Treatment option(s) for pulmonary lymphangioleiomyomatosis: Progress and current challenges

American Journal of Respiratory Cell and Molecular Biology

Volumn 46, Issue 5, 2012, Pages 563-565

  Krymskaya, Vera P ^a  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ATORVASTATIN; DOXYCYCLINE; HYDROXYMETHYLGLUTARYL COENZYME A REDUCTASE INHIBITOR; INITIATION FACTOR 4E BINDING PROTEIN 1; MATRIX METALLOPROTEINASE; MELAN A; MELANOCYTE PROTEIN PMEL 17; MELANOMA VACCINE; RAPAMYCIN; SIMVASTATIN; TYROSINASE RELATED PROTEIN 1; TYROSINASE RELATED PROTEIN 2;

CANCER CHEMOTHERAPY; CANCER INHIBITION; CELL SURVIVAL; EDITORIAL; FORCED EXPIRATORY VOLUME; HUMAN; LIPID METABOLISM; LUNG TUMOR; LYMPHANGIOLEIOMYOMATOSIS; NONHUMAN; PROTEIN PHOSPHORYLATION; PULMONARY LYMPHANGIOLEIOMYOMATOSIS; QUALITY OF LIFE;

HUMANS; LUNG NEOPLASMS; LYMPHANGIOLEIOMYOMATOSIS;

EID: 84860543952     PISSN: 10441549     EISSN: None     Source Type: Journal    
DOI: 10.1165/rcmb.2011-0381ED     Document Type: Editorial

References (43)

