Targeting BCR-ABL/CML Treatment

Chronic myelogenous leukemia (CML) is caused by the Philadelphia (Ph) chromosome-a mutation that can happen to stem cells in the bone marrow. Cells with the Philadelphia chromosome make an abnormal protein (BCR-ABL) that tells your bone marrow to make an increasing number of abnormal white blood cells. Over time, these abnormal cells crowd out healthy blood cells. CML usually stays in a "chronic" phase for a long time, but it can eventually become a life-threatening form of acute leukemia.

"TKI treatment"- is designed to block the action of BCR-ABL. Without this growth signal, the abnormal cells stop growing and begin to die. Within a few months, healthy cells begin replacing the abnormal cells, and blood returns to its normal mixture of cell types. Treatment for CML is based on a class of drugs called tyrosine kinase inhibitors (TKIs).

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Targeted Treatment and Blood Cells in CML

Healthy Blood

Healthy blood contains a mixture of cell types,
which grow from stem cells in the bone marrow.

CML

A mutation called the Ph chromosome encodes a protein
that sends a "growth signal". Cells with this chromosome
keep growing and crowd out healthy cells.

CML Controlled by Targeted Treatment

Targeted treatment blocks the growth signal. The mutated
cells stop growing, allowing healthy cells to replace them.