Acute promyelocytic leukemia future or investigational therapies

Jump to: navigation, search

Acute promyelocytic leukemia Microchapters

Home

Patient Information

Overview

Historical Perspective

Classification

Pathophysiology

Causes

Differentiating Acute promyelocytic leukemia from other Diseases

Epidemiology and Demographics

Risk Factors

Screening

Natural History, Complications and Prognosis

Diagnosis

History and Symptoms

Physical Examination

Laboratory Findings

Other Imaging Studies

Other Diagnostic Studies

Treatment

Medical Therapy

Interventions

Surgery

Primary PreventionSurgery

Secondary PreventionSurgery

Cost-Effectiveness of Therapy

Future or Investigational Therapies

Case Studies

Case #1

Acute promyelocytic leukemia future or investigational therapies On the Web

Most recent articles

Most cited articles

Review articles

CME Programs

Powerpoint slides

Images

American Roentgen Ray Society Images of Acute promyelocytic leukemia future or investigational therapies

All Images
X-rays
Echo & Ultrasound
CT Images
MRI

Ongoing Trials at Clinical Trials.gov

US National Guidelines Clearinghouse

NICE Guidance

FDA on Acute promyelocytic leukemia future or investigational therapies

CDC on Acute promyelocytic leukemia future or investigational therapies

Acute promyelocytic leukemia future or investigational therapies in the news

Blogs on Acute promyelocytic leukemia future or investigational therapies

Directions to Hospitals Treating Acute promyelocytic leukemia

Risk calculators and risk factors for Acute promyelocytic leukemia future or investigational therapies

Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief: Shyam Patel [2]; Grammar Reviewer: Natalie Harpenau, B.S.[3]

Overview

Investigational therapies for acute promyelocytic leukemia include bromodomain inhibitors and RNA-based silencing approaches (gene therapy).

Future or investigational therapies

Bromodomain inhibitors:

  • Studies have shown that the bromodomain inhibitor JQ1 may have efficacy in acute promyelocytic leukemia. This is based on the idea that bromodomain and extraterminal (BET) proteins normally function to maintain leukemia, so inhibition of these proteins can have anti-leukemic effect.[1]
  • Bromodomain proteins such as Brd4 play a major role in epigenetics, and the use of small molecule inhibitors like JQ1 may enhance the anti-cancer effect.[2]

RNA silencing of PML-RARalpha:

  • Studies have shown that the use of RNA interference technology can sensitive malignant promyelocytes to all-trans retinoic acid. A short hairpin RNA designed to the PML-RARalpha fusion transcript can result in degradation for the transcript and inhibition of leukemia growth, since the PML-RARalpha fusion product is the driver of the disease. The feasibility of short hairpin RNA to patients is currently unknown.[3] The concept of RNA therapeutics is currently being explored in treatment of acute leukemia. This is a form of gene therapy.

References

  1. Decker TM, Kluge M, Krebs S, Shah N, Blum H, Friedel CC; et al. (2017). "Transcriptome analysis of dominant-negative Brd4 mutants identifies Brd4-specific target genes of small molecule inhibitor JQ1". Sci Rep. 7 (1): 1684. doi:10.1038/s41598-017-01943-6. PMC 5431861. PMID 28490802.
  2. Pérez-Salvia M, Esteller M (May 2017). "Bromodomain inhibitors and cancer therapy: From structures to applications". Epigenetics. 12 (5): 323–339. doi:10.1080/15592294.2016.1265710. PMC 5453193. PMID 27911230.
  3. Casey NP, Woods GM (2012). "Anti-PML-RARα shRNA sensitises promyelocytic leukaemia cells to all-trans retinoic acid". J RNAi Gene Silencing. 8: 464–9. PMC 3522483. PMID 23300507.

Linked-in.jpg