Progress Shared and Future Course Charted
The Lupus Research Alliance (LRA) is pleased to report that the Lupus Accelerating Breakthroughs Consortium (Lupus ABC) held its second annual meeting in April in Bethesda, Maryland to share its substantial progress over the first year and chart future directions. Lupus ABC is a first-of-its-kind public-private partnership, uniting individuals with lupus and their advocates, industry, clinicians, researchers, and government representatives to identify and pursue the most effective ways to accelerate the development of urgently needed personalized treatments for people with lupus.
Annual Meeting Highlights
The second annual meeting was a cornerstone event, bringing together the diverse members of Lupus ABC to share progress, insights, and future directions. The two-day meeting began with a session for the Lupus Voices Council members to discuss and prioritize potential projects that they could champion.
The following day, all participants attended an open session kicked off by LRA President and Chief Executive Officer Albert Roy, LRA Vice President and Chief Scientific Officer Teodora Staeva, Ph.D., and Acting Director of the Office of Immunology and Inflammation in the Center for Drug Evaluation and Research (CDER) at the Food and Drug Administration (FDA) Nikolay Nikolov, M.D. “Through this partnership, the FDA will continue its engagement with the lupus community and reaffirm its longstanding commitment to work with researchers, drug developers, and patients to help make new treatments a reality,” noted Dr. Nikolov, setting the tone for an engaging, collaborative meeting.
A significant development highlighted at the meeting was the expansion of Lupus ABC’s partnership with the FDA to include the Center for Biologics Evaluation and Research (CBER). Peter Marks, M.D., Ph.D., Director of CBER, gave a compelling keynote address on advancing cell and gene therapies for autoimmune diseases. “The Center for Biologics Evaluation and Research is looking forward to working together to advance the development of cell and gene therapy products for the treatment of autoimmune diseases such as lupus,” commented Dr. Marks.
Moderated by Eric Morand, M.D., Ph.D., Monash University, a dynamic panel discussion addressed the challenges and opportunities of lupus clinical trial design and the inclusion of companion diagnostics, emphasizing the importance of patient perspectives in clinical trial design. The diverse panel included experts from academia, industry, and the patient community, offering a holistic view of the path forward.
Throughout the day, speakers highlighted accomplishments achieved during the first year of Lupus ABC, including the significantly expanded collaboration with the FDA to include the Center for Biologics Evaluation and Research (CBER) in addition to the initial liaison with the Center for Drug Evaluation and Research (CDER). Lupus ABC saw a surge in participation of biopharmaceutical companies committed to lupus, growing from 17 to 28 members as of April 2024. The most notable accomplishments were the four important projects that Lupus ABC has advanced. The co-chairs presented the projects’ progress and discussed the future plans.
“The power of the collective community cannot be understated, with the lupus patient voice at the center,” noted Teodora Staeva, Ph.D. “We originally defined success as launching one to two projects in the first 18 months. Now, we have launched four projects in just 12 months, paving the way to safer and more effective treatment options that our community seeks.”
Four Lupus ABC Projects’ Goals and Progress
Treatment Response Measure for Systemic Lupus Erythematosus (TRM-SLE)
Led by Eric Morand, M.D., Ph.D. (Monash University), this project aims to build a novel tool to better measure treatment response in SLE clinical trials and was already in progress with strong support from the global community. Lupus ABC became a partner to provide further support to bolster the project’s success. A representative from CDER has been appointed to the Steering Committee to offer regulatory insights. Additionally, members of the Lupus Voices Council serve on both the Steering Committee and the Patient Advisory Board to provide additional patient perspectives.
Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI) Working Group
Co-chaired by Benjamin Chong, M.D. (University of Texas Southwestern Medical Center) and Denesh Chitkara, M.D. (EMD Serono), this Working Group is dedicated to refining the common evaluation tool, CLASI, to align with FDA standards for use as an outcome measure in clinical trials testing potential treatments for cutaneous (skin) lupus. Based on previous feedback, this Working Group has identified the five main concerns that the FDA has with CLASI. Together, members are compiling published and unpublished data to address these concerns.
Chimeric Antigen Receptor (CAR) T Cell Working Group
Launched in March 2024 and co-chaired by Elena Hsieh, M.D. (University of Colorado) and James Chung, M.D., Ph.D. (Kyverna Therapeutics), this Working Group was established to address outstanding questions on the use of CAR T cell therapy in lupus. The group aims to build consensus and draft a position paper on the key considerations to advance CAR T cell therapies for SLE.
Patient Reported Outcomes (PRO) for Lupus
Prior to launching a Working Group on this topic, a planning committee, chaired by Patti Katz, Ph.D. (UCSF), is organizing a meeting on lupus PROs – reports of health status directly from the patient. Scheduled for October 2024, the meeting will bring together PRO experts to discuss the state of lupus PROs and establish a strategic roadmap for future working group efforts.
After the conclusion of the open session, Research Committee members convened to discuss clinical trial design as well as challenges, opportunities, and goals for current Lupus ABC Working Groups.
From advancing four projects in just 12 months to expanding partnerships with the FDA and welcoming new stakeholders, Lupus ABC demonstrates the power of uniting the community and the results of its collaborative effort.
