# LSD1 inhibitors

Source: https://onco.cc/technologies/lsd1-inhibitors/  
OnCo record `lsd1-inhibitors` (Technology). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Drugs against the histone demethylase LSD1, which keeps blood and neuroendocrine cancer cells from maturing; being tested in myelofibrosis, leukaemia and small cell lung cancer.

## Summary

Lysine-specific demethylase 1 (LSD1, gene KDM1A) removes methyl marks from histone H3 and supports the undifferentiated state of acute myeloid leukaemia blasts and small cell lung cancer cells. Inhibitors such as bomedemstat, iadademstat and pulrodemstat have shown differentiation responses and platelet-lowering effects, leading to trials in essential thrombocythaemia and myelofibrosis as well as combination studies in AML and small cell lung cancer. Thrombocytopenia is the on-target dose-limiting effect.

## Fields

- Kind: Technology
- Status: phase-2
- Last checked: 2026-09-04
- Principle: Irreversible or reversible inhibitors block LSD1's demethylase activity and its scaffolding role with GFI1, releasing differentiation programmes.
- Strengths: Differentiation rather than cytotoxicity; Activity in myeloproliferative neoplasms
- Limitations: Thrombocytopenia; No approval yet; Single-agent solid tumour activity limited

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/KDM1A
- Wikipedia: https://en.wikipedia.org/wiki/KDM1A

## Connected records

- cancers: [Acute myeloid leukaemia](https://onco.cc/cancers/aml/), [Myeloproliferative neoplasms (PV, ET, myelofibrosis)](https://onco.cc/cancers/myeloproliferative-neoplasms/), [Small-cell lung cancer](https://onco.cc/cancers/sclc/)
- fronts: [Epigenetic & Transcriptional Therapy](https://onco.cc/fronts/epigenetics/), [Targeted Therapy](https://onco.cc/fronts/targeted-therapy/)
- technologies: [Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET)](https://onco.cc/technologies/epigenetic-drugs/)
- drugs: [Bomedemstat](https://onco.cc/drugs/bomedemstat/)

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