Pilot Feasibility Core

PILOT FEASIBILITY ABSTRACT The pilot studies program for MGNet supports highly meritorious investigations addressing clinical trial readiness, biomarker discovery, and treatment of myasthenia gravis (MG). The core integrates with the MGNet’s overall activities, including the career enhancement program, when opportunities arise. The pilot studies program targets diverse disciplines including basic science investigators, clinicians, and allied health professionals. The core’s specific aims demonstrate the tight integration with MGNet’s overall aims of as well as the patient advocacy groups, MG Foundation of America (MGFA) and Conquer MG, which support the pilot program financially and promotion of the program to investigators. We have four specific aims. Aim 1: Strengthen and expand on existing hypothesis-driven, investigator-initiated discovery research program of our patient advocacy partners. Aim 2: Establish a mechanism to leverage the developing clinical data and specimen biobank of MGNet for broad use by the MG research community, in particular biomarker discovery and validation. Aim 3: Provide a formal mechanism for investigators with a broad spectrum of expertise in the MG and the greater scientific community to access the expertise of MGNet investigators and the broader RDCRN resources. Aim 4: Expand and improve pilot program science and reach by providing support for research development, rigorous peer-review and formal meetings of grantees during the course of their period of funding and at the MGNet Annual Meeting. In the first five years of funding, we have demonstrated that the pilot program has been successful in the areas of clinical research, basic science and clinical trial readiness for MG. We have integrated the funded investigators into MGNet’s support network.