← Back
Research and new methods

Molecular targets: the weak spot of cancer

A molecular target is a protein or gene that the leukaemia cell needs to survive and grow and that differs from normal cells. A targeted medicine acts on exactly that.

Last updated: ~1 min read
Ribbon model of the ABL kinase domain with imatinib bound in its active site
Figure 1.A molecular target: imatinib in the ABL kinase

01Short answer

Classic chemotherapy affects all rapidly dividing cells. Targeted medicines block a specific molecule: for example the BCR::ABL1 protein in CML, BTK or BCL-2 in CLL, and FLT3 and IDH in AML. That is why testing comes first, to see whether the target is present. Targeted medicines also have side effects, and cancer can become resistant over time. They are often combined with other treatments.

02Simple explanation

A distinct molecule.

Test first.

Precise action.

Side-effect free: no.

03Scientific explanation

NCI: targeted therapy acts on the proteins that control how cancer cells grow and spread.

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?

Selectivity

Less effect on normal cells.

A highlighted receptor
Figure 2.A highlighted receptor.

Resistance

The cell can change the target and escape the medicine.

Healthy cells
Figure 3.Healthy cells — Molecular targets: the weak spot of cancer

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
SelectivityLess effect on normal cells.
ResistanceThe cell can change the target and escape the medicine.
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

Targeted therapy
Treatment that acts on a specific molecule.
Receptor
An "antenna" protein on the cell surface.
Clinical trial
Testing a new treatment with volunteers.
Evidence
A result confirmed by reliable research.

08Key points

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

09Frequently asked questions

Do I have a target?

Genetic and molecular tests show this; ask your doctor.

Do targeted medicines replace chemotherapy?

Sometimes, sometimes together; it depends on the disease.

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. Do I have a target?

  2. Do targeted medicines replace chemotherapy?

  3. Is this advance right for me?

My questions

12Sources

  1. National Cancer Institute. Targeted Therapy to Treat Cancer
  2. National Cancer Institute. NCI Dictionary of Cancer Terms: targeted therapy
  3. National Cancer Institute. What Are Clinical Trials?
  4. National Cancer Institute. Phases of Clinical Trials
  5. National Cancer Institute. Using Trusted Resources
  6. European Medicines Agency. Clinical trials in human medicines
  7. National Cancer Institute. Informed Consent in Clinical Trials
  8. National Cancer Institute. NCI Dictionary of Cancer Terms: clinical trial
  9. National Cancer Institute (NCI). Paying for Clinical Trials
  10. National Cancer Institute (NCI). Find NCI-Supported Clinical Trials
Medical disclaimerThis information is for educational purposes only and does not replace medical advice.
Last updated: ← Back to the list

Open the interactive site →