Inhibiting drug efflux transporters improves efficacy of ALS therapeutics

Annals of Clinical and Translational Neurology

Volumn 1, Issue 12, 2014, Pages 996-1005

  Jablonski, Michael R ^a   Markandaiah, Shashirekha S ^a   Jacob, Dena ^a   Meng, Ni J ^a   Li, Ke ^a   Gennaro, Victoria ^a   Lepore, Angelo C ^a   Trotti, Davide ^a   Pasinelli, Piera ^a  

^a THOMAS JEFFERSON UNIVERSITY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADULT; AMYOTROPHIC LATERAL SCLEROSIS; ARTICLE; BIOAVAILABILITY; BLOOD BRAIN BARRIER; BRAIN DISEASE; CENTRAL NERVOUS SYSTEM; CONTROLLED STUDY; DISEASE COURSE; DRUG BIOAVAILABILITY; DRUG EFFLUX TRANSPORTER; EFFUSION; FEMALE; GRIP STRENGTH; IMMUNOFLUORESCENCE; MALE; MASS SPECTROMETRY; MOTONEURON; MOUSE; MUSCLE FUNCTION; NONHUMAN; PERIODIC ACID SCHIFF STAIN; PRIORITY JOURNAL; SURVIVAL ANALYSIS;

EID: 84933500368     PISSN: None     EISSN: 23289503     Source Type: Journal    
DOI: 10.1002/acn3.141     Document Type: Article

References (37)

