Leading the Way in Pediatric Cancer Research

At the Cincinnati Children’s Cancer and Blood Diseases Institute, we are on a mission to develop cures for children and young adults with relapsed and high-risk cancers. Our vision is a world in which all patients are cured of cancer and have the assurance of life-long wellness.

Families travel from around the world to meet with our nationally recognized team of cancer specialists, in part because we think outside the box to develop novel treatment strategies. Our physicians, consulting specialists, nurses, support staff and laboratory research scientists are working together to pioneer more effective therapies. For example, in just the past two years, research from our center led to Food and Drug Administration (FDA) approvals of two drugs: Vyxeos, for children and young adults with acute myeloid leukemia, and selumetinib, for children with inoperable neurofibromatosis tumors.

Advanced Research and New Therapies for Relapsed and Treatment-Resistant Cancers

Developing new cancer therapies requires intense basic research (in the lab) and clinical research (in the patient care setting). At Cincinnati Children’s, our lab scientists and physicians embrace new treatment approaches when current therapies are not leading to effective cures. We continue to push progress forward in the fight against cancer.

CAR T-Cell Therapy for Pediatric Leukemia and Lymphoma

Cincinnati Children’s is one of the few centers in the country comprehensively using CAR-T (chimeric antigen receptor T-cell) therapy for children with lymphoma and to treat relapsed and newly diagnosed high-risk B-cell acute lymphoblastic leukemia patients. We have been a leader in the use of CAR-T therapies since starting our program in 2014. In fact, we participated in the early research trials that led to the FDA’s approval of the therapy in 2017.

First-in-the-Nation Clinical Trials of New Treatments for High-Risk and Relapsed Acute Leukemia

Our portfolio of clinical trials for relapsed leukemia is broad and includes next-generation targeted drugs, immunotherapies and novel bone marrow transplant approaches.

One recent breakthrough relates to a new treatment for acute myeloid leukemia (AML) in children. Researchers at Cincinnati Children’s played a significant role in the FDA’s approval of Vyxeos, a targeted anti-leukemia nanoparticle drug that is shown to be effective and safe for AML.

In addition, our researchers are studying how AML cells are wired, in hopes of making Vyxeos more effective. That work has led to our new clinical trial that combines Vyxeos with a drug called Venetoclax, which targets a mechanism that leukemia cells use to escape being killed. This study, the first of its kind in the U.S. for children and young adults, was developed at Cincinnati Children’s and is only available here.

The First-in-Human FLASH Proton Therapy Clinical Trial

Proton therapy is the most advanced form of radiation therapy available anywhere, leading to fewer side effects and complications than traditional radiation therapy. In 2016, Cincinnati Children’s opened its state-of-the-art Proton Therapy Center, which includes a dedicated research facility. Researchers there are the first in the world to investigate novel applications of Flash proton therapy. Flash delivers radiation at ultra-high speeds up to 1,000 times faster than what is used clinically today. Early pre-clinical studies suggest it causes fewer side effects and kills cancers as well as or better than conventional proton therapy. Our researchers conducted the world’s first clinical trial of FLASH radiation therapy. It’s just the beginning of what could become a game-changing therapy in the treatment of cancer.

Our proton center research team also is developing new approaches to cure brain tumors such as diffuse intrinsic pontine glioma (DIPG) and glioblastoma, relapsed brain tumors, sarcomas and lymphomas.

New Therapies for High-Risk and Relapsed Brain Tumors

The Cure Starts Now Brain Tumor Center at Cincinnati Children’s has one of the nation’s largest programs for basic research and clinical trials of new therapies for pediatric brain tumors that resist treatment or have relapsed. The center leads significant research efforts for MEK protein inhibitors such as selumetinib in children with cancer. For example, the center’s co-director, Peter de Blank, MD, MSCE, is the principal investigator of an international phase III study comparing selumetinib to standard therapy in children with low-grade glioma. The Children’s Oncology Group (COG) and National Cancer Institute (NCI) sponsor the study.

Center co-director Trent Hummel, MD, is the principal site investigator for one of only a few studies in North America that offers radiation plus another therapy to patients who are newly diagnosed with DIPG or diffuse midline glioma (DMG). The phase 1 pediatric trial will evaluate the safety of a drug called BXQ-350 and determine the maximum tolerated dose in children with newly diagnosed DIPG or DMG.

Leading-Edge Neurofibromatosis and Neural Tumor Research

Basic science and clinical research breakthroughs from the Neurofibromatosis Program have led to FDA drug approvals. One example is a new treatment for neurofibromatosis 1 (NF1). As many as half of children born with the most common form of this disease develop one or more plexiform neurofibroma (PN) tumors, which can be life-threatening.

Several years ago, lab researchers led by Nancy Ratner, PhD, discovered that blocking the MEK protein could shrink the tumors by more than 70% in mice. Pediatric oncologist Brian Weiss, MD, and colleagues from the NCI led clinical trials showing that the drug selumetinib can shut down the MEK protein and shrink the PNs in children. Their findings appeared in the prestigious New England Journal of Medicine in April 2020. Within 24 hours, the FDA approved the therapy — the first for children with NF1 and inoperable plexiform neurofibromas. Our researchers continue to push toward the higher goal of curing NF1.

National Study for High-Risk Liver and Kidney Tumors

Cincinnati Children’s offers about 20 clinical trials for children and young adults with liver and kidney cancer. James Geller, MD, director of our Liver and Kidney Cancer Centers, is a pediatric oncologist and a global expert in liver and kidney tumor therapy and new anticancer drug development. He is the principal investigator for three new national trials studying a treatment for hepatoblastoma (a form of liver cancer) and anaplastic Wilms tumor, relapsed Wilms tumor and pediatric renal cell carcinoma (types of kidney cancer). The studies are investigating how well combination chemotherapy works in treating patients with these cancers. It is sponsored by COG and NCI and will include patients from more than 200 hospitals nationwide.

Young Adult Cancer Research

Young adults (ages 15-39) respond differently to cancer therapy than young children and older adults. Their side effects are different, and they have different emotional needs. Cincinnati Children’s is one of a handful of global centers with a dedicated young adult cancer facility and care team. We lead several research efforts:

• Our center was the first in the region to offer the NCI MATCH clinical trial, which uses tumor genetic testing to identify targeted therapies.
• We offer a combination immune therapy and radiation therapy clinical trial for treatment-resistant sarcomas and liver and kidney tumors.