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Review

Ongoing trials in motor neurone disease

Pages 885-902 | Published online: 23 Feb 2005

Bibliography

  • TANDAN R, BRADLEY W: Amyotrophic lateral sclerosis:Part 1. Clinical features, pathology, and ethical issues in management. Ann. Neurol (1985) 18:271–280.
  • WILLIAMS D, WINDEBANK A: Motor neuron disease(amyotrophic lateral sclerosis). Mayo Clin. Proc. (1991) 66:54–82.
  • ROTHSTEIN J: Excitotoxicity hypothesis. Neurology(1996) 47\(Suppl. 2):S19–S26.
  • •Discusses the excitotoxicity hypothesis of motor neurone death
  • CHOI D: Glutamate neurotoxicity and diseases of the nervous system. Neuron (1988) 1:623–634.
  • COYLE J, PUTTFARCKEN P: Oxidative stress, glutamate, and neurodegenerative disorders. Science (1993) 262:689–695.
  • SHAW P, INCE P: Glutamate, excitotoxicity and amyotrophic lateral sclerosis. J. Neurol. (1997) 244 (Suppl. 2):S3–S14.
  • ALEXIANU M, HO B-K, MOHAMED A, LA BELLA V, SMITH R, APPEL S: The role of calcium-binding proteins in selective motorneruon vulnerability in amyotrophic lateral sclerosis. Ann. Neurol (1994) 36:846–858.
  • ROSEN D, SIDDIQUE T, PATTERSON D et al: Mutations in Cu/Zn superoxide dismutase gene are associated with familial amyotrophic lateral sclerosis. Nature (1993) 362:59–62.
  • BROWN R: Superoxide dismutase and familialamyotrophic lateral sclerosis: New insights into mechanisms and treatments. Ann. Neurol (1996) 39:145–146.
  • ANDERSEN P, FORSGREN L, BINZER M et al.: Autosomalrecessive adult-onset amyotrophic lateral sclerosis associated with homozygosity for Asp90Ala CuZn-superoxide dismutase mutation. A clinical and genealogical study of 36 patients. Brain (1996) 119:1153–1172.
  • SIDDIQUE T, HONG S, BROOKS B et al: X-linked dominant ALS. Neurology (1998) 51:310.
  • BREDESEN D: Neural apoptosis. Ann. Neurol (1995) 38:839–851.
  • LEFEBVRE S, BYRGLEN L, REBOULLET S et al.: Identifica-tion and characterization of a spinal muscular atrophy-determining gene. Cell (1996) 80:155–165.
  • DRACHMAN D, KUNCL R: Amyotrophic lateral sclerosis: An unconventional autoimmune disease? Ann. Neurol (1989) 26:269–274.
  • GORDON P, ROWLAND L, YOUNGER D et al.: Lymphop-roliferative disorders and motor neuron disease: An update. Neurology (1997) 48:1671–1678.
  • SMITH R, HAMILTON S, HOFMANN F et al.: Serum antibodies to L-type calcium channels in patients with amyotrophic lateral sclerosis. New Engl. J. Med. 1992;327:1721–1728.
  • SMITH R, ALEXIANU M, CRAWFORD G, NYORMOI 0, STEFANI E, APPEL S: Cytotoxicity of immunoglobulins from amyotrophic lateral sclerosis patients on a hybrid motoneuron cell line. Proc. Natl. Acad. Sci. USA (1994) 91:3393–3397.
  • ARSAC C, RAYMOND C, MARTIN-MOUTOT N et al.: Immunoassays fail to detect antibodies against neuronal calcium channels in amyotrophic lateral sclerosis serum. Ann. Neurol (1996) 40:695–700.
  • DRACHMAN D, CHAUDHRY V, CORNBLATH D eta].: Trialof immunosuppression in amyotrophic lateral sclerosis using total lymphoid irradiation. Arch. Neurol (1994) 35:142–150.
