Treatments for Pediatric CML (Chronic Myeloid Leukemia)

Chemotherapy (chemo) is the use of anti-cancer drugs that reach all areas of the body, which is why it can be suitable for cancers that spread, such as CML. In general, chemo works to stop the growth of cancer cells, either by preventing them from dividing or killing them. However, unlike with acute leukemia, chemotherapy cannot cure childhood CML.

Targeted therapy uses drugs to locate and attack specific leukemia cells without harming normal cells in the body. The first treatment for patients with newly diagnosed CML is often tyrosine kinase inhibitors, such as Imatinib, which can eliminate leukemia from the blood and prolong the chronic phase of the disease – thereby delaying transformation to the accelerated phase. These drugs can often control CML for long periods of time, however patients usually need to keep taking them daily.

When CML does not respond sufficiently to TKI targeted therapy drugs, a stem cell transplant along with high-dose chemotherapy may be considered. An allogeneic bone marrow transplant (using stem cells from a genetically suitable donor) is regarded as the only potential cure for CML.

During this procedure, diseased bone marrow cells are replaced by specialized stem cells that grow and restore the body’s normal blood cells. At present, scientific research is investigating whether adding targeted therapy drugs to stem cell transplant treatment can help to raise cure rates