Accelerating Medicines Partnership – Schizophrenia

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Introduction

The Accelerating Medicines Partnership–Schizophrenia (AMP SCZ) is the first neuropsychiatric project of the landmark Accelerating Medicines Partnership (AMP) program managed by the Foundation for the National Institutes of Health (FNIH). This major public-private partnership between the National Institute of Mental Health (NIMH) of the National Institutes of Health, the FNIH, and diverse partners tackles the critical need for more effective treatments for people with schizophrenia. The five-year, $99 million global effort harnesses the capabilities of public and private partners to propel early interventions for individuals experiencing mental illness in the psychosis spectrum domain and improve their quality of life by reducing symptom burden. Detection and intervention before psychosis develops, when individuals are at clinical high risk (CHR) for psychosis, could attenuate, postpone or even prevent the transition to psychosis and improve individuals’ clinical and functional outcomes. AMP SCZ aims to develop measures that further define early stages of risk and predict the likelihood of progression to psychosis and other outcomes. Such tools will enable clinical trials to test new pharmacologic interventions that may prevent or delay the onset of psychosis. AMP SCZ partners will work toward the shared mission of discovering promising biological markers that can help identify those at risk of developing schizophrenia as early as possible; track the progression of symptoms and other clinical outcomes, including anxiety, depression and substance use disorders; and define targets for treatment development.

About Schizophrenia

Schizophrenia impacts the health and well-being of individuals and society. Schizophrenia is one of the top 15 leading causes of disability worldwide, and individuals with the disorder are at increased risk of premature death relative to the general population. In the United States, the estimated average potential life lost for individuals with schizophrenia is 28.5 years. Affecting approximately 20 million people worldwide, schizophrenia is one of the most recognized yet least-understood brain disorders. People diagnosed with schizophrenia often exhibit widely different symptoms that may present or subside along different timelines. Schizophrenia is most often associated with distortions in thinking and behavior, including delusions and hallucinations. Less recognized, however, are persistent cognitive symptoms—such as social withdrawal and diminished emotional expression—that have a profound adverse effect on patients’ ability to function across many domains of life and that are not effectively treated by current medications.

Need for New Therapies

Improving our understanding of the diverse nature of the disorder is critical to developing new treatments. While much is known about the risk factors for schizophrenia, including genetic links and differences in brain structure and function, more tools are needed to pinpoint those at clinical high risk for psychosis and to understand categories of such risk with increased accuracy and sensitivity. AMP SCZ will focus on those priority areas in order to refine clinical trials, enable evaluation of the efficacy of treatments, and support early detection and early intervention, all of which are key to better outcomes for patients. Dedicated funding provided by Wellcome helps leverage NIMH's significant contribution for AMP SCZ to set up an international research network focused on CHR populations, enhancing the applicability of research results to global clinical trials aimed at helping the approximately 1.5 million people diagnosed with schizophrenia worldwide each year. Data utility is further expanded through the use of a data platform allowing the broader biomedical research community open access to a robust collection of data and analysis extending reach and impact.

Data Access

A critical component of AMP SCZ is the rapid dissemination of research data to the scientific community using the National Institute of Mental Health (NIMH) Data Archive (NDA) platform, allowing faster translation of findings into tools and therapies. The NDA will provide cloud-based infrastructure to facilitate storage and analysis of AMP SCZ data. The data archive currently holds raw and derived data collected from a total of 500,000 research participants using a variety of measures, including clinical, biological, genetic and outcome measures. The NDA will work with a Data Processing, Analysis and Coordinating Center to make data analysis pipelines available to the research community. The archive, which was established in 2006, is sustained on an ongoing basis through NIMH funds.

Governance

The AMP SCZ steering committee (SC) is organized by the FNIH and includes representatives from each of the partner organizations. The SC operates under the direction of the overall AMP Executive Committee and is responsible for defining and maintaining the research plan, reviewing progress of the project and providing detailed assessment of milestones for AMP SCZ. Working groups created under the direction of the AMP SCZ SC will provide detailed technical recommendations for key scientific, policy or informatics issues that arise during implementation.

AMP Approach

AMP SCZ joins the four other AMP programs expediting discovery around Alzheimer’s disease, Parkinson’s disease, type 2 diabetes and rheumatoid arthritis and lupus, all coordinated by the FNIH since the 2014 launch of the large-scale public-private partnership initiative. The AMP partnerships use cutting-edge scientific approaches to bring new medicines and supports to patients by enhancing validation of novel, clinically relevant therapeutic targets and biomarkers. Click here to learn more about AMP.

Goals

Early intervention and treatment of mental health conditions are tied to better health outcomes, but no tools currently exist to identify individuals at early risk of developing schizophrenia. This makes it difficult to enroll at-risk individuals in clinical trials, which in turn hinders the development of new treatments. AMP SCZ will transform research in clinical high risk for psychosis and address crucial unmet patient needs by:

  • Spurring scientific discovery
    AMP SCZ will validate biomarkers that are needed to identify at-risk individuals and predict the likelihood of progression to psychosis and other outcomes. It will also enable the identification of these individuals for inclusion in clinical trials and pinpoint the metrics by which to assess early signs of a treatment’s efficacy.
  • Expanding reach
    In its first three years, the program will establish a research network with U.S. and international sites focused on clinical high-risk populations, making research results more applicable to clinical trials globally.
  • Setting the course for new research
    Upon completion of the project, AMP SCZ will have created a research framework that lays a foundation for future development of faster, more robust interventions.
  • Connecting the field
    Research data will be quickly disseminated to the broader scientific community through the NIMH Data Archive platform, allowing faster translation from findings to solutions.

Media

FNIH Press Release (September 15, 2020): Foundation for the NIH announces large-scale partnership to expedite treatments for schizophrenia

NIH Press Release (September 15, 2020): NIH partnership aims to advance early intervention for individuals at risk of developing schizophrenia

Partners

Public-Sector Partners:

National Institute of Mental Health (NIMH)
U.S. Food and Drug Administration (FDA)

Private-Sector Partners:

American Psychiatric Association Foundation*
Boehringer Ingelheim*
Janssen Research & Development, LLC*
National Alliance on Mental Illness*
One Mind*
Otsuka Pharmaceutical Development & Commercialization, Inc.*
Wellcome*

* Indicates partners that provide financial or in-kind support.

FNIH Contacts

Eline Appelmans, M.D., MPH, BMedSci, Scientific Program Manager, Neuroscience, eappelmans@fnih.org

Noam Keren, M.A., Scientific Project Manager, Neuroscience, nkeren@fnih.org