Screening for inhibitors of the SOD1 gene promoter: Pyrimethamine does not reduce SOD1 levels in cell and animal models

Neuroscience Letters

Volumn 482, Issue 3, 2010, Pages 188-192

  Wright, Paul D ^a   Huang, Mickey ^b   Weiss, Alexandra ^a   Matthews, Jonathan ^a   Wightman, Nicholas ^a   Glicksman, Marcie ^b   Brown, Robert H ^a  

^a UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL   (United States)

^b BRIGHAM AND WOMEN S HOSPITAL   (United States)

Author keywords

Amyotrophic lateral sclerosis; Assay; Compound screening; Pyrimethamine; SOD1; Superoxide dismutase

Indexed keywords

4 PHENYLBUTYRIC ACID; ACETYLCYSTEINE; ADENOSINE; CEFTRIAXONE; CELECOXIB; CHLORPROMAZINE; COLCHICINE; CREATINE; CYCLOHEXIMIDE; CYCLOSERINE; CYCLOSPORIN; DESIPRAMINE; FLUOROURACIL; GABAPENTIN; HYDROXYUREA; INDOMETACIN; LITHIUM; LITHIUM CITRATE; MEPACRINE; METHOTREXATE; MINOCYCLINE; PENICILLAMINE; PROGESTERONE; PUROMYCIN; PYRIMETHAMINE; RILUZOLE; SUPEROXIDE DISMUTASE; TAMOXIFEN; UNINDEXED DRUG; VINCRISTINE;

AMYOTROPHIC LATERAL SCLEROSIS; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; BRAIN; CELL STRAIN; CLINICAL TRIAL; CONTROLLED STUDY; CYTOTOXICITY; DRUG EFFECT; GENE EXPRESSION; HELA CELL; HUMAN; HUMAN CELL; LIVER HOMOGENATE; MOUSE; NONHUMAN; PRIORITY JOURNAL; PROMOTER REGION; SPINAL CORD; WILD TYPE;

AMYOTROPHIC LATERAL SCLEROSIS; ANIMALS; BLOTTING, WESTERN; DRUG EVALUATION, PRECLINICAL; FOLIC ACID ANTAGONISTS; GENE EXPRESSION; HELA CELLS; HUMANS; MICE; MICE, INBRED C57BL; PC12 CELLS; PROMOTER REGIONS, GENETIC; PYRIMETHAMINE; RATS; SUPEROXIDE DISMUTASE;

EID: 77955917878     PISSN: 03043940     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.neulet.2010.07.020     Document Type: Article

References (31)