1. Johnson SR. Lymphangioleiomyomatosis. Eur Respir J 2006;27:1056-1065. (Pubitemid 44911122)
2. Taveira-DaSilva AM, Moss J. Lymphangioleiomyomatosis. Cancer Contr 2006;13:276-285. (Pubitemid 44600939)
3. Juvet SC, McCormack FX, Kwiatkowski DJ, Downey GP. Molecular pathogenesis of lymphangioleiomyomatosis: lessons learned from orphans. Am J Respir Cell Mol Biol 2006;36:398-408.
4. Darling TN, Pacheco-Rodriguez G, Gorio A, Lesma E, Walker C, Moss J. Lymphangioleiomyomatosis and TSC-/- cells. Lymphat Res Biol 2010;8:59-69.
5. Krymskaya VP. Smooth muscle-like cells in lymphangioleiomyomatosis. Proc Am Thorac Soc 2008;5:119-126.
6. Klarquist J, Barfuss A, Kandala S, Reust MJ, Braun RK, Hu J, Dilling DF, McKee MD, Boissy RE, Love RB, et al. Melanoma-associated antigen expression in lymphangioleiomyomatosis renders tumor cells susceptible to cytotoxic T cells. Am J Pathol 2009;175:2463-2472.
7. Dilling DF, Gilbert ER, Picken MM, Eby J, Love RB, Le Poole IC. A current viewpoint of lymphangioleiomyomatosis supporting immunotherapeutic treatment options. Am J Respir Cell Mol Biol 2012;46:1-5.
8. Carsillo T, Astrinidis A, Henske EP. Mutations in the tuberous sclerosis complex gene TSC2 are a cause of sporadic pulmonary lymphangioleiomyomatosis. Proc Natl Acad Sci USA 2000;97:6085-6090. (Pubitemid 30367527)
9. Goncharova EA, Goncharov DA, Eszterhas A, Hunter DS, Glassberg MK, Yeung RS, Walker CL, Noonan D, Kwiatkowski DJ, Chou MM, et al. Tuberin regulates p70 S6 kinase activation and ribosomal protein S6 phosphorylation: a role for the TSC2 tumor suppressor gene in pulmonary lymphangioleiomyomatosis. J Biol Chem 2002;277:30958-30967. (Pubitemid 34970798)
10. McManus EJ, Alessi DR. TSC1-TSC2: A complex tale of PKB-mediated S6K regulation. Nat Cell Biol 2002;4:E214-E216. (Pubitemid 34993697)
11. Zoncu R, Efeyan A, Sabatini DM. Mtor: From growth signal integration to cancer, diabetes and ageing. Nat Rev Mol Cell Biol 2011;12:21-35.
12. Inoki K, Corradetti MN, Guan K-L. Dysregulation of the TSC-mTOR pathway in human disease. Nat Genet 2005;37:19-24. (Pubitemid 40070934)
13. Manning BD, Logsdon MN, Lipovsky AI, Abbott D, Kwiatkowski DJ, Cantley LC. Feedback inhibition of akt signaling limits the growth of tumors lacking TSC2. Genes Dev 2005;19:1773-1778. (Pubitemid 41126916)
14. Guertin DA, Sabatini DM. The pharmacology of mTOR inhibition. Sci Signal 2009;2:1-6.
15. Guertin DA, Sabatini DM. Defining the role of mTOR in cancer. Cancer Cell 2007;12:9-22. (Pubitemid 47001784)
16. Goncharova EA, Goncharov DA, Spaits M, Noonan D, Talovskaya E, Eszterhas A, Krymskaya VP. Abnormal smooth muscle cell growth in lymphangioleiomyomatosis (LAM): role for tumor suppressor TSC2. Am J Respir Cell Mol Biol 2006;34:561-572.
17. Krymskaya VP, Penn RB, Orsini MJ, Scott PH, Plevin RJ, Walker TR, Eszterhas AJ, Amrani Y, Chilvers ER, Panettieri RA. Phosphatidylinositol 3-kinase mediates mitogen-induced human airway smooth muscle cell proliferation. Am J Physiol 1999;277:L65-L78. (Pubitemid 29356752)
18. Goncharova EA, Ammit AJ, Irani C, Carroll RG, Eszterhas AJ, Panettieri RA, Krymskaya VP. PI3K is required for proliferation and migration of human pulmonary vascular smooth muscle cells. Am J Physiol 2002;283:L354-L363. (Pubitemid 34803992)
19. Kenerson HL, Aicher LD, True LD, Yeung RS. Activated mammalian target of rapamycin pathway in the pathogenesis of tuberous sclerosis complex renal tumors. Cancer Res 2002;62:5645-5650. (Pubitemid 35204715)
20. Lee L, Sudentas P, Donohue B, Asrican K, Worku A, Walker V, Sun Y, Scgmidt K, Albert MS, El-Hashemite N, et al. Efficacy of a rapamycin analog (CCI-779) and IFN-g in tuberous sclerosis mouse models. Genes Chromosomes Cancer 2005;42:213-227. (Pubitemid 40171186)
21. Krymskaya VP, Goncharova EA. PI3K/mTORC1 activation in hamartoma syndromes: therapeutic prospects. Cell Cycle 2009;8:403-413.
22. McCormack FX, Inoue Y, Moss J, Singer LG, Strange C, Nakata K, Barker AF, Chapman JT, Brantly ML, Stocks JM, et al. Efficacy and safety of sirolimus in lymphangioleiomyomatosis. N Engl J Med 2011; 364:1595-1606.
23. Taveira-DaSilva AM, Hedin C, Stylianou MP, Travis WD, Matsui K, Ferrans VJ, Moss J. Reversible airflow obstruction, proliferation of abnormal smooth muscle cells, and impairment of gas exchange as predictors of outcome of lymphangioleiomyomatosis. Am J Respir Crit Care Med 2001;164:1072-1076. (Pubitemid 32971661)
