Research Excellence, Outstanding Care, Advanced Technologies Drive Cancer Leadership
Post Date: May 2, 2025 | Publish Date:
Cancer
Research Annual Report 2024
Our top-ranked childhood cancer program continues to break ground with new discoveries and programs to build a better future for survivors. The Cancer and Blood Diseases Institute at Cincinnati Children’s has been renowned for years as a leader in scientific discovery, innovation and multidisciplinary collaboration. Researchers here constantly pursue new clues about the causes of cancer, blood diseases and immune deficiency disorders, and their work regularly leads to more effective, better-targeted therapies for children in need.
These successes combined with an ongoing commitment to invest in discovery helps explain why Cincinnati Children’s was rated in the past year as the nation’s top pediatric cancer program by U.S. News & World Report. The ranking reflects numerous measures of cancer care excellence, from survival outcomes to nurse staffing to the availability of advanced technologies. Not only does cancer care at Cincinnati Children’s reflect widescale use of best practices in the field, experts here often help establish those practices.
Underpinning it all: strong collaboration between clinical care and research programs that speed the transfer of knowledge from the laboratory to patient care.
In fiscal 2024, cancer and blood disease experts at Cincinnati Children’s launched far-reaching new programs, developed advanced research tools, played leading roles in clinical trials and authored or co-authored hundreds of research papers. Highlights include:
Cincinnati Children’s Launches Advanced Leukemia Therapies and Research Center
Our new Advanced Leukemia Therapies and Research Center, directed by Daniel Starczynowski, PhD, seeks to improve outcomes for acute myeloid leukemia patients bybuilding on a foundation of groundbreaking research, including the use of novel cellular therapies and nanoparticle drugs.
“Translating cancer research into improved health outcomes for our patients has been a strength of our institution for quite some time,” says Tina Cheng, MD, MPH, chair of Pediatrics, Research Foundation director, and chief medical officer at Cincinnati Children’s. “The Advanced Leukemia Therapies and Research Center and the progress we are making there sets Cincinnati Children’s apart from other pediatric academic research centers.”
Advanced Map of Human Blood Stem Cells Could Guide Highly Targeted Leukemia Care
Researchers at Cincinnati Children’s have published the world’s most detailed atlas of hematopoietic stem and progenitor cells (HSPCs), identifying over 80 distinct subsets. Details were published in Nature Immunology.
This advanced map, created using game theory and machine learning, provides a deep view of bone marrow stem and progenitor compartments. The findings could guide highly targeted leukemia care by revealing new stem/progenitor cell populations and strategies for isolating novel cell populations.
The study was led by co-first authors Xuan Zhang, PhD, and Baobao Song, PhD, and co-corresponding authors Nathan Salomonis, PhD, and H. Leighton Grimes, PhD.
Research Reveals Potential Achilles’ Heel in Treatment-Resistant MPN
This study identifies DUSP1 as a potential target for treating myeloproliferative neoplasms (MPNs) that are resistant to JAK2 inhibitors. The findings, published in the journal Leukemia, showed that targeting DUSP1 eradicates JAK2 mutated MPN in mice, providing a novel therapeutic approach.
“This study, for the first time, provides mechanistic understanding about why JAK2 inhibitors are ineffective in vivo and how JAK2V617F signaling suppresses P53 function required for MPN transformation and progression,” says senior author Mohammad Azam, PhD. “Selective targeting of DUSP1 opens up a completely novel therapeutic approach and a potentially curative treatment outcome in MPNs.”
First Skeleton-Wide Study of Blood Cell Formation Yields Surprising Findings
Cincinnati Children’s researchers conducted the first skeleton-wide study of blood cell formation. The study, published in Nature, showed that different bones have specialized responses to hematopoietic insults.
“For example, our data shows that biopsies that draw marrow from just one type of bone may not provide a full picture of how the blood production system has been affected by a disease or other insult,” says corresponding author Daniel Lucas, PhD.
“Meanwhile, efforts to stimulate production of certain blood cell types may be dramatically improved by focusing on specific bone types.”
