Precision medicine directed therapy enabling long-term survival in medulloblastoma: a case report
Subgroup-SHH of medulloblastoma (MB) is primarily found in children, with an increased propensity for metastatic presentation that contributes to its poor prognosis. Current therapy approaches may include surgery, radiation, systematic[...]
Establishment of Patient-Derived Organoids for Pediatric Cancer Research
Childhood cancer arises from disrupted developmental processes driven by genetic and epigenetic changes, making them fundamentally different from adult malignancies. Understanding this unique biology requires models that faithfully capture the[...]
In Vitro and In Vivo Efficacy of Romidepsin Alone and in Addition to Standard of Care for Treatment of Ewing Sarcoma
Smith KH, Trovillion EM, McKinney KQ, Gourabathini P, Wells K, Gandra D, Sholler C, Votruba I, Oesterheld J, Saulnier Sholler GL. In Vitro and In Vivo Efficacy of Romidepsin Alone and[...]
Molecular guided therapy leading to exceptional response in relapsed osteosarcoma
Osteosarcoma is the most common type of primary malignant bone tumor in children, adolescents and young adults and remains a significant clinical challenge, especially in the context of metastatic disease.[...]
Naxitamab-Combination Therapy for the Treatment of Patients With Refractory and/or Relapsed High-Risk Neuroblastoma
Approved treatments for patients with refractory and/or relapsed (R/R) high-risk (HR) neuroblastoma are limited, and there is a need for new treatment combinations. In this case series, 4 patients were[...]
Polyamine Inhibition with DFMO: Shifting the Paradigm in Neuroblastoma Therapy
Neuroblastoma is a common childhood malignancy, and high-risk presentations, including an MYCN amplified status, continue to result in poor survival. Difluoromethylornithine (DFMO) is a new and well-tolerated treatment for high-risk[...]
Panobinostat Synergizes with Chemotherapeutic Agents and Improves Efficacy of Standard-of-Care Chemotherapy Combinations in Ewing Sarcoma Cells
There has been little improvement in Ewing sarcoma survival rates over the past several decades and new therapeutic options are desperately needed. Histone deacetylases (HDACs) are known to play a[...]
DFMO inhibition of neuroblastoma tumorigenesis
Most high-risk neuroblastoma patients who relapse succumb to disease despite the existing therapy. We recently reported increased event-free and overall survival in neuroblastoma patients receiving difluoromethylornithine (DFMO) during maintenance therapy.[...]
Molecular-guided therapy for the treatment of patients with relapsed and refractory childhood cancers: a Beat Childhood Cancer Research Consortium trial
A total of 186 eligible patients were enrolled with 144 evaluable for safety and 124 evaluable for response. The average number of days from biopsy to initiation of the MTB‑recommended[...]
Maintenance DFMO Increases Survival in High Risk Neuroblastoma
High risk neuroblastoma (HRNB) accounts for 15% of all pediatric cancer deaths. Despite aggressive therapy approximately half of patients will relapse, typically with only transient responses to second-line therapy. This[...]