At The Leukemia & Lymphoma Society (LLS), we celebrate every advancement toward our mission of curing blood cancer and improving the quality of life for patients and their families. Today’s reason to celebrate is the news that the U.S. Food and Drug Administration (FDA) has approved a new treatment for children with a rare type of cancer called ALK-positive anaplastic large cell lymphoma (ALCL).

ALCL constitutes approximately 10-15% of non-Hodgkin lymphoma in the pediatric population. This rare lymphoma is curable with standard chemotherapy for most children, but the newly approved drug from Pfizer, crizotinib (XALKORI®), is now an option for those whose cancer returned or worsened despite earlier treatment. This is a long-awaited treatment option for the pediatric population, as anaplastic lymphoma kinase (ALK) inhibitors have been approved for adults with metastatic non-small cell lung cancer and lymphoma for years. ALK, the molecular target for crizotinib is altered in 85% of pediatric ALCL patients. This alteration in ALK is much less prevalent in adult ALCL; treatments for ALK-negative ALCL is the subject of a current LLS grant provided to Dr. Andrew Feldman (Mayo Clinic, Rochester, MN).

In clinical studies for the pediatric indication, crizotinib was efficacious based on objective response rate (ORR) and duration of response as assessed by an independent review committee. The ORR in the 26 patients was 88% (95% CI: 71, 96), with a complete remission rate of 81%. Of the 23 patients who achieved a response, 39% maintained response for at least 6 months, and 22% maintained response for at least 12 months.

Medical advancements are never more important than when they lead to cures for children. LLS has been at the forefront of advances in pediatric blood cancer since its founding in 1949, and with the launch of the Children’s Initiative in 2019, continues to take on children’s cancer from every direction. Most children with blood cancers are treated with the same toxic combinations of chemotherapies developed decades ago. LLS is committed to revolutionizing the standard of care for children with cancer and providing them with safer, more effective treatments that rely less on chemotherapies that harm healthy cells in the body.

While we celebrate today’s good news and what it means for children with ALK-positive ALCL and their families, we at LLS also remain focused on how much more there is to do.

Through the LLS Children’s Initiative, an unprecedented collaboration, we are setting out to transform pediatric blood cancer treatment and care. The initiative is focused on saving even more children from blood cancers, but our focus goes deeper. We want 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 striving to raise $100 million for this 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.

LLS financial investments are strategically designed to fuel scientific breakthroughs, but breakthroughs, despite how they sound, do not happen quickly. The journey from laboratory discovery to the ability to control or even cure a disease is a long road. LLS, on behalf of blood cancer patients, our volunteers, and our advocates, provides sustained funding in research programs that can make cures happen.

Lee Greenberger, Ph.D., is the Chief Scientific Officer of The Leukemia & Lymphoma Society

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