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Research and new methods

Menin inhibitors: a new class

Menin is a protein that the disease genes need in order to work in leukaemias with a KMT2A rearrangement (formerly called MLL) or an NPM1 mutation. Menin inhibitors break this link.

Last updated: ~1 min read
A gloved hand holding a test tube while a pipette adds a sample
Figure 1.Studying a new target in the laboratory

01Short answer

This is a relatively new class of medicine. The first medicines have begun to be approved for relapsed or resistant acute leukaemia with a KMT2A rearrangement; combinations in NPM1-mutated AML and as first treatment are being studied in clinical trials. As with IDH inhibitors, differentiation syndrome can occur, and some medicines can affect heart rhythm. Only genetic testing shows whether one is suitable, and availability depends on the country.

02Simple explanation

KMT2A and NPM1.

A new class.

Much is in trials.

Differentiation syndrome: be alert.

03Scientific explanation

NCI: a menin inhibitor is a targeted medicine that blocks the menin protein and is being studied in some acute leukaemias.

NCI: new treatments are tested step by step, from the laboratory to clinical trials; only those proven safe and beneficial become standard care.

04What happens in the body?

KMT2A

A gene often rearranged in leukaemia.

Genes dimming
Figure 2.Genes dimming — Menin inhibitors: a new class

NPM1

A gene often mutated in AML.

A new path
Figure 3.A new path — Menin inhibitors: a new class

How to read the news

Research news brings hope, but not every advance suits everyone. Look at who it was tested in, at what phase and in how many people.

Talk to your doctor

Show your doctor what you have read; they will explain how it relates to your diagnosis and treatment. Do not change your treatment on your own.

05From research news to a conversation: four steps

Read, check, ask, decide together.

Read — From a trusted source.1ReadFrom a trusted source.
Check — Phase, participants, result.2CheckPhase, participants, result.
Ask — Show your doctor.3AskShow your doctor.
Decide — Together, without rushing.4DecideTogether, without rushing.
SourceA trusted site.
DoctorA personal explanation.
SafetyNot changing treatment yourself.

06Key points at a glance

TopicIn brief
KMT2AA gene often rearranged in leukaemia.
NPM1A gene often mutated in AML.
How to read the newsResearch news brings hope, but not every advance suits everyone. Look at who it was tested in, at what phase and in how many people.
Talk to your doctorShow your doctor what you have read; they will explain how it relates to your diagnosis and treatment. Do not change your treatment on your own.

07Key terms

Menin
A protein that regulates gene activity.
KMT2A rearrangement
The KMT2A gene fused with another gene.
Clinical trial
Testing a new treatment with volunteers.
Evidence
A result confirmed by reliable research.

08Key points

  • Test.
  • Ask.
  • Monitor.
  • Check the source.
  • Talk to your doctor.

09Frequently asked questions

Is this medicine right for me?

Only with a KMT2A or NPM1 change and by your doctor's decision.

Is it available in my country?

Often through trials or special programmes; ask your doctor.

Is this advance right for me?

Only your treating doctor can say, based on your diagnosis, stage and condition.

10Questions for your doctor

Take these questions to your appointment. The ones you save are collected on the “My questions” page and stay on this device only.

  1. Is this medicine right for me?

  2. Is it available in my country?

  3. Is this advance right for me?

My questions

12Sources

  1. National Cancer Institute. NCI Dictionary of Cancer Terms: menin inhibitor
  2. National Cancer Institute. Adult Acute Myeloid Leukemia Treatment (PDQ®) – Patient Version
  3. National Cancer Institute. What Are Clinical Trials?
  4. National Cancer Institute. Phases of Clinical Trials
  5. National Cancer Institute. Using Trusted Resources
  6. U.S. National Library of Medicine. ClinicalTrials.gov
  7. Deutsches Krebsforschungszentrum. Krebsinformationsdienst
  8. KWF / NFK / IKNL. Kanker.nl
  9. U.S. Food and Drug Administration. The Drug Development Process
  10. National Cancer Institute. NCI Dictionary of Cancer Terms: preclinical study
Medical disclaimerThis information is for educational purposes only and does not replace medical advice.
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