Therapeutic Development in the Absence of Predictive Animal Models of Nervous System Disorders Proceedings of a Workshop (2017) / Chapter Skim
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2 Drug Development for Nervous System Disorders: Overview of Challenges and Potential Opportunities
2 Drug Development for Nervous System Disorders: Overview of Challenges and Potential Opportunities
Pages 5-14
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From page 5... ...
Steven Hyman added that the process of target validation requires understanding disease mechanisms, and traditionally this has been accomplished in animal models. Zorn said there are currently no, and may never be, predictive animal models of complicated human diseases such as nervous system disorders.
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Noonetheless, annimal modelss remain criti cal to drug ddiscovery, esppecially for lead optimmization, said d Michelson. T They are a hiighly predictiive means to assessing the safety, metabolism, m annd absorptionn characteristiics of a coompound, alllowing decon nstruction of a drug's pharrmacology inn a way thhat cannot bee done in hum mans.
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From page 7... ...
A better approach, he said, would be to have discovery scientists work together with clinical scientists and clinical pharmacologists throughout the research and development process. In this workshop, the challenges facing drug development were broken down into three groups: those related to preclinical issues, including target identification and validation; those that specifically relate to new approaches in animal models of disease; and those that relate to research infrastructure and resources.
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From page 8... ...
The subsequent challenge is determining how preclinical markers identified in these models can be translated into clinical endpoints, he added. Many animal models are based on an increased understanding of human genetics; however, these genetic models present many challenges, said Steven McCarroll, director of genetics at the Stanley Center for Psychiatric Research at the Broad Institute of Massachusetts Institute of Technology and Harvard University.
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From page 9... ...
Research Infrastructure and Resources Several research collaborations and partnerships were represented at the workshop, and a number of participants spoke of the need for improved infrastructure and resources, including a broader array of collaborative ventures, such as those that would integrate research and discovery with clinical testing efforts. Several participants, including Frances Jensen, professor and chair of neurology at the University of Pennsylvania Perelman School of Medicine, also commented on the inadequacy of workforce training for neuroscience research and the lack of trained clinicians working at the preclinical‒experimental medicine interface to better enable translation of preclinical findings to clinical studies.
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From page 10... ...
The potential opportunities summarized here are discussed in greater detail throughout the proceedings. A major theme throughout the workshop, articulated by Nita Farahany, professor of law and philosophy at the Duke University School of Law, was the need to ensure that ethical and regulatory issues are considered throughout the research process, beginning at the preclinical stage of research.
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From page 11... ...
. • Integrating human observational assessments in biomarker stud ies might help researchers identify translatable biomarkers for validation (Zorn)
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From page 12... ...
, or monogenic diseases might provide the best opportunity for advancing CNS drug development (Feng, Jensen, Rubin, Yocca)
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From page 13... ...
. • Instituting policies that encourage risk sharing, improve access to data, simplify clinical study requirements, invest in regulatory science, and extend patent protection could increase the proba bility of success for drug development (Martin)
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