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Enabling partnerships for Alzheimer's disease drug development—meeting report

Enabling partnerships for Alzheimer's disease drug development—meeting report

It was the best of times, it was the worst of times... --Charles Dickens

These words sum up the state of Alzheimer’s disease (AD) research, specifically therapy development, over the last few years.

On the one hand, the budget climate and dismal therapeutic results cloud the future. On the other hand, there are tremendous opportunities presented by the U.S. National Plan to Address Alzheimer’s Disease and by the emergence of systems and precision medicine. These could transform AD research and drug development.

Why hold another meeting?

Failure is humbling. And we (government, academia, industry, and others) have failed to date to find an effective therapy for AD patients. We are looking for new ways to work together to meet this grand challenge.

What are we trying to accomplish?

Diagram of intersecting shapes reading 'Partnerships for Enabling Alzheimer's Disease Drug Development', April 20th, 2013, Bethesda, MD, also including logos for the National Institute on Aging, the National Institutes of Health, and the Department of Health and Human Services.Our goal is to gear up collaborations that will enable a well-integrated and seamless approach to AD research and drug development.

Toward this end, the NIA hosted a meeting in April of this year: Enabling Partnerships for AD Drug Development. We brought together more than 60 leaders from academia, the NIH, the FDA, foundations, public advocacy groups, and the biotech and pharmaceutical industries.

The meeting program was developed around key recommendations from the NIH AD Research Summit 2012. You can read more about those Summit recommendations and view the Summit videocast.

A series of thought-provoking presentations opened the meeting:

  • Stephen Friend of Sage Bionetworks shared his vision for making biomedical research more open source and illustrated how computational challenges can be used to crowdsource innovation. He talked about the need to redefine data sharing if we are to build predictive models of disease, given ever-increasing amounts of human “omic” data.
  • Joel Dudley of Mount Sinai Icahn Institute for Genomics and Multiscale Biology illustrated how network biology and translational bioinformatics can inform the selection of therapeutic targets and rational drug repurposing.
  • Michael Rogers of NIGMS and Julie Stone of Merck described the emergence of quantitative and systems pharmacology (PDF 1,294K), a model based, data-driven approach to drug development that integrates cell biology, tissue and organ physiology and pharmacology with various bioinformatic and “omic” approaches, and how this approach might inform key steps in drug development.
  • Chas Bountra of the Oxford University Structural Genomics Consortium spoke about precompetitive partnerships as tools for knowledge creation and presented a model of a partnership for clinical validation of novel targets that would expand the precompetitive space through phase II trials.

Spirited discussion and breakout sessions followed the talks. Participants explored how these new models of data sharing, translational science, and partnering can be adopted and adapted to AD research and drug development. We hope that these discussions will lead to new collaborations and ultimately new partnerships to accelerate AD therapy development.

Participants saw the meeting as an important source of new connections and opportunities. It was an important program development tool for us as well. Please get in touch with me or my colleagues Laurie Ryan and Larry Refolo if you want more information about the content of the meeting.

How can you learn about NIA meetings?

The best way to find out about NIH meetings in your area of interest is to ask your program officer. There are a number of us who facilitate AD and aging research at the NIA. You can ask me, or my colleagues.

You can also learn about Institute-Sponsored Meetings, Workshops, and Conferences in the thrice yearly National Advisory Council on Aging meeting updates.

Recent NIH meetings involving dementias and AD included Alzheimer's Disease-Related Dementias: Research Challenges and Opportunities, and Advancing Treatments for Alzheimer’s Disease in Individuals with Down Syndrome.

Have I answered all of your questions? Please join me in a conversation about AD drug development and partnerships in the comments section, below.

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Posted by Penny Dacks on Jul 12, 2013 - 1:58 pm
This was an important meeting that highlighted the potential opportunities of diverse computational approaches to better utilize the extensive datasets that have been gathered for dementia research. The Alzheimer's Drug Discovery Foundation, an independent non-profit that participated in this panel, has released a new request for proposals opportunity to encourage creative computational approaches to accelerate drug discovery for the treatment and prevention of dementia. More information about this funding opportunity and others can be found at www.alzdiscovery.org/research-and-grants/request-for-proposal.

Posted by Rima Kaddurah Daouk on Jul 16, 2013 - 12:27 pm
The issues raised at this meeting are of great importance and critical for bringing catalysis that can lead to change in current affairs where our limited understanding of disease pathogenesis continues to lead to failures in clinical trials. We have known for many years that there are metabolic failures that include glucose utilization and lipid metabolism in patients with Alzheimer’s disease and yet we have very limited knowledge about metabolic and signaling pathway failures that lead to these well documented observations. We have also known for decades that pathways are interconnected by utilization of shared substrates and by use of common cofactors suggesting that one needs to map metabolic failures at a pathway and network level. The detailed mapping of these biochemical changes is key for understanding at the molecular level mechanisms of disease and for developing novel approaches for drug discovery and drug development. >>> Recent advances in analytical chemistry led to the emergence of a new field called metabolomics. Metabolomics allows simultaneous measurement of 100’s to 1000’s of metabolites for mapping perturbations in interconnected pathways and in metabolic networks enabling a systems approach to the study of AD. To enable that approach sophisticated computational tools are needed as well as tolls for engineering among other tools. Also all this data can and should be used to inform other rich layers of data such as genomics transcriptomics proteomics and imaging. It would be poor investments if collective data cannot be synthesized and integrated to gain most knowledge and of course should be shared and made publically available for the research community at large. At these most difficult financial times we can’t afford to work in isolated islands it simply does not yield high return on investments. >>> Over the past decade we have started to assemble interdisciplinary research teams that include experts in metabolomics, genetics, engineering, imaging, AD clinical and basic research, biochemistry and bioinformatics, and have started to define perturbations in metabolic networks across the trajectory of several neurodegenerative and neuropsychiatric diseases. The idea is to leverage large investments that went into clinical studies and where samples are available and where layers of data exists and is underutilized. Totally new insights are emerging about pathway affected in each disease and some common across neuropsychiatric diseases. Such approach to research is impactful and can possibly transform our understanding of disease and can provide new roadmap for drug discovery yet it involves some fishing expedition and getting out of comfort zones. It creates new hypothesis that challenges old ones or improves upon them. It takes a community effort, openness, collaborations that break boundaries. We believe it is time for change and indeed at the most difficult times great opportunities emerge. The workshops that NIA has hosted over the past year and the effort of NIGMS in building systems biology and computational tools for biology are fantastic and open the door for a real change. The participants in this last workshop address right on many of the issues we are struggling with and need solutions for to enable development of more effective therapies for prevention and treatment of AD.