Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET)
Drugs that change how genes are switched on and off without changing the DNA itself.
Azacitidine/decitabine (DNMT) in MDS/AML, HDAC inhibitors in T-cell lymphoma, tazemetostat (EZH2; withdrawn worldwide March 2026 over secondary blood cancers), ivosidenib/vorasidenib (IDH), revumenib/ziftomenib (menin), and BET inhibitors (pelabresib in myelofibrosis). Solid tumour activity is limited so far except for IDH and EZH2 in defined subsets; combinations to re-sensitise to immunotherapy or hormone therapy are the hope.
How it works
Inhibit writers, erasers, or readers of DNA and histone marks, or scaffold proteins that tether them.
- Differentiation rather than cytotoxicity
- Defined genetic subsets respond
- Broad effects; modest solid tumour activity
An ancient poison turned cure: with retinoic acid it cures more than 95% of acute promyelocytic leukaemia without conventional chemotherapy.
A gentle chemotherapy that switches silenced genes back on. With venetoclax it became the standard for older people with AML who cannot take intensive treatment.
Belinostat is an HDAC inhibitor for relapsed peripheral T-cell lymphoma; about a quarter of patients respond, and it can be used in patients with low platelets.
Oral decitabine-cedazuridine is a pill version of the hypomethylating chemotherapy that, since May 2026, lets older AML patients take their whole venetoclax regimen at home.
Enasidenib is the IDH2 counterpart of ivosidenib, approved in the US for relapsed disease but never approved in Europe after it failed to extend survival in a confirmatory trial.
Ivosidenib is a pill that blocks the mutant IDH1 enzyme, approved in bile duct cancer and in leukaemia.
An epigenetic drug that may re-sensitise prostate cancer to hormone therapy, in three phase 3 trials with enzalutamide.
Olutasidenib is a second IDH1 inhibitor for relapsed AML, with a higher response rate in its pivotal cohort than ivosidenib had in its own.
An HDAC inhibitor for relapsed myeloma approved in 2015 and withdrawn in 2021 after its confirmatory trial was never completed.
Revumenib (Revuforj) is the first menin inhibitor (2024), for acute leukaemias with KMT2A rearrangements or NPM1 mutations.
Romidepsin is an epigenetic drug for T-cell lymphomas of the skin and lymph nodes; its US lymph-node indication was withdrawn when a confirmatory trial failed.
Tazemetostat was the first EZH2 inhibitor, approved for epithelioid sarcoma and follicular lymphoma in 2020 and withdrawn worldwide in 2026 after secondary blood cancers.
The vitamin-A derivative that made acute promyelocytic leukaemia the first cancer cured by differentiation therapy rather than by killing cells.
The first targeted therapy for low-grade brain tumours, delaying the need for radiation and chemotherapy by years.
Ziftomenib is the second menin inhibitor for leukaemia, approved in November 2025 as a once-daily pill for relapsed NPM1-mutated AML.
Latest papers
topQuery for this technology: (TITLE:"epigenetic therapy" OR ABSTRACT:"epigenetic therapy" OR TITLE:"HDAC inhibitor" OR ABSTRACT:"HDAC inhibitor" OR TITLE:"EZH2 inhibitor" OR ABSTRACT:"EZH2 inhibitor" OR TITLE:"menin inhibitor" OR ABSTRACT:"menin inhibitor" OR TITLE:"IDH inhibitor" OR ABSTRACT:"IDH inhibitor") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET), not a curated reading list.