Guidelines for the preclinical in vivo evaluation of pharmacological active drugs for ALS/MND: Report on the 142nd ENMC international workshop

References

• Rosen D. R., Siddique T., Patterson D., Figlewicz D. A., Sapp P., Hentati A., et al. Mutations in the Cu/Zn superoxide dismutase gene are associated with familial amyotrophic lateral sclerosis. Nature 1993; 362: 59–62
   PubMed Web of Science ®Google Scholar
• Gurney M. E., Pu H., Chiu A. Y., Dal Canto M. C., Polchow C. Y., Alexander D. D., et al. Motor neuron degeneration in mice that express a human Cu/Zn superoxide dismutase mutation. Science 1994; 264: 1772–5
   PubMed Web of Science ®Google Scholar
• Philips G. C., Porter R. Cortical neurons: their role in movement. Academic Press, London
   Google Scholar
• Bendotti C., Carri M. T. Lessons from models of SOD1‐linked familial ALS. Trends Mol Med 2004; 10: 393–400
   PubMed Web of Science ®Google Scholar
• Rothstein J. D. Of mice and men: reconciling preclinical ALS mouse studies and human clinical trials. Ann Neurol 2003; 53: 423–6
   PubMed Web of Science ®Google Scholar
• Bensimon G., Lacomblez L., Meininger V. A controlled trial of riluzole in amyotrophic lateral sclerosis. ALS/Riluzole Study Group. N Engl J Med 1994; 330: 585–91
   PubMed Web of Science ®Google Scholar
• Lacomblez L., Bensimon G., Leigh P. N., Guillet P., Meininger V. Dose‐ranging study of riluzole in amyotrophic lateral sclerosis. ALS/Riluzole Study Group II Lancet 1996; 347: 1425–31
   PubMed Web of Science ®Google Scholar
• Klivenyi P., Ferrante R. J., Matthews R. T., Bogdanov M. B., Klein A. M., Andreassen O. A., et al. Neuroprotective effects of creatine in a transgenic animal model of amyotrophic lateral sclerosis. Nat Med 1999; 5: 347–50
   PubMed Web of Science ®Google Scholar
• Groeneveld G. J., Veldink J. H., van der Tweel I., Kalmijn S., Beijer C., de Visser M., et al. A randomized sequential trial of creatine in amyotrophic lateral sclerosis. Ann Neurol 2003; 53: 437–45
   PubMed Web of Science ®Google Scholar
• Gurney M. E., Cutting F. B., Zhai P., Doble A., Taylor C. P., Andrus P. K., et al. Benefit of vitamin E, riluzole, and gabapentin in a transgenic model of familial amyotrophic lateral sclerosis. Ann Neurol 1996; 39: 147–57
   PubMed Web of Science ®Google Scholar
• Desnuelle C., Dib M., Garrel C., Favier A. A double‐blind, placebo‐controlled randomized clinical trial of alpha‐tocopherol (vitamin E) in the treatment of amyotrophic lateral sclerosis. Amyotroph Lateral Scler Other Motor Neuron Disord 2001; 2: 9–18
   PubMed Web of Science ®Google Scholar
• Graf M., Ecker D., Horowski R., Kramer B., Riederer P., Gerlach M., et al. High dose vitamin E therapy in amyotrophic lateral sclerosis as add‐on therapy to riluzole: results of a placebo‐controlled double‐blind study. J Neural Transm 2005; 112: 649–60
   PubMed Web of Science ®Google Scholar
• Miller R. G., Moore D. H., Gelinas D. F., Dronsky V., Mendoza M., Barohn R. J., et al. Phase III randomized trial of gabapentin in patients with amyotrophic lateral sclerosis. Neurology 2001; 56: 843–8
   PubMed Web of Science ®Google Scholar
• Drachmen D. B., Frank K., Dykes‐Hoberg M., Teismenn P., Almer G., Przedborski S., et al. Cyclooxygenase 2 inhibition protects motor neurons and prolongs survival in a transgenic mouse model of ALS. Ann Neurol 2002; 52: 771–8
   PubMed Web of Science ®Google Scholar
• Kiaei M., Kipiani K., Petri S., Choi D. K., Chen J., Calingasan N. Y., et al. Integrative role of cPLA with COX‐2 and the effect of non‐steroidal anti‐inflammatory drugs in a transgenic mouse model of amyotrophic lateral sclerosis. J Neurochem 2005; 93: 403–11
   PubMed Web of Science ®Google Scholar
• Cudkowicz M. E., Shefner J. M., Schoenfeld D. A., Zhang H., Andreasson K. I., Rothstein J. D., et al. Trial of celecoxib in amyotrophic lateral sclerosis. Ann Neurol 2006; 60: 22–31
   PubMed Web of Science ®Google Scholar
• Heimen‐Patterson T. D., Deitch J. S., Blankenhorn E. P., Erwin K. L., Perreault M. J., Alexander B. K., et al. Background and gender effects on survival in the TgN(SOD1‐G93A)1Gur mouse model of ALS. J Neurol Sci 2005; 236: 1–7
   PubMed Web of Science ®Google Scholar
• Angelov D. N., Waibel S., Guntinas‐Lichius O., Lenzen M., Neiss W. F., Tomov T. L., et al. Therapeutic vaccine for acute and chronic motor neuron diseases: implications for amyotrophic lateral sclerosis. Proc Natl Acad Sci. U S A 2003; 100: 4790–5
   PubMed Web of Science ®Google Scholar
• Schwalenstöcker B., Danzeisen R., Ludolph A. C. Copaxone treatment in a mouse model of ALS: effect of gender and genetic background. Amyotroph Lateral Scler Other Motor Neuron Disord 2004; 5((Suppl 2))91
