Adult Stem Cell Research
Stem cells possess the potential to develop into many different types of cells in the body, and thus, serve as a repair system. The LFA takes a strategic approach to stem cell research by calling on some of the greatest minds in the field to address issues that hold the greatest promise for accelerating our understanding of the potential uses of stem cells as a therapeutic for lupus.
We continue to break new ground by funding research to understand the role of stem cells in treating lupus and severe manifestations of the disease. These studies have the potential to lead to the next breakthrough in the field.
Learn more about Stem Cells and Lupus
2012 Grant Award
Human Mesenchymal Stem Cells in Lupus 
Gary S. Gilkeson, M.D.
Associate Dean for Faculty Affairs and Faculty Development
Medical University of South Carolina
This grant is in honor of The Cooper Family Foundation
2011 Grant Award
“Hematopoietic Stem Cell Transplantation Induces Remission in Lupus”
Richard K. Burt, M.D.
Chief, Division of Immunotherapy
Professor of Immunotherapy
Northwestern University School of Medicine
This grant award is made possible through support of the LFA, Illinois Chapter Grant Period: February 1, 2010 – January 31, 2011
2008 Grant Awards
“Mechanisms of Abnormal Phenotype and Function of Bone Marrow Endothelial Progenitor Stem Cells”
Mariana Kaplan, MD
University of Michigan Health Systems
Ann Arbor, Michigan
“Mechanistic Studies of Type I IFN Gene Expression in SLE and the Role of IKK-related Kinases and Interferon Regulator Factor 3 During and After Reconstitution of the Immune Response Following Non-ablative Therapy and Autologous Hematopoietic Precursor Stem Cell Replacement in Patients with Systemic Lupus Erythematosus”
Kenneth Kalunian, MD
University of California, San Diego
San Diego, California
2009 Grant Awards
These grant awards are made possible through support by the Cooper Family Foundation
"Unusual Regulatory T Cells in Patients with Severe Lupus Following HSCT"
Richard Burt, M.D.
Chief, Division of Immunotherapy
Northwestern University (IL)
Lay Abstract:
Researchers at Northwestern University have found that regulatory T cells (Treg) return in patients with refractory lupus after stem cell transplantation, and both CD4+CD25HighFoxP3+, and an unusual CD8+FoxP3+ Treg subsets are increased. Observations in mouse models of SLE suggest that induced CD8+ Treg cells are important in controlling lupus. Researchers aim to characterize the CD8 Treg subset of post-transplant lupus patients with regards to their unique surface markers, cytokine/chemokine profile, and mechanism of action. Clinical correlations based on gender and disease activity will be done. Molecular requirements for both nuclear autoantigen-specific and non-specific suppressive function of the Treg will be determined. Significance to gaps in knowledge relevant to lupus. The mechanisms of long-term remission of lupus after stem cell transplantation are unknown. Our hypothesis is that the therapy generates a newly differentiated population of Treg cells, which repairs the Treg deficiency in lupus. Defects in Treg cells are beginning to be described in lupus patients. Most of those studies deal with phenotyping and enumerating the CD4+CD25High Treg subset by surface markers. Functional studies, especially autoantigen-specific suppression have not been done. Moreover, very little is known about unusual CD8+ Treg cells in humans and how they could control lupus autoimmunity, although animal models indicate their importance. Investigators will evaluate the quality and quantity of Treg cells before and after HSCT and compare their recovery to gender and clinical disease status. Therefore, this proposal will address new issues critical for immunoregulation of lupus autoimmunity and therapy.
"Induced Pluripotent Stem (iPS) Cell-Based Therapy for SLE"
Igor Slukvin, M.D., Ph.D.
Assistant Professor
University of Wisconsin, Madison
Lay Abstract:
The major goal of this proposal is to significantly advance the stem cell-based therapies for drug-resistant lupus through development of novel technologies for generation of HSCs and immunotherapeutic cells from induced pluripotent cells (iPSCs). The iPSCs are stem cells artificially derived from somatic fibroblasts through insertion of genes critical for the maintenance of pluripotency. These cells can be generated from SLE patients and differentiated into blood cells. Because iPSCs and sustainably expanded and genetically altered in a highly controlled way that is not yet possible with more committed HSCs, iPSCs could provide important advantages over somatic HSCs as target cells for stem cell based therapy of SLE. In this application we propose to characterize in vivo repopulating potential of primitive hematopoietic cells derived from iPSCs, develop a system for differentiation of iPSCs into dendritic cells and T cells, and demonstrate the feasibility of iPSC-based gene transfer into hematopoietic cells. The proposed studies could ultimately lead to novel adult stem cell-based and gene therapies for SLE.
2008 Grant Awards
These grant awards are made possible through support by the Cooper Family Foundation
"Mechanisms of Abnormal Phenotype and Function of Bone Marrow Endothelial Progenitor Stem Cells"
Mariana Kaplan, M.D.
Assistant Professor of Internal Medicine, Division of Rheumatology
University of Michigan Health Systems
Lay Abstract:
Systemic lupus is associated with significant increases in premature heart attacks. We propose that an important factor implicated in heart disease in lupus is the abnormal function of stem cells that repair damaged blood vessels. This proposal will investigate the potential mechanisms by which abnormal vascular repair occurs in lupus and could contribute to designing treatments aimed at decreasing the risk of this potentially fatal complication.
Watch this video of Dr. Kaplan speaking about her research
"Mechanistic Studies of Type I IFN Gene Expression in SLE and the Role of IKK-related Kinases and Interferon Regulator Factor 3 During and After Reconstitution of the Immune Response Following Non-ablative Therapy and Autologous Hematopoietic Precursor Stem Cell Replacement in Patients with Systemic Lupus Erythematosus"
Kenneth Kalunian, M.D.
Professor of Medicine
University of California, San Diego
Lay Abstract:
Lupus (SLE) is a chronic autoimmune disease that causes significant disability and organ damage and, in some cases, fails to respond to available treatments. Targeted therapeutics with increased efficacy and fewer side effects are needed to improve patient care. Studies suggest a role for the cytokine interferon a (IFNa) in the pathogenesis of SLE. We hypothesize that the IKK-related kinases and interferon regulatory factor activate the signaling pathway that leads to increase IFN gene expression in patients with SLE and we plan to study of role of this pathway in SLE in patients who undergo therapy that severe suppresses the immune system with stem cell transplantation in order to understand the effects of this therapy on the signaling pathway and how the pathway may become active again as the immune system returns after stem cell transplantation.
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