Publications


An open label study of a novel immunosuppression intervention for the treatment of amyotrophic lateral sclerosis
Fournier, CN, Schoenfeld, D, Berry, JD, Cudkowicz, ME, Chan, J, Quinn, C, Brown, RH, Salameh, JS, Tansey, MG, Beers, DR, Appel, SH & Glass, JD 2018, Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, pp. 1-8. DOI: 10.1080/21678421.2017.1421666

Phase IIa trial of fingolimod for amyotrophic lateral sclerosis demonstrates acceptable acute safety and tolerability
Berry, JD, Paganoni, S, Atassi, N, Macklin, EA, Goyal, N, Rivner, M, Simpson, E, Appel, S, Grasso, DL, Mejia, NI, Mateen, F, Gill, A, Vieira, F, Tassinari, V & Perrin, S 2017, Muscle and Nerve, vol 56, no. 6, pp. 1077-1084. DOI: 10.1002/mus.25733

Introduction to supplement: the current status of treatment for ALS
Miller, RG & Appel, SH 2017, Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, vol 18, pp. 1-4. DOI: 10.1080/21678421.2017.1361447

TDP-43 Depletion in Microglia Promotes Amyloid Clearance but Also Induces Synapse Loss
Paolicelli, RC, Jawaid, A, Henstridge, CM, Valeri, A, Merlini, M, Robinson, JL, Lee, EB, Rose, J, Appel, S, Lee, VMY, Trojanowski, JQ, Spires-Jones, T, Schulz, PE & Rajendran, L 2017, Neuron. DOI: 10.1016/j.neuron.2017.05.037

Pyrimethamine Significantly Lowers CSF/SOD1 in ALS Patients With SOD1 Mutations
Lange, DJ, Shahbazi, M, Silani, V, Ludolph, AC, Weishaupt, JH, Ajroud-Driss, S, Fields, KG, Remanan, R, Appel, SH, Morelli, C, Doretti, A, Maderna, L, Messina, S, Weiland, U, Marklund, SL & Andersen, PM 2017, Annals of Neurology. DOI: 10.1002/ana.24950

Characterization of Gene Expression Phenotype in Amyotrophic Lateral Sclerosis Monocytes
Zhao, W, Beers, DR, Hooten, KG, Sieglaff, DH, Zhang, A, Kalyana-Sundaram, S, Traini, CM, Halsey, WS, Hughes, AM, Sathe, GM, Livi, GP, Fan, GH & Appel, SH 2017, JAMA Neurology, vol 74, no. 6, pp. 677-685. DOI: 10.1001/jamaneurol.2017.0357

Serum C-Reactive Protein as a Prognostic Biomarker in Amyotrophic Lateral Sclerosis
Lunetta, C, Lizio, A, Maestri, E, Sansone, VA, Mora, G, Miller, RG, Appel, SH & Chiò, A 2017, JAMA Neurology, vol 74, no. 6, pp. 660-667. DOI: 10.1001/jamaneurol.2016.6179

Clinical Significance of TDP-43 Neuropathology in Amyotrophic Lateral Sclerosis
Cykowski, MD, Powell, SZ, Peterson, LE, Appel, JW, Rivera, AL, Takei, H, Chang, E & Appel, SH 2017, Journal of Neuropathology and Experimental Neurology, vol 76, no. 5, pp. 402-413. DOI: 10.1093/jnen/nlx025

ALS patients' regulatory T lymphocytes are dysfunctional, and correlate with disease progression rate and severity
Beers, DR, Zhao, W, Wang, J, Zhang, X, Wen, S, Neal, D, Thonhoff, JR, Alsuliman, AS, Shpall, EJ, Rezvani, K & Appel, SH 2017, JCI insight, vol 2, no. 5, pp. e89530. DOI: 10.1172/jci.insight.89530

A robust, good manufacturing practice–compliant, clinical-scale procedure to generate regulatory T cells from patients with amyotrophic lateral sclerosis for adoptive cell therapy
Alsuliman, A, Appel, SH, Beers, DR, Basar, R, Shaim, H, Kaur, I, Zulovich, J, Yvon, E, Muftuoglu, M, Imahashi, N, Kondo, K, Liu, E, Shpall, EJ & Rezvani, K 2016, Cytotherapy, vol 18, no. 10, pp. 1312-1324. DOI: 10.1016/j.jcyt.2016.06.012

Stem cells in amyotrophic lateral sclerosis: Ready for prime time?
Appel, SH & Armon, C 2016, Neurology, vol 87, no. 4, pp. 348-349. DOI: 10.1212/WNL.0000000000002906

