Accelerating Medicines Partnership Advances Research for Autoimmune Diseases

Autoimmune diseases are receiving record levels of national attention during this time of unparalleled scientific discovery. Last fall, National Institutes of Health (NIH) Director Francis Collins, MD, PhD, spoke at a conference in New York presented jointly by the Lupus Research Institute and the Alliance for Lupus Research, in part, to promote that rheumatoid arthritis (RA) and systemic lupus erythematosus (SLE) have been included in the diseases to be targeted by the Accelerating Medicines Partnership (AMP). AMP is a partnership between the NIH, the U.S. Food and Drug Administration, 10 biopharmaceutical companies and multiple nonprofit organizations to find new diagnostics and treatments by jointly identifying and validating promising biological targets for therapeutics.

For an update, The Rheumatologist recently spoke with Robert Carter, MD, deputy director of the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), one branch of the NIH.

Question: Why were RA and SLE included in the debut of AMP?
Answer: The selection of autoimmune diseases as an AMP focus was based on extensive polling of the NIH and industry partners to identify the most compelling research needs and opportunities. Given the genetic overlap in lupus and RA, it was felt that it would be advantageous to work on them together. This approach could point to targets that are shared across several autoimmune conditions.

Q: Should rheumatologists be hopeful that when the initial five-year program expires, future rounds of the program may also include autoimmune diseases?
A: It was the hope and expectation that the pilot projects would set the stage for broadening AMP to other diseases and conditions. The partnership will make the data available to the broad research community for further analysis. We expect [those data] to benefit the entire community and spur future research. However, it is important to note that, at least in this round, both NIH and industry developed the project, and future projects may also need to identify potentially interested partners.

Q: For rank-and-file rheumatologists, what is the takeaway from AMP?
A: As a rheumatologist, I understand the many challenges in caring for patients with diseases, such as RA and lupus. Most people with RA have only a partial response to available drugs and many only respond to drugs for a limited period of time. In the case of lupus, no effective targeted therapies exist for the most severe forms of the disease.

With AMP, the focus is clearly on the disease tissues and associated changes in the blood. We believe that our ability to develop better biomarkers and design better clinical trials will be made possible through the AMP. Insights gained should help reveal the most promising new biological targets for drug development and match existing drugs to patients with specific molecular profiles who are the most likely to benefit. Essentially, the AMP helps lay a foundation for precision medicine for autoimmune diseases. NIH Director Francis Collins has said that precision medicine can be thought of as the opposite of the standard one-size-fits-all medicine. We hope that the AMP and other efforts will enable doctors to better tailor treatment for each individual patient.

Q: Dr. Collins has spoken frequently on the leaps scientific discovery has taken in recent years. How encouraged are you about the possibility of autoimmune disease research moving forward?
A: We live in a period of tremendous opportunity in science, technology and medicine. I would encourage researchers to focus on what they see as the most exciting scientific questions. The AMP provides an excellent example of how technological advances enable researchers to ask and answer questions that we did not have the tools to address previously. New technologies to analyze single cells and small groups of cells, and the ability to develop computational tools to integrate different data types to understand the molecular pathways are particularly exciting.

The AMP is also a great model of how innovative partnerships are advancing research. It draws on the strengths of the public sector, industry and academic institutions to overcome many of the barriers that have impeded autoimmune disease research. AMP, both with the focus on disease tissues and the application of emerging technologies, is already influencing the landscape.

Q: Do you see cures as feasible for rheumatic and other autoimmune diseases? Or is strong management the area that needs to be focused on, and why?
A: Treatments for RA and lupus have typically focused on decreasing inflammation and pain by slowing down tissue damage and reducing complications. We believe that if we drill down on the single cells of the immune system and try to understand how they change during disease, we will be able to restore normal immune function and bring back quality of life, potentially even reversing the disease process in RA and lupus.

Q: What would you say to rheumatologists frustrated by rejected research grants because NIH has limited funding?
A: Each year, the NIH receives many worthy grant applications from talented researchers, but budgetary constraints prevent us from funding them all. Partnering with organizations that share our goals, including other government agencies, industry, patient groups and professional organizations, helps us extend our impact beyond our own resources and leverage our investments in research and training, which benefits everyone. With the AMP partnership, we are able to fund research that would not otherwise have been possible.

Professional organizations can also help augment opportunities for promising early-stage investigators who may need additional support to ensure steady footing on their career path. For example, the Rheumatology Research Foundation provides bridge funding to talented investigators who are in a transitional career period in which a steady stream of funding may not yet be available to them. These awards can help researchers stay the course of their chosen careers and, perhaps one day, make a significant discovery.

Richard Quinn is a freelance writer in New Jersey.