| Scleroderma Clinical Trials (MAIN MENU) |
|
|
|
| Stem Cell (Bone Marrow) Transplantation |
|
|
| About Stem Cells and Stem Cell Tranplants |
| About Stem Cells. Topics include embryonic stem cells, adult stem cells, stem cells from baby teeth and umbilical cords, cures, therapies, therapeutic cloning, reproductive cloning, somatic cell nuclear transfer, and regenerative medicine. International Society for Stem Cell Research. |
| Stem Cells. This site provides many links about stem cells, stem cell transplants, research, and the latest news. MedlinePlus. |
| Stem cell transplant. A stem cell transplant is a complex procedure to replace unhealthy stem cells with healthy ones. Find out what the process is like, including conditioning, and what complications may arise. Mayo Clinic. April 11, 2006. |
| Haematopoietic stem cell transplantation (HSCT) for autoimmune disorders. Updates of published trials and data from the registries indicate a long-lasting, immunosuppression-free condition in about 50% of the patients who underwent an HSCT for a severe, progressive autoimmune disease. (PubMed) Saccardi R. Curr Opin Hematol. November 2008. (Also see: Autoimmunity) |
| Technology Insight: hematopoietic stem cell (HSCs) transplantation for systemic rheumatic disease. Transplantation of HSCs for the treatment of autoimmune diseases aims to fundamentally correct the dysregulated immune system, which could result in sustained clinical remission or potential cure. (PubMed) Nat Clin Pract Rheumatol. 2008 Feb 19. |
| Q&A: Stem cells. Stem cells are thought to hold huge potential for treating a wide range of disease and disability. When a stem cell divides, each new cell has the potential to either remain a stem cell or become another type of cell with a more specialised function. BBC News. May 2005. |
| Open Enrollments for Early Diffuse Progressive Systemic Sclerosis |
| Stem Cell Transfusion is a form of bone marrow transplantation which uses a patient's own cells, thus avoiding rejection problems. This is now being used, under a National Institute of Health Program, for early diffuse progressive systemic sclerosis. |
| Adult stem cells in the treatment of autoimmune diseases. During the past 10 yrs, over 700 patients suffering from severe autoimmune disease (AD) have received an autologous haematopoietic stem cell transplant as treatment of their disorder with durable remission being obtained in around one-third. Rheumatology 2006 45(10):1187-1193. |
| SCOT Clinical Trial: Open Enrollment, 2005-2008 |
| SCOT Clinical Trial, which stands for "Scleroderma Cyclophosphamide Or Transplantation", will compare two potential therapies: autologous stem cell transplantation versus high-dose monthly cyclophosphamide. Study dates 2005-2008. (Also see: Scleroderma Clinical Trials: Current and Cyclophos-phamide, Cytoxan) Reposted 9/18/08. |
| Allogeneic Hematopoietic Stem Cell Transplantation |
| Allogeneic Hematopoietic Stem Cell Transplantation (NST) for Patients With Systemic Sclerosis This study is designed to examine whether treating patients with high dose Cyclophosphamide and Fludarabine (drugs which reduce the function of your immune system) and CAMPATH-1H (a protein that kills the immune cells that are thought to be causing the disease), followed by return of blood stem cells that have been previously collected from patients brother or sister will stop or reverse the disease. Northwestern University, Chicago, Illinois. ClinicalTrials.gov. |
| U.S. Centers Doing Stem Cell Transplantations for Scleroderma |
| MI: University of Michigan - Ann Arbor, Dr. James R. Seibold |
| Research Studies on Stem Cell Transplantation |
| Stem cell transplantation for rheumatic autoimmune diseases. Long-term and marked improvements of disease activity have been observed, notably in systemic sclerosis, systemic lupus erythematosus, and juvenile idiopathic arthritis, and treatment-related morbidity and mortality have improved due to better patient selection and modifications of transplant regimens. T. Hügle. Arthritis Research & Therapy. October 10, 2008. |
| Mesenchymal stem cells (MSC) in arthritic diseases. MSCs, the nonhematopoietic progenitor cells found in various adult tissues, are characterized by their ease of isolation and their rapid growth in vitro while maintaining their differentiation potential, allowing for extensive culture expansion to obtain large quantities suitable for therapeutic use. These properties make MSCs an ideal candidate cell type as building blocks for tissue engineering efforts to regenerate replacement tissues and repair damaged structures as encountered in various arthritic conditions. F. H Chen. Arthritis Research & Therapy. October 10, 2008. (Also see: Arthritis) |
