For children and families facing cancer, progress increasingly depends on connecting patients with treatments informed by the biology of their disease. At Nicklaus Children’s Health System, the Helen & Jacob Shaham Cancer & Blood Disorders Institute is expanding the research capabilities needed to make that increasingly possible while broadening access to specialized pediatric oncology care across South Florida.
Nicklaus Children’s is South Florida’s leader in the diagnosis and treatment of pediatric cancer and is involved in more than 40 cancer-related clinical studies. As a full member of the nationally funded Children’s Oncology Group (COG), the health system participates in a network of leading pediatric cancer specialists and institutions working together to develop safer and more effective therapies for children and adolescents.
That research mission received a major boost in 2025, when the Helen & Jacob Shaham Cancer & Blood Disorders Institute was awarded a $7.5 million appropriation from the State of Florida through the Cancer Connect Collaborative Research Incubator Fund. The investment supports expanded clinical trial capabilities while strengthening the infrastructure required to connect more Florida children with advanced treatment options close to home.
“This investment gives us an opportunity to build the research foundation needed to bring more promising therapies to children in South Florida and across the state,” said Marcos A. Mestre, senior vice president and chief clinical officer at Nicklaus Children’s Health System. “It is about expanding access while moving scientific discovery toward better care for our patients.”
A major component of that work is development of an early-phase pediatric oncology program that will expand first-in-child and Phase I/II clinical trial capabilities. Such studies are particularly important for children with relapsed or refractory disease and for those with rare cancers, where established treatment options are often limited.
Nicklaus Children’s is also expanding its precision medicine efforts. Researchers and clinicians are bringing together genomics and transcriptomics with proteomics and microbiome research. Drug-sensitivity testing adds another layer of information by helping researchers assess how an individual tumor responds to potential therapies. Together, these approaches provide a more individualized understanding of both the child and the cancer, helping inform medication selection and dosing while supporting treatment decisions.
That work builds on research conducted by Maggie Fader, M.D., pediatric hematologist/oncologist and medical director of the Sarcoma and Solid Tumors Program, and collaborators at Florida International University Herbert Wertheim College of Medicine. In a study published in Nature Medicine, the team combined genomic profiling with ex vivo drug-sensitivity testing for children and adolescents with relapsed or refractory cancers. Among six patients who received treatment guided by the functional precision medicine approach, five experienced improved progression-free survival compared with their previous therapy.
The institute is also developing a comprehensive cancer data repository supported by advanced bioinformatics and artificial intelligence analytics. These capabilities will help researchers work with larger volumes of clinical and research data while supporting stronger collaboration across Florida. The infrastructure also creates opportunities for new academic and industry partnerships focused on pediatric cancer research.
Innovation extends beyond systemic therapy. Toba Niazi, M.D., chief of neurosurgery and Co-Director of the Neuroscience Institute, is leading research involving magnetic resonance-guided focused ultrasound for aggressive pediatric brain tumors. Current work includes approaches designed to improve drug delivery as well as research evaluating sonodynamic therapy.
Thomas Utset-Ward, M.D., one of less than 10pediatric orthopedic oncologists in the nation, is advancing care for children with bone and soft-tissue tumors. His work includes complex surgical planning alongside research focused on pediatric sarcomas.
“Research is increasingly integrated into how we approach pediatric cancer care,” said [Dr. Ziad Khatib, medical director of hematology-oncology. “Our goal is to give families access to established therapies while also offering carefully designed research opportunities that expand what is possible for children with difficult-to-treat cancers.”
The institute itself is growing alongside its research portfolio. Nicklaus Children’s recently broke ground on a new home for the Helen & Jacob Shaham Cancer & Blood Disorders Institute on the hospital’s Miami campus. The new facility represents a significant investment in the future of pediatric cancer care and research in South Florida.
Pediatric hematology and oncology services are also available at Nicklaus Children’s Hospital Fort Lauderdale at Broward Health Medical Center, led by Hector Rodriguez-Cortes, M.D. This expansion helps families in Broward County access specialized pediatric cancer and blood disorders expertise closer to home while remaining connected to the broader resources of Nicklaus Children’s specialty services
That growth is also receiving national recognition. Nicklaus Children’s is nationally ranked by U.S. News & World Report in eight pediatric specialties, including pediatric oncology, reflecting the strength of its clinical program and its commitment to advancing care for children with cancer and blood disorders.
For Nicklaus Children’s, the future of pediatric oncology is closely tied to research embedded in clinical care. Continued investment in early-phase trials and precision medicine, supported by a growing research infrastructure, is helping bring new treatment possibilities and renewed hope to children and families across South Florida and beyond.