1. Bechhofer D.H. Triple post-transcriptional control. Mol. Microbiol. 1990, 4(9):1419-1423.
2. Boillee S., Yamanaka K., Lobsiger C.S., Copeland N.G., Jenkins N.A., Kassiotis G., Kollias G., Cleveland D.W. Onset and progression in inherited ALS determined by motor neurons and microglia. Science 2006, 312(5778):1389-1392.
3. Broom W.J., Auwarter K.E., Ni J., Russel D.E., Yeh L.A., Maxwell M.M., Glicksman M., Kazantsev A.G., Brown R.H. Two approaches to drug discovery in SOD1-mediated ALS. J. Biomol. Screen. 2006, 11(7):729-735.
4. Cavallito J.C., Nichol C.A., Brenckman W.D., Deangelis R.L., Stickney D.R., Simmons W.S., Sigel C.W. Lipid-soluble inhibitors of dihydrofolate reductase. I. Kinetics, tissue distribution, and extent of metabolism of pyrimethamine, metoprine, and etoprine in the rat, dog, and man. Drug Metab. Dispos. 1978, 6(3):329-337.
5. Dal Canto M.C., Gurney M.E. Neuropathological changes in two lines of mice carrying a transgene for mutant human Cu, Zn SOD, and in mice overexpressing wild type human SOD: a model of familial amyotrophic lateral sclerosis (FALS). Brain Res. 1995, 676(1):25-40.
6. Ferone R., Burchall J.J., Hitchings G.H. Plasmodium berghei dihydrofolate reductase. Isolation, properties, and inhibition by antifolates. Mol. Pharmacol. 1969, 5(1):49-59.
7. Gong Y.H., Parsadanian A.S., Andreeva A., Snider W.D., Elliott J.L. Restricted expression of G86R Cu/Zn superoxide dismutase in astrocytes results in astrocytosis but does not cause motoneuron degeneration. J. Neurosci. 2000, 20(2):660-665.
8. Greenway M.J., Alexander M.D., Ennis S., Traynor B.J., Corr B., Frost E., Green A., Hardiman O. A novel candidate region for ALS on chromosome 14q11.2. Neurology 2004, 63(10):1936-1938.
9. http://alsod.iop.kcl.ac.uk/.
10. Kwiatkowski T.J., Bosco D.A., Leclerc A.L., Tamrazian E., Vanderburg C.R., Russ C., Davis A., Gilchrist J., Kasarskis E.J., Munsat T., Valdmanis P., Rouleau G.A., Hosler B.A., Cortelli P., de Jong P.J., Yoshinaga Y., Haines J.L., Pericak-Vance M.A., Yan J., Ticozzi N., Siddique T., McKenna-Yasek D., Sapp P.C., Horvitz H.R., Landers J.E., Brown R.H. Mutations in the FUS/TLS gene on chromosome 16 cause familial amyotrophic lateral sclerosis. Science 2009, 323(5918):1205-1208.
11. Lange D. Abstract C46: pyrimethamine as a therapy for SOD1 associated FALS: Early findings. Amyotroph. Lateral Scler. 2008, 9(Suppl. 1):45-47.
12. Lino M.M., Schneider C., Caroni P. Accumulation of SOD1 mutants in postnatal motoneurons does not cause motoneuron pathology or motoneuron disease. J. Neurosci. 2002, 22(12):4825-4832.
13. Miller R.G., Mitchell J.D., Lyon M., Moore D.H. Riluzole for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND). Amyotroph. Lateral Scler. Other Motor Neuron Disord. 2003, 4(3):191-206.
14. Nagai M., Aoki M., Miyoshi I., Kato M., Pasinelli P., Kasai N., Brown R.H., Itoyama Y. Rats expressing human cytosolic copper-zinc superoxide dismutase transgenes with amyotrophic lateral sclerosis: associated mutations develop motor neuron disease. J. Neurosci. 2001, 21(23):9246-9254.
15. Pajouhesh H., Lenz G.R. Medicinal chemical properties of successful central nervous system drugs. NeuroRx 2005, 2(4):541-553.
16. Pierdominici M., Giammarioli A.M., Gambardella L., De Felice M., Quinti I., Iacobini M., Carbonari M., Malorni W., Giovannetti A. Pyrimethamine (2,4-diamino-5-p-chlorophenyl-6-ethylpyrimidine) induces apoptosis of freshly isolated human T lymphocytes, bypassing CD95/Fas molecule but involving its intrinsic pathway. J. Pharmacol. Exp. Ther. 2005, 315(3):1046-1057.
17. Pramatarova A., Laganiere J., Roussel J., Brisebois K., Rouleau G.A. Neuron-specific expression of mutant superoxide dismutase 1 in transgenic mice does not lead to motor impairment. J. Neurosci. 2001, 21(10):3369-3374.
