New Leukemia Research Center Changing the Outcome for AML Patients

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Key Takeaways

The new Advanced Leukemia Therapies and Research Center at Cincinnati Children’s integrates world-class research, clinical programs and innovative treatments to improve outcomes for patients with acute myeloid leukemia (AML).
The new center collaborates across science and clinical divisions to accelerate therapy development and link research to patient care.

Established in 2024, the new Advanced Leukemia Therapies and Research Center at Cincinnati Children’s integrates the expertise of its researchers with the health system’s world-class clinical programs to accelerate new areas of research and lifesaving treatments. The center also increases patient access to cutting-edge clinical trials.

“We want to improve the outcomes of acute myeloid leukemia (AML) patients by spearheading innovative and transformative research that brings new treatment options to blood cancer patients of any age,” says Daniel Starczynowski, PhD, director of the Advanced Leukemia Therapies and Research Center, associate director of the Cancer and Blood Diseases Institute and associate director for basic science research at the University of Cincinnati Cancer Center. “The center eliminates barriers and initiates new laboratory research and treatment approaches informed by our AML patients.”

Leaders in the Cincinnati Children’s Cancer and Blood Diseases Institute designed the center to foster research collaborations across basic science and clinical divisions. An end-to-end leukemia team within the institute leverages extensive scientific research discoveries with translational and clinical initiatives. The center’s integrated structure brings together leukemia research initiatives spanning fundamental mechanisms of leukemia biology, drug discovery and development, and early and late clinical development teams.

In addition, the Advanced Leukemia Therapies and Research Center:

Facilitates translational research to support early drug development
Provides disease-specific computational support
Links research projects that require patient specimens at Cincinnati Children’s
Offers seed funding
Serves as a hub for investigators dedicated to leukemia research and novel therapies through targeted new recruitments and current core faculty at Cincinnati Children’s

To accelerate the delivery of new treatments, center staff members work closely with the Cancer and Blood Diseases Institute, other research divisions within Cincinnati Children’s, and the University of Cincinnati Cancer Center.

A Bench-to-Bedside Approach

The new center builds on a foundation of ongoing, groundbreaking AML research at Cincinnati Children’s. For example, in 2018, researchers here led the first pediatric phase I clinical trial of Vyxeos, an engineered anti-cancer nanoparticle delivery system, in children with AML. Three years later, as a result of that study and a phase II Children’s Oncology Group study, the U.S. Food and Drug Administration approved a new pediatric indication for Vyxeos.

Cincinnati Children’s is in the vanguard for new AML therapies, and we are thrilled to see the intersection of research and treatment come to life.

John Perentesis, MD

Now, researchers at Cincinnati Children’s are testing a novel cellular therapy—using cytokine-induced, memory-like natural killer cells—for children ages 2–18 and adults with treatment-resistant AML. In studies, these cells have exhibited a response against AML.

“Our bench-to-bedside approach personalizes treatment for our AML patients,” says John Perentesis, MD, executive co-director of the Cancer and Blood Diseases Institute and director of the Division of Oncology. “Cincinnati Children’s is in the vanguard for new AML therapies, and we are thrilled to see the intersection of research and treatment come to life.”

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