24. Taveira-DaSilva AM, Stylianou MP, Hedin CJ, Hathaway O, Moss J. Decline in lung function in patients with lymphangioleiomyomatosis treated with or without progesterone. Chest 2004;126:1867-1874. (Pubitemid 39643992)
25. Goncharova E, Goncharov D, Noonan D, Krymskaya VP. TSC2 modulates actin cytoskeleton and focal adhesion through TSC1-binding domain and the Rac1 gtpase. J Cell Biol 2004;167:1171-1182. (Pubitemid 40066631)
26. Goncharova EA, Goncharov DA, Lim PN, Noonan D, Krymskaya VP. Modulation of cell migration and invasiveness by tumor suppressor TSC2 in lymphangioleiomyomatosis. Am J Respir Cell Mol Biol 2006;34:473-480.
27. Goncharova EA, Goncharov DA, Li H, Pimtong W, Lu S, Khavin I, Krymskaya VP. mTORC2 is required for proliferation and survival of TSC2-null cells. Mol Cell Biol 2011;31:2484-2498.
28. Demierre M-F, Higgins PDR, Gruber SB, Hawk E, Lippman SM. Statins and cancer prevention. Nat Rev Cancer 2005;5:930-942. (Pubitemid 41766782)
29. Lee N, Woodrum C, Nobil A, Rauktys A, Messina M, Dabora S. Rapamycin weekly maintenance dosing and the potential efficacy of combination sorafenib plus rapamycin but not atorvastatin or doxycycline in tuberous sclerosis preclinical models. BMC Pharmacol 2009;9:8.
30. Finlay G, Malhowski AJ, Polizzi K, Malinowska-Kolodziej I, Kwiatkowski DJ. Renal and liver tumors in TSC2(+/-) mice, a model of tuberous sclerosis complex, do not respond to treatment with atorvastatin, a 3-hydroxy-3- methylglutaryl coenzyme a reductase inhibitor. Mol Cancer Ther 2009;8:1799-1807.
31. Howe SR, Gottardis MM, Everitt JI, Goldsworthy TL, Wolf DC, Walker CL. Rodent model of reproductive tract leiomyomata: establishment and characterization of tumor-derived cell lines. Am J Pathol 1995;146:1568-1579.
32. El-Chemaly S, Taveira-DaSilva A, Stylianou MP, Moss J. Statins in lymphangioleiomyomatosis: a word of caution.Eur Respir J 2009;34:513-514.
33. Jiang X, Kenerson HL, Yeung RS. Glucose deprivation in tuberous sclerosis complex-related tumors. Cell Biosci 2011;1:34.
34. Parkhitko A, Myachina F, Morrison TA, Hindi KM, Auricchio N, Karbowniczek M, Wu JJ, Finkel T, Kwiatkowski DJ, Yu JJ, et al. Tumorigenesis in tuberous sclerosis complex is autophagy and p62/ sequestosome 1 (sqstm1)-dependent. Proc Natl Acad Sci USA 2011; 108:12455-12460.
35. Kwiatkowski DJ. Animal models of lymphangioleiomyomatosis (LAM) and tuberous sclerosis complex (TSC). Lymphat Res Biol 2010;8:51-57.
36. Kobayashi E, Minowa O, Kuno J, Mitani H, Hino O, Noda T. Renal carcinogenesis, hepatic hemangiomatosis, and embryonic lethality caused by a germ-line TSC2 mutation in mice. Cancer Res 1999;59:1206-1211. (Pubitemid 29136248)
37. Onda H, Lueck A, Marks PW, Warren HB, Kwiatkowski DJ. TSC2+/- mice develop tumors in multiple sites that express gelsolin and are influenced by genetic background. J Clin Invest 1999;104:687-695. (Pubitemid 29536354)
38. Wilson C, Idziaszczyk S, Parry L, Guy C, Griffiths DFR, Lazda E, Bayne RAL, Smith AJH, Sampson JR, Cheadle JP. A mouse model of tuberous sclerosis 1 showing background specific early post-natal mortality and metastatic renal cell carcinoma. Hum Mol Genet 2005;14:1839-1850. (Pubitemid 41418055)
39. Eker R, Mossige J, Johannessen JV, Aars H. Hereditary renal adenomas and adenocarcinomas in rats. Diagn Histopathol 1981;4:99-110. (Pubitemid 11191589)
40. Everitt J, Wolf D, Howe S, Goldsworthy T, Walker C. Rodent model of reproductive tract leiomyomata: clinical and pathological features. Am J Pathol 1995;146:1556-1567.
41. Yu JJ, Robb VA, Morrison TA, Ariazi EA, Karbowniczek M, Astrinidis A, Wang C, Hernandez-Cuebas L, Seeholzer LF, Nicolas E, et al. Estrogen promotes the survival and pulmonary metastasis of tuberin-null cells. Proc Natl Acad Sci USA 2009;106:2635-2640.
42. Zhe X, Schuger L. Combined smooth muscle and melanocytic differentiation in lymphangioleiomyomatosis. J Histochem Cytochem 2004;52:1537-1542. (Pubitemid 39603253)
43. Schwartzentruber DJ, Lawson DH, Richards JM, Conry RM, Miller DM, Treisman J, Gailani F, Riley L, Conlon K, Pockaj B, et al. Gp100 peptide vaccine and interleukin-2 in patients with advanced melanoma. N Engl J Med 2011;364:2119-2127.