18. Ralph G.S., Radcliffe P.A., Day D.M., Carthy J.M., Leroux M.A., Lee D.C., Wong L.F., Bilsland L.G., Greensmith L., Kingsman S.M., Mitrophanous K.A., Mazarakis N.D., Azzouz M. Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model. Nat. Med. 2005, 11(4):429-433.
19. Raoul C., Abbas-Terki T., Bensadoun J.C., Guillot S., Haase G., Szulc J., Henderson C.E., Aebischer P. Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS. Nat. Med. 2005, 11(4):423-428.
20. Reaume A.G., Elliott J.L., Hoffman E.K., Kowall N.W., Ferrante R.J., Siwek D.F., Wilcox H.M., Flood D.G., Beal M.F., Brown R.H., Scott R.W., Snider W.D. Motor neurons in Cu/Zn superoxide dismutase-deficient mice develop normally but exhibit enhanced cell death after axonal injury. Nat. Genet. 1996, 13(1):43-47.
21. Rosen D.R. Mutations in Cu/Zn superoxide dismutase gene are associated with familial amyotrophic lateral sclerosis. Nature 1993, 364(6435):p362.
22. Smith R.A., Miller T.M., Yamanaka K., Monia B.P., Condon T.P., Hung G., Lobsiger C.S., Ward C.M., McAlonis-Downes M., Wei H., Wancewicz E.V., Bennett C.F., Cleveland D.W. Antisense oligonucleotide therapy for neurodegenerative disease. J. Clin. Invest. 2006, 116(8):2290-2296.
23. Sreedharan J., Blair I.P., Tripathi V.B., Hu X., Vance C., Rogelj B., Ackerley S., Durnall J.C., Williams K.L., Buratti E., Baralle F., de Belleroche J., Mitchell J.D., Leigh P.N., Al-Chalabi A., Miller C.C., Nicholson G., Shaw C.E. TDP-43 mutations in familial and sporadic amyotrophic lateral sclerosis. Science 2008, 319(5870):1668-1672.
24. Vance C., Rogelj B., Hortobagyi T., De Vos K.J., Nishimura A.L., Sreedharan J., Hu X., Smith B., Ruddy D., Wright P., Ganesalingam J., Williams K.L., Tripathi V., Al-Saraj S., Al-Chalabi A., Leigh P.N., Blair I.P., Nicholson G., de Belleroche J., Gallo J.M., Miller C.C., Shaw C.E. Mutations in FUS, an RNA processing protein, cause familial amyotrophic lateral sclerosis type 6. Science 2009, 323(5918):1208-1211.
25. Wang H., Ghosh A., Baigude H., Yang C.S., Qiu L., Xia X., Zhou H., Rana T.M., Xu Z. Therapeutic gene silencing delivered by a chemically modified small interfering RNA against mutant SOD1 slows amyotrophic lateral sclerosis progression. J. Biol. Chem. 2008, 283(23):15845-15852.
26. Weiss L.M., Harris C., Berger M., Tanowitz H.B., Wittner M. Pyrimethamine concentrations in serum and cerebrospinal fluid during treatment of acute Toxoplasma encephalitis in patients with AIDS. J. Infect. Dis. 1988, 157(3):580-583.
27. Welch E.M., Barton E.R., Zhuo J., Tomizawa Y., Friesen W.J., Trifillis P., Paushkin S., Patel M., Trotta C.R., Hwang S., Wilde R.G., Karp G., Takasugi J., Chen G., Jones S., Ren H., Moon Y.C., Corson D., Turpoff A.A., Campbell J.A., Conn M.M., Khan A., Almstead N.G., Hedrick J., Mollin A., Risher N., Weetall M., Yeh S., Branstrom A.A., Colacino J.M., Babiak J., Ju W.D., Hirawat S., Northcutt V.J., Miller L.L., Spatrick P., He F., Kawana M., Feng H., Jacobson A., Peltz S.W., Sweeney H.L. PTC124 targets genetic disorders caused by nonsense mutations. Nature 2007, 447(7140):87-91.
28. Yamamoto A., Lucas J.J., Hen R. Reversal of neuropathology and motor dysfunction in a conditional model of Huntington's disease. Cell 2000, 101(1):57-66.
29. Yamanaka K., Chun S.J., Boillee S., Fujimori-Tonou N., Yamashita H., Gutmann D.H., Takahashi R., Misawa H., Cleveland D.W. Astrocytes as determinants of disease progression in inherited amyotrophic lateral sclerosis. Nat. Neurosci. 2008, 11(3):251-253.
30. Zhong Z., Ilieva H., Hallagan L., Bell R., Singh I., Paquette N., Thiyagarajan M., Deane R., Fernandez J.A., Lane S., Zlokovic A.B., Liu T., Griffin J.H., Chow N., Castellino F.J., Stojanovic K., Cleveland D.W., Zlokovic B.V. Activated protein C therapy slows ALS-like disease in mice by transcriptionally inhibiting SOD1 in motor neurons and microglia cells. J. Clin. Invest. 2009, 119(11):3437-3449.
31. 20060211645, U.S.P.A., 2006.