Children diagnosed with an aggressive form of leukemia now have survival odds squarely in their favour as the result of a major clinical drug trial led by CFRI pediatric cancer researcher Dr. Kirk Schultz.
On January 25, 2013, the United States Food and Drug Administration (FDA) approved the use of the drug imatinib for the treatment of children newly diagnosed with Philadelphia chromosome positive (Ph+) acute lymphoblastic leukemia (ALL).
ALL is the most common type of pediatric cancer, affecting about 3200 children in North America each year. Of these, about four per cent are Ph+ ALL, for which treatment options lagged behind other less aggressive forms of ALL.
The FDA’s decision was based on the 2009 results of a clinical trial led by Dr. Schultz and conducted through the Children’s Oncology Group, involving over 50 pediatric cancer centres across North America.
The trial revealed that adding imatinib to an intense chemotherapy regime improves kids’ three-year survival rates from the historical average of 25 per cent to about 70 per cent – with no increased toxic effects.
“This is one of the biggest improvements in the treatment of a type of childhood cancer achieved in the early 21st century,” says Dr. Schultz, for whom the FDA’s blessing was the conclusion of almost 15 years of study planning, research and analysis.
The Children’s Oncology Group Study evaluated imatinib in 92 pediatric and young-adult patients at oncology centres across North America, including at BC Children’s Hospital.
Imatinib (commercial name Gleevec®) blocks proteins that promote the development of cancerous cells.
"The addition of that single drug was what changed the survival but it required to combination of both,” says Dr. Schultz. “This treatment strategy is now being adopted by the entire Children’s Oncology Group and applied to a number of high risk childhood cancers.”