By Nanotechnology Characterization Laboratory (NCL)
This application is intended for nanomedicines that address an unmet clinical need in cancer through a defined mechanism of action. Ideal candidates should have well-established synthesis consistently resulting in nanoparticles with expected physicochemical and biological properties, demonstrate proven in vivo efficacy, and be on a clear path toward clinical translation. NCL characterization is positioned to enable and accelerate this critical transition. Demonstrated Efficacy: Discuss findings from safety and efficacy studies. Studies should include all appropriate controls (e.g., standard of care comparison, nontargeted controls), use a cancer model appropriate to your intended indication, and use the intended clinical route of administration. Given the competitive nature of the application process, preference is given to applications that have in vivo efficacy data as opposed to only in vitro data and to innovative concepts containing novel active pharmaceutical ingredients designed to overcome current biological barriers for cancer drug delivery and provide substantial improvement in cancer therapy or diagnosis. Reformulation of legacy drugs that are already approved by the FDA for clinical use is no longer being considered. Anticipated Impact of Strategy: Describe the projected clinical use of the material and the intended biological mechanism(s) of action. Discuss the strategy's advantage when compared with existing therapeutics and diagnostics. If the strategy has benefits due to targeting or specificity, discuss the specific underlying mechanisms and include data to support these claims. Describe any significant findings related to safety or pharmacology endpoints, comparing these results with current therapeutics or devices. Previous Characterization of Material: Supply detailed information on assays that were previously used to characterize the material and discuss lot-to-lot variability. If the variability is large, physical and biological assays will not provide meaningful data, and the concept is not likely to be accepted into the program. In addition, discuss the shelf-life stability of the formulation. Since the NCL Assay Cascade involves extended characterization efforts, a minimal shelf-life of 3 months will be required for acceptance into the program. Manufacturing Process; Compatibility with Scale-Up: Briefly describe the manufacturing process and steps used during purification. Discuss impurities that may be present in the final product. Provide information on the current batch size of nanomaterial (e.g., milligram, gram, or kilogram scale). Discuss potential obstacles associated with producing enough material for completing the NCL Assay Cascade evaluation studies. Inherent Toxicity of Nanotechnology Concept: Include information on relevant safety issues related to the production, purification, handling and use of the nanomaterial. For example, if the nanomaterial contains a known toxic compound, discuss how the strategy overcomes or mitigates potential adverse health effects. Plan or Strategy to Transition the Concept to Clinical Use: Information related to teaming with industry, non-profit, academic, or other government partners in the translation effort is of interest to the NCL. If applicable, describe steps previously taken toward translation of the nanomaterial to clinical use. Discuss possible sponsors for future studies or trials and arrangements with commercial production firms. Discuss intellectual property issues related to the material, especially if the material uses licenses or represents an improvement or modification of an existing material or production process. If applicable, a brief summary of similar or closely related antecedents or approaches to the submitted nanomaterial should be described.
