DRAFT – WORK IN PROGRESS
Neural Phenotyping Core for Human and Mouse StudiesFrom Discovery to Human Clinical Trials
The path to move laboratory discoveries into human clinical trials is not clear or simple because basic researchers and clinicians reside in separate worlds that rarely intersect. Furthermore, clinicians who are skillful at evaluating and managing patients with diseases typically lack the skills required to design and implement clinical trials. To facilitate the movement of innovative basic research into clinical practice, infrastructure routes must be constructed to connect laboratory investigators with clinicians and to facilitate longitudinal studies and early clinical trials.
The most productive connections will likely be between basic scientists and clinical researchers and then between clinical researchers and clinicians.Creating a Neural Phenotyping Core
At CUMC and NYSPI, the clinical neurosciences – neurology, psychiatry, and neurosurgery – have excelled in defining and caring for disorders of the nervous system. CTNI proposes to marshal these outstanding resources to create a first-rate human Neural Phenotyping Core (NPC). This NPC will facilitate natural history studies and early clinical trials across the range from abnormal neurodevelopment to neurological and neuropsychiatric disorders to neurotrauma.
The NPC will serve as a central information repository of clinical neuroscience resources and as a catalyst to connect basic researchers involved in neurobiology of disease with expert clinical researchers. It will also enable clinicians to develop and implement effective and scientifically rigorous studies. The NPC will be a virtual core with:
- Centralized data on the clinical neuroscience activities at CUMC,
- A well-managed website with up-to-date information on clinicians and resources,
- A role as a clearing house with an understanding of the broad translational neuroscience landscape and expertise in the management of funds and collaborations,
- Funding to initiate small projects (e.g. to facilitate investigator-initiated, single-site, Phase 2 trials),
- Biostatistical and clinical trial design expertise to ensure that studies are adequately powered and efficient.
This initiative is expected to reduce costs and labor by facilitating efforts that are currently being performed by individuals who are not directly funded for these efforts but rather supported by resources borrowed from other projects.Overarching Extension to Animal Models
Another goal of the NPC is to develop probes to assess neural function (e.g. neurobehavioral, neurocognitive, neurophysiological, tasks for fMRI) that can be applied across species. We wish to validate and share these probes across groups/labs/departments and to develop new probes of general relevance. This will be achieved by developing a rodent NPC that will:
- Help with the design and implementation of rodent phenotyping tests that have translational relevance to the human disease modeled by the rodent and
- Be a centralized database of rodent models being used at CUMC to help direct PIs to existing models, and identify labs with overlapping interests. This should significantly streamline the process of obtaining and generating rodent models.