Of mice and men: Reconciling preclinical ALS mouse studies and human clinical trials

Annals of Neurology

Volumn 53, Issue 4, 2003, Pages 423-426

  Rothstein, Jeffrey D ^a  

^a JOHNS HOPKINS UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CREATINE; GLUTAMIC ACID; MINOCYCLINE; NIFEDIPINE; RILUZOLE; SUPEROXIDE DISMUTASE;

AMYOTROPHIC LATERAL SCLEROSIS; ANIMAL USE; CELL SURVIVAL; CLINICAL TRIAL; DISEASE ACTIVITY; DISEASE COURSE; DISEASE MODEL; DRUG APPROVAL; DRUG EFFECT; DRUG EFFICACY; DRUG SCREENING; EDITORIAL; FOOD AND DRUG ADMINISTRATION; GENE MUTATION; HUMAN; IMMUNOHISTOCHEMISTRY; LIFESPAN; MOTONEURON; MOUSE; NERVE CELL CULTURE; NEUROPIL; NONHUMAN; OXIDATIVE STRESS; PATHOGENESIS; PRIORITY JOURNAL; SURVIVAL TIME;

AMYOTROPHIC LATERAL SCLEROSIS; ANIMALS; DISEASE MODELS, ANIMAL; HUMANS; MICE; MICE, TRANSGENIC;

EID: 0037380729     PISSN: 03645134     EISSN: None     Source Type: Journal    
DOI: 10.1002/ana.10561     Document Type: Editorial

References (26)

1. Groeneveld GJ, Veldink JH, van der Tweel I. A randomized sequential trial of creatine monohydrate in amyotrophic lateral sclerosis. Ann Neurol 2003;53:437-445.
2. Cleveland DW, Rothstein JD. From Charcot to Lou Gehrig: deciphering selective motor neuron death in ALS. Nat Rev Neurosci 2001;2:806-819.
3. Henderson CE. Neurotrophic factors as therapeutic agents in amyotrophic lateral sclerosis. Potential and pitfalls. Adv Neurol 1995;68:235-240.
4. Wong PC, Pardo CA, Borchelt DR, et al. An adverse property of a familial ALS-linked SOD1 mutation causes motor neuron disease characterized by vacuolar degeneration of mitochondria. Neuron 1995;14:1105-1116.
5. Gurney ME, Pu H, Chiu AY, et al. Motor neuron degeneration in mice that express a human Cu,Zn superoxide dismutase mutation. Science 1994;264:1772-1775.
6. Bruijn LI, Becher MW, Lee MK, et al. ALS-linked SOD1 mutant G85R mediates damage to astrocytes and promotes rapidly progressive disease with SOD1-containing inclusions. Neuron 1997;18:327-338.
7. Howland DS, Liu J, She Y, et al. Focal loss of the glutamate transporter EAAT2 in a transgenic rat model of SOD1 mutant- mediated amyotrophic lateral sclerosis (ALS). Proc Natl Acad Sci USA 2002;99:1604-1609.
8. Julien JP. Amyotrophic lateral sclerosis. Unfolding the toxicity of the misfolded. Cell 2001;104:581-591.
9. Shaw PJ. Excitatory amino acid neurotransmission, excitotoxicity and excitotoxins. Curr Opin Neurol Neurosurg 1992;5:383-390.
10. Camu W, Henderson CE. Rapid purification of embryonic rat motoneurons: an in vitro model for studying MND/ALS pathogenesis. J Neurol Sci 1994;124(suppl):73-74.
11. Rothstein JD, Jin L, Dykes-Hoberg M, et al. Chronic inhibition of glutamate uptake produces a model of slow neurotoxicity. Proc Natl Acad Sci USA 1993;90:6591-6595.
12. Ferrante RJ, Andreassen OA, Jenkins BG, et al. Neuroprotective effects of creatine in a transgenic mouse model of Huntington's disease. J Neurosci 2000;20:4389-4397.
13. Gurney ME, Pu H, Chiu AY, et al. Motor neuron degeneration in mice that express a human Cu,Zn superoxide dismutase mutation. Science 1994;264:1772-1775.
14. Gurney ME, Cutting FB, Zhai P, et al. Benefit of vitamin E, riluzole, and gabapentin in a transgenic model of familial amyotrophic lateral sclerosis. Ann Neurol 1996;39:147-157.
15. Gong YH, Parsadanian AS, Andreeva A, et al. Restricted expression of G86R Cu/Zn superoxide dismutase in astrocytes results in astrocytosis but does not cause motoneuron degeneration. J Neurosci 2000;20:660-665.
16. Lino MM, Schneider C, Caroni P. Accumulation of SOD1 mutants in postnatal motoneurons does not cause motoneuron pathology or motoneuron disease. J Neurosci 2002;22:4825-4832.
17. Raoul C, Estevez AG, Nishimune H, et al. Motoneuron death triggered by a specific pathway downstream of Fas. Potentiation by ALS-linked SOD1 mutations. Neuron 2002;35:1067-1083.
18. Haenggeli C, Kato AC. Differential vulnerability of cranial motoneurons in mouse models with motor neuron degeneration. Neurosci Lett 2002;335:39-43.
19. Sagot Y, Vejsada R, Kato AC. Clinical and molecular aspects of motoneurone diseases: animal models, neurotrophic factors and Bcl-2 oncoprotein. Trends Pharmacol Sci 1997;18:330-337.
20. Mockett RJ, Radyuk SN, Benes JJ, et al. Phenotypic effects of familial amyotrophic lateral sclerosis mutant Sod alleles in transgenic Drosophila. Proc Natl Acad Sci USA 2003;100: 301-306.
21. Kriz J, Gowing G, Julien J. An efficient three-drug cocktail for motor neuron disease induced by mutant superoxide dismutase. Ann Neurol 2003;53:429-436.
22. Chen M, Ona VO, Li M, et al. Minocycline inhibits caspase-1 and caspase-3 expression and delays mortality in a transgenic mouse model of Huntington disease. Nat Med 2000;6:797-801.
23. Kriz J, Nguyen MD, Julien JP. Minocycline slows disease progression in a mouse model of amyotrophic lateral sclerosis. Neurobiol Dis 2002;10:268-278.
24. Van den BL, Tilkin P, Lemmens G, et al. Minocycline delays disease onset and mortality in a transgenic model of ALS. Neuroreport 2002;13:1067-1070.
25. Heemskerk J, Tobin AJ, Ravina B. From chemical to drug: neurodegeneration drug screening and the ethics of clinical trials. Nat Neurosci 2002;5(suppl):1027-1029.
26. Zhang W, Narayanan M, Friedlander RM. Additive neuroprotective effects of minocycline with creatine in a mouse model of ALS. Ann Neurol 2003;53:267-270.