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IDH1 and IDH2: restoring maturation

IDH1 and IDH2 are enzymes of normal cell metabolism. When mutated, they produce a substance (2-HG) that blocks cell maturation. Some people with AML have these mutations.

Last updated: ~1 min read
A scientist in blue gloves pipetting coloured samples into test tubes
Figure 1.The laboratory finds IDH1/IDH2 mutations

01Short answer

IDH inhibitors reduce production of this faulty substance and allow leukaemia cells to mature again. The response often appears gradually over weeks to months. A particular side effect is differentiation syndrome: fever, breathlessness, weight gain and swelling, which must be reported to your doctor at once. IDH inhibitors are used alone or with other medicines, and new combinations are being studied.

02Simple explanation

A faulty substance: 2-HG.

Maturation restored.

Response: gradual.

Differentiation syndrome: report at once.

03Scientific explanation

NCI: targeted medicines can be used in AML with an IDH1 or IDH2 mutation.

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?

2-HG

An oncometabolite made by mutant IDH.

Stuck rungs
Figure 2.Stuck rungs — IDH1 and IDH2: restoring maturation

Differentiation syndrome

The body's reaction to cells maturing quickly.

Climbing again
Figure 3.Climbing again — IDH1 and IDH2: restoring maturation

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
2-HGAn oncometabolite made by mutant IDH.
Differentiation syndromeThe body's reaction to cells maturing quickly.
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

IDH
The enzyme isocitrate dehydrogenase.
Differentiation
Cell maturation.
Clinical trial
Testing a new treatment with volunteers.
Evidence
A result confirmed by reliable research.

08Key points

  • Test.
  • Take it.
  • Report symptoms.
  • Check the source.
  • Talk to your doctor.

09Frequently asked questions

When will I see results?

Often after weeks to months; your doctor will monitor.

I am breathless and gaining weight.

This may be differentiation syndrome; call at once.

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. When will I see results?

  2. I am breathless and gaining weight.

  3. Is this advance right for me?

My questions

12Sources

  1. National Cancer Institute. Adult Acute Myeloid Leukemia Treatment (PDQ®) – Patient Version
  2. National Cancer Institute. What Are Clinical Trials?
  3. National Cancer Institute. Phases of Clinical Trials
  4. National Cancer Institute. Using Trusted Resources
  5. National Cancer Institute. NCI Dictionary of Cancer Terms: mutation
  6. MedlinePlus (U.S. National Library of Medicine). Evaluating Health Information
  7. National Cancer Institute (NCI). NCI Dictionary of Cancer Terms: flow cytometry
  8. National Cancer Institute (NCI). NCI Dictionary of Cancer Terms: next-generation sequencing
  9. National Cancer Institute (NCI). Find NCI-Supported Clinical Trials
  10. National Cancer Institute (NCI). Biomarker Testing / Precision Medicine
Medical disclaimerThis information is for educational purposes only and does not replace medical advice.
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