Center Unites Global Experts to Advance Clinical Trials and Transform Patient Care Across All Ages

The Advanced Leukemia Therapies and Research Center at Cincinnati Children’s is accelerating the path from discovery to treatment for patients with myeloid malignancies, including acute myeloid leukemia and myelodysplastic syndromes. Established in 2023, the Advanced Leukemia Therapies and Research Center unites world-class researchers and clinicians to drive innovation, expand access to leading-edge clinical trials, and deliver next-generation therapies for pediatric, adolescent, young adult and adult patients.

“We want to improve the outcomes of acute myeloid leukemia 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 Cincinnati Children's 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 acute myeloid leukemia patients.”

Leaders in the Cancer and Blood Diseases Institute designed the Advanced Leukemia Therapies and Research Center to foster research collaborations across basic science and clinical divisions. An end-to-end leukemia team within the Cancer and Blood Diseases 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 for innovative and high-risk projects
• 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, 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 Advanced Leukemia Therapies and Research Center builds on the foundation of ongoing, groundbreaking acute myeloid leukemia research at Cincinnati Children’s. For example, in 2018, our researchers led the first pediatric phase I clinical trial of Vyxeos, an engineered anti-cancer nanoparticle delivery system, in children with acute myeloid leukemia. Three years later, because 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.

Now, researchers within the Advanced Leukemia and Therapies Research Center are pioneering a clinical study using cytokine-induced memory-like natural killer cells from haploidentical donors to treat children, adolescents, young adults and adults (ages 2-45) with relapsed or refractory acute myeloid leukemia who have not undergone bone marrow transplantation. This novel therapy offers a potentially safer alternative to traditional cellular therapies, such as CAR T-cell therapy, with lower risks of cytokine release syndrome and neurotoxicity.

The study’s first patient was successfully infused in May 2023, marking a major milestone in cell therapy manufacturing and delivery. The protocol involves reduced-intensity chemotherapy followed by infusion of donor-derived cytokine-induced memory-like natural killer cells. Study results prompted U.S. Food and Drug Administration approval to lower the minimum age to 2, further expanding access to more pediatric patients.

Another promising initiative at the Advanced Leukemia Therapies and Research Center is the PARPAML study, a phase I clinical trial evaluating the safety and efficacy of combining talazoparib—a poly ADP-ribose polymerase inhibitor—with chemotherapy agents topotecan and gemcitabine in pediatric patients with relapsed or refractory acute myeloid leukemia and acute leukemia of ambiguous lineage. By blocking DNA repair in cancer cells, talazoparib may enhance the effectiveness of chemotherapy. This is the first time this drug combination is being tested in children and young adults with these leukemias.

The PARPAML trial represents a significant step in targeted therapy for pediatric acute myeloid leukemia. Researchers aim to improve outcomes by combining molecularly targeted agents with chemotherapy while minimizing toxicity. The study reflects the center’s commitment to translating scientific discoveries into new treatment options for patients with limited responses to standard therapies.

“Our bench-to-bedside approach personalizes treatment for our acute myeloid leukemia 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 acute myeloid leukemia therapies, and we are thrilled to see the intersection of research and treatment come to life.”

(Published November 2025)

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