  • ELLIOTT J, SNIDER W: Motor neuron growth factors.Neurology (1996) 47\(Suppl. 2):S47–S53.
  • SENDTNER M, KREUTZBERG G, THOENEN H: Ciliary neurotrophic factor prevents the degeneration of motor neurons after axotomy. Nature (1990) 345:440–441.
  • NEFF N, PREVETTE D, HOUENOU L et al.: Insulin-likegrowth factors: putative muscle-derived trophic agents that promote motoneuron survival. J. Neurobiol (1993) 24:1558–1566.
  • YAN Q, ELLIOT J, SNIDER W: Brain-derivedneurotrophic factor rescues spinal motor neurons from axotomy-induced cell death. Nature (1992) 360:753–755.
  • MITSUMOTO H, IKEDA K, HOLMLUND T eta].: The effectsof ciliary neurotrophic factor on motor dysfunction in wobbler mouse motor neuron disease. Ann. Neurol 1994;36:142–148.
  • IKEDA K, KLINKOSZ B, GREENE T et al.: Effects of brain-derived neurotrophic factor on motor dysfunction in wobbler mouse motor neuron disease. Ann. Neurol (1995) 37:505–511.
  • WORLD FEDERATION OF NEUROLOGY RESEARCH GROUP ON NEUROMUSCULAR DISEASES: El Escorial World Federation of Neurology criteria for the diagnosis of amyotrophic lateral sclerosis. J. Neurol Sci. (1994) 124 (Suppl.):96–107.
  • •Current set of diagnostic criteria for ALS for clinical trials. Criteria were reviewed in April, 1998, and a revised version will be published in the same journal in the near future.
  • ROSS M, MILLER R, BERCHERT L et al.: Toward earlier diagnosis of amyotrophic lateral sclerosis. Revised criteria. Neurololgy (1998) 50:768–772.
  • MUNSAT T, DAVIES K: Spinal muscular atrophy. In: Diagnostic criteria for neuromuscular disorders 2nd Edition. Emery A (Ed.) London: Royal Society of Medicine Press (1997).
  • •Current set of diagnostic criteria for SMA.
  • IANNACCONE S, BROWNE R, SAMAHA F, BUNCHER C,GROUP DS. Prospective study of spinal muscular atrophy before age 6 years. Pediat. Neurol. (1993) 9:187–193.
  • RUSSMAN B, IANNACONE S, BUNCHER C et al.: Spinalmuscular atrophy: New thoughts on the pathogenesis and classification schema. J. Child Neurol. (1992) 7:347–353.
  • ZERRES K, RUDNIK-SCHNEBORN: Natural history in proximal spinal muscular atrophy. Arch. Neurol. (1995) 52:518–523.
  • BEASLEY W: Quantitative muscle testing: Principles and applications to research and clinical services. Arch. Phys. Med. Rehab. (1961) 42:398–425.
  • STALBERG E: Electrophysiological studies of reinner-vation in ALS. In: Human motor neuron disease. Rowland L (Ed.) Raven Press, New York (1982).
  • DOHERTY T, VANDERVOORT A, TAYLOR A, BROWN W:Effects of motor unit losses on strength in older men and women. J. Appl. Physiol. (1993) 74:868–874.
  • ANDRES P, FINISON L, CONLON T, THIBODEAU L, MUNSAT T: Use of composite scores (megascores) to measure deficit in amyotrophic lateral sclerosis. Neurology (1988) 38:405–408.
  • MUNSAT T, ANDRES P, FINISON L, CONLON T, THIBODEAU L: The natural history of motoneuron loss in amyotrophic lateral sclerosis. Neurology (1988) 38:409–413.
  • •Original study showing linear decline in quantitatively measured muscle strength in ALS. This led to the use of quantitatively measured muscle strength as an end-point measure in clinical trials.
