While the use of highly active anti-viral therapy (HAART) has lowered the level of virus in the blood stream, immune reconstitution may offer additional benefits in maintaining viral suppression and enhancing immune function. Clinical trials in immune-based therapy (IBT) must to be designed to capture endpoints that will allow approval of these agents by regulatory agencies. Appropriate indices of immune function and competence need to be identified and validated.
In December 2000, the Forum held a meeting for invited scientists, researchers, and pharmaceutical, government, and community representatives, on Immune-Based Therapies and HIV Disease. The focus of the meeting was on immune-based therapies (IBTs) and indices of immune competency for HIV research. Participants discussed indices of immune function, biomarkers that can be used to evaluate the efficacy of immunologic agents in HIV disease and the validation of these indices along with questions of how to overcome barriers in developing immune-based agents and designing and implementing clinical trials. The participants identified the gaps in the research and made recommendations to advance our understanding of immune pathogenesis in HIV disease, identify and validate appropriate markers, and determine how these markers can be used in research of immune-based therapies. They also outlined some general considerations for the design clinical trials of IBTs based on surrogate markers of bioactivity.
As background information for participants attending the Forum's workshop, a report Immune-based therapy for HIV treatment and prevention: A review of clinical endpoints for trials of immunologic agents was prepared to address issues related to the discussion questions. The report reviewed study designs and endpoints used in phase II and phase III trials for 13 immunologic agents.
One outcome of the December 2000 meeting was the suggestion to hold small focused meetings to concentrate on well-defined topics. On February 6, 2001, the Forum held a meeting in Chicago, IL to discuss ways to promote the selection, refinement, and evaluation of immunologic markers used to assess immune-based therapies in people with HIV infection.