Pulmonary alveolar proteinosis – current and future therapeutical strategies

Jehn, Lutz-Bernhard; Bonella, Francesco

doi:10.1097/mcp.0000000000000982

Cited by 4 publications

(3 citation statements)

References 111 publications

(130 reference statements)

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“…In the presence of anti-GM-CSF autoantibodies, efflux of cholesterol in AMs is limited, resulting in interrupted surfactant catabolism and excessive accumulation of surfactant lipids and proteins in the alveolar space 7 12 13 14 . Today, the therapy of first choice for autoimmune PAP is whole-lung lavage (WLL), which involves the repetitive intra-pulmonary installation of saline solution to remove the accumulated surfactant 15 16 . New attempts concentrate on the direct inhalation of high dose GM-CSF, showing promising results in patients with autoimmune PAP 17 18 and several clinical trials are ongoing (see 16 ).…”

Section: The Current Clinical View On Pulmonary Alveolar Proteinosis ...mentioning

confidence: 99%

“…Today, the therapy of first choice for autoimmune PAP is whole-lung lavage (WLL), which involves the repetitive intra-pulmonary installation of saline solution to remove the accumulated surfactant 15 16 . New attempts concentrate on the direct inhalation of high dose GM-CSF, showing promising results in patients with autoimmune PAP 17 18 and several clinical trials are ongoing (see 16 ).…”

Section: The Current Clinical View On Pulmonary Alveolar Proteinosis ...mentioning

confidence: 99%

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Pulmonary Alveolar Proteinosis and new therapeutic concepts

Rodriguez Gonzalez,

Schevel,

Hansen

et al. 2024

Klin Padiatr

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Pulmonary alveolar proteinosis (PAP) is an umbrella term used to refer to a pulmonary syndrome which is characterized by excessive accumulation of surfactant in the lungs of affected individuals. In general, PAP is a rare lung disease affecting children and adults, although its prevalence and incidence is variable among different countries. Even though PAP is a rare disease, it is a prime example on how modern medicine can lead to new therapeutic concepts, changing ways and techniques of (genetic) diagnosis which ultimately led into personalized treatments, all dedicated to improve the function of the impaired lung and thus life expectancy and quality of life in PAP patients. In fact, new technologies, such as new sequencing technologies, gene therapy approaches, new kind and sources of stem cells and completely new insights into the ontogeny of immune cells such as macrophages have increased our understanding in the onset and progression of PAP, which have paved the way for novel therapeutic concepts for PAP and beyond. As of today, classical monocyte-derived macrophages are known as important immune mediator and immune sentinels within the innate immunity. Furthermore, macrophages (known as tissue resident macrophages (TRMs)) can also be found in various tissues, introducing e. g. alveolar macrophages in the broncho-alveolar space as crucial cellular determinants in the onset of PAP and other lung disorders. Given recent insights into the onset of alveolar macrophages and knowledge about factors which impede their function, has led to the development of new therapies, which are applied in the context of PAP, with promising implications also for other diseases in which macrophages play an important role. Thus, we here summarize the latest insights into the various forms of PAP and introduce new pre-clinical work which is currently conducted in the framework of PAP, introducing new therapies for children and adults who still suffer from this severe, potentially life-threatening disease.

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Section: The Current Clinical View On Pulmonary Alveolar Proteinosis ...mentioning

confidence: 99%

Section: The Current Clinical View On Pulmonary Alveolar Proteinosis ...mentioning

confidence: 99%

Pulmonary Alveolar Proteinosis and new therapeutic concepts

Rodriguez Gonzalez,

Schevel,

Hansen

et al. 2024

Klin Padiatr

View full text Add to dashboard Cite

show abstract

“…Pulmonary alveolar proteinosis represents another rare lung disease [40]. To date, whole lung lavage (WLL) remains the gold standard treatment for PAP syndrome.…”

Section: Other Genetic Lung Diseasesmentioning

confidence: 99%

Unlocking the Future: Pluripotent Stem Cell-Based Lung Repair

Goecke,

Ius,

Ruhparwar

et al. 2024

Cells

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The human respiratory system is susceptible to a variety of diseases, ranging from chronic obstructive pulmonary disease (COPD) and pulmonary fibrosis to acute respiratory distress syndrome (ARDS). Today, lung diseases represent one of the major challenges to the health care sector and represent one of the leading causes of death worldwide. Current treatment options often focus on managing symptoms rather than addressing the underlying cause of the disease. The limitations of conventional therapies highlight the urgent clinical need for innovative solutions capable of repairing damaged lung tissue at a fundamental level. Pluripotent stem cell technologies have now reached clinical maturity and hold immense potential to revolutionize the landscape of lung repair and regenerative medicine. Meanwhile, human embryonic (HESCs) and human-induced pluripotent stem cells (hiPSCs) can be coaxed to differentiate into lung-specific cell types such as bronchial and alveolar epithelial cells, or pulmonary endothelial cells. This holds the promise of regenerating damaged lung tissue and restoring normal respiratory function. While methods for targeted genetic engineering of hPSCs and lung cell differentiation have substantially advanced, the required GMP-grade clinical-scale production technologies as well as the development of suitable preclinical animal models and cell application strategies are less advanced. This review provides an overview of current perspectives on PSC-based therapies for lung repair, explores key advances, and envisions future directions in this dynamic field.

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Learning from cystic fibrosis: How can we start to personalise treatment of Children’s Interstitial Lung Disease (chILD)?

Bush

2024

Paediatric Respiratory Reviews

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Pulmonary alveolar proteinosis – current and future therapeutical strategies

Cited by 4 publications

References 111 publications

Pulmonary Alveolar Proteinosis and new therapeutic concepts

Pulmonary Alveolar Proteinosis and new therapeutic concepts

Unlocking the Future: Pluripotent Stem Cell-Based Lung Repair

Learning from cystic fibrosis: How can we start to personalise treatment of Children’s Interstitial Lung Disease (chILD)?

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