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MEDICATION — TARGETED THERAPY

Tyrosine kinase inhibitor (TKI) therapy

Targeted pills that block the abnormal BCR-ABL1 protein driving Philadelphia-chromosome–positive (Ph+) leukemia — added to chemotherapy to greatly improve outcomes.
Treatments & procedures › Tyrosine kinase inhibitor (TKI) therapy

What it is

Some B-ALL is “Philadelphia-chromosome positive” (Ph+), meaning the leukemia cells carry a BCR-ABL1 fusion gene that makes an always-on growth signal. Tyrosine kinase inhibitors — imatinib, dasatinib, ponatinib — block that signal. Added to chemotherapy, and increasingly paired with immunotherapy, they have turned a once high-risk subtype into one with much better outcomes. The same drug class is the mainstay of chronic myeloid leukemia (CML).

Why it's done

  • Philadelphia-positive (BCR-ABL1–positive) B-ALL
  • Some “Ph-like” ALL with targetable ABL-class fusions
  • Given together with chemotherapy, and increasingly with immunotherapy

How it’s taken

Taken by mouth every day, alongside chemotherapy and continued per protocol. The specific TKI is chosen by the leukemia’s mutation profile and side-effect considerations, and response is followed with molecular (BCR-ABL1) testing.

What to expect

Ongoing daily therapy with molecular monitoring to track response. Depending on protocol and whether a transplant is planned, some patients can eventually reduce or stop under specialist guidance.

Risks & side effects

  • Fluid retention, low blood counts, rash and GI upset
  • Dasatinib — fluid around the lungs (pleural effusions)
  • Ponatinib — cardiovascular and clotting risks
  • Drug interactions; resistance mutations (e.g., T315I) can emerge

Alternatives

Chemotherapy
The backbone TKIs are added to
Immunotherapy
Blinatumomab is increasingly combined with TKIs in Ph+ ALL
Stem-cell transplant
Considered in some Ph+ cases