FDA Approval of New Treatment Option for Children with Secondary Acute Myeloid Leukemia is a Welcome Step Forward

By Gwen Nichols, MD, LLS Chief Medical Officer | March, 2021

Here at The Leukemia & Lymphoma Society (LLS), we applaud every advance for children with blood cancers. Today’s reason to celebrate is the news that the U.S. Food and Drug Administration (FDA) expanded the approval of daunorubicin and cytarabine (Vyxeos®) to include treatment of pediatric patients aged one year and older with newly-diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC). This is a welcome and necessary advancement in support of some of our most vulnerable patients.

The therapy was previously approved in 2017 for adult patients who acquired AML after being treated for a previous cancer or whose AML developed after progressing from a diagnosis of myelodysplastic syndrome. LLS recognized this therapy’s potential back in 2009 when it invested in it through its Therapy Acceleration Program® (TAP), which invests in cutting-edge companies working in areas of high unmet need for blood cancer patients. LLS partnered with Celator Pharmaceuticals, which Jazz Pharmaceuticals later acquired, on the clinical development of daunorubicin and cytarabine (Vyxeos®) for adults with AML.

The approval is supported by safety data from two trials conducted by the Children’s Oncology Group (COG) and Cincinnati Children’s Hospital (CCH), which established safety for relapsed/refractory patients. Both studies did report adverse reactions, including bleeding events, fever, and rash. The efficacy data was based on the CPX351–301 study in adults with secondary AML, which LLS TAP supported.

While we celebrate today’s news and what it means for children with AML and their families, and all the work being done to provide safe and effective treatments, LLS remains focused on how much more there is to do. Today, many children do not survive pediatric blood cancer, and survivors are often left with lifelong complications from treatment. When it comes to AML, the five-year survival rate for children younger than 15 years old diagnosed is only 68.7 percent.

Children have been treated with a one-size-fits-all approach for too long, despite cancer behaving differently in children than in adults. For many childhood cancers, chemotherapies developed decades ago remain the standard of care and simply don’t work well enough. In this era of precision medicine — giving the right treatment to the right patient at the right time — it’s critical for children to receive new therapies designed just for them.

LLS is determined to usher in a new era of drug development for pediatric blood cancers that puts children at the center of their care. Through the LLS Children’s Initiative, we seek to shift the standard of care for children, moving them away from toxic chemotherapies and toward effective and safe precision medicine treatments that target their cancer without harming the rest of their bodies. LLS is currently laying the groundwork for LLS PedAL, the first global precision medicine clinical trial for children with relapsed acute leukemia, which is being informed by its success with the Beat AML® Master Clinical Trial that was launched in 2016 for newly diagnosed AML adult patients 60 or older. Further, LLS is funding more than 30 research grants focused on deepening our understanding of the unique genetic causes and molecular biomarkers of pediatric blood cancer and how best to target them.

LLS is striving to raise $100 million for The LLS Children’s Initiative, which is a multi-year collaboration to expand our investment in leading-edge research, provide a comprehensive array of free information, resources, and financial support for affected children and families, and support policies that break down barriers to accessing care and developing new treatments.

Our goal is not only for children to survive blood cancer but for them to thrive in their lives after treatment.

The Leukemia & Lymphoma Society (LLS) is dedicated to funding research, finding cures and ensuring access to treatments for blood cancer patients.