| Bone marrow stem cells may help control inflammatory bowel disease (IBD). Massachusetts General Hospital investigators have found that infusions of a particular bone marrow stem cell appeared to protect gastrointestinal tissue from autoimmune attack in a mouse model. Findings suggest that mesenchymal stem cells therapy could become a useful treatment for inflammatory bowel disease. EurekAlert. August 20, 2008. (Also see: Inflammatory Bowel Disease) |
| Exercise tolerance in children with juvenile idiopathic arthritis (JIA) after autologous stem cell transplant (ASCT). The study group showed significantly reduced exercise tolerance compared to healthy subjects. Functional ability and joint status were also decreased in patients after ASCT. Children with JIA post ASCT have impaired exercise tolerance even 9 years post ASCT. T Takken. Bone Marrow Transplantation. June 30, 2008. (Also see: Arthritis) |
| The Development of Inflammatory Arthritis and Other Rheumatic Diseases Following Stem Cell Transplantation (SCT). Immunologic manipulation during the SCT process raises the question of whether there is an increased propensity to develop autoimmune disease posttransplant. (PubMed) Semin Arthritis Rheum. 2008 May 31. (Also see: Arthritis). |
| Bone Marrow-Derived Mesenchymal Stem Cells (MSC) Ameliorate Autoimmune Enteropathy Independent of Regulatory T Cells. Data implicates the intestine as a new site of MSC tolerance induction and should motivate further studies evaluating the use of MSCs as a treatment for autoimmune enteropathies. (PubMed) Parekkadan B. Stem Cells. 2008 Apr 17. (Also see: Autoimmunity) |
| Autologous stem cell transplantation (ASCT) in diffuse scleroderma: impact on hand structure and function. ASCT improved hand scleroderma over 12 months and resolved previously refractory tenosynovitis. ASCT secondarily improved hand function (paid employment, followed by self-care, home care, then by sport/hobbies). (PubMed) Intern Med J. 2008 Mar 11. (Also see: Joint Contratures, and Sclerodactyly) |
| Multiple Autoimmune Diseases after Autologous Stem-Cell Transplantation. We report on a 19-year-old woman with systemic sclerosis who underwent CD34+-selected autologous hematopoietic stem-cell transplantation in March 2001. The dermal sclerosis improved immediately after transplantation, but thrombocytopenia and Graves' disease developed. The New England Journal of Medicine. Volume 357:2734-2736 December 27, 2007 Number 26. |
| Adult Stem Cells May Improve Treatment Of Certain Cardiovascular Disorders And Autoimmune Diseases. When this team examined studies related to autoimmune diseases, 26 reports representing 854 patients reported nonmyeloablative transplants with a treatment-related mortality rate of less than one percent; dose-reduced myeloablative transplants with a treatment-related mortality rate of less than two percent; and intense myeloablative regiments with a mortality rate of 18 percent. Medical News Today. 03/08/08. |
| Stem cell transplantation has long-term benefits in systemic sclerosis. Autologous hematopoietic stem cell transplantation improves survival and reduces skin thickening and other symptoms of systemic sclerosis. TheJobCure (Reuters) 01/09/08. |
| High-dose immunosuppressive therapy and autologous hematopoietic cell transplantation for severe systemic sclerosis: long-term follow-up of the US multicenter pilot study. The estimated progression-free survival was 64% at 5 years. Sustained responses including a decrease in dermal fibrosis were observed exceeding those previously reported with other therapies. Blood, 15 August 2007, Vol. 110, No. 4, pp. 1388-1396. |
| Local implantation of autologous mononuclear cells (MNCs) from bone marrow and peripheral blood for treatment of ischaemic digits in patients with connective tissue diseases. Implantation of autologous MNCs from peripheral and bone marrow into the ischaemic digits was very effective in pain-relief and more clinical trials would be warranted to see whether this could be a new treatment modality for angiogenesis in connective tissue diseases as in Buerger's disease. PubMed. Rheumatology (Oxford). 2007 Feb 19. (Also see: Digital Ulcers) |
