Children with childhood diffuse and interstitial lung diseases (chILD) are afflicted with rare, life-threatening, high-morbidity disorders that are poorl understood, under-recognized, and have no known evidence-based therapy. Adult ILD paradigms do not generally apply to chILD entities, which include recently-described disorders such as surfactant dysfunction mutations (SDM), Pulmonary Interstitial Glycogenosis (PIG), and Neuroendocrine Cell Hyperplasia of Infancy (NEHI). However, many of these rare disorders hold clues to understanding normal lung biology, and may unravel aspects of more common diseases. Rare disorders have limited patient populations, requiring novel and interdisciplinary approaches to define clinical phenotypes, genotypes, molecular pathogenesis, predictive factors, biomarkers, and therapeutic targets. Breakthroughs in other rare diseases have recently been catalyzed by the efforts of multidisciplinary teams spurred on by passionate patient advocacy groups. There has not been an interdisciplinary scientific meeting focused on these entities in over five years. This workshop will bring together leaders from clinical, basic science and biotechnology fields, together with chILD family advocates, to promote novel cross-cutting approaches to promote scientific breakthroughs for these clinically vexing disorders. The conference will take place in the summer of 2012 in La Jolla, CA. The objectives of the conference are to: 1) showcase cutting-edge scientific knowledge regarding lung biology relevant to childhood diffuse and interstitial lung disease, including lung development, cellular programming, remodeling and regeneration, 2) promote and encourage young scientists, underrepresented minorities, and persons with disabilities to continue their work in this field and to pursue careers in science, 3) review emerging technologies that will drive breakthroughs for these rare disorders, and 4) promote novel interactions among the fields of developmental biology, stem cell biology, respiratory medicine, neonatology, genomics, epigenetics and bioengineering, that will ultimately facilitate development of innovative and effective therapies. The conference will bring together leaders and future leaders from these disciplines to address core questions surrounding lung growth, molecular pathogenesis of chILD disorders, and application of emerging technologies for molecular phenotyping and therapeutic discovery. The workshop will provide attendees with a look beyond current state of the art and a pathway to the future.
Relevance to Public Health: Childhood diffuse and interstitial lung diseases (chILD), although rare, often hold biological clues for understanding diseases which are common and challenging to treat, such as idiopathic pulmonary fibrosis and chronic obstructive pulmonary disease. Scientific breakthroughs for chILD may come from a broad spectrum of scientific disciplines. By bringing together leaders from the fields of developmental biology, stem cell biology, respiratory medicine, neonatology, genomics, epigenetics and bioengineering, along with parent advocates of children affected by these disorders, this unique conference will spur new collaborative approaches that may provide breakthroughs for many individuals struggling with chILD or other diffuse lung diseases.
|Hamvas, Aaron; Deterding, Robin; Balch, William E et al. (2014) Diffuse lung disease in children: summary of a scientific conference. Pediatr Pulmonol 49:400-9|