1. Robberecht W, Philips T. The changing scene of amyotrophic lateral sclerosis. Nat Rev Neurosci 2013;14:248–264.
2. Sreedharan J, Brown RH. Amyotrophic lateral sclerosis: problems and prospects. Ann Neurol 2013;74:309–316.
3. Gurney ME, et al. Benefit of vitamin E, riluzole, and gabapentin in a transgenic model of familial amyotrophic lateral sclerosis. Ann Neurol 1996;39:147–157.
4. Del Signore SJ, et al. Combined riluzole and sodium phenylbutyrate therapy in transgenic amyotrophic lateral sclerosis mice. Amyotroph Lateral Scler 2009;10:85–94.
5. Scott S, et al. Design, power, and interpretation of studies in the standard murine model of ALS. Amyotroph Lateral Scler 2008;9:4–15.
6. Waibel S, Reuter A, Malessa S, et al. Rasagiline alone and in combination with riluzole prolongs survival in an ALS mouse model. J Neurol 2004;251:1080–1084.
7. Milane A, et al. Minocycline and riluzole brain disposition: interactions with P-glycoprotein at the blood-brain barrier. J Neurochem 2007;103:164–173.
8. Milane A, et al. Interactions between riluzole and ABCG2/BCRP transporter. Neurosci Lett 2009;452:12–16.
9. Dunlop J, Beal McIlvain H, She Y, Howland DS. Impaired spinal cord glutamate transport capacity and reduced sensitivity to riluzole in a transgenic superoxide dismutase mutant rat model of amyotrophic lateral sclerosis. J Neurosci 2003;23:1688–1696.
10. Zoccolella S, et al. Riluzole and amyotrophic lateral sclerosis survival: a population-based study in southern Italy. Eur J Neurol 2007;14:262–268.
11. Bensimon G, Lacomblez L, Meininger V; Group AS. A controlled trial of riluzole in amyotrophic lateral sclerosis. N Engl J Med 1994;330:585–591.
12. Jablonski MR, et al. Selective increase of two ABC drug efflux transporters at the blood-spinal cord barrier suggests induced pharmacoresistance in ALS. Neurobiol Dis 2012;47:194–200.
13. Miller DS. Regulation of P-glycoprotein and other ABC drug transporters at the blood-brain barrier. Trends Pharmacol Sci 2010;31:246–254.
14. Winkler EA, et al. Blood-spinal cord barrier breakdown and pericyte reductions in amyotrophic lateral sclerosis. Acta Neuropathol 2013;125:111–120.
15. Garbuzova-Davis S, et al. Impaired blood-brain/spinal cord barrier in ALS patients. Brain Res 2012;1469:114–128.
16. Garbuzova-Davis S, et al. Reduction of circulating endothelial cells in peripheral blood of ALS patients. PLoS One 2010;5:e10614.
17. Nicaise C, et al. Impaired blood-brain and blood-spinal cord barriers in mutant SOD1-linked ALS rat. Brain Res 2009;1301:152–162.
18. Zhong Z, et al. ALS-causing SOD1 mutants generate vascular changes prior to motor neuron degeneration. Nat Neurosci 2008;11:420–422.
19. Garbuzova-Davis S, et al. Ultrastructure of blood-brain barrier and blood-spinal cord barrier in SOD1 mice modeling ALS. Brain Res 2007;1157:126–137.
20. Brandt C, Bethmann K, Gastens AM, Löscher W. The multidrug transporter hypothesis of drug resistance in epilepsy: proof-of-principle in a rat model of temporal lobe epilepsy. Neurobiol Dis 2006;24:202–211.
21. Bankstahl JP, Hoffmann K, Bethmann K, Loscher W. Glutamate is critically involved in seizure-induced overexpression of P-glycoprotein in the brain. Neuropharmacology 2008;54:1006–1016.
22. Agarwal S, Hartz AMS, Elmquist WF, Bauer B. Breast cancer resistance protein and P-glycoprotein in brain cancer: two gatekeepers team up. Current Pharm Des 2011;17:2793–2802.
23. Potschka H. Role of CNS efflux drug transporters in antiepileptic drug delivery: overcoming CNS efflux drug transport. Adv Drug Deliv Rev 2012;64:943–952.
24. Kuntner C, et al. Dose-response assessment of tariquidar and elacridar and regional quantification of P-glycoprotein inhibition at the rat blood-brain barrier using (R)-[(11)C]verapamil PET. Eur J Nucl Med Mol Imaging 2010;37:942–953.
25. Shaik N, Giri N, Pan G, Elmquist WF. P-glycoprotein-mediated active efflux of the anti-HIV1 nucleoside abacavir limits cellular accumulation and brain distribution. Drug Metab Dispos 2007;35:2076–2085.
26. Kruijtzer CMF. Increased oral bioavailability of topotecan in combination with the breast cancer resistance protein and P-glycoprotein inhibitor GF120918. J Clin Oncol 2002;20:2943–2950.
27. Kuppens IELM, et al. A phase I, randomized, open-label, parallel-cohort, dose-finding study of elacridar (GF120918) and oral topotecan in cancer patients. Clin Cancer Res 2007;13:3276–3285.
28. Stokvis E, Rosing H, Causon RC, et al. Quantitative analysis of the P-glycoprotein inhibitor Elacridar (GF120918) in human and dog plasma using liquid chromatography with tandem mass spectrometric detection. J Mass Spectrom 2004;39:1122–1130.
29. Kawamura K, et al. Evaluation of limiting brain penetration related to P-glycoprotein and breast cancer resistance protein using [(11)C]GF120918 by PET in mice. Mol Imaging Biol 2011;13:152–160.
30. Gurney ME, Fleck TJ, Himes CS, Hall ED. Riluzole preserves motor function in a transgenic model of familial amyotrophic lateral sclerosis. Neurology 1998;50:62–66.
31. Kriz J, Gowing G, Julien J-P. Efficient three-drug cocktail for disease induced by mutant superoxide dismutase. Ann Neurol 2003;53:429–436.
32. Benatar M. Lost in translation: treatment trials in the SOD1 mouse and in human ALS. Neurobiol Dis 2007;26:1–13.
33. Traynor BJ, et al. Neuroprotective agents for clinical trials in ALS: a systematic assessment. Neurology 2006;67:20–27.
34. Gould TW, et al. Complete dissociation of motor neuron death from motor dysfunction by Bax deletion in a mouse model of ALS. J Neurosci 2006;26:8774–8786.
35. Parone P, et al. Enhancing mitochondrial calcium buffering capacity reduces aggregation of misfolded SOD1 and motor neuron cell death without extending survival in mouse models of inherited amyotrophic lateral sclerosis. J Neurosci 2013;33:4657–4671.
36. Liu X, Chen C, Smith BJ. Progress in brain penetration evaluation in drug discovery and development. J Pharmacol Exp Ther 2008;325:349–356.
37. Bruno R, et al. Population pharmacokinetics of riluzole in patients with amyotrophic lateral sclerosis. Clin Pharmacol Ther 1997;62:518–526.