Phase 2 study of sodium phenylbutyrate in ALS

References

• Mulder D. Clinical limits of amyotrophic lateral sclerosis. Human motor neuron diseases, L Rowland. Raven Press, New York 1982; 15–22
   Google Scholar
• Bensimon G, Lacomblez L, Meininger V. A controlled trial of riluzole in amyotrophic lateral sclerosis. ALS/Riluzole Study Group. New England Journal of Medicine. 1994; 330: 585–91
   PubMed Web of Science ®Google Scholar
• Hadano S, Hand C, Osuga H, Yanagisawa Y, Otomo A, Devon RS, et al. A gene encoding a putative GTPase regulator is mutated in familial amyotrophic lateral sclerosis 2. Nat Genet. 2001; 29: 166–73
   PubMed Web of Science ®Google Scholar
• Chang J, Hsieh-Li H, Jong Y, Wang N, Tsai C, Li H. Treatment of spinal muscular atrophy by sodium butyrate. Proc Natl Acad Sci USA. 2001; 98: 9808–13
   PubMed Web of Science ®Google Scholar
• Gonzalez de Aguilar J, Gordon J, Rene F, Lutz-Bucher B, Gaiddon C, Loeffler JP. Alteration of the Bcl-x/Bax ratio in a transgenic mouse model of amyotrophic lateral sclerosis: evidence for the implication of the p53 signaling pathway. Neurobiology of Disease. 2000; 7: 406–15
   PubMed Web of Science ®Google Scholar
• Malaspina A, Kaushik N, de Belleroche J. Differential expression of 14 genes in amyotrophic lateral sclerosis spinal cord detected using gridded cDNA arrays. Journal of Neurochemistry. 2001; 77: 132–45
   PubMed Web of Science ®Google Scholar
• Yoshihara T, Ishigaki S, Yamamoto M, Liang Y, Niwa J, Takeuchi H, et al. Differential expression of inflammation and apoptosis-related genes in spinal cords of a mutant SOD1 transgenic mouse model of familial amyotrophic lateral sclerosis. J Neurochem. 2002; 80: 158–67
   PubMed Web of Science ®Google Scholar
• Petricoln EF, Ardekani AM, Hitt BA, Levine PJ, Fusaro VA, Steinberg SM, et al. Use of proteomic patterns in serum to identify ovarian cancer. Lancet. 2002; 359: 572–7
   PubMed Web of Science ®Google Scholar
• Dangond F, Hwang D, Camelo S, Pasinelli P, Frosch MP, Stephanopoulos G, et al. Microarray analysis shows distinct changes in the molecular signature of gene expression in both ALS animal models and patients. Physiol Genomics. 2004; 16: 229–39
   PubMed Web of Science ®Google Scholar
• Dasgupta S, Zhou Y, Jana M, Banik NL, Pahan K. Sodium phenylacetate inhibits adoptive transfer of experimental allergic encephalomyelitis in SJL/J mice at multiple steps. Journal of Immunology. 2003; 170: 3874–82
   PubMed Web of Science ®Google Scholar
• Ferrante R, Kubilus J, Lee J, Ryu H, Beesen A, Zucker B, et al. Histone deacetylase inhibition by sodium butyrate chemotherapy ameliorates the neurodegenerative phenotype in Huntington's disease mice. J Neurosci. 2003; 23: 9418–27
   PubMed Web of Science ®Google Scholar
• Ryu H, Smith K, Camelo SI, Carreras I, Lee J, Iglesias AH, et al. Sodium phenylbutyrate prolongs survival and regulates expression of anti-apoptotic genes in transgenic amyotrophic lateral sclerosis mice. Journal of Neurochemistry. 2005; 93: 1087–98
   PubMed Web of Science ®Google Scholar
• Brooks B. El Escorial World Federation of Neurology criteria for the diagnosis of amyotrophic lateral sclerosis. Subcommittee on Motor Neuron Diseases/Amyotrophic Lateral Sclerosis of the World Federation of Neurology Research Group on Neuromuscular Diseases and the El Escorial'Clinical limits of amyotrophic lateral sclerosis'. J Neurol Sci. 1994; 124: 96–107
