Treatment of Cystic Fibrosis and Other Rare Lung Diseases

Treatment of Cystic Fibrosis and Other Rare Lung Diseases

This volume describes the pathogenesis and pathophysiology of several pulmonary diseases as well as their treatment. It also discusses the underlying genetic and molecular biological basis, which opens the way for new treatments for these conditions.

It focuses on the treatment of cystic fibrosis including CFTR (cystic fibrosis transmembrane-conductance regulator) modulator therapies, drug therapies that augment airway surface liquid as well as anti-inflammatory and anti-infective therapies. Further topics include long-term, low-dose macrolide therapy for diffuse panbronchiolitis; novel agents for previously untreatable idiopathic pulmonary fibrosis; possible new treatments for pulmonary alveolar proteinosis (PAP); and multiple novel therapeutic targets for treating lymphangiomyomatosis. Research into these conditions has led to major advances in our understanding of the underlying genetic and molecular basis of this disease, and to dramatic improvements in survival and quality of life for affected individuals.

Some of the most exciting discoveries in pulmonary medicine have come from studying rare diseases. Insights gained from uncommon lung diseases often shed light on normal physiology as well as the mechanism of more common lung diseases. For example, investigations into cystic fibrosis (CF) have clarified the role of innate defense and mechanism of mucociliary clearance in the airway, as well as the function of the cystic fibrosis transmembrane conductance regulator protein in maintenance of airway surface liquid. The study of lymphangioleiomyomatosis (LAM) has led to an understanding of genes that control cell energy utilization, growth, and movement, potentially lending insights into the cellular and molecular basis of cancers. An understanding of the role of granulocyte macrophage colony-stimulating factor (GM-CSF) in the regulation of surfactant and other components of the complex biological systems in lung host defense as seen in pulmonary alveolar proteinosis (PAP) has led to GM-CSF being developed as an immunity-enhancing treatment for several other diseases. Drug development for these conditions has been limited by a lack of understanding of the underlying mechanisms of disease and the relative unavailability of
subjects for clinical trials, as well as the prohibitive cost of investing in novel pharmaceutical agents with poor market potential. Over the last several decades, however, legislation has been created in the USA (1983), Japan (1993), Australia (1998), and Europe (2000) to provide incentives for the commercial development of new “orphan drugs” to treat rare diseases, including those of the lung. In the USA, the National Institutes of Health has established the Rare Lung Diseases Consortium within its Office of Rare Diseases, and patient advocacy organizations such as EURORDIS are valuable allies in the fight against rare lung disease, by educating, supporting, and organizing patients and families in a manner that facilitates research. Central databases, registries, and research networks such as the Rare Lung Diseases Consortium in the USA and Orphanet in Europe are also useful adjuncts. With such rare disorders, international cooperation is critical for accumulating sufficient numbers of patients for research.
This volume of  Milestones in Drug Therapy is dedicated to a discussion of a somewhat arbitrarily chosen group of rare lung disease—we must point out that many others, in which exciting new research is currently being performed, could have been chosen, but were not, in the interest of keeping the volume from being too overwhelming. We have included introductory chapters on the pathogenesis and current standard treatment of the diseases of interest, followed by chapters discussing the biologic basis of current and new investigational treatments for those conditions. We pay special attention to CF, which incidentally serves as a
special example of successful alignment of governmental, academic, foundational, and pharma resources; research into this condition has led to extraordinary advances in our understanding of the underlying genetic and molecular basis of this disease and to dramatic improvements in survival and quality of life for affected individuals. Chapters in Part II focus on treatments directed toward the sequential well-defined steps in the pathogenic pathway of the disease. Additional chapters in Parts I and III discuss diffuse panbronchiolitis, for which the salutary effect of macrolides has been brought to attention due to their anti-inflammatory effect and potential benefit in a host of other inflammatory conditions; idiopathic pulmonary fibrosis, a previously untreatable condition that is now becoming better characterized and for which several effective drugs have become available, with others on the horizon; PAP, a disease for which the discovery of the underlying
abnormality related to GM-CSF offers promise of effective treatment; and LAM, for which identification of the key role of dysregulation of the mTOR pathway has identified multiple novel therapeutic targets.We wish to thank Jutta Lindenborn, our editorial contact from Springer, for keeping us on track and assisting in the organization of contributions from our valued chapter contributors, who of course did most of the “heavy lifting” for this volume. We hope this contribution will be of benefit to clinicians, students, and researchers looking for an introduction into the current investigations that are taking place in regard to the diseases discussed

View Fullscreen

Treatment of Cystic Fibrosis and Other Rare Lung Diseases PDF

Treatment of Cystic Fibrosis and Other Rare Lung Diseases Ebook

Treatment of Cystic Fibrosis and Other Rare Lung Diseases PDF Free

Download Treatment of Cystic Fibrosis and Other Rare Lung Diseases

List Price:

$159.99 USD

New From:

$119.38 USD

Download Ebook pdf

Get it $10 USD

Medical Books

I' m Library book medical. Download free.............

Leave a Reply

Your email address will not be published. Required fields are marked *