Therapeutic Development for Nervous System Disorders in the Absence of Animal Models: A Workshop

Workshop

September 12 - 13, 2016

Past

Materials

About

Although the prevalence and burden of nervous system disorders remains high, development of new therapeutics lags behind other disease areas. Gaps in understanding of the underlying mechanisms of disease, a dearth of biomarkers, and limitations in the capacity of animal models to predict drug efficacy for human brain disorders have contributed to a high rate of late stage failures in drug development. In 2012, the Forum on Neuroscience and Nervous System Disorders hosted a public workshop on Improving the Utility and Translation of Animal Models for Nervous System Disorders to discuss potential opportunities for maximizing the translation of effective therapies from animal models to clinical practice. During the workshop, several participants emphasized the utility of animal models for investigating basic neural processes, but their limitations for fully recapitulating nervous system disorders, and predicting therapeutic efficacy in human clinical trials. Given these concerns, the Forum hosted a second public workshop on Improving and Accelerating Therapeutic Development for Nervous System Disorders to explore opportunities and challenges in neuroscience research for accelerating entry of potential treatments into first-in-human trials. Workshop participants explored the potential usefulness of supplementing animal models of basic mechanisms with new technologies ranging from use of human induced pluripotent stem cells [iPSCs], to partially humanized animal models, to a greater emphasis on advancing human experimental biology.

Building on the discussions from these two activities, the Forum will host a public workshop to more deeply explore ways to motivate and accelerate drug development for nervous system disorders. The workshop will consider the evidence needed to bring compounds that appear to be safe into human efficacy trials both from an ethical and regulatory point of view and from a pragmatic and financial point of view in the absence of a predictive animal model. The workshop will bring together key stakeholders to discuss scientific, regulatory, and business challenges and to identify potential opportunities in this domain to motivate and accelerate therapeutic development to address unmet medical needs.

Meeting objectives:

Explore the utility of novel approaches to the process of target validation and biomarker development including human genetics, stem cell technologies, including use of iPSCs and human brain organoids, experimental human biology such as molecular imaging and neurophysiology, and computational modeling.
Discuss future technological developments that would facilitate bringing compounds that appear to be safe into human dose finding and efficacy trials, even if an animal model of the human disease is not achievable.
Discuss the regulatory landscape and what would be needed for regulatory agencies and institutional review boards to consider these approaches.
Explore the private sector environment for proceeding with drug development approaches in situations that lack animal models to predict drug efficacy.
Consider ethical issues, including for exploratory trials in pediatric populations.

As of March 2016, the Health and Medicine Division continues the consensus studies and convening activities previously undertaken by the Institute of Medicine (IOM).

Contact

Contact us

(202) 334-3265
neuroforum@nas.edu

Location

National Academy of Sciences Building

2101 Constitution Ave NW

Washington DC 20418, USA

Disclaimer

It is essential to the National Academies mission of providing evidence-based advice that participants in any of our meetings or events avoid political or partisan statements or commentary and maintain a culture of mutual respect. The statements and presentations during our meetings or events are solely those of the individual participants and do not necessarily represent the views of other participants or the National Academy of Sciences, which is a non-partisan, tax exempt organization that includes under its Charter the National Academy of Engineering and National Academy of Medicine, and that operates the National Research Council.

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Therapeutic Development for Nervous System Disorders in the Absence of Animal Models: A Workshop

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Therapeutic Development for Nervous System Disorders in the Absence of Animal Models: A Workshop