  • PRADAS J, FINISON L, ANDRES P, THORNELL B,HOLLANDER D, MUNSAT T: The natural history of amyotrophic lateral sclerosis and the use of natural history controls in therapeutic trials. Neurology (1993) 43:751–755.
  • FALLAT R, JEWITT B, BASS M, KAMM B, NORRIS F:Spirometry in amyotrophic lateral sclerosis. Arch. Neurol (1979) 36:74–80.
  • ALS CNTF TREATMENT STUDY (ACTS) PHASE I-II STUDYGROUP: The amyotrophic lateral sclerosis functional rating scale. Arch. Neurol. (1996) 53:141–147.
  • APPEL V, STEWART S, SMITH G, APPEL S: A rating scale for amyotrophic lateral sclerosis: Description and preliminary experience. Ann. Neurol. (1987) 22:328–333.
  • STALBERG E, THIELE B: Motor unit fibre density in the extensor digitorum communis muscle. J. Neurol. Neurosurg. Psychiat. (1975) 38:874–880.
  • MCCOMAS A: Invited review: Motor unit estimation:Methods, results, and present status. Musc. Nerve (1991) 14:585–597.
  • BROMBERG M: Electrodiagnostic studies in clinicaltrials for motor neuron disease. J. Clin. Neurophysiol. (1998) 15:117–128.
  • •Discusses end-point measures in clinical trials in MND.
  • DANTES M, MCCOMAS A: The extent and time course ofmotorneuron involvement in amyotrophic lateral sclerosis. Musc. Nerve (1991) 14:416–421.
  • KUETHER G, LIPINSKI H-G: Computer simulation ofneuron degeneration in motor neuron disease. In: Amyotrophic lateral sclerosis. Tsubaki T, Yase Y (Eds.) Elsevier (1988).
  • WFN RESEARCH GROUP ON NEUROMUSCULAR DISEASES SUBCOMMITTEE ON MOTOR NEURON DISEASE. AIRLIE HOUSE GUIDELINES: Therapeutic trials in amyotrophic lateral sclerosis. J. Neurol Sci. (1995) 129(Suppl.):1–10.
  • •Discusses guidelines for clinical trials in ALS.
  • MUNSAT T: Issues in clinical trial design I: Use ofnatural history controls. A protagonist view. Neurology (1996) 47 (Suppl. 2):S96–S97.
  • SUFIT R: Issues in clinical trial design I: Use of naturalhistory controls. An antagonist view. Neurology (1996) 47 (Suppl. 2):S98–S99.
  • RINGEL S, MURPHY J, ALDERSON M et al.: The natural history of amyotrophic lateral sclerosis. Neurology (1993) 43:1316–1322.
  • BROMBERG M, NAU K, FORSHEW D: End point measure-ments in clinical trials in amyotrophic lateral sclerosis. In: ALS-from Charcot to the present and into the future. Rose F (Ed.) Smith-Gordon, London (1994).
  • ANDRES P, HEDLUND W, FINISON L, CONLON T, FELMUS M, MUNSAT T: Quantitative motor assessment in amyotrophic lateral sclerosis. Neurology (1986) 36:937–941.
  • SMITH B, STEVENS J, LITCHY W et al.: Longitudinal electrodiagnostic studies in amyotrophic lateral sclerosis patients treated with recombinant human ciliary neurotrophic factor. Neurology (1995) 45 (Suppl. 4):A448.
  • BROMBERG M, FRIES T: Relationships between changes in strength and electromyographic measures in amyotrophic lateral sclerosis patients over a six-month interval. Musc. Nerve (1992) 15:1189.
  • FELICE K: A longitudinal study comparing thenar motor unit number estimates to other quantitative tests in patients with amyotrophic lateral sclerosis. Musc. Nerve (1997) 20:179–185.
  • YUEN E, OLNEY R: Longitudinal study of fibre density and motor unit number estimate in patients with amyotrophic lateral sclerosis. Neurology (1997) 49:573–578.
