Publications


Transplantation of human bone marrow stem cells into symptomatic ALS mice enhanced structural and functional blood-spinal cord barrier repair
Garbuzova-Davis, S, Haller, E, Navarro, S, Besong, TE, Boccio, KJ, Hailu, S, Khatib, M, Sanberg, PR, Appel, SH & Borlongan, CV 2018, Experimental Neurology. DOI: 10.1016/j.expneurol.2018.08.012

Head-to-head comparison of 11C-PBR28 and 18F-GE180 for quantification of the translocator protein in the human brain
Zanotti-Fregonara, P, Pascual, B, Rizzo, G, Yu, M, Pal, N, Beers, D, Carter, R, Appel, SH, Atassi, N & Masdeu, JC 2018, Journal of Nuclear Medicine, vol. 59, no. 8, pp. 1260-1266. DOI: 10.2967/jnumed.117.203109

Neuroinflammatory mechanisms in amyotrophic lateral sclerosis pathogenesis
Thonhoff, JR, Simpson, EP & Appel, SH 2018, Current Opinion in Neurology. DOI: 10.1097/WCO.0000000000000599

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.

Expanded autologous regulatory T-lymphocyte infusions in ALS A phase I, first-in-human study
Thonhoff, JR, Beers, DR, Zhao, W, Pleitez, M, Simpson, EP, Berry, JD, Cudkowicz, ME & Appel, SH 2018, Neurology: Neuroimmunology and NeuroInflammation, vol. 5, no. 4, e465. DOI: 10.1212/NXI.0000000000000465

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

Genome-wide Analyses Identify KIF5A as a Novel ALS Gene
Nicolas, A, Kenna, KP, Renton, AE, Ticozzi, N, Faghri, F, Chia, R, Dominov, JA, Kenna, BJ, Nalls, MA, Keagle, P, Rivera, AM, van Rheenen, W, Murphy, NA, van Vugt, JJFA, Geiger, JT, Van der Spek, RA, Pliner, HA, Shankaracharya, Smith, BN, Marangi, G, Topp, SD, Abramzon, Y, Gkazi, AS, Eicher, JD, Kenna, A, Logullo, FO, Simone, IL, Logroscino, G, Salvi, F, Bartolomei, I, Borghero, G, Murru, MR, Costantino, E, Pani, C, Puddu, R, Caredda, C, Piras, V, Tranquilli, S, Cuccu, S, Corongiu, D, Melis, M, Milia, A, Marrosu, F, Marrosu, MG, Floris, G, Cannas, A, Capasso, M, Caponnetto, C, Appel, SH, Simpson, E, ITALSGEN Consortium, Genomic Translation for ALS Care (GTAC) Consortium, ALS Sequencing Consortium, NYGC ALS Consortium, Answer ALS Foundation, Clinical Research in ALS and Related Disorders for Therapeutic Development (CReATe) Consortium, SLAGEN Consortium, French ALS Consortium & Project MinE ALS Sequencing Consortium 2018, Neuron, vol. 97, no. 6, pp. 1268-1283.e6. DOI: 10.1016/j.neuron.2018.02.027

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

Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study
Howard, JF, Utsugisawa, K, Benatar, M, Murai, H, Barohn, RJ, Illa, I, Jacob, S, Vissing, J, Burns, TM, Kissel, JT, Muppidi, S, Nowak, RJ, OBrien, F, Wang, JJ, Mantegazza, R, Mazia, CG, Wilken, M, Ortea, C, Saba, J, Rugiero, M, Bettini, M, Vidal, G, Garcia, AD, Lamont, P, Leong, WK, Boterhoven, H, Fyfe, B, Roberts, L, Jasinarachchi, M, Willlems, N, Wanschitz, J, Löscher, W, De Bleecker, J, Van den Abeele, G, de Koning, K, De Mey, K, Mercelis, R, Wagemaekers, L, Mahieu, D, Van Damme, P, Smetcoren, C, Stevens, O, Verjans, S, DHondt, A, Tilkin, P, Alves de Siqueira Carvalho, A, Hasan, R, Dias Brockhausen, I, Simpson, E, Appel, SH & REGAIN Study Group 2017, The Lancet Neurology, vol. 16, no. 12, pp. 976-986. DOI: 10.1016/S1474-4422(17)30369-1

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

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

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

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