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.
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页数:14
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共 41 条
  • [1] Phenotypic variability in ALS-FTD and effect on survival
    Ahmed, Rebekah M.
    Devenney, Emma M.
    Strikwerda-Brown, Cherie
    Hodges, John R.
    Piguet, Olivier
    Kiernan, Matthew C.
    [J]. NEUROLOGY, 2020, 94 (19) : E2005 - E2013
  • [2] Randomized, double-blind, placebo-controlled trial of arimoclomol in rapidly progressive SOD1 ALS
    Benatar, Michael
    Wuu, Joanne
    Andersen, Peter M.
    Atassi, Nazem
    David, William
    Cudkowicz, Merit
    Schoenfeld, David
    [J]. NEUROLOGY, 2018, 90 (07) : E565 - E574
  • [3] Kennedy's disease (spinal and bulbar muscular atrophy): a clinically oriented review of a rare disease
    Breza, Marianthi
    Koutsis, Georgios
    [J]. JOURNAL OF NEUROLOGY, 2019, 266 (03) : 565 - 573
  • [4] El Escorial revisited: Revised criteria for the diagnosis of amyotrophic lateral sclerosis
    Brooks, BR
    Miller, RG
    Swash, M
    Munsat, TL
    [J]. AMYOTROPHIC LATERAL SCLEROSIS AND OTHER MOTOR NEURON DISORDERS, 2000, 1 (05): : 293 - 299
  • [5] Onco-Multi-OMICS Approach: A New Frontier in Cancer Research
    Chakraborty, Sajib
    Hosen, Md. Ismail
    Ahmed, Musaddeque
    Shekhar, Hossain Uddin
    [J]. BIOMED RESEARCH INTERNATIONAL, 2018, 2018
  • [6] New and Developing Therapies in Spinal Muscular Atrophy: From Genotype to Phenotype to Treatment and Where Do We Stand?
    Chen, Tai-Heng
    [J]. INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 2020, 21 (09)
  • [7] Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial
    Day, John W.
    Finkel, Richard S.
    Chiriboga, Claudia A.
    Connolly, Anne M.
    Crawford, Thomas O.
    Darras, Basil T.
    Iannaccone, Susan T.
    Kuntz, Nancy L.
    Pena, Loren D. M.
    Shieh, Perry B.
    Smith, Edward C.
    Kwon, Jennifer M.
    Zaidman, Craig M.
    Schultz, Meredith
    Feltner, Douglas E.
    Tauscher-Wisniewski, Sitra
    Ouyang, Haojun
    Chand, Deepa H.
    Sproule, Douglas M.
    Macek, Thomas A.
    Mendell, Jerry R.
    [J]. LANCET NEUROLOGY, 2021, 20 (04) : 284 - 293
  • [8] Dharmadasa T, 2018, HAND CLINIC, V159, P345, DOI 10.1016/B978-0-444-63916-5.00022-7
  • [9] Dutasteride:: A dual 5-α reductase inhibitor for the treatment of symptomatic benign prostatic hyperplasia
    Dolder, CR
    [J]. ANNALS OF PHARMACOTHERAPY, 2006, 40 (04) : 658 - 665
  • [10] Efficacy and safety of dutasteride in patients with spinal and bulbar muscular atrophy: a randomised placebo-controlled trial
    Fernandez-Rhodes, Lindsay E.
    Kokkinis, Angela D.
    White, Michelle J.
    Watts, Charlotte A.
    Auh, Sungyoung
    Jeffries, Neal O.
    Shrader, Joseph A.
    Lehky, Tanya J.
    Li, Li
    Ryder, Jennifer E.
    Levy, Ellen W.
    Solomon, Beth I.
    Harris-Love, Michael O.
    La Peon, Alison
    Schindler, Alice B.
    Chen, Cheunju
    Di Prospero, Nicholas A.
    Fischbeck, Kenneth H.
    [J]. LANCET NEUROLOGY, 2011, 10 (02) : 140 - 147