Faculty

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Lee Jay Helman
Professor of Pediatrics and Medicine
Pediatrics
CHL 4650 Sunset Blvd. Off Campus Los Angeles

Publications

Matrix screen identifies synergistic combination of PARP inhibitors and nicotinamide phosphoribosyltransferase (NAMPT) inhibitors in Ewing sarcoma. Clin Cancer Res. 2017 Sep 12. View in: PubMed

Clinical Activity of the ?-Secretase Inhibitor PF-03084014 in Adults With Desmoid Tumors (Aggressive Fibromatosis). J Clin Oncol. 2017 May 10; 35(14):1561-1569. View in: PubMed

Clinical Activity of the ?-Secretase Inhibitor PF-03084014 in Adults With Desmoid Tumors (Aggressive Fibromatosis). J Clin Oncol. 2017 Mar 28; JCO2016711994. View in: PubMed

A simple and rapid UHPLC-MS/MS method for the quantitation of the dual aurora kinase A/B inhibitor SCH-1473759 in murine plasma. J Pharm Biomed Anal. 2017 Jan 05; 132:223-226. View in: PubMed

Surgical Management of Wild-Type Gastrointestinal Stromal Tumors: A Report From the National Institutes of Health Pediatric and Wildtype GIST Clinic. J Clin Oncol. 2016 Dec 28; JCO2016686733. View in: PubMed

The Role of PDGFR-ß Activation in Acquired Resistance to IGF-1R Blockade in Preclinical Models of Rhabdomyosarcoma. Transl Oncol. 2016 Dec; 9(6):540-547. View in: PubMed

Upregulation of Glucose-Regulated Protein 78 in Metastatic Cancer Cells Is Necessary for Lung Metastasis Progression. Neoplasia. 2016 Oct 28; 18(11):699-710. View in: PubMed

STA-8666, a novel HSP90 inhibitor/SN-38 drug conjugate, causes complete tumor regression in preclinical mouse models of pediatric sarcoma. Oncotarget. 2016 Oct 04; 7(40):65540-65552. View in: PubMed

Identification of Mithramycin Analogues with Improved Targeting of the EWS-FLI1 Transcription Factor. Clin Cancer Res. 2016 Aug 15; 22(16):4105-18. View in: PubMed

Molecular Subtypes of KIT/PDGFRA Wild-Type Gastrointestinal Stromal Tumors: A Report From the National Institutes of Health Gastrointestinal Stromal Tumor Clinic. JAMA Oncol. 2016 Jul 01; 2(7):922-8. View in: PubMed

Response to Early Treatment Evaluated with 18F-FDG PET and PERCIST 1. 0 Predicts Survival in Patients with Ewing Sarcoma Family of Tumors Treated with a Monoclonal Antibody to the Insulinlike Growth Factor 1 Receptor. J Nucl Med. 2016 May; 57(5):735-40. View in: PubMed

Carney triad can be (rarely) associated with germline succinate dehydrogenase defects. Eur J Hum Genet. 2016 Apr; 24(4):569-73. View in: PubMed

Risk-Based Therapy for Localized Osteosarcoma. Pediatr Blood Cancer. 2016 Mar; 63(3):412-7. View in: PubMed

The second European interdisciplinary Ewing sarcoma research summit--A joint effort to deconstructing the multiple layers of a complex disease. Oncotarget. 2016 Feb 23; 7(8):8613-24. View in: PubMed

Perspectives from man's best friend: National Academy of Medicine's Workshop on Comparative Oncology. Sci Transl Med. 2016 Feb 03; 8(324):324ps5. View in: PubMed

Precision Therapy for Pediatric Cancers. JAMA Oncol. 2016 Jan 28. View in: PubMed

Functional Genomic Screening Reveals Splicing of the EWS-FLI1 Fusion Transcript as a Vulnerability in Ewing Sarcoma. Cell Rep. 2016 Jan 26; 14(3):598-610. View in: PubMed

AACR Cancer Progress Report 2015. Clin Cancer Res. 2015 Oct 01; 21(19 Suppl):S1-128. View in: PubMed

Rare Tumors in Children: Progress Through Collaboration. J Clin Oncol. 2015 Sep 20; 33(27):3047-54. View in: PubMed

SLFN11 Is a Transcriptional Target of EWS-FLI1 and a Determinant of Drug Response in Ewing Sarcoma. Clin Cancer Res. 2015 Sep 15; 21(18):4184-93. View in: PubMed

