From Pathways of Motor Neuron Degeneration to Interventional Strategies

The goal of this meeting is to provide a dynamic forum for the active exchange of new discoveries and research tools in the advancing field of motor neuron diseases. Motor neuron diseases are a family of inherited and sporadic diseases affecting children and adults, typically are progressive and often fatal. The last 5+ years has seen tremendous growth in the understanding and development of relevant models for motor neuron diseases, such as amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA). This reflects the discovery of disease causing gene mutations, multiple transgenic animal, fish and fly models and disease relevant human patient derived iPS cell models of brain neurons and glia. The field has seen a convergence of multiple different threads of basic and clinical science disciplines- basic synaptic circuitry, RNA processing, nuclear trafficking, neuron-glial interactions, to whole genome, transcriptome, epigenome and proteomic approaches to diverse pathways, clinical subtypes and disease relevant drug targeting pathways. More recently, even wearable devices monitoring health- are intersecting these basic biological approaches. The conference will bring together experts in a wide range of basic, clinical and translational neuroscience to discuss advances in the progress of understanding common and rare motor neuron diseases. Exciting new areas of research in these disease include dysfunction in RNA metabolism and protein homeostasis, with specific defects in nucleocytoplasmic trafficking, induction of endoplasmic reticulum stress, and impaired dynamics of ribonucleoprotein bodies such as RNA granules. In addition, the use and validation of imaging modalities, such as PET, as well as large population multi-omics data generation, to identify and validate dysfunctional pathways in animal models and patients, as well as the development of new therapies and biomarkers will be discussed. The extraordinary recent progress in understanding the biology of ALS provides new grounds for optimism that meaningful therapies for ALS will be identified. Recent studies indicate that pathways and drugs that may be relevant to these diseases, have far wider applicability to other neurological injuries.