FDA Approves Drug for Rare Cancer

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CHOP led key trial for the treatment, which targets unresectable, recurrent, or refractory inflammatory anaplastic lymphoma kinase (ALK)-positive myofibroblastic tumors (IMT).

The FDA has approved crizotinib (brand name Xalkori®), a first generation ALK inhibitor, for the treatment of adult and pediatric patients 1 year of age and older with unresectable, recurrent, or refractory inflammatory anaplastic lymphoma kinase (ALK)-positive myofibroblastic tumors (IMT), a rare soft tissue tumor that has a predilection for children and adolescents and for which the mainstay of curative therapy is complete surgical resection.

Two multicenter clinical trials ultimately led to the approval of crizotinib for IMT: one led by researchers at Children’s Hospital of Philadelphia (CHOP) through the Children’s Oncology Group (COG), which included 14 pediatric patients, and another trial that included 7 adult patients.

Yael P. Mossé, MD, attending physician at CHOP's Cancer Center, and Professor of Pediatrics at the University of Pennsylvania School of Medicine, has studied ALK alterations in the context of several cancers, including neuroblastoma, and was the principal investigator for the pediatric trial that led to crizotinib’s FDA approval for anaplastic large cell lymphoma (ALCL) in January 2021, and now for IMT.

Yael P. Mossé, MD Yael P. Mossé, MD “The approval of Xalkori for ALK-positive IMT will bring hope to many pediatric patients and families who previously had highly limited treatment options,” Mossé said. “It is very exciting to have approval for frontline unresectable IMTs. This is an important milestone for these patients, and for pediatric oncology researchers within COG and CHOP, who continue to seek out more rational therapies.”

ALK fusions were originally discovered in 1994 in a pediatric lymphoma model and have since been identified as playing a role in other cancers, including certain forms of neuroblastoma, the majority of ALCLs, and IMTs.

Xalkori® inhibits ROS1, a tyrosine kinase closely related to ALK, and has already been approved for the treatment of patients with metastatic non-small cell lung cancer that is ALK or ROS1-positive, and for the treatment of relapsed or refractory systemic ALCL that is ALK-positive.

Read more about this approval here.

Contact: Jennifer Lee, The Children’s Hospital of Philadelphia, 267-426-6084 or leej41@chop.edu

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