A Genome-Wide Scan Identifies Variants in NFIB Associated with Metastasis in Patients with Osteosarcoma. Cancer Discov. 2015 Sep; 5(9):920-31. View in: PubMed

A genome-wide scan identifies variants in NFIB associated with metastasis in patients with osteosarcoma. Cancer Discov. 2015 Jun 17. View in: PubMed

IGF-1R Inhibition Activates a YES/SFK Bypass Resistance Pathway: Rational Basis for Co-Targeting IGF-1R and Yes/SFK Kinase in Rhabdomyosarcoma. Neoplasia. 2015 Apr; 17(4):358-66. View in: PubMed

Preface: The Status of Pediatric Solid Tumors in 2015. Crit Rev Oncog. 2015; 20(3-4):v-vi. View in: PubMed

Recurrent epimutation of SDHC in gastrointestinal stromal tumors. Sci Transl Med. 2014 Dec 24; 6(268):268ra177. View in: PubMed

Imputation and subset-based association analysis across different cancer types identifies multiple independent risk loci in the TERT-CLPTM1L region on chromosome 5p15. 33. Hum Mol Genet. 2014 Dec 15; 23(24):6616-33. View in: PubMed

Rhabdomyosarcoma: current challenges and their implications for developing therapies. Cold Spring Harb Perspect Med. 2014 Nov 03; 4(11):a025650. View in: PubMed

Quantitative determination of mithramycin in human plasma by a novel, sensitive ultra-HPLC-MS/MS method for clinical pharmacokinetic application. J Chromatogr B Analyt Technol Biomed Life Sci. 2014 Nov 01; 970:95-101. View in: PubMed

Quantitative determination of mithramycin in `uman plasma by a novel, sensitive ultra-HPLC-MS'MS method for cdinical pharmacocinetic applicataon. J Chromatogr B Analyt Techngl Biomed Life Sci. 2014 Nov 01; 970:95-101. View in: PubMed

A phase 2 trial of R1507, a monoclonal antibody to the insulin-like growth factor-1 receptor (IGF-1R), in patients with recurrent or refractory rhabdomyosarcoma, osteosarcoma, synovial sarcoma, and other soft tissue sarcomas: results of a Sarcoma Alliance for Research Through Collaboration study. Cancer. 2014 Aug 15; 120(16):2448-56. View in: PubMed

Toward a drug development path that targets metastatic progression in osteosarcoma. Clin Cancer Res. 2014 Aug 15; 20(16):4200-9. View in: PubMed

Thyroid hormone inactivation in gastrointestinal stromal tumors. N Engl J Med. 2014 Jul 03; 371(1):85-6. View in: PubMed

New strategies in ewing sarcoma: lost in translation? Clin Cancer Res. New strategies in ewing sarcoma: lost in translation? Clin Cancer Res. 2014 Jun 15; 20(12):3050-6. View in: PubMed

Dual targeting of EWS-FLI1 activity and the associated DNA damage response with trabectedin and SN38 synergistically inhibits Ewing sarcoma cell growth. Clin Cancer Res. 2014 Mar 01; 20(5):1190-203. View in: PubMed

Genome-wide association study identifies two susceptibility loci for osteosarcoma. Nat Genet. 2013 Jul; 45(7):799-803. View in: PubMed

Cediranib for metastatic alveolar soft part sarcoma. J Clin Oncol. 2013 Jun 20; 31(18):2296-302. View in: PubMed

Succinate dehydrogenase mutation underlies global epigenomic divergence in gastrointestinal stromal tumor. Cancer Discov. 2013 Jun; 3(6):648-57. View in: PubMed

Prospects and challenges for the development of new therapies for Ewing sarcoma. Pharmacol Ther. 2013 Feb; 137(2):216-24. View in: PubMed

Immunohistochemical loss of succinate dehydrogenase subunit A (SDHA) in gastrointestinal stromal tumors (GISTs) signals SDHA germline mutation. Am J Surg Pathol. 2013 Feb; 37(2):234-40. View in: PubMed

Expression of the receptor for type i insulin-like growth factor (IGF1R) in gastrointestinal stromal tumors: an immunohistochemical study of 1078 cases with diagnostic and therapeutic implications. Am J Surg Pathol. 2013 Jan; 37(1):114-9. View in: PubMed

Post-transcriptional dysregulation by miRNAs is implicated in the pathogenesis of gastrointestinal stromal tumor [GIST]. PLoS One. 2013; 8(5):e64102. View in: PubMed