| High-dose immunosuppressive therapy and autologous hematopoietic cell transplantation for severe systemic sclerosis: long-term follow-up of the U.S. multicenter pilot study. These are the results of a phase II, single arm study of high-dose immunosuppressive therapy (HDIT) and autologous CD34-selected hematopoietic cell transplantation (HCT) conducted in 34 patients with diffuse cutaneous SSc. PubMed. Blood. 2007 Apr 23. |
| Autologous hematopoietic stem cell transplantation in systemic lupus erythematosus patients with cardiac dysfunction: feasibility and reversibility of ventricular and valvular dysfunction with transplant-induced remission. These data suggest that auto-HSCT is feasible in selected patients with lupus-related cardiac dysfunction, and with control of disease activity, may improve. PubMed. Bone Marrow Transplant. 2007 May 7. (Also see: Lupus) |
| Cell therapy for autoimmune diseases. Autologous haemopoietic stem cell transplantation (HSCT) has become a curative option for conditions with very poor prognosis such as severe forms of scleroderma, multiple sclerosis, and lupus, in which targeted therapies have little or no effect. PubMed. Arthritis Res Ther. 2007 Mar 14;9(2):206. |
| High-Dose Immune Suppressive Therapy And Autologous Peripheral Blood Stem Cell Transplantation (PBSCT) For Systemic Sclerosis : Analysis of Immune Reconstitution. These observations suggest that high-dose immune suppressive therapy with auto-PBSCT improves the disease activity of patients with SSc. Hiroshi Tsukamoto. 690 ACR 2006. (Also see: Causes of Scleroderma: Cytokines) |
| Topical Application of Autologous Bone Marrow-Derived Cultured Mesenchymal Stem Cells to Scleroderma Ulcers. Novel techniques have been developed for culturing and delivering autologous bone marrow-derived mesenchymal stem cells to scleroderma ulcers. The phenotype of these cells has been characterized both in vitro and in vivo. The outcome seems very promising. Vincent Falanga. 1138/397 ACR 2006. (Also see: Digital Ulcers) |
| Initial Trials Indicate Reduced Intensity Stem Cell Transplant Safe for Scleroderma Patients. Within 12 months most patients showed a significant improvement in skin scores and their cardiac, pulmonary and renal functions remained stable. Two years later, 7 out of 10 had no signs of worsening disease. Walter G. Barr, MD. ACR Annual Scientific Meeting. 11/12/06. |
| Autologous Non-Myeloablative Peripheral Blood Stem Cell (PBSC) Transplantation in Patients with Systemic Sclerosis. Autologous PBSC transplantation with a non-myeloablative conditioning regimen of cyclophosphamide and rabbit antithymocyte globulin appears safe with no treatment related mortality in our first 10 patients. Walter G. Barr. 688 ACR 2006. |
| Stretching bone marrow stem cells pushes them towards becoming blood vessel. When stretched, a type of adult stem cell taken from bone marrow can be nudged towards becoming the type of tissue found in blood vessels. Mesenchymal stem cells have the ability to turn into different types of connective tissue including bone, cartilage and muscle. EurekAlert! 10/23/06. |
| Autologous Hematopoietic Stem Cell Transplantation in the Treatment of Systemic Sclerosis: Follow-Up Results From an International Phase l-ll Study. Autologous peripheral blood stem cell transplantation is feasible in selected patients with SSc with acceptable toxicity and significant clinical and functional benefits during the five years of follow up. M. C. Vonk. SAT0259 EULAR 2006. |
| Adult stem cells in the treatment of autoimmune diseases (AD). During the past 10 yrs, over 700 patients with severe ADs have received an autologous haematopoietic stem cell transplant as treatment with durable remission being obtained in around one-third. PubMed. Rheumatology (Oxford). 2006 Jun 15. |
| Scientists Find Previously Unknown Receptors On Adult Stem Cells. Scientists have discovered that marrow stem cells, undifferentiated cells that eventually give rise to the blood cells that fight infection, possess receptors that recognize bacteria and viruses. The findings could have important implications for treating autoimmune diseases. Medical News Today. 06/23/06. (Also see: Causes of Scleroderma: Infections) |
| Allogeneic marrow transplantation in patients with severe systemic sclerosis: Resolution of dermal fibrosis. Allogeneic HCT (hematopoietic cell transplantation) may result in sustained remission of SSc. Graft-versus-host disease and opportunistic infections are the major risks associated with allogeneic HCT for SSc, as for allogeneic HCT in general. Arthritis & Rheumatism Journal. Volume 54, Issue 6:1982 - 1986.June 2006. |
