New drugs for children and adolescents with cancer: The need for novel development pathways

The Lancet Oncology

Volumn 14, Issue 3, 2013, Pages

  Vassal, Gilles ^a   Zwaan, C Michel ^b   Ashley, David ^c   Le Deley, Marie Cecile ^a   Hargrave, Darren ^d   Blanc, Patricia ^e   Adamson, Peter C ^f  

^a INSTITUT GUSTAVE ROUSSY   (France)

^b SOPHIA CHILDREN S HOSPITAL   (Netherlands)

^c DEAKIN UNIVERSITY   (Australia)

^d GREAT ORMOND STREET HOSPITAL   (United Kingdom)

^e Imagine for Margo   (France)

^f CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTINEOPLASTIC AGENT; BLINATUMOMAB; BRENTUXIMAB VEDOTIN; CRIZOTINIB; CYTOTOXIC AGENT; DABRAFENIB; DALOTUZUMAB; DASATINIB; EVEROLIMUS; FIGITUMUMAB; GANITUMAB; IMATINIB; MIDOSTAURIN; NICOTINAMIDE ADENINE DINUCLEOTIDE ADENOSINE DIPHOSPHATE RIBOSYLTRANSFERASE INHIBITOR; R 1507; RITUXIMAB; SORAFENIB; UNCLASSIFIED DRUG; VEMURAFENIB; VISMODEGIB;

ACUTE GRANULOCYTIC LEUKEMIA; ACUTE LYMPHOBLASTIC LEUKEMIA; ADOLESCENT; BASAL CELL CARCINOMA; BREAST CANCER; CANCER MORTALITY; CANCER SURVIVOR; CHILD; CHILD HEALTH CARE; CHILDHOOD CANCER; CHRONIC MYELOID LEUKEMIA; CLINICAL RESEARCH; COOPERATION; DOSE CALCULATION; DRUG CONTROL; DRUG DOSE ESCALATION; DRUG EFFICACY; DRUG INDUSTRY; DRUG RESEARCH; DRUG SAFETY; DRUG TARGETING; EUROPE; EWING SARCOMA; GOVERNMENT REGULATION; HEALTH CARE ACCESS; HEALTH ECONOMICS; HODGKIN DISEASE; HUMAN; KIDNEY CANCER; KIDNEY CARCINOMA; LARGE CELL LYMPHOMA; LIVER CELL CARCINOMA; LONG TERM CARE; LUNG CANCER; LUNG NON SMALL CELL CANCER; MALIGNANT NEOPLASTIC DISEASE; MEDULLOBLASTOMA; MELANOMA; NEPHROBLASTOMA; NEUROBLASTOMA; NONHODGKIN LYMPHOMA; PANCREATIC NEUROENDOCRINE TUMOR; PATIENT SELECTION; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PREDICTION; PRIORITY JOURNAL; PROSTATE CANCER; PUBLIC HEALTH; PUBLIC-PRIVATE PARTNERSHIP; REVIEW; STUDY DESIGN; SUBEPENDYMAL GIANT CELL ASTROCYTOMA; SURVIVAL RATE; TUBEROUS SCLEROSIS; TUMOR BIOPSY; UNITED STATES;

ADOLESCENT; CHILD; CLINICAL TRIALS AS TOPIC; DEVELOPED COUNTRIES; DRUG INDUSTRY; EUROPE; GOVERNMENT; HUMANS; NEOPLASMS; PUBLIC-PRIVATE SECTOR PARTNERSHIPS; RESEARCH; UNITED STATES;

EID: 84875246025     PISSN: 14702045     EISSN: 14745488     Source Type: Journal    
DOI: 10.1016/S1470-2045(13)70013-5     Document Type: Review

References (74)

