University of Rochester
Grant: $1,113,166 - National Institutes of Health - Sep. 20, 2009
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Award Description: This application responds to RFA OD-08-001 Rare Diseases Clinical Research Consortia (RDCRC) for the Rare Diseases Clinical Research Network and proposes the renewal of U54 # NS059065 Nervous System Channelopathies: Pathogenesis and Treatment, focusing on the prototypic episodic disorders periodic paralysis, episodic ataxia and nondystrophic myotonia. It exploits the strengths of 10 collaborating centers in 4 countries to link molecular scientists with clinical investigators experienced in the development of new treatments for neurological disease. The training plan builds on the successful mentoring skills of translational scientists to develop a cadre of patient-oriented investigators prepared for careers in rare disease research. The Clinical Investigation of Neurologic Channelopathies (CINCH) has mutually supportive relationships with US and international patient advocacy groups: the MDA's of the US, Canada, and UK; the National Ataxia Foundation; and the international Periodic Paralysis Association. 4 US CINCH centers have recently funded CTSAs; the other 2 have CRCs (CTSA's pending). In the UK the National Hospital Queens Square investigator has one of 6 newly-funded translational Research Centers providing a rich infrastructure for research and training including the support of year-out medical student trainees. New CINCH projects will address the development of validated therapeutic targets in episodic disorders with innovative trial designs using novel patient-reported outcome measures piloted in the initial 6 years to better characterize episodic phenotypes and to devise treatments for Andersen-Tawil syndrome, other periodic paralysis patients currently without treatment options, and episodic ataxia-1. Companion studies funded by other grants will characterize the physiologic implications of channel dysfunction, identify the risk/triggering factors for the intermittent, recurrent symptoms, and collaborate with industry partners to bring new therapeutic agents to market. Insights from CINCH investigations hold promise for providing a window for a better understanding of many other episodic Channelopathies such as epilepsy and migraine; CINCH trainees will be well-positioned to pursue innovative treatments for both rare and more common diseases.
Project Description: This proposal completes the longitudinal and therapeutic clinical studies in three rare neurologic channelopathies, Andersen-Tawil Syndrome, episodic ataxias, and the non-dystrophic myontonias, initiated in the initial funding cycle. These studies will pave the way for phase 1, 2 clinical trials for neuromuscular channelopathies, employing novel outcome measures to establish treatment and working with industry and patient advocacy groups to bring treatment to patients. New applications will be submitted to fund these trials during the next 2 years.
Jobs Summary: This award was just issued no jobs have been created or retained to date. (Total jobs reported: 0)
Project Status: More than 50% Completed
This award's data was last updated on Sep. 20, 2009. Help expand these official descriptions using the wiki below.
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