HEMATOLOGY, ONCOLOGY & STEM CELL TRANSPLANT
RESEARCH HIGHLIGHTS
Our team is participating in 95 open clinical trials. Select study topics include: Phase III studies
Expert hematologists, oncologists and stem cell transplant specialists at Riley Children’s Health combine leading-edge medicine with holistic, personalized care to treat children with cancer and blood disorders. With access to the latest clinical trials and therapeutic options, our skilled specialists treat the full spectrum of pediatric cancers, including aggressive disease and cancers that are no longer responsive to conventional therapy.
Phase I studies ■ GRN163L (imetelstat) in combination with fludarabine and cytarabine for patients with acute myeloid leukemia ■ Intra-tumoral injections of ex vivo expanded natural killer cells in children and young adults with recurrent or progressive supratentorial malignant brain tumors ■ Targeted immunotherapy with daratumumab following myeloablative total body irradiation-based conditioning and allogeneic hematopoietic cell transplantation in children, adolescents and young adults with high-risk T-cell acute lymphoblastic leukemia and lymphoma
■ Dinutuximab added to intensive multimodal therapy for children with newly diagnosed high-risk neuroblastoma ■ Efficacy and safety of prophylaxis with vonicog alfa in children diagnosed with severe von Willebrand disease ■ Inotuzumab ozogamicin for newly diagnosed high-risk B-cell acute lymphoblastic leukemia (B-ALL); risk- adapted post-induction therapy for high-risk B-ALL, mixed phenotype acute leukemia and disseminated B-lymphoblastic lymphoma Prospective study ■ Evaluate the disease status in patients with sickle cell disease: A globin regional data and discovery network
Premier programs
Advanced therapies and procedures ■ CAR-T trials for leukemia (PLAT Phase I and Phase II trials) ■ Gene therapy for beta thalassemia and sickle cell disease (Bluebird Bio and Vertex) ■ NK cell infusions following stem cell transplant Our program is accredited or certified by: ■ Foundation for the Accreditation of Cellular Therapy (FACT) ■ Association for the Advancement of Blood & Biotherapies (AABB) ■ American Society for Histocompatibility and Immunogenetics (ASHI) ■ National Cancer Institute Comprehensive Cancer Center ■ Health Care Financing Administration
TOP DISTINCTIONS
■ Precision Genomics Program ■ Neurofibromatosis Program ■ Stem Cell/Immunotherapy Program ■ Comprehensive Bleeding Program and Women and Girls Blood Disorder Clinic of Excellence ■ Thrombosis and Thrombophilia Program ■ Sickle Cell Disease Program (includes a multidisciplinary program for high-risk patients) ■ Onco-Fertility Program ■ Developmental Therapeutics Program ■ Adolescent and Young Adult Oncology Program ■ Survivorship Program Our program is affiliated with the Brown Center for Immunotherapy at IU School of Medicine (offering access to novel immunotherapy and gene therapy clinical trials only available at IU) and the highly regarded Indiana University Gene Therapy Testing Laboratory .
■ Home to the only pediatric stem cell transplant and CAR-T cell therapy programs in Indiana ■ Part of the only National Cancer Institute-designated Comprehensive Cancer Center in Indiana ■ Largest comprehensive sickle cell program in Indiana: – Only National Institutes of Health-funded telemedicine program in the nation for pediatric sickle cell disease – One of 40 institutions in the National Alliance of Sickle Cell Disease Centers – Only Lifespan Sickle Cell Disease Center of Excellence in the state ■ Among the 10% of Children’s Oncology Group (COG) members that have early access to clinical trials and new treatments through the COG Pediatric Early Phase Clinical Trials Network ■ One of the few children’s hospitals in the nation with a multidisciplinary treatment program for neurofibromatosis ■ Sole Indiana program (and one of the few regionally) to offer experimental testicular
Program Volume: 2022 – 2025
16,149
1,594
1,581
1,460
14,256 14,915
13,702
We offer co-location of subspecialists to provide convenient and accessible care to individuals living with sickle cell disease.
1,211
Outpatient visits (All disease types)
New patient visits (All disease types)
2022
2023
2024
2025
2022
2023
2024
2025
93 research publications in 2025
NOTABLE PUBLICATIONS
Leukemia: February 2026 Targeting IL-1/IRAK1/4 signaling in acute myeloid leukemia stem cells following treatment and relapse The Oncologist: January 2026 A phase I study targeting the APE1/ref-1 redox signaling protein with APX3330: First clinical agent targeting APE1/ref-1 in cancer Science Advances: January 2026 Inhibition of focal adhesion kinase impairs tumor formation and preserves hearing in a murine model of NF2-related schwannomatosis Redox Biology: October 2025 The absence of Peroxiredoxin-1 in human pancreatic ductal adenocarcinoma (PDAC) markedly reduces cell survival and tumor growth when coupled with the inhibition of Ref-1 redox signaling Journal of Clinical Oncology: August 2025 Phase I study of 131I-metaiodobenzylguanidine with dinutuximab ± vorinostat for patients with relapsed or refractory neuroblastoma: A new approaches to neuroblastoma therapy trial
DIVISION CHIEF Brian D. Weiss, MD Nora Letzter Professor of Pediatrics, IU School of Medicine > Connect with Dr. Weiss on Doximity.
A founding hospital of The H.E.A.L. Network, a national learning
tissue and ovarian tissue cryopreservation protocols
34 board certified physicians
health network dedicated to “improving sickle cell disease outcomes through collaboration and innovation.”
See full team member list >
OUR TEAM ALSO PUBLISHED IN: Global Advances in Integrative Medicine and Health Research and Practice in Thrombosis and Haemostasis
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