylase inhibitor (HDACI), shows manageable side effects and encouraging responses in various hematologic cancers. This oral agent demonstrates efficacy in clinical trials for treating conditions like Hodgkin lymphoma and cutaneous T-cell lymphoma.

Area of Science:

  • Oncology
  • Epigenetics
  • Pharmacology

Background:

  • Histone deacetylase inhibitors (HDACIs) are epigenetic antineoplastic agents that modulate gene expression.
  • Panobinostat is a potent pan-histone inhibitor of HDAC enzymes involved in cancer progression.
  • HDACIs have demonstrated activity in hematological malignancies such as cutaneous T-cell lymphoma (CTCL), Hodgkin lymphoma (HL), and myeloma.

Purpose of the Study:

  • To review the pharmacology of panobinostat.
  • To analyze Phase II trial results for panobinostat in various hematologic cancers.
  • To discuss future directions in drug development, including predictive biomarkers.

Main Methods:

  • Review of basic pharmacology.
  • Analysis of early phase and large Phase II clinical trials.
  • Consideration of future drug development strategies.

Main Results:

  • Oral panobinostat is deliverable with manageable dosing regimens.
  • Myelosuppression (thrombocytopenia) is the primary hematologic side effect, but it is transient and manageable.
  • Encouraging responses observed in HL, CTCL, myelofibrosis, and Waldenstrom's macroglobulinemia (WM).

Conclusions:

  • Panobinostat demonstrates a favorable safety and efficacy profile in Phase II trials for hematologic cancers.
  • Combination therapy with azacitidine shows promise in acute myeloid leukemia and myelodysplastic syndromes.
  • Panobinostat may become a valuable treatment option for a range of hematologic malignancies.

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