Management of adrenoleukodystrophy: From pre-clinical studies to the development of new therapies

Yan, Chui; Li, Cheng; Zhou, Xin; Zhang, Zhao; Jiang, Hua; Liu, Huan; Chen, Huanhuan Joyce; Tse, Hung‐Fat; Liao, Can; Lian, Qizhou

doi:10.1016/j.biopha.2021.112214

Cited by 15 publications

(4 citation statements)

References 93 publications

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“…Skysona изготавливается специально для каждого пациента с использованием собственных стволовых клеток крови пациента. Функциональные копии гена ABCD1 добавляются к стволовым клеткам пациента, которые затем могут помочь организму разрушить VLCFAs, чтобы замедлить прогрессирование повреждения головного мозга и замедлить снижение неврологической функции [82]. Пакеты Skysona вводят внутривенно в течение менее 60 мин каждый.…”

Section: обзорunclassified

Overview of drugs approved by the FDA in 2022

Kurkin,

Morkovin,

Bakulin

et al. 2023

Farm. farmakol. (Pâtigorsk)

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The aim of the work is to conduct a review of medications approved by the FDA in 2022.Materials and methods. In searching for the materials to write this review article, bibliographic databases including PubMed, Google Scholar and e-library.ru were utilized. The search was conducted for the publications spanning the period from 2008 to 2023. Herewith, the following keywords and word combinations were used: new drug approval, NDA, drug authorization, approval package, breakthrough medicine.Results. The discovery and development of medications are among the most crucial scientific processes in healthcare. Developing a new drug is a highly intricate, expensive, and time-consuming process. Nowadays, the problem of costs reduction and the process of expedited discovering of new medications are particularly pertinent. To optimize the search for active compounds, virtual and high-throughput screenings, machine learning, artificial intelligence, cryo-electron microscopy, and drug repurposing are employed. Simultaneously, the search for original molecules to serve as the basis for innovative drugs continues. This article presents a review of medications approved by the FDA in 2022 for the treatment of various pathologies.Conclusion. A drug development is a complex and resource-intensive process, with only a small fraction of candidates advancing to clinical trials. A drug design evolves in tandem with societal needs, and this review highlights some of the medications approved by the FDA in 2022. Technological advancements are expected to expedite drug development, potentially reducing the time to the market. Biotechnology, including cell therapy, holds significant prospects, and achievements in genetic mapping and chip technologies will enhance the accessibility of personalized pharmacology.

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Section: обзорunclassified

Overview of drugs approved by the FDA in 2022

Kurkin,

Morkovin,

Bakulin

et al. 2023

Farm. farmakol. (Pâtigorsk)

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“…Diagnosis is suggested by a persistent serum transferrin saturation of ≥ 45% and/or unexplained serum ferritin of ≥ 200 ng/ml in premenopausal woman. The clinical penetrance of disease is variable [18,155].…”

Section: Hereditary Hemochromatosis (Hh)mentioning

confidence: 99%

Therapeutic Apheresis and Human Monoclonal Antibodies in Non-Immunologic Diseases. TA, HMA in non-immunologic diseases

2023

JCEI

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Non-immunologic diseases such as nephrological, blood diseases, dyslipoproteinemia, cancer, infections, intoxications etc. have often a severe course or a bad prognosis. Since more than 45 years, therapeutic apheresis has led in combination with other therapies to a steady increase in survival rates. Therapeutic apheresis is indicated in the most of the non-immunologic diseases, and in all antibody-mediated diseases. Since the introduction of hollow fiber membranes, a complete separation of the corpuscular components, pathologic substances, antibodies, toxins etc. is reached. The mortality rate of severe non-immunologic diseases is reduced by therapeutic apheresis. Other new therapies are various human monoclonal antibodies alone or in combination with therapeutic apheresis. The pathological aspects, the first-line and second-lines therapies for non-immunologic such as nephrological, neurological, gastroenterological, hematological diseases, and others are shown. The guidelines of the Apheresis Applications Committee of the American Society for Apheresis are cited. Therapeutic apheresis and/or human monoclonal antibodies have shown to effectively cure severe diseases without immunologic origin.

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“…earlier and even worse symptoms that are suitable for therapeutic assays at 12 months but also in the absence of inflammatory demyelination (77). Similarly, studies have shown that ABCD1 knockout or ABCD1/2 double-knockout rabbit models still fail to recapitulate human CALD phenotypes (78).…”

Section: The Underlying Mechanisms Of Onset and Damage Involved In Ox...mentioning

confidence: 99%

The Role of Oxidative Stress and Inflammation in X-Link Adrenoleukodystrophy

Chen

Guo

et al. 2022

Front. Nutr.

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X-linked adrenoleukodystrophy (X-ALD) is an inherited disease caused by a mutation in the ABCD1 gene encoding a peroxisomal transmembrane protein. It is characterized by the accumulation of very-long-chain fatty acids (VLCFAs) in body fluids and tissues, leading to progressive demyelination and adrenal insufficiency. ALD has various phenotypes, among which the most common and severe is childhood cerebral adrenoleukodystrophy (CCALD). The pathophysiological mechanisms of ALD remain unclear, but some in vitro/in vivo research showed that VLCFA could induce oxidative stress and inflammation, leading to damage. In addition, the evidence that oxidative stress and inflammation are increased in patients with X-ALD also proves that it is a potential mechanism of brain and adrenal damage. Therefore, normalizing the redox balance becomes a critical therapeutic target. This study focuses on the possible predictors of the severity and progression of X-ALD, the potential mechanisms of pathogenesis, and the promising targeted drugs involved in oxidative stress and inflammation.

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Management of adrenoleukodystrophy: From pre-clinical studies to the development of new therapies

Cited by 15 publications

References 93 publications

Overview of drugs approved by the FDA in 2022

Overview of drugs approved by the FDA in 2022

Therapeutic Apheresis and Human Monoclonal Antibodies in Non-Immunologic Diseases. TA, HMA in non-immunologic diseases

The Role of Oxidative Stress and Inflammation in X-Link Adrenoleukodystrophy

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