  • TESTA M, SIMONSON D: Assessment of quality-of-lifeoutcomes. New Engl. J. Med. (1996) 334:220–233.
  • BERGNER M, BOBBITT R, CARTER R, GILSON B: The Sickness Impact Profile: development and final revision of a health status measure. Med. Care (1981) 19:787–805.
  • WARE J, SHERBOURNE C: The MOS 36-item short-formsurvey (SF-36). I. Conceptual framework and item selection. Med. Care. (1992) 30:473–483.
  • WARE J, KOSINSKI M, KELLER S: A 12-item short-formhealth survey. Med. Care (1996) 34:220–233.
  • MCGUIRE D, GARRISON L, ARMON C et al.: A briefquality-of-life measure for ALS clinical trials based on a subset of items from the sickness impact profile. J. Neurol. Sci. (1997) 152:S18–522.
  • JENKINSON C, FITZPATRICK R, BRENNAN C, BROMBERG M, SWASH M. Development and validation of a short measure of health status for individuals with amyotrophic lateral sclerosis/motor neuron disease: The ALSAQ-40. J. Neurol. (1999) (In Press).
  • HICKEY A, BURY G, O'BOYLE C, BRADLEY F, O'KELLY F, SHANNON W: A new short form individual quality of life measure (SEIQoL-DW): applicaiton in a cohort of individuals with HIV?AIDS. BMJ (1996) 313:29–33.
  • BROMBERG M, FORSHEW D: Comparison of quality of life (QoL) instruments in ALS patients. Neurology (1999) 52 (Suppl. 2):A527.
  • MILLER R, ROSENBERG J, GELINAS D et al.: Practiceparameter: The care of the patient with amyotrophic lateral sclerosis (an evidence-based review). Neurology (1999) 52:1311–1323.
  • NAU K, BROMBERG M, FORSHEW D, KATCH V: Individuals with amyotrophic lateral sclerosis are in caloric balance despite losses in mass. J. Neurol Sci. (1995) 129(Suppl.):47–49.
  • MAZZINI L, CORR" T, ZACCALA M, MORA G, DEL PIANO M, GALANTE M: Percutaneous endoscopic gastrostomy and enteral nutrition in amyolateral sclerosis. J. Neurol (1995) 242:695–698.
  • ABOUSSOUAN L, KHAN S, MEEKER D, STELMACH K, MITSUMOTO H. Effects of noninvasive positive-pressure ventilation on survival in amyotrophic lateral sclerosis. Ann. Intern. Med. (1997) 127:450–453.
  • ROTHSTEIN J, JEN L, DYKES-HOBERG M, KUNCL R: Chronic inhibition of glutamate uptake produces a model of slow neurotoxicity. Proc. Natl. Acad. Sci. USA (1993) 90:6591–6595.
  • ROTHSTEIN J, KUNCL R: Neuroprotective strategies in amodel of chronic glutamate-mediated motor neuron toxicity. J. Neurochem. (1995) 65:643–651.
  • LUDOLPH A: Animal models for motor neuro diseases:Research directions. Neurology (1996) 47\(Suppl. 4):5228–S232.
  • SCHMALBRUCH H, JENSEN H-JS, BJAERG M, KAMIENIECKA Z, KURLAND L: A new mouse mutant with progressive motor neuropathy. J. Neuropathol. Exp. Neurol. (1991) 50:192–204.
  • MITSUMOTO H, BRADLEY W: Murine motor neuron disease (The wobbler mouse) Degeneration and regeneration of the lower motor neuron. Brain (1982) 105:811–834.
  • GURNEY M: The use of transgenic mouse models ofamyotrophic lateral sclerosis in preclinical drug studies. J. Neurol. Sci. (1997) 152 (Suppl. 1):S67–S73.
