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


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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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