Lupus research, up until this point, has not been part of such a large-scale and close collaboration among the government, industry, nonprofit research organizations, academics and patients, Dr. Bell says. The AMP’s emphasis on collaboration among those parties should help propel research to the “next level by generating huge datasets on signaling pathways in human lupus.”
“In our work we have learned that breakthroughs often come where least expected from informed inferences. Importantly, the AMP datasets will be available not just to the NIH and large pharma companies but to all who are interested to analyze it, including innovative biotech companies, researchers from diverse academic disciplines, and patients,” she says.
The Arthritis Foundation, the Lupus Foundation of America and the Rheumatology Research Foundation are also partners in the project. David Karp, MD, PhD, president of the Rheumatology Research Foundation, says this collaboration could yield major breakthroughs. “If the program is successful, it may also open the door for many similar discoveries in other autoimmune diseases that would mean better treatments for even more patients, which is an important goal for the Foundation.”
The entire biomedical research community, along with the entire patient advocacy community, has a shared interest in compressing the timeline, reducing the cost & increasing the success rate of new targeted therapies.
5-Year Program
AMP’s focus on RA and SLE is divided into three research phases, with Phase 0 testing the different ways to obtain and prep tissue. Initial analytic runs will be conducted with the aim of developing standardized methods in small numbers of homogenous samples in at least two diseases. Phase 1 will analyze the standardized analytics developed in Phase 0 in blood and tissue cells and will conduct trial runs of additional selected analytics. The number of samples will be sufficient to inform power calculations and establish feasibility studies of the tissue analytic and sample type, source, and acquisition approach.
At least one Phase 1 study in RA and one in SLE should be completed by the end of the second year of the project, according to the NIH. This phase will include analysis of samples from people without RA or SLE to identify pathways that distinguish disease and nondisease tissue.
In Phase 2, studies will be conducted on the recommendation of the AMP RA/SLE Steering Committee and approval by the NIH Program Official. Testing will be conducted in larger patient populations, with patient stratification expected for comparisons within a disease, such as treatment responders vs. nonresponders in RA, and before-treatment vs. after-treatment results for SLE. The final 12 months of the project, projected for 2019, will include preliminary target validation.