Inhibiting the hedgehog pathway in patients with the basal-cell nevus syndrome

New England Journal of Medicine

Volumn 366, Issue 23, 2012, Pages 2180-2188

  Tang, Jean Y ^a,b   Mackay Wiggan, Julian M ^e   Aszterbaum, Michelle ^c   Yauch, Robert L ^d   Lindgren, Joselyn ^a   Chang, Kris ^a   Coppola, Carol ^e   Chanana, Anita M ^a   Marji, Jackleen ^e   Bickers, David R ^e   Epstein, Ervin H ^a  

^a CHILDREN S HOSPITAL OAKLAND RESEARCH INSTITUTE   (United States)

^b STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

^c UNIVERSITY OF CALIFORNIA   (United States)

^d GENENTECH INC   (United States)

^e NewYork Presbyterian Hospital   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

PLACEBO; SONIC HEDGEHOG PROTEIN; VISMODEGIB;

ADULT; ANTINEOPLASTIC ACTIVITY; APOPTOSIS; ARTICLE; BASAL CELL CARCINOMA; BASAL CELL NEVUS SYNDROME; CANCER INCIDENCE; CANCER REGRESSION; CANCER SIZE; CANCER SURGERY; CELL PROLIFERATION; CLINICAL ARTICLE; CONTROLLED STUDY; DOUBLE BLIND PROCEDURE; DRUG EFFICACY; DRUG SAFETY; DRUG WITHDRAWAL; DYSGEUSIA; FEMALE; GENE EXPRESSION; HAIR LOSS; HUMAN; HUMAN TISSUE; IN VITRO STUDY; MALE; MUSCLE CRAMP; OUTCOME ASSESSMENT; PLASMA CONCENTRATION-TIME CURVE; PRIORITY JOURNAL; PROTEIN TARGETING; RANDOMIZED CONTROLLED TRIAL; RISK REDUCTION; SIDE EFFECT; SIGNAL TRANSDUCTION; TASTE DISORDER; TREATMENT DURATION; TREATMENT RESPONSE; WEIGHT REDUCTION;

EID: 84861876867     PISSN: 00284793     EISSN: 15334406     Source Type: Journal    
DOI: 10.1056/NEJMoa1113538     Document Type: Article

References (25)

1. Miller DL, Weinstock MA. Nonmelanoma skin cancer in the United States: incidence. J Am Acad Dermatol 1994;30:774-8. (Pubitemid 24157120)
2. Gorlin RJ. Nevoid basal cell carcinoma syndrome. Medicine (Baltimore) 1987;66:98-113. (Pubitemid 17059640)
3. Johnson RL, Rothman AL, Xie J, et al. Human homolog of patched, a candidate gene for the basal cell nevus syndrome. Science 1996;272:1668-71. (Pubitemid 26200029)
4. Hahn H, Wicking C, Zaphiropoulous PG, et al. Mutations of the human homolog of Drosophila patched in the nevoid basal cell carcinoma syndrome. Cell 1996;85:841-51. (Pubitemid 26192135)
5. Epstein EH. Basal cell carcinomas: attack of the hedgehog. Nat Rev Cancer 2008;8:743-54.
6. Gailani MR, Ståhle-Bäckdahl M, Leffell DJ, et al. The role of the human homologue of Drosophila patched in sporadic basal cell carcinomas. Nat Genet 1996;14:78-81. (Pubitemid 26301550)
7. Aszterbaum M, Rothman A, Johnson RL, et al. Identification of mutations in the human PATCHED gene in sporadic basal cell carcinomas and in patients with the basal cell nevus syndrome. J Invest Dermatol 1998;110:885-8. (Pubitemid 28237693)
8. Ling G, Ahmadian A, Persson A, et al. PATCHED and p53 gene alterations in sporadic and hereditary basal cell cancer. Oncogene 2001;20:7770-8. (Pubitemid 33121950)
9. Von Hoff DD, LoRusso PM, Rudin CM, et al. Inhibition of the hedgehog pathway in advanced basal-cell carcinoma. N Engl J Med 2009;361:1164-72.
10. Kimonis VE, Goldstein AM, Pastakia B, et al. Clinical manifestations in 105 persons with nevoid basal cell carcinoma syndrome. Am J Med Genet 1997;69:299-308. (Pubitemid 27154674)
11. McCullagh P, Nelder JA. Generalized linear models. 2nd ed. London: Chapman and Hall, 1989.
12. Sekulic A, Migden MR, Oro AE, et al. Efficacy and safety of vismodegib in advanced basal-cell carcinoma. N Engl J Med 2012;366:2171-9.
13. Ahnert JR, Baselga J, Tawbi HA, et al. A phase I dose-escalation study of LDE225, a smoothened (Smo) antagonist, in patients with advanced solid tumors. Presented at the 2010 annual meeting of the American Society of Clinical Oncology, Chicago, June 4-8, 2010. abstract.
14. Skvara H, Kalthoff F, Meingassner JG, et al. Topical treatment of basal cell carcinomas in nevoid basal cell carcinoma syndrome with a Smoothened inhibitor. J Invest Dermatol 2011;131:1735-44.
15. Xie J, Murone M, Luoh SM, et al. Activating Smoothened mutations in sporadic basal-cell carcinoma. Nature 1998;391:90-2. (Pubitemid 28079221)
16. Reifenberger J, Wolter M, Weber RG, et al. Missense mutations in SMOH in sporadic basal cell carcinomas of the skin and primitive neuroectodermal tumors of the central nervous system. Cancer Res 1998;58:1798-803. (Pubitemid 28217449)
17. Yauch RL, Dijkgraaf GJP, Alicke B, et al. Smoothened mutation confers resistance to a hedgehog pathway inhibitor in medulloblastoma. Science 2009;326:572-4.
18. Chen JK, Taipale J, Young KE, Maiti T, Beachy PA. Small molecule modulation of Smoothened activity. Proc Natl Acad Sci U S A 2002;99:14071-6. (Pubitemid 35257626)
19. Blackburn EH. Cancer interception. Cancer Prev Res (Phila) 2011;4:787-92.
20. Engelman JA, Zejnullahu K, Mitsudomi T, et al. MET amplification leads to gefitinib resistance in lung cancer by activating ERBB3 signaling. Science 2007;316:1039-43. (Pubitemid 46799492)
21. Gorre ME, Mohammed M, Ellwood K, et al. Clinical resistance to STI-571 cancer therapy caused by BCR-ABL gene mutation or amplification. Science 2001;293:876-80. (Pubitemid 32743979)
22. Davari P, Hebert JL, Albertson DG, et al. Loss of Blm enhances basal cell carcinoma and rhabdomyosarcoma tumorigenesis in Ptch1+/- mice. Carcinogenesis 2010;31:968-73.
23. Takezaki T, Hide T, Takanaga H, Nakamura H, Kuratsu J, Kondo T. Essential role of the Hedgehog signaling pathway in human glioma-initiating cells. Cancer Sci 2011;102:1306-12.
24. Zhao C, Chen A, Jamieson CH, et al. Hedgehog signalling is essential for maintenance of cancer stem cells in myeloid leukaemia. Nature 2009;458:776-9. [Erratum, Nature 2009;460:652.]
25. Hutchin ME, Kariapper MST, Grachtchouk M, et al. Sustained Hedgehog signaling is required for basal cell carcinoma proliferation and survival: conditional skin tumorigenesis recapitulates the hair growth cycle. Genes Dev 2005;19:214-23. (Pubitemid 40111117)