DoD Amyotrophic Lateral Sclerosis, Therapeutic Development Award - 0 views
-
MiamiOH OARS on 12 Apr 18The Therapeutic Development Award supports research ranging from validation of therapeutic leads to U.S. Food and Drug Administration (FDA) Investigational New Drug (IND)-enabling studies. The proposed studies are expected to be empirical in nature and product-driven. Applicants with limited ALS experience are strongly encouraged to collaborate with those having substantial expertise in ALS research and/or ALS model systems. Examples of activities that will be supported by this award include: * Confirmation of candidate therapeutics obtained from screening or by other means, including optimization of potency and pharmacological properties and testing of derivatives and sister compounds * Validation of early pilot studies, including the use of multiple ALS model systems and/or replicating preliminary data with more time points or additional doses * Studies on formulation and stability leading to Good Manufacturing Practice (GMP) production methods * IND-enabling studies, to include compound characterization, absorption, distribution, metabolism, and excretion (ADME) studies, and dose/response and toxicology studies in relevant model systems Validation of treatment approaches in appropriately powered and controlled studies using biological correlates of disease activity and progression in pre-existing, de-identified human specimens from well-characterized patient cohorts is encouraged. Examples of acceptable cohorts for study include controlled clinical trials, observational studies, and registries (e.g., Centers for Disease Control and Prevention National ALS Registry and/or Biorepository; https://wwwn.cdc.gov/als/).