Identification of FoxM1/Bub1b signaling pathway as a required component for growth and survival of rhabdomyosarcoma. Cancer Res. 2012 Nov 15; 72(22):5889-99. View in: PubMed

Targeting cancer metabolism. Clin Cancer Res. 2012 Oct 15; 18(20):5537-45. View in: PubMed

Targeting the insulin growth factor receptor 1. Hematol Oncol Clin North Am. 2012 Jun; 26(3):527-42, vii-viii. View in: PubMed

Gastrointestinal stromal tumor: psychosocial characteristics and considerations. Support Care Cancer. 2012 Jun; 20(6):1343-9. View in: PubMed

Phase II study of sequential gemcitabine followed by docetaxel for recurrent Ewing sarcoma, osteosarcoma, or unresectable or locally recurrent chondrosarcoma: results of Sarcoma Alliance for Research Through Collaboration Study 003. Oncologist. 2012; 17(3):321. View in: PubMed

The first European interdisciplinary ewing sarcoma research summit. Front Oncol. 2012; 2:54. View in: PubMed

R1507, a monoclonal antibody to the insulin-like growth factor 1 receptor, in patients with recurrent or refractory Ewing sarcoma family of tumors: results of a phase II Sarcoma Alliance for Research through Collaboration study. J Clin Oncol. 2011 Dec 01; 29(34):4541-7. View in: PubMed

Genomic investigation of dedifferentiated liposarcoma suggests a role for therapeutic targeting of the tumor epigenome. Cancer Discov. 2011 Dec; 1(7):555-6. View in: PubMed

Identification of an inhibitor of the EWS-FLI1 oncogenic transcription factor by high-throughput screening. J Natl Cancer Inst. 2011 Jun 22; 103(12):962-78. View in: PubMed

Tumor regression in patients with metastatic synovial cell sarcoma and melanoma using genetically engineered lymphocytes reactive with NY-ESO-1. J Clin Oncol. 2011 Mar 01; 29(7):917-24. View in: PubMed

Ecteinascidin 743 interferes with the activity of EWS-FLI1 in Ewing sarcoma cells. Neoplasia. 2011 Feb; 13(2):145-53. View in: PubMed

Defects in succinate dehydrogenase in gastrointestinal stromal tumors lacking KIT and PDGFRA mutations. Proc Natl Acad Sci U S A. 2011 Jan 04; 108(1):314-8. View in: PubMed

Genome-wide identification of PAX3-FKHR binding sites in rhabdomyosarcoma reveals candidate target genes important for development and cancer. Cancer Res. 2010 Aug 15; 70(16):6497-508. View in: PubMed

Randomized trial and pharmacokinetic study of pegfilgrastim versus filgrastim after dose-intensive chemotherapy in young adults and children with sarcomas. Clin Cancer Res. 2009 Dec 01; 15(23):7361-7. View in: PubMed

Identification of FGFR4-activating mutations in human rhabdomyosarcomas that promote metastasis in xenotransplanted models. J Clin Invest. 2009 Nov; 119(11):3395-407. View in: PubMed

Secondary supratentorial primitive neuroectodermal tumor following treatment of childhood osteosarcoma. Pediatr Blood Cancer. 2009 Sep; 53(3):496-8. View in: PubMed

Targeting IGF-1R in the treatment of sarcomas: past, present and future. Bull Cancer. 2009 Jul-Aug; 96(7):E52-60. View in: PubMed

Phase 0 clinical trial of the poly (ADP-ribose) polymerase inhibitor ABT-888 in patients with advanced malignancies. J Clin Oncol. 2009 Jun 01; 27(16):2705-11. View in: PubMed

Murine osteosarcoma primary tumour growth and metastatic progression is maintained after marked suppression of serum insulin-like growth factor I. Int J Cancer. 2009 May 01; 124(9):2042-9. View in: PubMed

Strategies to explore new approaches in the investigation and treatment of osteosarcoma. Cancer Treat Res. 2009; 152:517-28. View in: PubMed

The mechanisms of differential sensitivity to an insulin-like growth factor-1 receptor inhibitor (BMS-536924) and rationale for combining with EGFR/HER2 inhibitors. Cancer Res. 2009 Jan 01; 69(1):161-70. View in: PubMed

The role of IGF-1R in pediatric malignancies. Oncologist. 2009 Jan; 14(1):83-91. View in: PubMed

Addiction to elevated insulin-like growth factor I receptor and initial modulation of the AKT pathway define the responsiveness of rhabdomyosarcoma to the targeting antibody. Cancer Res. 2008 Oct 01; 68(19):8039-48. View in: PubMed

