Keck School Faculty

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

A Phase II Trial of Vandetanib in Children and Adults with Succinate Dehydrogenase-Deficient Gastrointestinal Stromal Tumor Clin Cancer Res. 2019 Nov 01; 25(21):6302-6308. . View in PubMed

Insights into pediatric rhabdomyosarcoma research: Challenges and goals Pediatr Blood Cancer. 2019 Oct; 66(10):e27869. . View in PubMed

Glutamine synthetase is necessary for sarcoma adaptation to glutamine deprivation and tumor growth Oncogenesis. 2019 Feb 26; 8(3):20. . View in PubMed

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

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

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

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

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

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

Downregulation of IGFBP2 is associated with resistance to IGF1R therapy in rhabdomyosarcoma Oncogene. 2014 Dec 11; 33(50):5697-705. . 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

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

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

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

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

Global methylation analysis identifies PITX2 as an upstream regulator of the androgen receptor and IGF-I receptor genes in prostate cancer Horm Metab Res. 2012 Jun; 44(7):511-9. . 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

Insulin-like growth factor 1 receptor antibody induces rhabdomyosarcoma cell death via a process involving AKT and Bcl-x(L) Oncogene. 2010 Dec 02; 29(48):6367-77. . 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

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

Beta4 integrin promotes osteosarcoma metastasis and interacts with ezrin Oncogene. 2009 Sep 24; 28(38):3401-11. . 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

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

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

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

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

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

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

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

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

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

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

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

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

Insulin-like growth factor type 1 (IGF-1) and IGF binding protein-3 in patients with Ewing sarcoma family of tumors Cancer. 2001 Dec 01; 92(11):2941-7. . View in PubMed

Insulin and IGF-1 induce different patterns of gene expression in mouse fibroblast NIH-3T3 cells: identification by cDNA microarray analysis Endocrinology. 2001 Nov; 142(11):4969-75. . View in PubMed

Intramedullary Ewing sarcoma of the spinal cord: consequences of molecular diagnosticsCase report. J Neurosurg. 2001 Oct; 95(2 Suppl):270-5. . View in PubMed

Antigenicity of fusion proteins from sarcoma-associated chromosomal translocations Cancer Res. 2001 Sep 15; 61(18):6868-75. . View in PubMed

Immunomagnetic purging of Ewing's sarcoma from blood and bone marrow: quantitation by real-time polymerase chain reaction J Clin Oncol. 2001 Aug 15; 19(16):3649-59. . View in PubMed

A phase I/II dose escalation and activity study of intravenous injections of OCaP1 for subjects with refractory osteosarcoma metastatic to lung Hum Gene Ther. 2001 Aug 10; 12(12):1591-3. . View in PubMed

Insulin-like growth factor 1 (IGF-1)-induced twist expression is involved in the anti-apoptotic effects of the IGF-1 receptor J Biol Chem. 2001 Jul 13; 276(28):26699-707. . View in PubMed

High-dose chemotherapy for rhabdomyosarcoma: where do we go from here J Pediatr Hematol Oncol. 2001 Jun-Jul; 23(5):266-7. . View in PubMed

Inguinal hernia in patients with Ewing sarcoma: a clue to etiology Med Pediatr Oncol. 2000 Mar; 34(3):195-9. . View in PubMed

An orthotopic model of murine osteosarcoma with clonally related variants differing in pulmonary metastatic potential Clin Exp Metastasis. 2000; 18(3):261-71. . View in PubMed

Autocrine Transforming Growth Factor-beta Growth Pathway in Murine Osteosarcoma Cell Lines Associated with Inability to Affect Phosphorylation of Retinoblastoma Protein Sarcoma. 2000; 4(3):93-102. . View in PubMed

Diminished G1 checkpoint after gamma-irradiation and altered cell cycle regulation by insulin-like growth factor II overexpression J Biol Chem. 1999 May 07; 274(19):13118-26. . View in PubMed

Insulin-like growth factor II and PAX3-FKHR cooperate in the oncogenesis of rhabdomyosarcoma Cancer Res. 1998 Oct 01; 58(19):4426-33. . View in PubMed

AP-2 may contribute to IGF-II overexpression in rhabdomyosarcoma Oncogene. 1998 Sep 10; 17(10):1261-70. . View in PubMed

Biallelic expression of all four IGF-II promoters and its association with increased methylation of H19 gene in human brain Brain Res. 1998 May 11; 792(2):283-90. . View in PubMed

Glimpsing the cause of rhabdomyosarcoma Nat Med. 1998 May; 4(5):559-60. . View in PubMed

Expression of a kinase-deficient IGF-I-R suppresses tumorigenicity of rhabdomyosarcoma cells constitutively expressing a wild type IGF-I-R Int J Cancer. 1998 Apr 13; 76(2):223-7. . View in PubMed

Cytogenetics and experimental models Curr Opin Oncol. 1997 Jul; 9(4):342-7. . View in PubMed

Expression of PAX3 in Ewing's sarcoma family of tumors Biochem Mol Med. 1997 Apr; 60(2):121-6. . View in PubMed

