October 23, 2014
Photo: Stephen I. Katz M.D. Ph.D.
Photo: Stephen I. Katz M.D. Ph.D.

Dear Colleagues:

In my April letter, I wrote about a new program to speed research that will lead to new diagnostics and therapeutics for the autoimmune diseases rheumatoid arthritis (RA) and lupus. I am pleased to report that the launch of the Accelerating Medicines Partnership (AMP) RA/Lupus Network was announced on September 24, 2014. This innovative initiative draws on the strengths of the public sector, industry, and academic institutions to address many of the barriers that have impeded autoimmune disease research. In this month’s letter, I will discuss a few of the exceptional aspects of this multi-sector, "team science" effort that aims to transform our understanding of and research on autoimmune diseases.

The goal of the AMP RA/Lupus Network is to define the shared and disease-specific biological pathways that drive the development and progression of these autoimmune diseases. Because achieving this goal will require the coordinated efforts of a broad range of clinical, experimental, technical, and analytical experts, we have assembled 11 multi-disciplinary teams with complementary strengths and resources. It is important to note that the Network is not intended to be a simple collection of related, but independent projects. Instead, it represents a new approach in which all activities will be selected, coordinated, and integrated by the Network investigators, rather than by individual sites. The research will also be guided by input from government, industry, and foundation experts, as well as by the Network investigators.

An important objective of autoimmune disease research is to understand the molecular and cellular mechanisms of tissue damage in patients with these diseases. For example, researchers are interested in mechanisms that contribute to kidney damage in lupus and joint damage in patients with RA. Working together, we are poised to overcome many of the challenges that have hampered such research. In this regard, the limited availability of clinical samples has been a significant barrier; as a result, much of the research to date has used animal models or generic cell lines that may not accurately reflect biological processes in patients. The Network includes several groups with expertise in clinical research, patient recruitment, and tissue acquisition, and these groups will help to ensure an adequate supply of clinical samples for studies, and to engage diverse cohorts of research participants.

Another technical limitation facing researchers has been the lack of tools to explore molecular events occurring in single cells or small subpopulations of cells within affected tissues. Most studies to date that have obtained data from whole tissues or blood samples, may miss important changes occurring at the single-cell level. The Network includes researchers with expertise in cutting-edge technologies to identify key targets that regulate disease pathways in single cells. Another challenge has been the lack of experimental, analytical, and computational approaches for developing a systems-level understanding of autoimmune diseases. The Network includes a systems biology group that will integrate data from multiple studies to generate a comprehensive, multi-dimensional map of RA and lupus.

The National Institute of Arthritis and Musculoskeletal and Skin Diseases is pleased to be a leader in this unprecedented public-private partnership, along with the National Institute of Allergy and Infectious Diseases. Although we expect that this Network will lead to vital new insights into the biology of RA and lupus, we hope that the model—integrating basic research with the study of well-defined patient cohorts and cutting-edge analytic and bioinformatic approaches—will be broadly applicable to research on other complex diseases. We also hope that this will be a useful example of robust cross-sector collaboration to advance collective goals and ultimately improve the lives of patients.

Stephen I. Katz, M.D., Ph.D.
National Institute of Arthritis and Musculoskeletal and Skin Diseases
National Institutes of Health

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