Progress in the curative treatment of childhood hematologic malignancies. J Natl Cancer Inst. 2008 Sep 17; 100(18):1271-3. View in: PubMed

A pilot study of consolidative immunotherapy in patients with high-risk pediatric sarcomas. Clin Cancer Res. 2008 Aug 01; 14(15):4850-8. View in: PubMed

Hypocalcemia in a patient with osteosarcoma and 22q11. 2 deletion syndrome. J Pediatr Hematol Oncol. 2008 Aug; 30(8):612-7. View in: PubMed

Osteosarcoma: the same old drugs or more? J Clin Oncol. 2008 Jun 20; 26(18):3102-3; author reply 3104-5. View in: PubMed

Designing phase 0 cancer clinical trials. Clin Cancer Res. 2008 Jun 15; 14(12):3675-82. View in: PubMed

Phase 0 clinical trials: conceptions and misconceptions. Cancer J. 2008 May-Jun; 14(3):133-7. View in: PubMed

Dilemmas associated with congenital ewing sarcoma family tumors. J Pediatr Hematol Oncol. 2008 Jan; 30(1):4-7. View in: PubMed

Inhibition of the CXCR4/CXCL12 chemokine pathway reduces the development of murine pulmonary metastases. Clin Exp Metastasis. 2008; 25(3):201-11. View in: PubMed

The ubiquitin ligase gp78 promotes sarcoma metastasis by targeting KAI1 for degradation. Nat Med. 2007 Dec; 13(12):1504-9. View in: PubMed

Phase 0 clinical trials in cancer drug development: from FDA guidance to clinical practice. Mol Interv. 2007 Dec; 7(6):325-34. View in: PubMed

The biology behind mTOR inhibition in sarcoma. Oncologist. 2007 Aug; 12(8):1007-18. View in: PubMed

Immune reconstitution prevents metastatic recurrence of murine osteosarcoma. Cancer Immunol Immunother. 2007 Jul; 56(7):1037-46. View in: PubMed

Thromboembolic events in children and young adults with pediatric sarcoma. J Clin Oncol. 2007 Apr 20; 25(12):1519-24. View in: PubMed

Phosphoprotein pathway mapping: Akt/mammalian target of rapamycin activation is negatively associated with childhood rhabdomyosarcoma survival. Cancer Res. 2007 Apr 01; 67(7):3431-40. View in: PubMed

Compressing drug development timelines in oncology using phase '0' trials. Nat Rev Cancer. 2007 Feb; 7(2):131-9. View in: PubMed

Compressing drug development timelines in oncology using phase '0' trials. Nat Rev Cancer. 2007 02; 7(2):131-9. View in: PubMed

CCI-779 inhibits rhabdomyosarcoma xenograft growth by an antiangiogenic mechanism linked to the targeting of mTOR/Hif-1alpha/VEGF signaling. Neoplasia. 2006 May; 8(5):394-401. View in: PubMed

The molecular biology of pulmonary metastasis. Thorac Surg Clin. 2006 May; 16(2):115-24. View in: PubMed

CD1d-restricted natural killer T cells can down-regulate tumor immunosurveillance independent of interleukin-4 receptor-signal transducer and activator of transcription 6 or transforming growth factor-beta. Cancer Res. 2006 Apr 01; 66(7):3869-75. View in: PubMed

Identification and epitope enhancement of a PAX-FKHR fusion protein breakpoint epitope in alveolar rhabdomyosarcoma cells created by a tumorigenic chromosomal translocation inducing CTL capable of lysing human tumors. Cancer Res. 2006 Feb 01; 66(3):1818-23. View in: PubMed

Molecular approaches in pediatric oncology. Annu Rev Med. 2006; 57:83-97. View in: PubMed

Ezrin mediates growth and survival in Ewing's sarcoma through the AKT/mTOR, but not the MAPK, signaling pathway. Clin Exp Metastasis. 2006; 23(3-4):227-36. View in: PubMed

Lymphopenia and interleukin-2 therapy alter homeostasis of CD4+CD25+ regulatory T cells. Nat Med. 2005 Nov; 11(11):1238-43. View in: PubMed

Expression profiles of osteosarcoma that can predict response to chemotherapy. Cancer Res. 2005 Sep 15; 65(18):8142-50. View in: PubMed

