scholarly journals Towards a global initiative for fibrosis treatment (GIFT)

2017 ◽  
Vol 3 (4) ◽  
pp. 00106-2017 ◽  
Author(s):  
Maria Molina-Molina ◽  
Alvar Agusti ◽  
Bruno Crestani ◽  
David A. Schwartz ◽  
Melanie Königshoff ◽  
...  
Keyword(s):  
Systems Biology ◽  
Research Strategy ◽  
New Drugs ◽  
Emergent Properties ◽  
Slow Disease Progression ◽  
European Respiratory Society ◽  
Disease Pathogenesis ◽  
Novel Drugs ◽  
Global Initiative ◽  
Society Research

Idiopathic pulmonary fibrosis (IPF) is a progressive lung disease characterised by increased scarring of lung tissue. Despite the recent introduction of novel drugs that slow disease progression, IPF remains a deadly disease, and the benefits of these new drugs differ markedly between patients.Human diseases arise due to alterations in an almost limitless network of interconnected genes, proteins, metabolites, cells and tissues, in direct relationship with a continuously changing macro- or microenvironment. Systems biology is a novel research strategy that seeks to understand the structure and behaviour of the so-called “emergent properties” of complex systems, such as those involved in disease pathogenesis, which are most often overlooked when just one element of disease pathogenesis is observed in isolation.This article summarises the debate that took place during a European Respiratory Society research seminar in Barcelona, Spain on December 15–16, 2016, which focused on how systems biology could generate new data by integrating the different IPF pathogenic levels of complexity. The main conclusion of the seminar was to create a global initiative to improve IPF outcomes by integrating cutting-edge international research that leverages systems biology to develop a precision medicine approach to tackle this devastating disease.

scholarly journals From systems biology to P4 medicine: applications in respiratory medicine

2018 ◽  
Vol 27 (147) ◽  
pp. 170110 ◽  
Author(s):  
Guillaume Noell ◽  
Rosa Faner ◽  
Alvar Agustí
Keyword(s):  
Systems Biology ◽  
Medical Diagnostics ◽  
Research Strategy ◽  
Emergent Properties ◽  
Chronic Obstructive ◽  
Network Systems ◽  
Obstructive Pulmonary Disease ◽  
P4 Medicine ◽  
Future Goals ◽  
Health And Disease

Human health and disease are emergent properties of a complex, nonlinear, dynamic multilevel biological system: the human body. Systems biology is a comprehensive research strategy that has the potential to understand these emergent properties holistically. It stems from advancements in medical diagnostics, “omics” data and bioinformatic computing power. It paves the way forward towards “P4 medicine” (predictive, preventive, personalised and participatory), which seeks to better intervene preventively to preserve health or therapeutically to cure diseases. In this review, we: 1) discuss the principles of systems biology; 2) elaborate on how P4 medicine has the potential to shift healthcare from reactive medicine (treatment of illness) to predict and prevent illness, in a revolution that will be personalised in nature, probabilistic in essence and participatory driven; 3) review the current state of the art of network (systems) medicine in three prevalent respiratory diseases (chronic obstructive pulmonary disease, asthma and lung cancer); and 4) outline current challenges and future goals in the field.

Novel Drugs and Biologics of 2016: A Boom for Neurodrugs in the Pipelin

2017 ◽  
Vol 10 (5) ◽  
pp. 3809-3814
Author(s):  
D Samba Reddy
Keyword(s):  
Muscular Atrophy ◽  
Clinical Care ◽  
New Drugs ◽  
Essential Medicines ◽  
High Demand ◽  
The Past ◽  
Novel Drugs ◽  
Innovative Products ◽  
Cns Drugs ◽  
The U.S

