Gary S. Gilkeson, MD, Medical University of South Carolina
"Human Mesenchymal Stem Cells in Lupus"
2012 Adult Stem Cell Grant Award
This grant is in honor of The Cooper Family Foundation
Mesenchymal stromal cells (MSC) are unique adult stem cells with immune suppressive properties that make them attractive as an adoptive cell therapy for the treatment of lupus. MSC induced remission of autoimmune disease in multiple animal models, including lupus models. Their use in early phase human clinical trials is promising. Dr. Linyung Sun, in Nanjing, China, has treated 89 lupus patients with MSC. These uncontrolled trials of bone marrow derived or umbilical cord derived MSC in severe cytoxan resistant lupus patients suggest efficacy in over 70% of patients, with minimal side effects, with patients out 3 years from transplantation. His results support the continued development of MSC as a cellular biopharmaceutical for the treatment of lupus. There are no active trials in the United States for treating autoimmune diseases with MSC. The major roadblock to MSC trials in the US is that MSC for human use must be derived in a U.S. Food and Drug Administration (FDA) approved clean cell facility with separate individual FDA approval for each trial. This proposal seeks to remove many of the roadblocks of studies of MSCs in lupus. The proposed studies will be used to support an FDA Investigational New Drug (IND) application for the first Phase I US clinical trial of MSC in lupus. The ultimate goal is to perform definitive controlled trials, comparing MSC treatment to standard of care.
Listen to a Podcast with Dr. Gilkenson