1. Cancer incidence and survival among children and adolescents: United States SEER program 1975-1995 1999, National Cancer Institute SEER Program, National Cancer Institute, Bethesda.
2. Figlin R, Sternberg C, Wood CG Novel agents and approaches for advanced renal cell carcinoma. J Urol 2012, 188:707-715.
3. Flaherty KT, Puzanov I, Kim KB, et al. Inhibition of mutated, activated BRAF in metastatic melanoma. N Engl J Med 2010, 363:809-819.
4. Kwak EL, Bang YJ, Camidge DR, et al. Anaplastic lymphoma kinase inhibition in non-small-cell lung cancer. N Engl J Med 2010, 363:1693-1703.
5. Druker BJ, Sawyers CL, Kantarjian H, et al. Activity of a specific inhibitor of the BCR-ABL tyrosine kinase in the blast crisis of chronic myeloid leukemia and acute lymphoblastic leukemia with the Philadelphia chromosome. N Engl J Med 2001, 344:1038-1042.
6. Jones DT, Jäger N, Kool M, et al. Dissecting the genomic complexity underlying medulloblastoma. Nature 2012, 488:100-105.
7. Making better drugs for children with cancer 2005, Committee on shortening the time line for new cancer treatments, National Academies Press, Washington DC. P.C. Adamson, S.L. Weiner, J.V. Simone, H. Gelband (Eds.).
8. Smith M, Bernstein M, Bleyer WA, et al. Conduct of phase I trials in children with cancer. J Clin Oncol 1998, 16:966-978.
9. Prahallad A, Sun C, Huang S, et al. Unresponsiveness of colon cancer to BRAF(V600E) inhibition through feedback activation of EGFR. Nature 2012, 483:100-103.
10. Skolnik JM, Barrett JS, Jayaraman B, Patel D, Adamson PC Shortening the timeline of pediatric phase I trials: the rolling six design. J Clin Oncol 2008, 26:190-195.
11. Mossé YP, Lipsitz E, Fox E, et al. Pediatric phase I trial and pharmacokinetic study of MLN8237, an investigational oral selective small-molecule inhibitor of aurora kinase A: a Children's Oncology Group phase I consortium study. Clin Cancer Res 2012, 18:6058-6064.
12. Mosse YP, Balis FM, Lim MS, et al. Efficacy of crizotinib in children with relapsed/refractory ALK-driven tumors including anaplastic large cell lymphoma and neuroblastoma: a Children's Oncology Group phase I consortium study. Proc Am Soc Clin Oncol 2012, 30(suppl). abstr 9500.
13. Mauguen A, Le Deley MC, Zohar S Dose-finding approach for dose escalation with overdose control considering incomplete observations. Stat Med 2011, 30:1584-1594.
14. Doussau A, Asselain B, Le Deley MC, et al. Dose-finding designs in pediatric phase I clinical trials: comparison by simulations in a realistic timeline framework. Contemp Clin Trials 2012, 33:657-665.
15. Ezzalfani M, Zohar S, Qin R, Mandrekar, Le Deley MC Dose-finding designs using a novel quasi-continuous endpoint for multiple toxicities. Stat Med 2013, published online Jan 21. 10.1002/sim.5737.
16. Zwaan CM, Rizzari C, Mechinaud F, et al. Dasatinib in children and adolescents with relapsed or refractory leukemia: results of the CA180018 phase 1 dose-escalation study of the Innovative Therapies for Children with Cancer (ITCC) consortium. Blood 2010, 116:2265. (abstr).
17. Horton TM, Sposto R, Brown P, et al. Toxicity assessment of molecularly targeted drugs incorporated into multiagent chemotherapy regimens for pediatric acute lymphocytic leukemia (ALL): review from an international consensus conference. Pediatr Blood Cancer 2010, 54:872-878.
18. Roujeau T, Machado G, Garnett MR, et al. Stereotactic biopsy of diffuse pontine lesions in children. J Neurosurg 2007, 107(suppl 1):1-4.
