Keck School Faculty

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

Corrigendum: Positively selected enhancer elements endow osteosarcoma cells with metastatic competence Nat Med. 2018 Apr 10; 24(4):525. . View in PubMed

Positively selected enhancer elements endow osteosarcoma cells with metastatic competence Nat Med. 2018 Feb; 24(2):176-185. . View in PubMed

Matrix Screen Identifies Synergistic Combination of PARP Inhibitors and Nicotinamide Phosphoribosyltransferase (NAMPT) Inhibitors in Ewing Sarcoma Clin Cancer Res. 2017 Dec 01; 23(23):7301-7311. . View in PubMed

A phase I/II trial and pharmacokinetic study of mithramycin in children and adults with refractory Ewing sarcoma and EWS-FLI1 fusion transcript Cancer Chemother Pharmacol. 2017 Sep; 80(3):645-652. . View in PubMed

Surveillance Recommendations for Children with Overgrowth Syndromes and Predisposition to Wilms Tumors and Hepatoblastoma Clin Cancer Res. 2017 Jul 01; 23(13):e115-e122. . View in PubMed

J Clin Oncol. 2017 May 10; 35(14):1561-1569. . 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. 2017 Feb 10; 35(5):523-528. . 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

Surveillance Recommendations for Children with Overgrowth Syndromes and Predisposition to Wilms Tumors and Hepatoblastoma Clin Cancer Res. 2017 07 01; 23(13):e115-e122. . View in PubMed

SARC006: Phase II Trial of Chemotherapy in Sporadic and Neurofibromatosis Type 1 Associated Chemotherapy-Naive Malignant Peripheral Nerve Sheath Tumors Sarcoma. 2017; 2017:8685638. . 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 10 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

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

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 5p1533. 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

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

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

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

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

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 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

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

Hypocalcemia in a patient with osteosarcoma and 22q112 deletion syndrome. J Pediatr Hematol Oncol. 2008 Aug; 30(8):612-7. . 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

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

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

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 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

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 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

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

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

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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