Publications


Rare variant analyses validate known ALS genes in a multi-ethnic population and identifies ANTXR2 as a candidate in PLS
The New York Genome Center ALS Sequencing Consortium, Scottish Genomes Partnership, ALS COSMOS Study Group, PLS COSMOS Study Group & GTAC Investigators 2024, , BMC genomics, vol. 25, no. 1, 651. https://doi.org/10.1186/s12864-024-10538-1

Safety and tolerability of tegoprubart in patients with amyotrophic lateral sclerosis: A Phase 2A clinical trial
Perrin, S, Ladha, S, Maragakis, N, Rivner, MH, Katz, J, Genge, A, Olney, N, Lange, D, Heitzman, D, Bodkin, C, Jawdat, O, Goyal, NA, Bornstein, JD, Mak, C, Appel, SH & Paganoni, S 2024, , PLoS Medicine, vol. 21, no. 10, e1004469. https://doi.org/10.1371/journal.pmed.1004469



The Miami Framework for ALS and related neurodegenerative disorders: an integrated view of phenotype and biology
Attendees of the Second International Pre-Symptomatic ALS Workshop 2024, , Nature Reviews Neurology, vol. 20, no. 6, pp. 364-376. https://doi.org/10.1038/s41582-024-00961-z

A phase 1 proof-of-concept study evaluating safety, tolerability, and biological marker responses with combination therapy of CTLA4-Ig and interleukin-2 in amyotrophic lateral sclerosis
Thonhoff, JR, Beers, DR, Zhao, W, Faridar, A, Thome, A, Wen, S, Zhang, A, Wang, J & Appel, SH 2024, , Frontiers in Neurology, vol. 15, 1415106, pp. 1415106. https://doi.org/10.3389/fneur.2024.1415106

Barriers to Tofersen Therapy for Variant SOD1-Mediated ALS
Appel, SH & Thonhoff, JR 2024, , JAMA Neurology, vol. 81, no. 12, pp. 1239-1240. https://doi.org/10.1001/jamaneurol.2024.3331

A phase 1 open-label pilot study of low-dose interleukine-2 immunotherapy in patients with Alzheimer’s disease
Faridar, A, Eid, AM, Thome, AD, Zhao, W, Beers, DR, Pascual, MB, Nakawah, MO, Roman, GC, Davis, CS, Grundman, M, Masdeu, JC & Appel, SH 2023, , Translational Neurodegeneration, vol. 12, no. 1, 54, pp. 54. https://doi.org/10.1186/s40035-023-00387-5

A phase 1 open-label pilot study of low-dose interleukine-2 immunotherapy in patients with Alzheimer's disease
Faridar, A, Eid, AM, Thome, AD, Zhao, W, Beers, DR, Pascual, MB, Nakawah, MO, Roman, GC, Davis, CS, Grundman, M, Masdeu, JC & Appel, SH 2023, , Translational Neurodegeneration, vol. 12, no. 1, pp. 54. https://doi.org/10.1186/s40035-023-00387-5


Primary visual cortex pathology in ALS patients with C9ORF72 expansion
Cykowski, MD, Arumanayagam, AS, Powell, SZ & Appel, SH 2024, , Brain Pathology, vol. 34, no. 5, e13229. https://doi.org/10.1111/bpa.13229

Neuroinflammation co-localizes highly with tau in amnestic mild cognitive impairment
Appleton, J, Funk, Q, Bradbury, K, Yu, M, Faridar, A, Beers, D, Appel, SH, Fujita, M, Masdeu, JC & Pascual, B 2022, , Alzheimers and Dementia, vol. 18, no. S1, e068025. https://doi.org/10.1002/alz.068025

Combined Regulatory T-Lymphocyte and IL-2 Treatment Is Safe, Tolerable, and Biologically Active for 1 Year in Persons With Amyotrophic Lateral Sclerosis
Thonhoff, JR, Berry, JD, Macklin, EA, Beers, DR, Mendoza, PA, Zhao, W, Thome, AD, Triolo, F, Moon, JJ, Paganoni, S, Cudkowicz, M & Appel, SH 2022, , Neurology: Neuroimmunology and NeuroInflammation, vol. 9, no. 6, e200019. https://doi.org/10.1212/NXI.0000000000200019

Ex vivo expanded human regulatory T cells modify neuroinflammation in a preclinical model of Alzheimer’s disease
Faridar, A, Vasquez, M, Thome, AD, Yin, Z, Xuan, H, Wang, JH, Wen, S, Li, X, Thonhoff, JR, Zhao, W, Zhao, H, Beers, DR, Wong, STC, Masdeu, JC & Appel, SH 2022, , Acta Neuropathologica Communications, vol. 10, no. 1, 144. https://doi.org/10.1186/s40478-022-01447-z

Extracellular Vesicles Derived From Ex Vivo Expanded Regulatory T Cells Modulate In Vitro and In Vivo Inflammation
Thome, AD, Thonhoff, JR, Zhao, W, Faridar, A, Wang, J, Beers, DR & Appel, SH 2022, , Frontiers in immunology, vol. 13, 875825, pp. 875825. https://doi.org/10.3389/fimmu.2022.875825

Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development
for the Healey ALS Platform Trial Study Group, HEALEY ALS Platform Trial Study Group, Biostatisticians, Investigators, Clinical Operations Team & Medical Monitors 2022, , Annals of Neurology, vol. 91, no. 2, pp. 165-175. https://doi.org/10.1002/ana.26285

Tregs Attenuate Peripheral Oxidative Stress and Acute Phase Proteins in ALS
Beers, DR, Thonhoff, JR, Faridar, A, Thome, AD, Zhao, W, Wen, S & Appel, SH 2022, , Annals of Neurology, vol. 92, no. 2, pp. 195-200. https://doi.org/10.1002/ana.26375


The role of inflammation in neurodegenerative diseases
Appel, SH, Beers, DR & Zhao, W 2022, . in Neurobiology of Brain Disorders: Biological Basis of Neurological and Psychiatric Disorders, Second Edition. Elsevier, pp. 403-421. https://doi.org/10.1016/B978-0-323-85654-6.00036-8