| Allogeneic Bone Marrow Transplantation for Autoimmune Disease — The Jury Is Still Out. The results so far, on mostly autologous HSCT-treated patients, of the immune system "resetting" to a tolerant state, suggest this to be so in over 30%. J. Rheumatol 2006 April;33:644. Editorial. A. Tyndall. |
| Successful autologous peripheral blood stem cell transplantation using Thiotepa in a patient with systemic sclerosis and cardiac involvement. The present case report demonstrates that thiotepa can be employed as a conditioning regimen for auto-PBSCT in SSc patients with cardiac involvement in order to reduce cyclophosphamide-induced cardiotoxicity. PubMed. Tohoku J Exp Med. 2006;209(1):61-67. (Also see: Cardiac Involvement) |
| Immune Reconstitution After Autologous Hematopoietic Stem Cells Transplantation in Systemic Sclerosis. B and T lymphocyte populations remained disturbed after autologous HSCT in SSc. The positive correlation of high B lymphocyte counts with TREC values suggests that an excessive thymic function could help pathogenic autoantibody producing B cells in the first period after HSCT. D. Farge. FRI0142 EULAR 2005. |
| A Phase I-II Trial of Autologous Peripheral Blood Stem Cell Transplantation in the Treatment of Refractory Autoimmune Disease. These observations suggest that high-dose CY (cyclophosphamide) with autologous PBSCT (peripheral blood stem cell transplantation) is feasible and may be effective in the treatment of severe and refractory AD. PubMed. Ann Rheum Dis. 2005 Aug 26. |
| Hemopoietic stem cell transplantation in rheumatic diseases-an update. Hematopoietic stem cell transplant (HSCT) for autoimmune diseases has been recognized as a potential treatment for patients who have failed conventional therapy. Transplant in SSc patients has offered durable responses with improving transplant-related mortality related to careful patient selection. PubMed. Autoimmun Rev. 2005 Sep;4(7):442-9. |
| Pulmonary function in long-term survivors of pediatric hematopoietic cell transplantation (HCT). In multivariate analysis, risk factors associated with restrictive lung disease (RLD)included transplant regimen, transplant diagnosis, scleroderma/contracture, and donor relation. Fifty-five percent of long-term pediatric HCT survivors had pulmonary dysfunction. These findings stress the need for long-term follow-up to detect pulmonary dysfunction. PubMed. Pediatr Blood Cancer. 2005 Aug 5. |
| Haemopoietic Stem Cells Transplantation Improves Microcirculation in Systemic Sclerosis (SSc). These results indicate that NVC (nailfold videocapillaroscopy) pattern reflects the dynamics of the vascular changes in SSc and that a vascular remodelling following HSCT may be taking place. I. Miniati. FRI0107 EULAR 2005. |
| Autologous Stem Cells Transplantation for Severe Diffuse Systemic Sclerosis (SSc): 2 Years of Follow Up. Our results show that HSCT may significantly improve skin involvement, stabilize lung function and reduce PAH, contributing to the amelioration of quality of life. I. Miniati. FRI0108 EULAR 2005. (Also see: Pulmonary Hypertension) |
| Autologous Stem Cell Transplantation for Severe Systemic Sclerosis: Update on the ASTIS Trial. The absence of TRM (treatment-related mortality) and unexpected toxicities with 42 SSc patients enrolled underscores the feasibility of the ongoing ASTIS-trial. J. M. Van Laar. FRI0077 EULAR 2005. (Also see: ASTIS Clinical Trial) |
| Cardiopulmonary function before and after cyclophosphamide treatment in severe systemic sclerosis: comparison of monthly intravenous bolus and autologous haematopoietic stem cell transplantation. In spite of higher cyclophosphamide doses during autologous haematopoietic stem cells transplantation than bolus treatment, cardiopulmonary toxicity appeared not to be increased. PubMed. Rev Med Interne. 2005 Jun;26(6):444-452. (Also see: ASTIS Trial for Scleroderma) |
| Analysis of immune reconstitution after autologous bone marrow transplantation in systemic sclerosis. B and T lymphocyte populations remained disturbed for at least 1 year after HSCT in SSc patients, which may reflect the persistence of an underlying disease mechanism. PubMed. Arthritis Rheum. 2005 May;52(5):1555-63. |
