Dana-Farber Scientist Awarded $400K to Seek New Therapies for Childhood AML

Dana-Farber Scientist Awarded $400K to Seek New Therapies for Childhood AML
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Kimberly Stegmaier, MD, of the Dana-Farber Cancer Institute, has been awarded a $400,000 research grant to continue her work to find new treatments for pediatric acute myeloid leukemia (AML) — a childhood cancer long in need of therapeutic options.

The two-year grant is funded by the Leukemia & Lymphoma Society (LLS) and the Rally Foundation for Childhood Cancer Research, and was peer-reviewed by both organizations. It will support Stegmaier’s preclinical project, titled “Advancing New Therapeutic Strategies for Pediatric Acute Leukemias.”

Stegmaier is co-director of Dana-Farber’s Hematologic Malignancy Center and a professor of pediatrics at Harvard Medical School.

“Children fighting AML are in desperate need of new targeted treatments and we are so pleased to partner with LLS in supporting this promising, target-focused work of Dr. Stegmaier,” Dean Crowe, Rally founder and CEO, said in a press release. “Dr. Stegmaier’s groundbreaking research will provide us critical data that will hopefully change the paradigm for kids fighting AML and possibly acute lymphoblastic leukemia, too.”

AML accounts for about 20% of childhood cancers. In most cases, gene mutations and chromosome abnormalities in leukemia cells occur sporadically, meaning by chance. New therapies are needed for childhood AML cases because roughly one-third of patients relapse after initial chemotherapy treatment.

There has been some progress. The U.S. Food and Drug Administration recently granted Rare Pediatric Disease designation to Mateon Therapeutics’ OXi-4503 (combretastatin A1-diphosphate, CA1P) for the treatment of children and teens with AML who carry genetic mutations that disproportionately affect this demographic.

The research project is part of the LLS Children’s Initiative, a $100 million comprehensive effort that aims to battle pediatric blood cancers on every front. The initiative includes new research grants to advance potential treatments, enhanced free education and support services for children and their families, and policy and advocacy endeavors.

A clinical trial in pediatric acute leukemia, called LLS PedAL, also is included in the initiative. LLS PedAL is a global precision medicine master study that will test multiple targeted therapies at up to 200 clinical sites globally. Stegmaier is a medical doctor and member of the LLS PedAL team. The hope is that her research will lead to treatment candidates being evaluated in the clinical trial.

“We are extraordinarily hopeful that Dr. Stegmaier’s trailblazing research offers promise for less toxic and more effective treatment options for children,” said Gwen Nichols, MD, LLS chief medical officer. “As well, we are grateful to announce this collaboration with the Rally Foundation, who shares the same relentless commitment as LLS to help children not only survive their cancer but thrive in their lives after treatment.”

The nonprofit LLS works to ultimately cure leukemia, lymphoma, and multiple myeloma, and improve the quality of life for patients and their families.

The Rally Foundation For Childhood Cancer Research aims to empower researchers nationwide to find better therapies with fewer long-term side effects. It has awarded $20 million in childhood cancer research grants, supporting 364 projects.

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