Co-authors say this groundbreaking research opens new paths for diagnosing and treating blood-related conditions.
New Research Tool Accelerates Hunt for Cancer Immunotherapy Targets
An innovative computational tool dubbed “SNAF” may help the research world bring the emerging promise of cancer immunotherapy to a wider range of patients, according to a study published in Science Translational Medicine.
The research tool, called the Splicing Neo Antigen Finder (SNAF), was developed by researchers from Cincinnati Children’s and the University of Virginia. The project was led by Guangyuan Li, PhD, and Nathan Salomonis, PhD, both with the Division of Biomedical Informatics.
“The implications of this discovery are significant,” says H. Leighton “Lee” Grimes, PhD, a co-author of the study and director of the Cancer Pathology Program at Cincinnati Children’s. “By identifying shared splicing neoantigens present in up to 90% of cancer patients, SNAF not only presents new targets for therapy but also challenges and expands our understanding of cancer biology.”
Clinical Trial Begins for Novel Natural Killer Cell Therapy
Cincinnati Children’s is testing a novel therapy using cytokine-induced memory-like natural killer (CIML-NK) cells from haploidentical donors to treat acute myeloid leukemia (AML).
This study is open to patients aged 12 to 45 years with relapsed or refractory AML who have not undergone bone marrow transplantation. The first patient was infused in May 2023. The therapy aims to offer AML patients a less risky treatment.
“To date, cellular therapy options for patients with AML have been limited by toxicity from the cellular therapy,” says principal investigator YunZu Wang, MD. “NK cell therapy may be a better option for them.”
Novel Therapy for Langerhans Cell Histiocytosis Maintains 100% Response Rate
A novel therapy using kinase inhibitors dabrafenib and trametinib has shown a 100% success rate in treating Langerhans cell histiocytosis (LCH) at Cincinnati Children’s.
The therapy targets BRAF and MAP2K1 mutations and has been used on more than 60 patients with no incidence of disease recurrence, according to a September 2023 retrospective study in Haematologica.
“The evidence for this protocol is so strong that encouraging its widespread adoption has become my life’s work,” says Ashish Kumar, MD, PhD, director of Cincinnati Children’s Histiocytosis Center. “Our protocol is as good as or better than chemotherapy, with fewer side effects. Not using these effective lifesaving drugs to treat children with treatment-resistant, high-risk LCH is almost negligent.”
Proton Therapy Excels at Treating Cancers of Head and Neck
Proton therapy can be an effective and less-toxic alternative to conventional radiation therapy for patients with advanced cancers of the head and neck. Researchers at the Proton Therapy Center on the Liberty Campus of Cincinnati Children’s found that proton therapy can be delivered with precision, sparing surrounding normal tissue and causing fewer side effects than X-ray-based photon therapy.
The study, published in Cancers, showed treatment significantly reducing production of small extracellular vesicles from cancer cells, which usually suppress the immune system.
Head and neck squamous cell carcinoma is the sixth-most prevalent cancer worldwide and among the most immunosuppressive.
“Our findings suggest that proton therapy might be better at reducing the immune-suppressing effects of cancer exosomes by producing fewer of them,” the authors wrote.
Improved Chemotherapy and Surgery Eliminating Need for Radiation for Low-Grade Gliomas
Evolving surgical and chemotherapy treatments for pediatric low-grade glioma (pLGG) have greatly reduced the need for radiation therapy, according to a study published in Nature Cancer, led by corresponding author Peter de Blank, MD, MSCE, co-medical director at Cincinnati Children’s The Cure Starts Now Brain Tumor Center.
The study followed 2,501 adult survivors of childhood glioma and showed that the proportion of survivors exposed to cranial radiation decreased over time, while the use of surgery and chemotherapy increased. This shift has led to improved long-term outcomes for survivors.
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About this blog
The Research Horizons blog features news and insights about the latest discoveries and innovations developed by the scientists of Cincinnati Children's. This blog does not provide medical advice, diagnosis, or treatment.
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