Publications


Expanded autologous regulatory T-lymphocyte infusions in ALS
Thonhoff, JR, Beers, DR, Zhao, W, Pleitez, M, Simpson, EP, Berry, JD, Cudkowicz, ME & Appel, SH 2018, Neurology - Neuroimmunology Neuroinflammation, vol 5, no. 4.

The role of regulatory T lymphocytes in amyotrophic lateral sclerosis
Beers, DR, Zhao, W & Appel, SH 2018, JAMA Neurology, vol 75, no. 6, pp. 656-658. DOI: 10.1001/jamaneurol.2018.0043

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

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

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

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

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

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

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

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

Amyotrophic lateral sclerosis
Simpson, EP & Appel, SH 2016, . in Neuroimmune Pharmacology. Springer International Publishing, pp. 493-502. DOI: 10.1007/978-3-319-44022-4_32

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