Effect of the Butyrate Prodrug Pivaloyloxymethyl Butyrate (AN9) on a Mouse Model for Spinal Muscular Atrophy

Journal of Neuromuscular Diseases

Volumn 3, Issue 4, 2016, Pages 511-515

  Edwards, Jonathan D ^a   Butchbach, Matthew E R ^a,b,c,d  

^a THE OHIO STATE UNIVERSITY WEXNER MEDICAL CENTER   (United States)

^b NEMOURS ALFRED I DUPONT HOSPITAL FOR CHILDREN   (United States)

^c THOMAS JEFFERSON UNIVERSITY   (United States)

^d UNIVERSITY OF DELAWARE   (United States)

Author keywords

AN9; Motor neuron disease; neonatal mouse; preclinical drug trial; spinal muscular atrophy

Indexed keywords

INTRALIPID; PIVALOYLOXYMETHYL BUTYRATE; SURVIVAL MOTOR NEURON PROTEIN; BUTYRIC ACID DERIVATIVE; PIVALYLOXYMETHYL BUTYRATE; PRODRUG;

ANIMAL MODEL; ANTINEOPLASTIC ACTIVITY; ARTICLE; CONTROLLED STUDY; DRUG METABOLISM; FEMALE; FOREBRAIN; GROWTH RATE; HIGH PERFORMANCE LIQUID CHROMATOGRAPHY; LIFESPAN; MALE; MOUSE; MUSCLE FUNCTION; NEWBORN; NONHUMAN; PHENOTYPE; PRIORITY JOURNAL; SPINAL MUSCULAR ATROPHY; ANIMAL; BODY WEIGHT; DISEASE MODEL; DRUG EFFECTS; MORTALITY; PATHOPHYSIOLOGY; SURVIVAL RATE;

ANIMALS; BODY WEIGHT; BUTYRATES; DISEASE MODELS, ANIMAL; MICE; MUSCULAR ATROPHY, SPINAL; PHENOTYPE; PRODRUGS; SURVIVAL RATE;

EID: 85035789881     PISSN: 22143599     EISSN: 22143602     Source Type: Journal    
DOI: 10.3233/JND-160187     Document Type: Article

References (26)

