This Gordon Research Conference (GRC) on Lysosomal Disease, with its inaugural meeting scheduled for January, 2011, is one of the newest of over 170 annual conferences organized by the GRC, an organization known world-wide because of the high-quality, cutting-edge nature of its conferences. This new Lysosomal Disease meeting has evolved from the longstanding and highly successful Lysosomes and Endocytosis GRC, which for many years now has focused principally on the burgeoning field of intracellular trafficking and endocytic mechanisms, rather than lysosomal function/disease. Yet Lysosomal Disease represents a group of more than 50 disorders caused by defects in a wide spectrum of proteins, many of which remain poorly understood, and all of which compromise the function of the lysosomal system in critical ways and lead to serious illness and death of affected individuals. This meeting thus offers a critical new venue for addressing the major challenges involving lysosomal diseases, including the need for better understanding of (1) pathogenic mechanisms underlying dysfunction of neurons and brain, as well as other cell types/ organs/systems, (2) specific functions of non-enzymatic, enigmatic proteins causing some of the most difficult to treat lysosomal disorders (NPC1 and NPC2, CLN3, TRPML1, others) and (3) the relationship between specific lysosomal diseases and other chronic neurodegenerative conditions (e.g., Niemann-Pick type C and Alzheimer's, Gaucher and Parkinson's, and so forth). Additionally there is the challenge to develop (4) new and innovative therapies for these disorders coupled with (5) appropriate biomarker identification and validation, with this followed by (6) effective clinical trial development. To address these important topics our goal is to invite 40+ leading internationally known scientists and clinicians working in the field of lysosomal disease to join with approximately another 100 attendees, fully one-third of whom we anticipate will be junior investigators, postdoctoral fellows and graduate students who are at the beginning stages of their careers. The resulting discussion and cross fertilization of ideas and approaches we believe will accelerate not only our understanding of the role of the lysosomal system in health and in disease, but also move us closer to effective therapies for many of these disorders. Based on the large number of supporting emails that we have received from across the globe since the announcement of this new meeting by the GRC, we fully believe that this conference is going to meet a real unmet need for a genuine academic forum for the Lysosomal Disease field. Significance: Lysosomal diseases, while individually rare, together exhibit an incidence of 1:5000 to 7000 live births, making them as a group one of the more common families of genetic disorders. Yet academic meetings focused on the molecular and cellular pathogenesis and treatment of these diseases as a group is uncommon at best. We anticipate that the scientific discussions, research presentations and informal interactions between the participants in this new GRC will significantly contribute to advancing our understanding of these important and understudied disorders, and will also set the stage for development of innovative new therapies. This meeting, furthermore, will also foster the development of the all important next generation of scientists and clinicians focused on these disorders.
The Gordon Research Conference on Lysosomal Disease will bring together major leaders who are contributing to the understanding and treatment of lysosomal diseases, including those involved in basic science disciplines (genetics, biochemistry, cell biology, pathology, development and characterization of animal disease models etc.) as well as in the clinical sciences (pediatric geneticists and neurologists, clinical trial designers, etc.) along with graduate students and junior investigators who represent the future generation of researchers with interest in Lysosomal Disease. The scientific presentations, discussions and workshops during this conference will significantly contribute to advancing our understanding of the pathogenesis of lysosomal diseases, in terms of brain and other organs/systems, and to developing natural history studies and biomarker discovery, as well as new and innovative therapeutic strategies. Importantly, we are confident that the collegial and cooperative atmosphere of the GRC and the sharing of ideas and approaches, the capacity for cross fertilization between scientists and clinicians working on different lysosomal diseases with common challenges, will provide a basis for advancement of understanding and treating Lysosomal Disease in a manner unparalleled by other meetings.