  • GURNEY M, CUTTING F, ZHAI P et al: Benefit of vitaminE, riluzole, and gabapentin in a transgenic model of familial amyotrophic lateral sclerosis. Ann. Neurol. (1996) 39:147–157.
  • MILLER R, PETAJAN J, BRYAN W et al.: A placebo-controlled trial of recombinant human ciliary neurotrophic (rhCNTF) factor in amyotrophic lateral sclerosis. Ann. Neurol (1996) 39:256–260.
  • MITSUMOTO H, HANSEN M, CHAD D: Amyotrophic lateral sclerosis. Recent advances in pathogenesis and therapeutic trials. Arch. Neurol. (1988) 45:189–202.
  • FESTOFF B: Amyotrophic lateral sclerosis. Current and future treatment strategies. Drugs (1996) 51:28–44.
  • GREENSMITH L, VRBOV G: Disturbances of neuromus-cular interaction may contribute to muscle weakness in spinal muscular atrophy. Neuromusc. Disord. (1997) 7:369–372.
  • CUDKOWICZ M, MCKENNA-YASEK D, CHEN C, HEDLEY-WHYTE E, BROWN R: Limited corticospinal tract involvement in amyotrophic lateral sclerosis subjects with the A4V mutation in the copper/zinc superoxide dismutase gene. Ann. Neurol. (1998) 43:703–710.
  • APPLEBAUM J, ROOS R, SALAZAR-GRUESO E et al.: Intrafamilial heterogeneity in hereditary motor neuron disease. Neurology (1992) 42:1488–1492.
  • SHAW P, INCE P, GOODSHIP J et al.: Adult-onset motorneuron disease and infantile Werdnig-Hoffmann disease (spinal muscular atrophy Type 1) in the same family. Neurology (1992) 42:1477–1480.
  • BENSIMON G, LACOMBLEZ L, MEININGER V, GROUP TARS: A controlled trial of riluzole in amyotrophic lateral sclerosis. New Engl J. Merl. (1994) 330:585–591.
  • LACOMBLEZ L, BENSIMON G, LEIGH P, GUILLET P,MEININGER V, GROUP ALSRS: Dose-ranging study of riluzole in amyotrophic lateral sclerosis. Lancet (1996) 347:1425–1431.
  • •First clinical trial of a drug demonstrating efficacy in ALS.
  • LEWIS M, VAUGHT J, NEFF N: The potential of insulin-like growth factor-I as a therapeutic for the treatment of neuromuscular disorders. Ann. NY Acad Sci. (1993) 692:201–208.
  • LAI E, FELICE K, FESTOFF B et al.: Effect of recombinanthuman insulin-like growth factor-I on progression of ALS. A placebo-controlled study. Neurology (1997) 49:1621–1630.
  • BORASIO G, ROBBERECHT W, LEIGH P et al.: A placebo-controlled trial of insulin-like growth factor-I in amyotrophic lateral sclerosis. Neurology (1998) 51:583–586.
  • TAYLOR C: Gabapentin-mechanisms action. In:Anti-epleptic drugs 4th Ed. Levy R, Mattson R, Meldrum B (Eds.) Raven Press, New York (1995).
  • MILLER R, MOORED, YOUNG L et al.: Placebo-controlledtrial of gabapentin in patients with amyotrophic lateral sclerosis. Neurology (1996) 47:1383–1388.
  • PRADINES A, MAGAZIN M, SCHILTZ P, LE FUR G, CAPUT D, FERRARA P: Evidence for nerve growth factor-potentiating activities of the nonpeptidic compound SR 57746A in PC12 cells. J. Neurochem. (1995) 64:1954–1964.
  • DUONG F, FOURNIER J, KEANE P et al.: The effect of the nonpeptide neurotrophic compound SR 57746A on the progression of the disease state of the pmn mouse. Br. J. Pharmacol. (1998) 124:811–817.
