Acute promyelocytic leukemia future or investigational therapies
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1] Shyam Patel [2]
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.
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.[2] The concept of RNA therapeutics is currently being explored in treatment of acute leukemia. This is a form of gene therapy.
References
- ↑ 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.
- ↑ 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.