19. Balic M, Williams A, Lin H, Datar R, Cote RJ Circulating tumor cells: from bench to bedside. Annu Rev Med 2013, 64:31-44.
20. Von Hoff DD, LoRusso PM, Rudin CM, et al. Inhibition of the hedgehog pathway in advanced basal-cell carcinoma. N Engl J Med 2009, 361:1164-1172.
21. Stegmeier F, Warmuth M, Sellers WR, Dorsch M Targeted cancer therapies in the twenty-first century: lessons from imatinib. Clin Pharmacol Ther 2010, 87:543-552.
22. Kim SY, Toretsky JA, Scher D, Helman LJ The role of IGF-1R in pediatric malignancies. Oncologist 2009, 14:83-91.
23. Franz DN, Belousova E, Sparagana S, et al. Efficacy and safety of everolimus for subependymal giant cell astrocytomas associated with tuberous sclerosis complex (EXIST-1): a multicentre, randomised, placebo-controlled phase 3 trial. Lancet 2013, 381:125-132.
24. Garnett MJ, Edelman EJ, Heidorn SJ, et al. Systematic identification of genomic markers of drug sensitivity in cancer cells. Nature 2012, 483:570-575.
25. Hauschild A, Grob JJ, Demidov LV, et al. Dabrafenib in BRAF-mutated metastatic melanoma: a multicentre, open-label, phase 3 randomised controlled trial. Lancet 2012, 380:358-365.
26. Tap WD, Demetri G, Barnette P, et al. Phase II study of ganitumab, a fully human anti-type-1 insulin-like growth factor receptor antibody, in patients with metastatic Ewing family tumors or desmoplastic small round cell tumors. J Clin Oncol 2012, 30:1849-1856.
27. Olmos D, Postel-Vinay S, Molife LR, et al. Safety, pharmacokinetics, and preliminary activity of the anti-IGF-1R antibody figitumumab (CP-751,871) in patients with sarcoma and Ewing's sarcoma: a phase 1 expansion cohort study. Lancet Oncol 2010, 11:129-135.
28. Pappo AS, Patel SR, Crowley J, et al. 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, 29:4541-4547.
29. Garnett MJ, Edelman EJ, Heidorn SJ, et al. Systematic identification of genomic markers of drug sensitivity in cancer cells. Nature 2012, 483:570-575.
30. Biondi A, Schrappe M, De Lorenzo P, et al. Imatinib after induction for treatment of children and adolescents with Philadelphia-chromosome-positive acute lymphoblastic leukaemia (EsPhALL): a randomised, open-label, intergroup study. Lancet Oncol 2012, 13:936-945.
31. Kantarjian H, Shah NP, Hochhaus A, et al. Dasatinib versus imatinib in newly diagnosed chronic-phase chronic myeloid leukemia. N Engl J Med 2010, 362:2260-2270.
32. Saglio G, Kim DW, Issaragrisil S, et al. ENESTnd Investigators. Nilotinib versus imatinib for newly diagnosed chronic myeloid leukemia. N Engl J Med 2010, 362:2251-2259.
33. Younes A, Bartlett NL, Leonard JP, et al. Brentuximab vedotin (SGN-35) for relapsed CD30-positive lymphomas. N Engl J Med 2010, 363:1812-1821.
34. Salles G, Seymour JF, Offner F, et al. Rituximab maintenance for 2 years in patients with high tumour burden follicular lymphoma responding to rituximab plus chemotherapy (PRIMA): a phase 3, randomised controlled trial. Lancet 2011, 377:42-51.
35. Fischer T, Stone RM, Deangelo DJ, et al. Phase IIB trial of oral Midostaurin (PKC412), the FMS-like tyrosine kinase 3 receptor (FLT3) and multi-targeted kinase inhibitor, in patients with acute myeloid leukemia and high-risk myelodysplastic syndrome with either wild-type or mutated FLT3. J Clin Oncol 2010, 28:4339-4345.