  • LACOMBLEZ L, DOPPLER V, BENSIMON G et al.: Safety and efficacy controlled trial of SR 57746A in amyotrophic lateral sclerosis. Meth. Find. Exp. Clin. Pharmacol. (1996) 18(Suppl B):206.
  • LOUWERSE E, WEVERLING G, BOSSUYT P, MEYJES E, DEJONG J: Randomized, double-blinded, controlled trial of acetylcysteine in amyotrophic lateral sclerosis. Arch. Neurol. (1995) 52:559–564.
  • KLIVENYI P, FERRANTE R, MATTHEWS R etal.: Neuropro-tective effects of creatine in a transgenic animal model of amyotrophic lateral sclerosis. Nature Med. (1999) 5:347–350.
  • MITSUMOTO H, OLNEY R: Drug combination treatmentin patients with ALS: Current status and future directions. Neurology (1996) 47 (Suppl. 2):S103–S107.
  • MITSUMOTO H, IKEDA K, KLINKOSZ B, CEDARBAUM J,WONG V, LINDSAY R: CNTF and BDNF co-treatment arrests loss of motor function in wobbler mice. Science (1994) 265:1107–1110.
  • HAASE G, PETTMANN B, BORDET T et al.: Therapeuticbenefit of ciliary neurotrophic factor in progressive motor neuronopathy depends on the route of delivery. Ann. Neurol (1999) 45:296–304.
  • PLAITAKIS A, SMITH J, MANDELI J, YAHR M: Pilot trial ofbranched-chain aminoacids in amyotrophic lateral sclerosis. Lancet (1988) 1:1015–1018.
  • TANDAN R, BROMBERG M, FORSHEW D et al.: A controlled trial of amino acid therapy in amyotrophic lateral sclerosis: I. Clinical, functional, and maximum isometric torque data. Neurology (1996) 47:1220–1226.
  • EISEN A, STEWART H, SCHULZER M, CAMERON D: Anti-glutamate therapy in amyotrophic lateral sclerosis: A trial using lamotrigine. Canadian J. Neurol. Sci. (1993) 20:297–301.
  • ASKMARK H, AQUILONIUS S-M, GILLBERG P-G, LIEDHOLM L, STALBERG E, WUOPIO R: A pilot trial of dextromethorphan in amyotrophic lateral sclerosis. J. Neurol Neurosurg. Psychial (1993) 56:197–200.
  • GREDAL 0, WERDELIN L, BBACK S et al.: A clinical trial of dextromethorphan in amyotrophic lateral sclerosis. Acta Neurologica Scandinavia. (1997) 96:8–13.
  • MAZZINI L, TESTA D, BALZARINI C, MORA G: An open-randomized clinical trial of selegiline in amyotrophic lateral sclerosis. J. Neurol. (1994) 241:223–227.
  • LANGE D, MURPHY P, DIAMOND B et al.: Selegiline is ineffective in a collaborative double-blind, placebo-controlled trial for treatment of amyotrophic lateral sclerosis. Arch. Neurol (1998) 55:93–96.
  • MILLER R, SMITH S, MURPHY J et al.: A clinical trial of verapamil in amyotrophic lateral sclerosis. Musc. Nerve (1996) 19:511–515.
  • SMITH S, MILLER R, MURPHY J, RINGLE S: Treatment of ALS with high dose pulse cyclophosphamide. J. Neurol Sci. (1994) 124(Suppl.):84–87.
  • DALAKAS M, STEIN D, OTERO C, SEKUL E, CUPLER E, MCCROSKY S: Effect of high-dose intravenous immunoglobulin on amyotrophic lateral sclerosis ands multifocal motor neuropathy. Arch. Neurol (1994) 51:861–864.
  • ALS CNTF TREATMENT STUDY GROUP: A double-blinded placebo-controlled clinical trial of subcuta-neous recombinant human ciliary neurotrophic factor (rHCNTF) in amyotrophic lateral sclerosis. Neurology (1996) 46:1244–1249.

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