The Advent of Omics Sciences in Clinical Trials of Motor Neuron Diseases

被引:7
|
作者
Ruffo, Paola [1 ]
Cavallaro, Sebastiano [2 ]
Conforti, Francesca Luisa [1 ]
机构
[1] Univ Calabria, Dept Pharm Hlth & Nutr Sci, Med Genet Lab, I-87036 Arcavacata Di Rende, Italy
[2] Natl Res Council CNR, Inst Biomed Res & Innovat IRIB, I-95126 Catania, Italy
来源
JOURNAL OF PERSONALIZED MEDICINE | 2022年 / 12卷 / 05期
关键词
clinical trials; omics; personalized medicine; neurodegenerative disease; motor neuron disease; amyotrophic lateral sclerosis; spinal muscular atrophy; genomics; transcriptomics; AMYOTROPHIC-LATERAL-SCLEROSIS; SPINAL MUSCULAR-ATROPHY; ABEPARVOVEC GENE-THERAPY; OPEN-LABEL; ANTISENSE OLIGONUCLEOTIDE; SINGLE-ARM; MULTICENTER;
D O I
10.3390/jpm12050758
中图分类号
R19 [保健组织与事业(卫生事业管理)];
学科分类号
摘要
The "omics revolution" has totally changed the scientific research approach and is contributing to the development of personalized therapies. In motor neuron diseases (MNDs), a set of complex, multifactorial, late-onset and chronic neurodegenerative diseases, the use of multi-omics approaches in clinical trials is providing new opportunities to stratify patients and develop target therapies. To show how omics science is gaining momentum in MNDs, in this work, we review the interventional clinical trials for MNDs based on the application of omics sciences. We analyze a total of 62 clinical trials listed in the ClinicalTrials database where different omics approaches have been applied in an initial phase, for diagnosis or patient selection, or in subsequent stages to cluster subjects, identify molecular signatures or evaluate drugs security or efficacy. The rise of omics sciences in clinical experimentation of MNDs is leading to an upheaval in their diagnosis and therapy that will require significant investments and means to ensure the correct and rapid evolution of personalized medicine.
引用
收藏
页数:14
相关论文
共 50 条
  • [41] Functional properties of motor units in motor neuron diseases and neuropathies
    Vogt, T
    Nix, WA
    ELECTROMYOGRAPHY AND MOTOR CONTROL-ELECTROENCEPHALOGRAPHY AND CLINICAL NEUROPHYSIOLOGY, 1997, 105 (04): : 328 - 332
  • [42] Hereditary motor neuropathies and motor neuron diseases: which is which
    Hanemann, C
    Ludolph, AC
    AMYOTROPHIC LATERAL SCLEROSIS AND OTHER MOTOR NEURON DISORDERS, 2002, 3 (04): : 186 - 189
  • [43] Therapeutic potential of ambroxol for motor neuron diseases
    Bouscary, Alexandra
    Quessada, Cyril
    M S-MEDECINE SCIENCES, 2020, 36 (05): : 447 - 448
  • [44] Critical analysis of the symptomology of motor neuron diseases
    Goldstein, K
    ARCHIV FUR PSYCHIATRIE UND NERVENKRANKHEITEN, 1907, 42 (03): : 1072 - 1085
  • [45] R-Loops in Motor Neuron Diseases
    Perego, Martina G. L.
    Taiana, Michela
    Bresolin, Nereo
    Comi, Giacomo P.
    Corti, Stefania
    MOLECULAR NEUROBIOLOGY, 2019, 56 (04) : 2579 - 2589
  • [46] MOTOR-NEURON DISEASES - INTERFERING WITH THE RUNNERS
    BRADY, S
    NATURE, 1995, 375 (6526) : 12 - 13
  • [47] Disruption of Axonal Transport in Motor Neuron Diseases
    Ikenaka, Kensuke
    Katsuno, Masahisa
    Kawai, Kaori
    Ishigaki, Shinsuke
    Tanaka, Fumiaki
    Sobue, Gen
    INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 2012, 13 (01) : 1225 - 1238
  • [48] Neurotoxic mechanisms of degeneration in motor neuron diseases
    Ludolph, AC
    Münch, C
    DRUG METABOLISM REVIEWS, 1999, 31 (03) : 619 - 634
  • [49] The Role of Sphingomyelin and Ceramide in Motor Neuron Diseases
    McCluskey, Gavin
    Donaghy, Colette
    Morrison, Karen E.
    McConville, John
    Duddy, William
    Duguez, Stephanie
    JOURNAL OF PERSONALIZED MEDICINE, 2022, 12 (09):
  • [50] HUMAN MOTOR NEURON DISEASES - ROWLAND,LP
    LEHMKUHL, D
    PHYSICAL THERAPY, 1984, 64 (03): : 425 - 425