Scleroderma Research Treatments Trials Positive

Stem Cell (Bone Marrow) Transplantation

Overview of Stem Cells and Stem Cell Transplants

Stem Cells. This site provides many links about stem cells, stem cell transplants, research, and the latest news. MedlinePlus.

Current immunosuppressive and antifibrotic therapies of systemic sclerosis (SSc) and emerging therapeutic strategies. A better understanding of SSc pathophysiology has enabled the identification of numerous new therapeutic targets. PubMed, Expert Rev Clin Pharmacol, 10/02/2020. (Also see Immunosuppressants)

Low B cell counts as risk factor for infectious complications in systemic sclerosis after autologous hematopoietic stem cell transplantation (aHSCT). After aHSCT, monitoring for infectious complications, especially for cytomegalovirus reactivations, is crucial as the reconstitution of the immune system takes longer than 12 months. PubMed, Arthritis Res Ther, 2020 Aug 8;22(1):183. (Also see B Cells and T Cells)

Advances in the Treatment of Scleroderma (SSc): Autologous Hematopoietic Stem Cell Transplant (HSCT). HSCT can be considered as an option in carefully selected patients with rapidly progressive SSc who have a risk of organ failure. Medscape, 1/2/2020.

Modeling Progressive Fibrosis with Pluripotent Stem Cells Identifies an Anti-fibrotic Small Molecule. We describe the generation and characterization of an in vitro progressive fibrosis model that uses cell types derived from induced pluripotent stem cells. PubMed, Cell Rep, 2019 Dec 10;29(11):3488-3505.e9.

Health-related quality of life (HRQoL) in systemic sclerosis (SSc) before and after autologous haematopoietic stem cell transplant (AHSCT) - a systematic review. AHSCT in severe SSc patients is associated with significant and durable improvement in physical HRQoL. PubMed, Rheumatology (Oxford), 08/27/2019. (Also see Quality of Life)

Stem cell enriched lipotransfer reverses the effects of fibrosis in systemic sclerosis. Autologous stem cell enriched lipotransfer significantly improved the effects of oro-facial fibrosis in SSc in this open cohort study. PubMed, PLoS One, 2019 Jul 17;14(7):e0218068.

Myeloablative Autologous Stem–Cell Transplantation for Severe Scleroderma. Myeloablative autologous hematopoietic stem–cell transplantation achieved long–term benefits in patients with scleroderma, including improved event–free and overall survival, at a cost of increased expected toxicity. PubMed, N Engl J Med, 2018 Jan 4;378(1):35-47.

Open Enrollments

Allogeneic Hematopoietic Stem Cell Transplantation

Currently recruiting. Last updated 10/15/12.

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.

ASSIST Trial: Now Recruiting

Autologous Stem Cell Systemic Sclerosis Immune Suppression Trial (DISclRituxan). ASSIST I was the first randomized trial in patients with scleroderma to not just slow disease progression but rather actually reverse it. It is the first treatment to have ever demonstrated reversal of lung disease in scleroderma with improvement in FVC, TLC, HRCT, and QOL. This study will compare the ASSIST I conditioning regimen of cyclophosphamide and rATG to the same regimen (rATG, cyclophosphamide) with rituximab (termed rituximab sandwich regimen). Northwestern.

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.

SCOT Clinical Trial: Enrollment Completed

Enrollment for the SCOT trial completed in 2011.

SCOT Clinical Trial, which stands for "Scleroderma Cyclophosphamide Or Transplantation", will compare two potential therapies: autologous stem cell transplantation versus high-dose monthly cyclophosphamide. This study has been completed. ClinicalTrials.gov. (Also see Scleroderma Clinical Trials: Current and Cyclophosphamide)

Research Studies

Machine learning predicts stem cell transplant (HSCT) response in severe scleroderma (SSc). This study suggests that intrinsic subset stratification of patients may be used to identify patients with SSc who receive significant benefit from HSCT. PubMed, Ann Rheum Dis, 2020 Sep 15;annrheumdis-2020-217033.

Antibody CD45-ADC Shows Potential to Treat Scleroderma and Other Diseases in Mice, Study Shows A single dose of an engineered antibody known as CD45-ADC can effectively eliminate disease-causing immune cells and ease the symptoms of scleroderma in mice, studies show. Scleroderma News, 12/04/2019.

New Computer–based Analysis Method Identifies Scleroderma Patients Who May Benefit Most from Stem Cell Transplant. The analysis of gene profile in blood samples may help identify scleroderma patients who could benefit most from stem cell transplant, a research study suggests. Scleroderma News, 11/01/2018.

Supportive Use of Adipose-Derived Stem Cells in Cell–Assisted Lipotransfer for Localized Scleroderma. Cell–assisted lipotransfer not only improves the survival rate of transplanted fat but also improves skin texture in bleomycin–induced skin fibrosis nude mice. PubMed, Plast Reconstr Surg, 2018 Jun;141(6):1395-1407.

Therapeutic Applications of Mesenchymal Stem Cells (MSCs) for Systemic Lupus Erythematosus (SLE). The immuno–modulatory and regenerative characteristics of MSCs make them as one promising candidate for treating SLE. PubMed, Adv Exp Med Biol, 05/17/2018.

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 Articles

Stem cell transplant for severe scleroderma improves survival, quality of life. New clinical trial findings show that a therapeutic regimen involving transplantation of a person’s own blood-forming stem cells can improve survival and quality of life for people with severe scleroderma.National Institutes of Health, 01/03/2018.

Patient Stories

Sadly, Doris Razo passed away in December 2012.

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.

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…

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