New NIH Network Will Support Development of Novel Treatments for Rheumatoid Arthritis and Lupus

The National Institutes of Health (NIH) announced today that it has “awarded grants to 11 research groups across the United States to establish the Accelerating Medicines Partnership in Rheumatoid Arthritis and Lupus (AMP RA/Lupus) Network.”

The main Accelerating Medicines Partnership (AMP), launched earlier this year, is “a bold new venture between the NIH, 10 biopharmaceutical companies, and several non-profit organizations to transform the current model for developing new diagnostics and treatments by jointly identifying and validating promising biological targets of disease.” The ultimate goal of AMP is to “increase the number of new diagnostics and therapies for patients and reduce the time and cost of developing them.”

A major goal of the program is to “generate pre-competitive, disease-specific data that will be publicly accessible to the broad biomedical community for further research.”

The AMP RA/Lupus program will address relevant challenges for rheumatoid arthritis (RA) and systemic lupus erythematosus (SLE), while trying to “ascertain and define shared and disease-specific biological pathways in order to identify relevant drug targets for the treatment of autoimmune diseases. This program will involve an enhanced systems-level understanding of gene expression and signaling in target tissues from affected end organs (synovium for RA, kidney and skin for lupus) and blood cells.”

Research conducted under the AMP RA/Lupus program will follow a “predetermined set of steps to ensure standardization of procedures to minimize technical variability,” organized into three phases:

Research Phase 0

Testing of different means of obtaining and prepping tissue and some initial analytic runs will be conducted with the goal of developing standardized methods in small number of homogenous samples in at least two diseases. Samples must include affected organ specimens in addition to peripheral blood and could be newly obtained or already available.

Research Phase I

Analysis of a standardized analytic(s) successfully established in Research Phase 0, in blood and tissue cells, plus trial runs of additional selected analytics. The number of samples will be sufficient to inform power calculations and establish the feasibility of larger studies of the tissue analytic and sample type, source and acquisition approach. At least one Research Phase I study in RA and one Research Phase I study in lupus are expected to be completed by the end of the second year of the project. Research Phase I will include analysis of samples from individuals without RA or lupus, such that a systems biology approach can be used to identify pathways that distinguish disease and non-disease tissue (ie, comparison between disease and non-disease).

Research Phase II

Testing in larger patient populations will be conducted as determined by power calculations, priority, and budget. Patient stratification is expected for comparison within a disease (eg, RA: treatment responder vs. non-responder, early vs. established RA, comparison among disease-modifying anti-rheumatic drugs (DMARD) treated groups; lupus: before treatment vs. after treatment, comparison among types of kidney disease, etc). Initiation of Research Phase II studies requires a favorable recommendation by the AMP RA/SLE Steering Committee and approval by the NIH Program Official.

In the news release announcing the launch of AMP RA/Lupus Network, Stephen I. Katz, MD, PhD, director of the NIH’s National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), said that to date, “treatments for RA and lupus have been aimed at decreasing inflammation and pain. For the first time, we are bringing together multidisciplinary research teams to achieve a broad, systems-level understanding of these diseases, setting the stage for the development of more effective diagnostic and treatment approaches.”

Anthony S. Fauci, MD, director of the NIH’s National Institute of Allergy and Infectious Diseases (NIAID), said “This program promises to lead to more diagnosis and treatment options for rheumatoid arthritis and lupus. We also anticipate that the flexibility of the program will enable investigators to advance research on related diseases, thus improving our overall understanding of autoimmunity.”

Funding is provided by NIAMS and NIAID, and the following members of the AMP: AbbVie, Bristol-Myers Squibb, Merck, Pfizer, Sanofi, Takeda, the Arthritis Foundation, the Lupus Foundation of America, the Lupus Research Institute/Alliance for Lupus Research, and the Rheumatology Research Foundation.