36. Topp MS, Gökbuget N, Zugmaier G, et al. Long-term follow-up of hematologic relapse-free survival in a phase 2 study of blinatumomab in patients with MRD in B-lineage ALL. Blood 2012, 120:5185-5187.
37. Carpenter EL, Mossé YP Targeting ALK in neuroblastoma-preclinical and clinical advancements. Nat Rev Clin Oncol 2012, 9:391-399.
38. Schindler G, Capper D, Meyer J, et al. Analysis of BRAF V600E mutation in 1,320 nervous system tumors reveals high mutation frequencies in pleomorphic xanthoastrocytoma, ganglioglioma and extra-cerebellar pilocytic astrocytoma. Acta Neuropathol 2011, 121:397-405.
39. Badalian-Very G, Vergilio JA, Degar BA, et al. Recurrent BRAF mutations in Langerhans cell histiocytosis. Blood 2010, 116:1919-1923.
40. Yee D Insulin-like growth factor receptor inhibitors: baby or the bathwater?. J Natl Cancer Inst 2012, 104:975-981.
41. Kim SY, Toretsky JA, Scher D, Helman LJ The role of IGF-1R in pediatric malignancies. Oncologist 2009, 14:83-91.
42. Motzer RJ, Escudier B, Oudard S, et al. Efficacy of everolimus in advanced renal cell carcinoma: a double-blind, randomised, placebo-controlled phase III trial. Lancet 2008, 372:449-456. for the RECORD-1 Study Group.
43. Baselga J, Campone M, Piccart M, et al. Everolimus in postmenopausal hormone-receptor-positive advanced breast cancer. N Engl J Med 2012, 366:520-529.
44. Yao JC, Shah MH, Ito T, et al. Everolimus for advanced pancreatic neuroendocrine tumors. N Engl J Med 2011, 364:514-523. for the RAD001 in Advanced Neuroendocrine Tumors, Third Trial (RADIANT-3) Study Group.
45. Sekulic A, Migden MR, Oro AE, et al. Efficacy and safety of vismodegib in advanced basal-cell carcinoma. N Engl J Med 2012, 366:2171-2179.
46. Rudin CM, Hann CL, Laterra J, et al. Treatment of medulloblastoma with hedgehog pathway inhibitor GDC-0449. N Engl J Med 2009, 361:1173-1178.
47. Escudier B, Eisen T, Stadler WM Sorafenib in advanced clear-cell renal-cell carcinoma. N Engl J Med 2007, 356:125-134. for the TARGET Study Group.
48. Llovet JM, Ricci S, Mazzaferro V, et al. Sorafenib in advanced hepatocellular carcinoma. N Engl J Med 2008, 359:378-390. for the SHARP Investigators Study Group.
49. Widemann BC, Kim A, Fox E, et al. A phase I trial and pharmacokinetic study of sorafenib in children with refractory solid tumors or leukemias: a Children's Oncology Group Phase I Consortium report. Clin Cancer Res 2012, 18:6011-6022.
50. Yu AL, Gilman AL, Ozkaynak MF, et al. Anti-GD2 antibody with GM-CSF, interleukin-2, and isotretinoin for neuroblastoma. N Engl J Med 2010, 363:1324-1334. for the Children's Oncology Group.
51. Zeng Y, Fest S, Kunert R, et al. Anti-neuroblastoma effect of ch14.18 antibody produced in CHO cells is mediated by NK-cells in mice. Mol Immunol 2005, 42:1311-1319.
52. Chesler L, Weiss WA Genetically engineered murine models-contribution to our understanding of the genetics, molecular pathology and therapeutic targeting of neuroblastoma. Semin Cancer Biol 2011, 21:245-255.
53. Missiaglia E, Williamson D, Chisholm J, et al. PAX3/FOXO1 fusion gene status is the key prognostic molecular marker in rhabdomyosarcoma and significantly improves current risk stratification. J Clin Oncol 2012, 30:1670-1677.
54. Toomey EC, Schiffman JD, Lessnick SL Recent advances in the molecular pathogenesis of Ewing's sarcoma. Oncogene 2010, 29:4504-4516.
55. Mano H ALKoma: a cancer subtype with a shared target. Cancer Discov 2012, 2:495-502.