This article provides a brief overview of novel drugs approved by the U.S. FDA in 2016.  It also focuses on the emerging boom in the development of neurodrugs for central nervous system (CNS) disorders. These new drugs are innovative products that often help advance clinical care worldwide, and in 2016, twenty-two such drugs were approved by the FDA. The list includes the first new drug for disorders such as spinal muscular atrophy, Duchenne muscular dystrophy or hallucinations and delusions of Parkinson’s disease, among several others. Notably, nine of twenty-two (40%) were novel CNS drugs, indicating the industry shifting to neurodrugs. Neurodrugs are the top selling pharmaceuticals worldwide, especially in America and Europe. Therapeutic neurodrugs have proven their significance many times in the past few decades, and the CNS drug portfolio represents some of the most valuable agents in the current pipeline. Many neuroproducts are vital or essential medicines in the current therapeutic armamentarium, including dozens of “blockbuster drugs” (drugs with $1 billion sales potential).  These drugs include antidepressants, antimigraine medications, and anti-epilepsy medications. The rise in neurodrugs’ sales is predominantly due to increased diagnoses of CNS conditions. The boom for neuromedicines is evident from the recent rise in investment, production, and introduction of new CNS drugs.  There are many promising neurodrugs still in the pipeline, which are developed based on the validated “mechanism-based” strategy. Overall, disease-modifying neurodrugs that can prevent or cure serious diseases, such as multiple sclerosis, epilepsy, and Alzheimer’s disease, are in high demand. 

Herbal Medicine: Clinical Perspective & Regulatory Status

2020 ◽  
Vol 23 ◽  
Author(s):  
Md Abul Barkat ◽  
Anjali Goyal ◽  
Harshita Abul Barkat ◽  
Mohammad Salauddin ◽  
Faheem Hyder Pottoo ◽  
...  
Keyword(s):  
Herbal Medicine ◽  
Herbal Medicines ◽  
New Drugs ◽  
Herbal Drugs ◽  
Clinical Perspective ◽  
Regulatory Status ◽  
Novel Drugs ◽  
History Of ◽  
Pros And Cons ◽  
Health Complications

Abstract:: Herbal medicines pays an important in treating the vaious diseases mainly due to the their potentially high therapeutic values and also due to the better acceptance of vaioruspatient under different health complications. The herbal medicine practice involves use of part of plant, entire plant or the selectctive isolated phytomedicineand the use and practices based on these has its pros and cons and has been greatly affected during the dawn. The search of new drugs during scientific era revives the interest in discovery of herbal drugs from different natural resources during 20th century. The present modern healthcare system invovlves utilization drugs and 50% of them are of ofnaural origin. Herbal drug disocovery found to be highly costly affair with low success rate and it hinders the further progress in utilizting the phytomedicine in treating the various deseases. But in recent years there is an increase in the search interest of herbal drugs mainly by the pharmaceutical industry and those invoves in the search of novel drugs from the herbs. Discovery of such new novel phytomedicines has to overcomes various challenges in indentification of active extracts and their toxicity, advereffects, herb drug interaction and importantly their regulatory requirments. The present review mainly focused on the history of herbal medicine, current clinical perspective, pharmaceutical, and regulatory challenges as well as its clinical presentation. Moreover, problems encountered in drug discovery from herbal resources and its possible solutions are delineated.

scholarly journals Rare Treatments for Rare Dyslipidemias: New Perspectives in the Treatment of Homozygous Familial Hypercholesterolemia (HoFH) and Familial Chylomicronemia Syndrome (FCS)

2021 ◽  
Vol 23 (11) ◽  
Author(s):  
Laura D’Erasmo ◽  
Simone Bini ◽  
Marcello Arca
Keyword(s):  
Familial Hypercholesterolemia ◽  
Treatment Options ◽  
New Drugs ◽  
Lipid Lowering ◽  
Homozygous Familial Hypercholesterolemia ◽  
Published Evidence ◽  
Novel Drugs ◽  
Life Threatening ◽  
History Of ◽  
Definition Of

