Targeted drugs
Targeted medicines block a specific change that drives the leukaemia cell.

01Short answer
02Simple explanation
Topic. targeted medicines.
Ask. Your team will explain your own situation.
Write down. Your questions.
Overview. The article “Targeted therapy”.
03Scientific explanation
Biomarker: a test showing whether a medicine fits.
Interactions: many are broken down by liver enzymes; see “Your full medication list and interactions”.
04What happens in the body?
Targeted medicines
They work when the cell carries a certain mutation or protein, for example BCR::ABL1 in CML, FLT3 or IDH in AML, and the BTK and BCL2 pathways in CLL. Most are tablets and may be taken for a long time. Their side effects differ from chemotherapy.
In more detail
Biomarker: a test showing whether a medicine fits. Interactions: many are broken down by liver enzymes; see “Your full medication list and interactions”.

The overview
This article goes deep into one subject. For the bigger picture, see “Targeted therapy”.

05Targeted medicines: the steps
Four steps.




06Examples
| Target | Where | Group |
|---|---|---|
| BCR::ABL1 | CML, Ph+ ALL | Tyrosine kinase inhibitors |
| BTK, BCL2 | CLL | BTK and BCL2 inhibitors |
| FLT3, IDH | AML | FLT3 and IDH inhibitors |
07Key terms
- Biomarker
- A feature measured in the cell.
- Inhibitor
- A medicine that blocks an enzyme.
08Key points
- Everyone’s plan is individual.
- Write down and bring your questions.
- Report new symptoms early.
- Do not change medicines yourself.
- For food, see “Handwashing: the simplest protection” and “Eggs and dairy: safe choices”.
- Overview: “Targeted therapy”.
09Frequently asked questions
Is a targeted medicine "gentler" than chemotherapy?
It is different; it has its own side effects.
Is this the same for everyone?
No; the plan depends on the type of leukaemia and your situation.
Where is the overview?
In the article “Targeted therapy”.
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.
Is a targeted medicine "gentler" than chemotherapy?
Is this the same for everyone?
Where is the overview?
12Sources
- National Cancer Institute (NCI). Targeted Therapy to Treat Cancer
- National Cancer Institute (NCI). Biomarker Testing / Precision Medicine
- MedlinePlus. Leukemia
- Leukemia & Lymphoma Society. Relapsed and refractory leukemia
- World Health Organization. Palliative care
- MedlinePlus. Cancer Chemotherapy
- American Cancer Society. How Chemotherapy Drugs Work
- Children’s Cancer and Leukaemia Group (CCLG). Acute lymphoblastic leukaemia (ALL)
- Cancer Research UK. Other ways of having chemotherapy
- Macmillan Cancer Support. Intrathecal chemotherapy