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FDA Approves Drug to Treat Pediatric Leukemia Subtype

The Leukemia & Lymphoma Society has a long history of funding research projects paving the way for today’s treatments, including for the latest FDA approval.

Rye Brook, N.Y., October 25, 2023 – The U.S. Food and Drug Administration (FDA) recently approved bosutinib (Bosulif®) for use in certain children aged 1 year and older with chronic myeloid leukemia (CML).

This marks the first indication of this drug to treat children, but its third since it was first approved for adults in 2012. Bosutinib is for use in children whose CML carries the Philadelphia chromosome (PH+ CML), including newly diagnosed children or those whose cancer stopped responding to past treatments.

“The biology of cancer is often different in children, and we need more treatments that are designed specifically for this vulnerable patient population,” said Gwen Nichols, M.D., Chief Medical Officer of The Leukemia & Lymphoma Society (LLS). “The LLS Dare to Dream Project is dedicated to transforming treatment and care for kids with blood cancer, so we are thrilled when a drug receives FDA approval for use in our youngest patients.”

Approximately three-quarters of children enrolled in the trial that resulted in bosutinib’s approval had a response to treatment, with reductions in the number of cancer cells in their bone marrow. Families should, however, be aware that children receiving bosutinib may experience certain side effects including abdominal pain, rash, fatigue, and vomiting. 

Bosutinib is a precision medicine called a Tyrosine kinase inhibitor that targets not only the tyrosine kinase ABL (similar to imatinib) but also a specific enzyme called SRC to limit cancer cell growth.

Over the past 20 years, LLS funded several research projects that helped define this new pathway and ways to target it that paved the way for the use of SRC inhibitors to treat several blood cancers. 

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