1. Crawford TO, Pardo CA. The neurobiology of childhood spinal muscular atrophy. Neurobiol Dis. 1996;3:97-110.
2. Pearn J. Incidence, prevalence and gene frequency studies of chronic childhood spinal muscular atrophy. JMed Genet. 1978;15:409-13.
3. Cuscó I, Barceló MJ, Soler C, et al. Prenatal diagnosis for risk of spinal muscular atrophy. Br J Obstet Gynaecol. 2002;109:1244-9.
4. Lefebvre S, Bürglen L, Reboullet S, et al. Identification and characterization of a spinal muscular atrophy-determining gene. Cell. 1995;80:155-65.
5. Burghes AHM, Beattie CE. Spinal muscular atrophy: Why do low levels of survival motor neuron protein make motor neurons sick? Nat Rev Neurosci. 2009;10:597-609.
6. Chang JG, Hsieh-Li HM, Jong YJ, et al. Treatment of spinal muscular atrophy by sodium butyrate. Proc Natl Acad Sci U S A. 2001;98:9808-13.
7. Butchbach MER, Lumpkin CJ, Harris AW, et al. Protective effects of butyrate-based compounds on a mouse model for spinal muscular atrophy. Exp Neurol. 2016;279:13-26.
8. Egorin MJ, Yuan ZM, Sentz DL, Plaisance K, Eiseman JL, Plasma pharmacokinetics of butyrate after intravenous administration of sodium butyrate or oral administration of tributyrin or sodium butyrate to mice and rats. Cancer Chemother Pharmacol. 1999;43:445-53.
9. Nudelman A, Ruse M, Aviram A, et al. Novel anticancer prodrugs of butyric acid. 2. J Med Chem. 1992;35:687-94.
10. Aviram A, Rephaeli A, Shaklai M, et al. Effect of the cytostatic butyric acid pro-drug, pivaloyloxymethyl butyrate, on the tumorigenicity of cancer cells. J Cancer Res Clin Oncol. 1997;123:267-71.
11. Batova A, Shao L, Diccianni MB, et al. The histone deacetylase inhibitor AN-9 has selective toxicity to acute leukemia and drug-resistant primary leukemia and cancer cell lines. Blood. 2002;100:3319-24.
12. Entin-Meer M, Yang X, VandenBerg SR, et al. In vivo efficacy of a novel histone deacetylase inhibitor in combination with radiation for the treatment of gliomas. Neuro-Oncology. 2007;9:82-8.
13. Tarasenko N, Nudelman A, Tarasenko I, et al. Histone deacetylase inhibitors: The anticancer, antimetastatic and antiangiogenic activities of AN-7 are superior to those of the clinically tested AN-9 (Pivanex). Clin Exp Metastasis. 2008;25:703-16.
14. Le TT, Pham LT, Butchbach MER, et al. SMN-7, the major product of the centromeric survival motor neuron gene (SMN2), extends survival in mice with spinal muscular atrophy and associates with full-length SMN. Hum Mol Genet. 2005;14:845-57.
15. Butchbach MER, Rose FF Jr, Rhoades S, et al. Effect of diet on the survival and phenotype of a mouse model for spinal muscular atrophy. Biochem Biophys Res Commun. 2010;391:835-40.
16. Narver HL, Kong L, Burnett BG, et al. Sustained improvement of spinal muscular atrophy mice treated with trichostatin A plus nutrition. Ann Neurol. 2008;64:465-70.
17. Butchbach MER, Singh J, GurneyME, Burghes AHM, The effect of diet on the protective action of D156844 observed in spinal muscular atrophy mice. Exp Neurol. 2014;256:1-6.
18. Butchbach MER, Edwards JD, Schussler KR, Burghes AHM. A novel method for oral delivery of compounds to the neonatal SMN-7 model of spinal muscular atrophy. J Neurosci Methods. 2007;161:285-90.
19. Butchbach MER, Edwards JD, Burghes AHM. Abnormal motor phenotype in the SMN-7 mouse model of spinal muscular atrophy. Neurobiol Dis. 2007;27:207-19.
20. Andreassi C, Angelozzi C, Tiziano FD, et al. Phenylbutyrate increases SMN expression in vitro: Relevance for treatment of spinal muscular atrophy. Eur J Hum Genet. 2004;12: 59-65.
21. Boffa LC, Vidali G, Mann RS, Allfrey VG. Suppression of histone deacetylation in vivo and in vitro by sodium butyrate. J Biol Chem. 1978;253:3364-66.
22. Vidali G, Boffa LC, Bradbury EM, Allfrey VG. Butyrate suppression of histone deacetylation leads to accumulation of multiacetylated forms of histones H3 and H4 and increased DNaseI sensitivity of the associated DNA sequences. Proc Natl Acad Sci U S A. 1978;75:2239-43.
23. Candido EPM, Reeves R, Davie JR. Sodium butyrate inhibits histone deacetylation in cultured cells. Cell. 1978;14:105-13.
24. Davis T, Kennedy C, Chiew YE, Clarke CL, deFazio A. Histone deacetylase inhibitors decrease proliferation and modulate cell cycle gene expression in normal mammary epithelial cells. Clin Cancer Res. 2000;6:4334-42.
25. Aviram A, Zimrah Y, Shaklai M, Nudelman A, Rephaeli A. Comparison between the effect of butyric acid and its prodrug pivaloyloxymethylbutyrate on histones hyperacetylation in an HL-60 leukemic cell line. Int J Cancer. 1994;56:906-09.
26. Calder AN, Androphy EJ, Hodgetts KJ. Small molecules in development for the treatment of spinal muscular atrophy. J Med Chem. 2016;in press.