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Published on: October 5, 2015
Cellular Therapy for the Treatment of Paediatric Respiratory Disease
Laura C Brennan1, Andrew O'Sullivan2, Ronan MacLoughlin2,3,4
1College of Medicine, Nursing & Health Sciences, National University of Ireland, H91 TK33 Galway, Ireland.
Insights
Cellular therapies show promise for curing pediatric respiratory diseases like bronchopulmonary dysplasia and cystic fibrosis. Further research is needed to optimize delivery methods for these lung conditions in children.
Area of Science:
- Pediatric Pulmonology
- Regenerative Medicine
- Cellular Therapy
Background:
- Respiratory diseases are a leading cause of mortality in children under five.
- Current treatments for pediatric lung diseases alleviate symptoms but do not provide cures.
- Cellular therapies offer potential curative treatment, halting disease progression into adulthood.
Purpose of the Study:
- To review the potential of cellular therapies for pediatric respiratory diseases.
- To highlight the need for pediatric-specific research and clinical trials.
- To emphasize the importance of optimizing cell dosage and delivery methods for children.
Main Methods:
- Review of preclinical models and existing research on cellular therapies (induced pluripotent stem cells, mesenchymal stromal cells, secretome).
- Analysis of current clinical trial focus on adult populations.
- Discussion of direct aerosol delivery to the lungs for enhanced efficiency and reduced systemic effects.
Main Results:
- Preclinical studies demonstrate the potential of cellular therapies in targeting key pathological features of lung disease.
- Research and clinical trials are predominantly focused on adult patients.
- Optimal cell type, dosage, and delivery methods require establishment for clinical translation.
Conclusions:
- Cellular therapies hold significant promise for treating pediatric respiratory diseases.
- A pediatric-centric approach is crucial due to differing pathology and anatomy.
- Further research and clinical trials are essential to translate these promising therapies into effective treatments for children.
Abstract:
Respiratory disease is the leading cause of death in children under the age of 5 years old. Currently available treatments for paediatric respiratory diseases including bronchopulmonary dysplasia, asthma, cystic fibrosis and interstitial lung disease may ameliorate symptoms but do not offer a cure. Cellular therapy may offer a potential cure for these diseases, preventing disease progression into adulthood. Induced pluripotent stem cells, mesenchymal stromal cells and their secretome have shown great potential in preclinical models of lung disease, targeting the major pathological features of the disease. Current research and clinical trials are focused on the adult population. For cellular therapies to progress from preclinical studies to use in the clinic, optimal cell type dosage and delivery methods need to be established and confirmed. Direct delivery of these therapies to the lung as aerosols would allow for lower doses with a higher target efficiency whilst avoiding potential effect of systemic delivery. There is a clear need for research to progress into the clinic for the treatment of paediatric respiratory disease. Whilst research in the adult population forms a basis for the paediatric population, varying disease pathology and anatomical differences in paediatric patients means a paediatric-centric approach must be taken.
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