Defining SOD1 ALS natural history to guide therapeutic clinical trial design
Bali, T, Self, W, Liu, J, Siddique, T, Wang, LH, Bird, TD, Ratti, E, Atassi, N, Boylan, KB, Glass, JD, Maragakis, NJ, Caress, JB, McCluskey, LF, Appel, SH, Wymer, JP, Gibson, S, Zinman, L, Mozaffar, T, Callaghan, B, McVey, AL, Jockel-Balsarotti, J, Allred, P, Fisher, ER, Lopate, G, Pestronk, A, Cudkowicz, ME & Miller, TM 2016, Journal of Neurology, Neurosurgery and Psychiatry. DOI: 10.1136/jnnp-2016-313521

The immune system continues to knock at the ALS door
Staff, NP & Appel, SH 2016, Neuromuscular Disorders, vol 26, no. 6, pp. 335-336. DOI: 10.1016/j.nmd.2016.04.010

TDP-43 activates microglia through NF-?B and NLRP3 inflammasome
Zhao, W, Beers, DR, Bell, S, Wang, J, Wen, S, Baloh, RH & Appel, SH 2015, Experimental Neurology, vol 273, pp. 24-35. DOI: 10.1016/j.expneurol.2015.07.019

Randomized phase 2 trial of NP001-a novel immune regulator: Safety and early efficacy in ALS
Miller, RG, Block, G, Katz, JS, Barohn, RJ, Gopalakrishnan, V, Cudkowicz, M, Zhang, JR, McGrath, MS, Ludington, E, Appel, SH & Azhir, A 2015, Neurology: Neuroimmunology and NeuroInflammation, vol 2, no. 3, pp. e100. DOI: 10.1212/NXI.0000000000000100

Protective and Toxic Neuroinflammation in Amyotrophic Lateral Sclerosis
Hooten, KG, Beers, DR, Zhao, W & Appel, SH 2015, Neurotherapeutics, vol 12, no. 2, pp. 364-375. DOI: 10.1007/s13311-014-0329-3

Exome sequencing in amyotrophic lateral sclerosis identifies risk genes and pathways
Cirulli, ET, Lasseigne, BN, Petrovski, S, Sapp, PC, Dion, PA, Leblond, CS, Couthouis, J, Lu, Y-F, Wang, Q, Krueger, BJ, Ren, Z, Keebler, J, Han, Y, Levy, SE, Boone, BE, Wimbish, JR, Waite, LL, Jones, AL, Carulli, JP, Day-Williams, AG, Staropoli, JF, Xin, WW, Chesi, A, Raphael, AR, McKenna-Yasek, D, Cady, J, Vianney de Jong, JMB, Kenna, KP, Smith, BN, Topp, S, Miller, J, Gkazi, A, Al-Chalabi, A, van den Berg, LH, Veldink, J, Silani, V, Ticozzi, N, Shaw, CE, Baloh, RH, Appel, S, Simpson, E, Lagier-Tourenne, C, Pulst, SM, Gibson, S, Trojanowski, JQ, Elman, L, McCluskey, L, Grossman, M, Shneider, NA, Chung, WK, Ravits, JM, Glass, JD, Sims, KB, Van Deerlin, VM, Maniatis, T, Hayes, SD, Ordureau, A, Swarup, S, Landers, J, Baas, F, Allen, AS, Bedlack, RS, Harper, JW, Gitler, AD, Rouleau, GA, Brown, R, Harms, MB, Cooper, GM, Harris, T, Myers, RM, Goldstein, DB & FALS Sequencing Consortium 2015, Science (New York, N.Y.), vol 347, no. 6229, pp. 1436-41. DOI: 10.1126/science.aaa3650

The Role of Regulatory T Cells and Microglia in Amyotrophic Lateral Sclerosis
Beers, DR, Zhao, W, Hooten, KG & Appel, SH 2015, . in S David (ed.), Neuroinflammation: New Insights into Beneficial and Detrimental Functions. John Wiley & Sons, Inc. pp. 235-249. DOI: 10.1002/9781118732748.ch15

Role of Inflammation in Neurodegenerative Diseases
Appel, SH, Beers, DR & Zhao, W 2014, . in Neurobiology of Brain Disorders: Biological Basis of Neurological and Psychiatric Disorders. Elsevier, pp. 380-395. DOI: 10.1016/B978-0-12-398270-4.00025-2

Neuroimmunology of Amyotrophic Lateral Sclerosis
Henkel, JS, Beers, DR, Zhao, W & Appel, SH 2014, . in Neuroinflammation and CNS Disorders. Wiley-Blackwell, pp. 185-209. DOI: 10.1002/9781118406557.ch8