Thanks to Four Diamonds, the Pediatric Cancer Experimental Therapeutics Program Collaborates to Save Lives

医学科学在对儿科癌症的治疗方面取得了重大进展;然而，某些肿瘤仍然存在抗性，导致预后差和生存率差。¹“With experimental therapeutics, we focus on those pediatric cancer patients for whom conventional treatment has failed,” saysValerie Brown, M.D., Ph.D., clinical director, experimental therapeutics, Penn State Children’s Hospital. “Our goal is to target these cancers more precisely to increase the cure rate and diminish the risk of patients developing short-term and long-term side effects.”

Since its inception in 2010, the Pediatric Cancer Experimental Therapeutics Program has benefited from funding provided through Four Diamonds. Funding to Four Diamonds comes through The Penn State IFC/Panhellenic Dance Marathon, affectionately known as THON™, Mini-THON^®and other community organizations.

发现effective therapies for highly treatment-resistant cancers requires a collaborative infrastructure of researchers to conduct clinical trials not typically available to a single institution. Currently, Children’s Hospital is a member of thePediatric Oncology Experimental Therapeutics Investigators’ Consortium (POETIC),Pediatric Blood and Marrow Transplant Consortium (PBMTC)and theChildren’s Oncology Group. Children’s Hospital also recently became a member of the Neuroblastoma and Medulloblastoma Translational Research Consortium (NMTRC), a network of more than 20 universities, children’s hospitals and institutions that offers a number of early phase clinical trials. Since October 2013, more than 20 patients have been enrolled in at least a dozen phase I and II trials, with five more set to begin shortly.

One pioneering trial conducted through the NMTRC, “Molecular-guided Therapy for the Treatment of Patients With Relapsed and Refractory Childhood Cancers,” uses an individualized approach to determine optimal therapy based upon the molecular aberrations of each patient’s tumor.²Biopsies of the tumor(s) are obtained and, from a genomic analysis (also known as personalized or precision medicine), a report is generated detailing potential responses to more than 200 drugs and naturally occurring compounds. A panel of NMTRC members holds a “virtual tumor board” where the patient’s case is reviewed, and a molecular-guided treatment plan is devised.

“最终，我们的目标很简单：未来的小儿癌患者现在具有生存希望的希望，”棕色博士说。“我们从四颗钻石收到的支持使我们能够提供这种希望。”

Read more about how Four Diamonds supports childhood cancer services at Penn State Children’s Hospital.

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Valerie I. Brown, M.D., Ph.D.
Associate Professor of Pediatrics, Division of Pediatric Oncology/Hematology Clinical Director, Experimental Therapeutics
PHONE:717-531-1042
E-MAIL:vbrown1@hmc.psu.edu
CLINICAL INTEREST:“我的重点是开发新颖，生物合理的儿童和青少年治疗方案，患有癌症，重点是复发急性淋巴细胞白血病和造血干细胞移植。我的总体目标是为无望的希望提供“希望”。“

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References:

1. Klimo P Jr, Nesvick CL, Broniscer A, Orr BA, Choudhri AF.Malignant brainstem tumors in children, excluding diffuse intrinsic pontine gliomas.J Neurosurg Pediatr.2015年10月16:1-9。
2. https://clinicaltrials.gov/ct2/show/NCT02162732. Accessed November 30, 2015.