Abstract Purpose of Review This review aims to summarize the most recent published literature concerning lomitapide and volanesorsen that are approved for the use in HoFH and FCS patients, respectively. Moreover, it will briefly revise the published evidence on novel, non-approved treatments that are under evaluation for the management of these rare forms of dyslipidemias Recent Findings The definition of rare dyslipidemias identifies a large number of severe disorders of lipid metabolism of genetic origin. Among them were homozygous familial hypercholesterolemia (HoFH) (OMIM #143890) and familial chylomicronemia syndrome (FCS) (OMIM #238600), which are characterized by a markedly impaired cholesterol- and triglyceride-containing lipoproteins metabolism. They are being particularly associated with poor health outcomes and quality of life. Considering the severity of these diseases, common lipid-lowering drugs are often ineffective or do not allow to achieve the recommended lipid targets to prevent the development of complications. Nowadays, several new drugs have been found to effectively treat HoFH and FCS with an acceptable safety profile. Summary Treating patients with HoFH and FCS remains very challenging. However, novel treatment options are emerging and might be considered in addition to conventional therapy for managing these diseases. These novel drugs will possibly change the natural history of these two rare and life-threatening diseases.

scholarly journals Empowering Melatonin Therapeutics with Drosophila Models

Diseases ◽  
2021 ◽  
Vol 9 (4) ◽  
pp. 67
Author(s):  
Cassandra Millet-Boureima ◽  
Caroline C. Ennis ◽  
Jurnee Jamison ◽  
Shana McSweeney ◽  
Anna Park ◽  
...  
Keyword(s):  
Circadian Rhythm ◽  
Model Organism ◽  
New Drugs ◽  
Renal Diseases ◽  
Disease Pathogenesis ◽  
Multiple Functions ◽  
Functional Conservation ◽  
Oxidative Response ◽  
Pharmacological Potential ◽  
Drosophila Models

Melatonin functions as a central regulator of cell and organismal function as well as a neurohormone involved in several processes, e.g., the regulation of the circadian rhythm, sleep, aging, oxidative response, and more. As such, it holds immense pharmacological potential. Receptor-mediated melatonin function mainly occurs through MT1 and MT2, conserved amongst mammals. Other melatonin-binding proteins exist. Non-receptor-mediated activities involve regulating the mitochondrial function and antioxidant cascade, which are frequently affected by normal aging as well as disease. Several pathologies display diseased or dysfunctional mitochondria, suggesting melatonin may be used therapeutically. Drosophila models have extensively been employed to study disease pathogenesis and discover new drugs. Here, we review the multiple functions of melatonin through the lens of functional conservation and model organism research to empower potential melatonin therapeutics to treat neurodegenerative and renal diseases.

scholarly journals First analysis of the Severe Paediatric Asthma Collaborative in Europe registry

2020 ◽  
Vol 6 (4) ◽  
pp. 00566-2020
Author(s):  
Norrice M. Liu ◽  
Karin C.L. Carlsen ◽  
Steve Cunningham ◽  
Grazia Fenu ◽  
Louise J. Fleming ◽  
...  
Keyword(s):  
Asthma Control ◽  
Severe Asthma ◽  
Suboptimal Control ◽  
High Dose ◽  
Asthma Control Test ◽  
European Respiratory Society ◽  
Paediatric Asthma ◽  
Global Initiative For Asthma ◽  
Global Initiative ◽  
Long Acting

New biologics are being continually developed for paediatric asthma, but it is unclear whether there are sufficient numbers of children in Europe with severe asthma and poor control to recruit to trials needed for registration. To address these questions, the European Respiratory Society funded the Severe Paediatric Asthma Collaborative in Europe (SPACE), a severe asthma registry. We report the first analysis of the SPACE registry, which includes data from 10 paediatric respiratory centres across Europe.Data from 80 children with a clinical diagnosis of severe asthma who were receiving both high-dose inhaled corticosteroid and long-acting β2-agonist were entered into the registry between January 2019 and January 2020. Suboptimal control was defined by either asthma control test, or Global Initiative for Asthma criteria, or ≥2 severe exacerbations in the previous 12 months, or a combination.Overall, 62 out of 80 (77%) children had suboptimal asthma control, of whom 29 were not prescribed a biologic. However, in 24 there was an option for starting a licensed biologic. 33 children with suboptimal control were prescribed a biologic (omalizumab (n=24), or mepolizumab (n=7), or dupilumab (n=2)), and for 29 there was an option to switch to a different biologic.We conclude that the SPACE registry provides data that will support the planning of studies of asthma biologics. Not all children on biologics achieve good asthma control, and there is need for new trial designs addressing biologic switching.