| Treatment of severe systemic autoimmune diseases with autologous peripheral blood stem cell transplantation. High dose immunosuppressive therapy followed by autologous peripheral blood stem cell transplantation with CD(34)(+) cell selection is feasible and relatively safe. Patients remain free from disease active and improved continuously. PubMed. Zhonghua Yi Xue Za Zhi. 2004 Dec 17;84(24):2077-81. |
| Autologous hematopoietic stem cell transplantation for autoimmune diseases. We have evaluated the toxicity and disease response in 473 patients with severe autoimmune disease treated with autologous HSCT between 1995 and 2003. This data supports the hypothesis that autologous HSCT can alter disease progression in severe autoimmune disease. PubMed. Bone Marrow Transplant. 2005 Mar 14. |
| Immunomodulatory Therapy for Systemic Sclerosis (SSc): Will High-intensity Immunosuppression with Stem Cell Rescue Improve Outcome? The types and intensities of immunomodulatory therapies employed in other connective tissue diseases have not proved as useful in patients with SSc. Preliminary studies of intensification of therapy to immunoablative and myeloablative levels with hematopoietic stem cell rescue have been performed and appear promising. Pub Med. Curr Rheumatol Rep. 2005 Apr;7(2):142-149. |
| Also see: Scleroderma Clinical Trials |
| Transplant Funding |
| National Foundation for Transplants (NFT). NFT has raised from $5,000 to $600,000 in individual transplant campaigns from Maine to Guam. |
| Related Media Articles |
| Stem cell banks could pay medical dividends. The cells are known as induced pluripotent stem cells, or IPS cells. They appear to be as versatile as embryonic stem cells, with the ability to transform themselves into virtually any tissue in the body. MSNBC. 05/23/08. |
| Amy Daniels: Stem Cell Transplant Speech on Capitol Hill. Amy is a member of Sclero Forums and has had a stem cell transplant for scleroderma. YouTube. 04/14/08. |
| Woman fights for an experimental procedure that could save her life. Chavez was diagnosed in 2003 with systemic scleroderma. The illness is creating what amounts to a tough inner shell of scar tissue in her lungs, hardening them, gradually reducing her ability to breathe. TuscloosaNews.com. March 13, 2008. |
| Bouncing back. Joe Dill had been diagnosed in March 2003 with diffuse scleroderma, and was about to embark as a test subject for a stem cell transplant with chemotherapy treatment. Three years later, Dill, 62, is still living with the effects of scleroderma, but he no longer has any diseased scleroderma cells and is living an active life in Halfway. The Herald-Mail. 11/06/06. |
| Stem Cells Fight Lupus. We've all heard a lot about stem cells. In what's being called a huge development, researchers have now found stem cells can wipe out lupus in certain patients. However, risks can include possible blood transfusions, infection, nausea and the risk of becoming sterile. Ivanhoe.com 05/19/06. (Also see: Lupus) |
| Patient Stories |
| Chasing the Cure. In this book, follow Doris' courageous journey in dealing with an autoimmune disease and sifting through the maze of doctors and treatments, including a stem cell transplant. Doris Razo. |
| Carmen Rijos |
| Carmen Rijos: Systemic Scleroderma Carmen is in need of a stem cell transplant for scleroderma. This is a request for donations for the Carmen Rijos Private Foundation... |
| Craig R. |
| Craig R: CREST Scleroderma I had my first scleroderma symptoms at age twenty-two, which included severe Raynaud's with some ulceration and tissue loss... |
| Elva |
| Elva: Mother of Diffuse Scleroderma Patient My son is unable to put on his own socks and shoes. He is very tired, and has tremendous pain... |
| Jennifer Weldon |
| Jennifer Weldon: Systemic Scleroderma/Stem Cell Transplant Patient My hope is that others who suffer with this disease find the courage to have the stem cell/bone marrow transplant. If the doctors can learn more about fighting and curing scleroderma, then my decision to have the transplant and go on living is worth the fight... ISN. |
| Kirstin Wallin |
| Kirstin Wallin's Home Page Kirstin Wallin |
| Krissy |
| Krissy: Systemic Scleroderma/Stem Cell Transplant Candidate Our home is like a hospital. We have oxygen tanks for when I need extra help breathing, and a lovely kangaroo pump which is how I get my continuous feeding in me... |
| Toni: Spouse of Stem Cell Transplant Survivor |
| Toni: Spouse of Stem Cell Transplant Survivor Jeff is now 17 months past his stem cell transplant and to look at him you'd never know he was sick... |