Disruption of the MyoD/p21 Pathway in Rhabdomyosarcoma Sarcoma. 1997; 1(3-4):135-41. . View in PubMed

Involvement of IGF-II in human cancer J Endocrinol. 1996 Jun; 149(3):367-72. . View in PubMed

1 alpha, 25-dihydroxy-16-ene-23-yne-26,27-hexafluorocholecalciferol (Ro24-5531) modulation of insulin-like growth factor-binding protein-3 and induction of differentiation and growth arrest in a human osteosarcoma cell line J Clin Endocrinol Metab. 1996 Jan; 81(1):93-9. . View in PubMed

Concordant loss of imprinting of the human insulin-like growth factor II gene promoters in cancer J Biol Chem. 1995 Nov 24; 270(47):27983-6. . View in PubMed

dlk, pG2 and Pref-1 mRNAs encode similar proteins belonging to the EGF-like superfamilyIdentification of polymorphic variants of this RNA. Biochim Biophys Acta. 1995 Apr 04; 1261(2):223-32. . View in PubMed

Insulin-like growth factor II overexpression in myoblasts induces phenotypic changes typical of the malignant phenotype Cell Growth Differ. 1995 Mar; 6(3):263-9. . View in PubMed

Insulin-like growth factors and cancer Ann Intern Med. 1995 Jan 01; 122(1):54-9. . View in PubMed

In vivo treatment with antibody against IGF-1 receptor suppresses growth of human rhabdomyosarcoma and down-regulates p34cdc2 Cancer Res. 1994 Nov 01; 54(21):5531-4. . View in PubMed

Activation of an imprinted allele of the insulin-like growth factor II gene implicated in rhabdomyosarcoma J Clin Invest. 1994 Jul; 94(1):445-8. . View in PubMed

Human osteosarcoma cell lines are dependent on insulin-like growth factor I for in vitro growth Cancer Res. 1994 May 15; 54(10):2803-7. . View in PubMed

Insulin-like growth factors stimulate growth and L-cysteine uptake by the intestinal parasite Giardia lamblia J Biol Chem. 1994 May 06; 269(18):13069-72. . View in PubMed

Diverse roles of insulin-like growth factors in pediatric solid tumors In Vivo. 1994 Jan-Feb; 8(1):81-90. . View in PubMed

Ara-C treatment leads to differentiation and reverses the transformed phenotype in a human rhabdomyosarcoma cell line Exp Cell Res. 1993 Feb; 204(2):210-6. . View in PubMed

IGF-II in the pathogenesis of rhabdomyosarcoma: a prototype of IGFs involvement in human tumorigenesis Adv Exp Med Biol. 1993; 343:327-43. . View in PubMed

Chromogranin-A expression in gastric and colon cancer tissues Int J Cancer. 1992 May 08; 51(2):189-94. . View in PubMed

Frequency and diversity of p53 mutations in childhood rhabdomyosarcoma Cancer Res. 1992 Apr 15; 52(8):2243-7. . View in PubMed

All-trans-retinoic acid inhibits the growth of human rhabdomyosarcoma cell lines Cancer Res. 1991 Sep 15; 51(18):4882-7. . View in PubMed

Insulin-like growth factor II-mediated proliferation of human neuroblastoma J Clin Invest. 1991 Feb; 87(2):648-57. . View in PubMed

Cytotoxic activity of a recombinant fusion protein between insulin-like growth factor I and Pseudomonas exotoxin Cancer Res. 1991 Jan 01; 51(1):174-80. . View in PubMed

Insulin-like growth factor II acts as an autocrine growth and motility factor in human rhabdomyosarcoma tumors Cell Growth Differ. 1990 Jul; 1(7):325-31. . View in PubMed

Expression of chromogranin-A messenger ribonucleic acid in parathyroid tissue from patients with primary hyperparathyroidism J Clin Endocrinol Metab. 1990 Jun; 70(6):1668-73. . View in PubMed

Human neuroblastoma tumor cell lines correspond to the arrested differentiation of chromaffin adrenal medullary neuroblasts Cell Growth Differ. 1990 Apr; 1(4):149-59. . View in PubMed

Autonomous growth of a human neuroblastoma cell line is mediated by insulin-like growth factor II J Clin Invest. 1989 Sep; 84(3):829-39. . View in PubMed

Chromogranin A expression in normal and malignant human tissues J Clin Invest. 1988 Aug; 82(2):686-90. . View in PubMed

Expression of neuroendocrine cell markers L-dopa decarboxylase, chromogranin A, and dense core granules in human tumors of endocrine and nonendocrine origin Cancer Res. 1988 Jul 15; 48(14):4078-82. . View in PubMed

Chromogranin A expression in childhood peripheral neuroectodermal tumors Prog Clin Biol Res. 1988; 271:175-84. . View in PubMed

Improving staff-student relations: effects of a humanistic medicine programme Med Teach. 1980; 2(1):32-9. . View in PubMed

Incidence of drug-related deaths secondary to high-dose methotrexate and citrovorum factor administration Cancer Treat Rep. 1977 Jul; 61(4):745-8. . View in PubMed

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