   PubMed Web of Science ®Google Scholar
• Cedarbaum J, Stambler N, Malta E, Fuller C, Hilt D, Thurmond B, et al. The ALSFRS-R: a revised ALS functional rating scale that incorporated assessments of respiratory function. J Neurol Sci. 1999; 169: 13–21
   PubMed Web of Science ®Google Scholar
• Cudkowicz M, Shefner J, Schoenfeld D, Zhang H, Andreassen KI, Rothstein JD, et al. Trial of celecoxib in amyotrophic lateral sclerosis. Annals of Neurology. 2006; 60: 22–31
   PubMed Web of Science ®Google Scholar
• Hogarth P, Lovrecic L, Krainc D. Sodium phenylbutyrate in Huntington's disease: a dose-finding study. Movement Disorders. 2007; 22: 1962–4
   PubMed Web of Science ®Google Scholar
• Gilbert J, Baker S, Bowling M, Grochow L, Figg WD, Zabelina Y, et al. A phase I dose escalation and bioavailability study of oral sodium phenylbutyrate in patients with refractory solid tumor malignancies. Clin Cancer Res. 2001; 7: 2292–300
   PubMed Web of Science ®Google Scholar
• Phuphanich S. Oral sodium phenylbutyrate in patients with recurrent malignant gliomas: a dose escalation and pharmacologic study. Neuro-Oncology. 2005; 7: 177–82
   PubMed Web of Science ®Google Scholar
• Carducci MA, Gilbert J, Bowling MK, Noe D, Eisenberger MA, Sinibaldi V, et al. A phase I clinical and pharmacological evaluation of sodium phenylbutyrate on an 120-h infusion schedule. Clin Cancer Res. 2001; 7: 3047–55
   PubMed Web of Science ®Google Scholar
• Savitsky K, Bar-Shira A, Gilad S, Rotman G, Ziv Y, Vanagaite L, et al. A single ataxia telangiectasia gene with a product similar to PI-3 kinase. Science. 1995; 268: 1749–53
   PubMed Web of Science ®Google Scholar
• Batshaw M, MacArthur R, Tuchman M. Alternative pathway therapy for urea cycle dis-orders: 20 years later. J Pediatr. 2001; 138: S46–55
   PubMed Web of Science ®Google Scholar
• Dover G, Brusilow S, Charache S. Induction of fetal hemoglobin production in subjects with sickle cell anemia by oral sodium phenylbutyrate. Blood. 1994; 84: 339–43
   PubMed Web of Science ®Google Scholar
• Iversen L, Foster A, Hill R, Iversen SD, Kemp JA, Leesen PD, et al. Neurotoxin-related research: from the laboratory to the clinic. Ann New York Acad Sci. 1992; 648: 207–18
   PubMed Web of Science ®Google Scholar
• Cudkowicz M, Shefner J, Schoenfeld D, Brown RH, Johnson H, Qureshi M, et al. A randomized, placebo-controlled trial of topiramate in amyotrophic lateral sclerosis. Neurology. 2003; 61: 456–64
   PubMed Web of Science ®Google Scholar
• Gordon PH, Moore DH, Gelinas DF, Qualls C, Meister ME, Mendoza M, et al. Placebo-controlled phase I/II studies of minocycline in amyotrophic lateral sclerosis. Neurology. 2004; 62: 1845–7
   PubMed Web of Science ®Google Scholar
• Gordon P, Moore D, Miller R, Florence JM, Verheijde JL, Doorish C, et al. Efficacy of minocycline in patients with amyotrophic lateral sclerosis: a phase III randomized trial. Lancet Neurology. 2007; 6: 1045–53
   PubMed Web of Science ®Google Scholar
• Berg S, Serabe B, Aleksic A, Bomgaars L, McGuffey L, Dauser R, et al. Pharmacokinetics and cerebrospinal fluid penetration of phenylacetate and phenylbutyrate in the non-human primate. Cancer Chemother Pharmacol. 2001; 47: 385–90
   PubMed Web of Science ®Google Scholar