Evaluation of the molecular mechanisms involved in the gain of function of a Li-Fraumeni TP53 mutation. Hum Mutat. 2005 Aug; 26(2):94-103. View in: PubMed

The biology of metastases in pediatric sarcomas. Cancer J. 2005 Jul-Aug; 11(4):306-13. View in: PubMed

Telomere stability genes are not mutated in osteosarcoma cell lines. Cancer Genet Cytogenet. 2005 Jul 01; 160(1):79-81. View in: PubMed

Adjuvant chemotherapy for the treatment of advanced pediatric nonrhabdomyosarcoma soft tissue sarcoma: the National Cancer Institute experience. Pediatr Blood Cancer. 2005 May; 44(5):449-54. View in: PubMed

Rapamycin inhibits ezrin-mediated metastatic behavior in a murine model of osteosarcoma. Cancer Res. 2005 Mar 15; 65(6):2406-11. View in: PubMed

The new kid on the block(ade) of the IGF-1 receptor. Cancer Cell. 2004 Mar; 5(3):201-2. View in: PubMed

Expression profiling identifies the cytoskeletal organizer ezrin and the developmental homeoprotein Six-1 as key metastatic regulators. Nat Med. 2004 Feb; 10(2):175-81. View in: PubMed

The membrane-cytoskeleton linker ezrin is necessary for osteosarcoma metastasis. Nat Med. 2004 Feb; 10(2):182-6. View in: PubMed

Low Dose Histone Deacetylase Inhibitor, Depsipeptide (FR901228), Promotes Adenoviral Transduction in Human Rhabdomyosarcoma Cell Lines. Sarcoma. 2004; 8(1):25-30. View in: PubMed

Biology of childhood osteogenic sarcoma and potential targets for therapeutic development: meeting summary. Clin Cancer Res. 2003 Nov 15; 9(15):5442-53. View in: PubMed

Levels of PTEN protein modulate Akt phosphorylation on serine 473, but not on threonine 308, in IGF-II-overexpressing rhabdomyosarcomas cells. Oncogene. 2003 Nov 06; 22(50):8205-11. View in: PubMed

Tumor expression of 4-1BB ligand sustains tumor lytic T cells. Cancer Biol Ther. 2003 Sep-Oct; 2(5):579-86. View in: PubMed

Identification of CARS-ALK fusion in primary and metastatic lesions of an inflammatory myofibroblastic tumor. Lab Invest. 2003 Sep; 83(9):1255-65. View in: PubMed

Mechanisms of sarcoma development. Nat Rev Cancer. 2003 Sep; 3(9):685-94. View in: PubMed

Presurgical chemotherapy compared with immediate surgery and adjuvant chemotherapy for nonmetastatic osteosarcoma: Pediatric Oncology Group Study POG-8651. J Clin Oncol. 2003 Apr 15; 21(8):1574-80. View in: PubMed

Testing of new agents in childhood cancer preclinical models: meeting summary. Clin Cancer Res. 2002 Dec; 8(12):3646-57. View in: PubMed

Synergism between INK4a/ARF inactivation and aberrant HGF/SF signaling in rhabdomyosarcomagenesis. Nat Med. 2002 Nov; 8(11):1276-80. View in: PubMed

Effect of insulin-like growth factor II on protecting myoblast cells against cisplatin-induced apoptosis through p70 S6 kinase pathway. Neoplasia. 2002 Sep-Oct; 4(5):400-8. View in: PubMed

Focus on sarcomas. Cancer Cell. 2002 Sep; 2(3):175-8. View in: PubMed

Treatment of metastatic osteosarcoma with the somatostatin analog OncoLar: significant reduction of insulin-like growth factor-1 serum levels. J Pediatr Hematol Oncol. 2002 Aug-Sep; 24(6):440-6. View in: PubMed

A randomized controlled trial of octreotide pamoate long-acting release and carboplatin versus carboplatin alone in dogs with naturally occurring osteosarcoma: evaluation of insulin-like growth factor suppression and chemotherapy. Clin Cancer Res. 2002 Jul; 8(7):2406-12. View in: PubMed

Pilot trial of tumor-specific peptide vaccination and continuous infusion interleukin-2 in patients with recurrent Ewing sarcoma and alveolar rhabdomyosarcoma: an inter-institute NIH study. Med Pediatr Oncol. 2002 Mar; 38(3):158-64. View in: PubMed

Seven BMPs and all their receptors are simultaneously expressed in osteosarcoma cells. Int J Oncol. 2002 Jan; 20(1):143-7. View in: PubMed

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