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Biomedicines
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Pathogenesis and Treatment of Amyotrophic Lateral Sclerosis (ALS)
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Pathogenesis and Treatment of Amyotrophic Lateral Sclerosis (ALS)

A Special Issue of Biomedicines (ISSN 2227-9059) belonging to the section "Neurobiology and Clinical Neuroscience".

Deadline for manuscript submissions: 31 December 2026 | Viewed by 1367

Editor

Prof. Dr. Dongsheng Fan

E-Mail Website
Guest Editor
Peking University Third Hospital, Beijing 100091, China
Interests: amyotrophic lateral sclerosis; escorial; advanced launch system (STS); stroke

Special Issue Information

Dear Colleagues,

This Special Issue provides a comprehensive platform for advancing the understanding of amyotrophic lateral sclerosis (ALS) pathogenesis and accelerating the development of effective treatments. As a multifactorial neurodegenerative disease, ALS involves complex interactions between genetic susceptibility, molecular pathways, and cellular processes. We welcome original contributions that explore multi-target therapeutic strategies, including novel drug development and rational combination therapies designed to address the disease's heterogeneity. Submissions utilizing cutting-edge methodologies—such as artificial intelligence-based prediction models, multi-omics data integration, and patient-derived iPSC platforms—for target discovery, drug repurposing use, and patient stratification are particularly encouraged. The issue also seeks innovative clinical trial designs incorporating adaptive protocols, novel endpoint selection, and biomarker-guided strategies to enhance trial efficiency and accuracy. We invite research on diverse intervention modalities ranging from pharmacological approaches (including small molecules, gene therapies, cell-based treatments, and traditional medicines) to non-pharmacological interventions such as neuromodulation and rehabilitation strategies. A key emphasis is placed on translational research that successfully bridges mechanistic insights from basic science with practical clinical applications, ultimately aiming to delay disease progression, extend survival, and improve patients' symptoms and quality of life. The Special Issue invites both original research articles and comprehensive review papers that address these critical areas in ALS research.

Prof. Dr. Dongsheng Fan
Guest Editor

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 250 words) can be sent to the Editorial Office for assessment.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-anonymized peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Biomedicines is an international peer-reviewed open access monthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2600 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

  • amyotrophic lateral sclerosis (ALS)
  • multi-target therapy
  • drug repurposing
  • translational research
  • biomarkers
  • induced pluripotent stem cells (iPSCs)
  • clinical trial design
  • AI-based prediction models
  • precise treatment

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Further information on MDPI's Special Issue policies can be found here.

Published Papers (2 papers)

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Research

25 pages, 2387 KB  
Article
Clinical, Social, and Healthcare Factors Associated with Nutritional Status in Amyotrophic Lateral Sclerosis: A Multidimensional Approach
by Diogo Sousa-Catita, Paulo Mascarenhas, Cátia Oliveira, Miguel Grunho, Filipe Gonçalves and Jorge Fonseca
Biomedicines 2026, 14(8), 1838; https://doi.org/10.3390/biomedicines14081838 - 15 Aug 2026
Viewed by 367
Abstract
Background: Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease where malnutrition significantly worsens clinical outcomes. We performed a multidimensional analysis to identify clinical, social, and healthcare factors associated with nutritional vulnerability in a real-world setting. Methods: This cross-sectional study included 97 adults with [...] Read more.
Background: Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease where malnutrition significantly worsens clinical outcomes. We performed a multidimensional analysis to identify clinical, social, and healthcare factors associated with nutritional vulnerability in a real-world setting. Methods: This cross-sectional study included 97 adults with ALS recruited through a national association (APELA) and a hospital-based outpatient consultation (ANOC). Nutritional status was assessed using the Mini Nutritional Assessment (MNA®), Body Mass Index (BMI), and Global Leadership Initiative on Malnutrition (GLIM) criteria. Associations with total MNA® scores were evaluated using a prespecified multivariable linear model with HC3 robust standard errors and bootstrap validation to assess inferential stability. Results: Malnutrition prevalence was high, with 42.3% of participants classified as moderately malnourished and 43.3% as severely malnourished under GLIM criteria. Mean MNA® score was 19.69 (SD 4.53). In the adjusted model, ANOC recruitment was associated, after adjustment, with a 4.477-point lower MNA® score compared with APELA (HC3 95% CI −7.071 to −1.884; p<0.001). Other sociodemographic and clinical factors yielded imprecise estimates. Although explanatory power was modest (Adjusted R2=0.067; optimism-corrected R2=−0.005), the recruitment setting remained a robust correlate across sensitivity analyses. Conclusions: Recruitment setting emerged as the most consistent adjusted correlate of MNA® scores, likely reflecting differences in clinical case mix and referral pathways in the Portuguese healthcare context. These findings highlight nutritional vulnerability in ALS as a multifaceted construct that should be interpreted in relation to both clinical and healthcare-contextual factors. This study supports considering systemic factors and the need for longitudinal research that incorporates comprehensive functional and social metrics to optimize multidisciplinary nutritional interventions. Full article
(This article belongs to the Special Issue Pathogenesis and Treatment of Amyotrophic Lateral Sclerosis (ALS))
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14 pages, 1535 KB  
Article
Retinal Thickness and Vascular Density Changes in Amyotrophic Lateral Sclerosis Assessed by Optical Coherence Tomography Angiography
by Abdelilah Assialioui, Mónica Povedano, Marta Senau, Isidro Ferrer and Luis Arias
Biomedicines 2026, 14(7), 1612; https://doi.org/10.3390/biomedicines14071612 - 17 Jul 2026
Viewed by 523
Abstract
Background: Amyotrophic lateral sclerosis (ALS) is increasingly recognized as a multisystem disorder involving neurovascular dysfunction. The retina allows in vivo assessment of neurovascular changes. This study evaluated retinal structural and microvascular alterations in ALS using optical coherence tomography (OCT) and optical coherence tomography [...] Read more.
Background: Amyotrophic lateral sclerosis (ALS) is increasingly recognized as a multisystem disorder involving neurovascular dysfunction. The retina allows in vivo assessment of neurovascular changes. This study evaluated retinal structural and microvascular alterations in ALS using optical coherence tomography (OCT) and optical coherence tomography angiography (OCT-A). Methods: This cross-sectional study included 46 participants with ALS and 19 healthy controls. Retinal thickness and vascular density in the superficial and deep retinal capillary plexuses and the choriocapillaris were quantified using OCT and OCT-A. Group comparisons and logistic regression analyses were performed to assess associations with ALS. Subgroup analyses were conducted according to clinical phenotype. Results: In total, 124 eyes were analyzed. ALS was associated with increased average retinal thickness (p = 0.023) and reduced vascular density in the superficial retinal capillary plexus (p = 0.005), deep retinal capillary plexus (p < 0.001), and choriocapillaris (p = 0.004). In logistic regression analyses, retinal thickness was positively associated with ALS status (OR = 1.42, p = 0.023), whereas higher vascular density in the superficial plexus, deep plexus, and choriocapillaris was associated with lower odds of ALS. No significant differences were observed between bulbar- and spinal-onset ALS phenotypes. Conclusions: ALS is associated with structural and microvascular retinal alterations detectable by OCT and OCT-A. These findings support the presence of systemic neurovascular dysfunction and highlight retinal imaging as a promising, non-invasive approach for investigating disease mechanisms and developing potential biomarkers. Full article
(This article belongs to the Special Issue Pathogenesis and Treatment of Amyotrophic Lateral Sclerosis (ALS))
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