   Google Scholar
• Kipnis J., Schwartz M. Dual action of glatiramer acetate (Cop‐1) in the treatment of CNS autoimmune and neurodegenerative disorders. Trends Mol Med 2002; 8: 319–23
   PubMed Web of Science ®Google Scholar
• Scott S. Design, power and interpretation of studies in the standard murine model of amyotrophic lateral sclerosis. Amyotrophic Lateral Sclerosis 2006; 7: 32–3
   PubMed Web of Science ®Google Scholar
• Alexander G. M., Erwin K. L., Byers N., Deitch J. S., Augelli B. J., Blankenhorn E. P., et al. Effect of transgene copy number on survival in the G93A SOD1 transgenic mouse model of ALS. Brain Res Mol Brain Res 2004; 130: 7–15
   PubMedGoogle Scholar
• Carri M. T., Grignaschi G., Bendotti C. Targets in ALS: designing multidrug therapies. Trends Pharmacol Sci 2006; 27: 267–73
   PubMed Web of Science ®Google Scholar
• Julien J. P., Kriz J. Transgenic mouse models of amyotrophic lateral sclerosis. Biochimica et Biophysica Acta 2006, in press
   PubMed Web of Science ®Google Scholar
• Pasinelli P., Brown R. H. Molecular biology of amyotrophic lateral sclerosis: insights from genetics. Nat Rev Neurosci 2006; 7: 710–23
   PubMed Web of Science ®Google Scholar
• Laird F. M., Ackerley S., Farah M. H., Hoke A., Maragakis N., Rothstein J., et al. Neuronal expression of an ALS‐linked mutant dynactin P150 glued in mice causes motor neuron disease. Program No. 131.1. 2005 Abstract Viewer/Itinerary Planner. Society for Neuroscience, Washington, DC 2005, Online
   Google Scholar
• Boillee S., Yamenaka K., Lobsiger C. S., Copeland N. G., Jenkins N. A., Kassiotis G., et al. Onset and progression in inherited ALS determined by motor neurons and microglia. Science 2006; 312: 1389–92
   PubMed Web of Science ®Google Scholar
• Ascherio A., Weisskopf M. G., O'Reilly E. J., Jacobs E. J., McCullough M. L., Calle E. E., et al. Vitamin E intake and risk of amyotrophic lateral sclerosis. Ann Neurol 2005; 57: 104–10
   PubMed Web of Science ®Google Scholar
• Waibel S., Reuter A., Malessa S., Blaugrund E., Ludolph A. C. Rasagiline alone and in combination with riluzole prolongs survival in an ALS mouse model. J Neurol 2004; 251: 1080–4
   PubMed Web of Science ®Google Scholar
• Grignaschi G., Tortarolo M., Calvaresi N., Bendotti C. Erythropoietin does not preserve motor neurons in a mouse model of familial ALS. Amyotrophic Lateral Sclerosis ALS 2006
   Google Scholar
• Pakkenberg B., Gundersen H. J. Solutions to old problems in the quantitation of the central nervous system. J Neurol Sci 1995; 129((Suppl))65–7
   PubMed Web of Science ®Google Scholar
• White C. M., Greensmith L., Vrbova G. Repeated stimuli for axonal growth causes motor neuron death in adult rats: the effect of botulinum toxin followed by partial denervation. Neuroscience 2000; 95: 1101–9
   PubMed Web of Science ®Google Scholar
• Kieran D., Kalmar B., Dick J. R., Riddoch‐Contreras J., Burnstock G., Greensmith L. Treatment with arimoclomol, a coinducer of heat shock proteins, delays disease progression in ALS mice. Nat Med 2004; 10: 402–5
   PubMed Web of Science ®Google Scholar
• Angenstein F., Niessen H. G., Goldschmidt J., Vielhaber S., Ludolph A. C., Scheich H. Age‐dependent changes in MRI of motor brainstem nuclei in a mouse model of ALS. Neuroreport 2004; 15: 2271–4
   PubMed Web of Science ®Google Scholar
• Niessen H. G., Angenstein F., Sander K., Kunz WSTeuchert M., Ludolph A. C., et al. In vivo quantification of spinal and bulbar motor neuron degeneration in the G93A–SOD1 transgenic mouse model of ALS by T2 relaxation time and apparent diffusion coefficient. Exp Neurol 2006; 201: 293–300
   PubMed Web of Science ®Google Scholar
• Dupuis L., Oudart H., Rene F., Gonzalez de Aguilar J. L., Loeffler J. P. Evidence for defective energy homeostasis in amyotrophic lateral sclerosis: benefit of a high‐energy diet in a transgenic mouse model. Proc Natl Acad Sci. U S A 2004; 101: 11159–64
   PubMed Web of Science ®Google Scholar
• Mahoney D. J., Kaczor J. J., Bourgeois J., Yasuda N., Tarnopolsky M. A. Oxidative stress and antioxidant enzyme up‐regulation in SOD1‐G93A mouse skeletal muscle. Muscle Nerve 2006; 33: 809–16
   PubMed Web of Science ®Google Scholar
• Veldink J. H., Kalmijn S., Groeneveld G. J., Titulaer M. J., Wokke J. H., van den Berg L. H. Physical activity and the association with sporadic ALS. Neurology 2005; 64: 241–5
   PubMed Web of Science ®Google Scholar
• van Den Bosch L., Tilkin P., Lemmens G., Robberecht W. Minocycline delays disease onset and mortality in a transgenic model of ALS. Neuroreport 2002; 13: 1067–70
   PubMed Web of Science ®Google Scholar