The urgency for therapeutics to prevent, delay and treat Alzheimer's is evident national and global as the epidemic of this disease grows with every passing minute. The overarching goal of the proposed planning grant is to address this need by building the foundation of intellectual, technological and infrastructure resources necessary to create an Alzheimer's Disease Translational Center for Predictive Drug Development. To capture the function of this future center, we refer to it as SysPharmRx-AD (Systems Pharmacology Therapeutics for Alzheimer's Disease). The SysPharmRx-AD vision is a portfolio of highly effective therapeutics personalized to etiology and stage of Alzheimer's disease. Ultimately, the mission of SysPharmRx-AD will be to provide expertise and resources that enable Alzheimer's researchers at all levels of translation from discovery to translational to clinical to effectively and efficiently develop therapeutics for AD. Goals of the planning grant wil be achieved through three Specific Aims designed to successively build the network of enabling technologies, infrastructure and organizational management required for SysPharmRx-AD, an Alzheimer's Disease Translational Center for Predictive Drug Development.
Specific Aim 1. Determine the enabling technologies and expertise required for identifying phenotypes of Alzheimer's disease risk, etiology of disease, stage of progression and systems based therapeutic targets. Objective of this aim is to develop the strategies required to know whom to treat, when to treat and what outcomes to measure as per NIA Alzheimer's Disease Summit recommendations.
Specific Aim 2 : Determine the enabling technologies and expertise required for translating systems biology targets into systems pharmacology based therapeutics. Objective of this aim is to develop quantitative systems pharmacology strategies for predictive drug development, whether novel or repurposed, that address the complex etiologies and progressive continuum of Alzheimer's disease, i.e. whom to treat, when to treat and with what to treat NIA Alzheimer's Disease Summit recommendations.
Specific Aim 3. Develop the organizational structure and function required to network enabling expertise, resources and infrastructure to create SysPharmRx-AD. Objectives of this aim are to: a) integrate capacities and resources to ensure seamless functionality and communication within SysPharmRx-AD that enable rapid and broad public sharing of data, analytical and research tools, and models prior to publication and b) address barriers to public private partnerships between academic, pharmaceutical / biotech and Alzheimer's philanthropic organizations to enable SysPharmRx-AD mission.
Our goal is to develop therapeutics to prevent, delay and cure Alzheimer's disease. The proposed planning grant to create an Alzheimer's disease Translational Center for Predictive Drug Development is a critical step to realizing this goal.