56. Downing JR, Wilson RK, Zhang J, et al. The pediatric cancer genome project. Nat Genet 2012, 44:619-622.
57. Houghton PJ, Morton CL, Tucker C, et al. The pediatric preclinical testing program: description of models and early testing results. Pediatr Blood Cancer 2007, 49:928-940.
58. Chesler L, Weiss WA Genetically engineered murine models-contribution to our understanding of the genetics, molecular pathology and therapeutic targeting of neuroblastoma. Semin Cancer Biol 2011, 21:245-255.
59. Patte C, Auperin A, Gerrard M, et al. Results of the randomized international FAB/LMB96 trial for intermediate risk B-cell non-Hodgkin lymphoma in children and adolescents: it is possible to reduce treatment for the early responding patients. Blood 2007, 109:2773-2780. for the FAB/LMB96 international study committee.
60. Tukenova M, Guibout C, Oberlin O, et al. Role of cancer treatment in long-term overall and cardiovascular mortality after childhood cancer. J Clin Oncol 2010, 28:1308-1315.
61. van der Pal HJ, van Dalen EC, Hauptmann M, et al. Cardiac function in 5-year survivors of childhood cancer: a long-term follow-up study. Arch Intern Med 2010, 170:1247-1255.
62. Brock PR, Knight KR, Freyer DR, et al. Platinum-induced ototoxicity in children: a consensus review on mechanisms, predisposition, and protection, including a new International Society of Pediatric Oncology Boston ototoxicity scale. J Clin Oncol 2012, 30:2408-2417.
63. Overbeek A, van den Berg MH, Kremer LC, et al. A nationwide study on reproductive function, ovarian reserve, and risk of premature menopause in female survivors of childhood cancer: design and methodological challenges. BMC Cancer 2012, 12:363.
64. Tukenova M, Guibout C, Hawkins M, et al. Radiation therapy and late mortality from second sarcoma, carcinoma, and hematological malignancies after a solid cancer in childhood. Int J Radiat Oncol Biol Phys 2011, 80:339-346.
65. Development of new therapeutic drugs and biologics for rare diseases. Rare diseases and orphan products: accelerating research and development 2011, Committee on accelerating rare diseases research and orphan product development, Institute of Medicine, The National Academies Press, Washington DC. M.J. Field, T.F. Boat (Eds.).
66. Goldman M The innovative medicines initiative: a European response to the innovation challenge. Clin Pharmacol Ther 2012, 91:418-425.
67. Zwaan CM, Kearns P, Caron H, et al. The role of the 'innovative therapies for children with cancer' (ITCC) European consortium. Cancer Treat Rev 2010, 36:328-334.
68. Hirschfeld S, Saint-Raymond A Pediatric regulatory initiatives. Handb Exp Pharmacol 2011, 205:245-268.
69. Connor E, Cure P "Creating hope" and other incentives for drug development in children. Sci Transl Med 2011, 3:1-4.
70. Vassal G, Geoerger B, Morland B Is the European pediatric medicine regulation working for children and adolescents with cancer. Clin Cancer Res 2013, published online Jan 17. 10.1158/1078-0432.CCR-12-2551.
71. McBride A, Johnson PE Drug shortages in oncology. Am J Health Syst Pharm 2012, 69:1190.
72. Altshuler JS, Balogh E, Barker AD, et al. Opening up to precompetitive collaboration. Sci Transl Med 2010, 2:1-4.
73. Munos B Can open-source drug R&D repower pharmaceutical innovation?. Clin Pharmacol Ther 2010, 87:534-536.
74. Woodcock J Precompetitive research: a new prescription for drug development?. Clin Pharmacol Ther 2010, 87:521-523.