Emergence

2017 ◽  
Author(s):  
Matthew Croasmun
Keyword(s):  
Systems Biology ◽  
Philosophy Of Science ◽  
Downward Causation ◽  
Feedback Loops ◽  
Emergent Properties ◽  
Middle Ground ◽  
Emergence Theory ◽  
Multiple Levels ◽  
Causal Structures

Emergence theory in philosophy of science is introduced, first in modest terms of the emergent properties exhibited by complex wholes that are not exhibited by their constituent parts. Then, emergence is treated as a trans-ordinal theory that stakes out a middle ground between reductionism and dualism. The tension between supervenience and downward causation is described as the generative dialectic of emergence. The coherence of downward causation is debated and ultimately affirmed on account of the prevalence of downward causation in the sorts of accounts produced by fields like systems biology. Racism is treated as a case study of the sorts of causal feedback loops generated by complex causal structures that operate at multiple levels of hierarchy.

scholarly journals An official European Respiratory Society/American Thoracic Society research statement: interstitial pneumonia with autoimmune features

2015 ◽  
Vol 46 (4) ◽  
pp. 976-987 ◽  
Author(s):  
Aryeh Fischer ◽  
Katerina M. Antoniou ◽  
Kevin K. Brown ◽  
Jacques Cadranel ◽  
Tamera J. Corte ◽  
...  
Keyword(s):  
Connective Tissue ◽  
Interstitial Pneumonia ◽  
Connective Tissue Disease ◽  
Task Force ◽  
American Thoracic Society ◽  
Classification Criteria ◽  
Future Research ◽  
Autoimmune Process ◽  
European Respiratory Society ◽  
Society Research

Many patients with an idiopathic interstitial pneumonia (IIP) have clinical features that suggest an underlying autoimmune process but do not meet established criteria for a connective tissue disease (CTD). Researchers have proposed differing criteria and terms to describe these patients, and lack of consensus over nomenclature and classification limits the ability to conduct prospective studies of a uniform cohort.The “European Respiratory Society/American Thoracic Society Task Force on Undifferentiated Forms of Connective Tissue Disease-associated Interstitial Lung Disease” was formed to create consensus regarding the nomenclature and classification criteria for patients with IIP and features of autoimmunity.The task force proposes the term “interstitial pneumonia with autoimmune features” (IPAF) and offers classification criteria organised around the presence of a combination of features from three domains: a clinical domain consisting of specific extra-thoracic features, a serologic domain consisting of specific autoantibodies, and a morphologic domain consisting of specific chest imaging, histopathologic or pulmonary physiologic features.A designation of IPAF should be used to identify individuals with IIP and features suggestive of, but not definitive for, a CTD. With IPAF, a sound platform has been provided from which to launch the requisite future research investigations of a more uniform cohort.

scholarly journals Strategy of medical treatment of pulmonary arterial hypertension in the current international recommendations

2016 ◽  
Vol 13 (2) ◽  
pp. 46-64
Author(s):  
T V Martyniuk ◽  
I E Chazova
Keyword(s):  
Pulmonary Arterial Hypertension ◽  
Arterial Hypertension ◽  
Optimal Choice ◽  
New Drugs ◽  
Reverse Remodeling ◽  
Significant Progress ◽  
European Respiratory Society ◽  
Specific Therapy ◽  
Pulmonary Arterial ◽  
European Society Of Cardiology

Significant progress in the treatment of pulmonary arterial hypertension (PAH), in recent years, is associated with the introduction into clinical practice of a number of drugs pathogenetic action, can cause vasodilation and reverse remodeling of pulmonary vessels. Recently PAH-specific therapy was replenished with new drugs. This review is created as a result of the analysis of modern American recommendations CHEST and recommendations of the European society of cardiology and the European respiratory society (ESC/ERS) in order to provide all professionals involved in the maintenance of the PAH, data on the main approaches to the pharmacotherapy and the optimal choice of pharmacological treatment methods.

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