Fact Sheet: Adult Stem Cell Research and Transplants …

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Last updated: November 21, 2017

Adult stem cell transplants are already widely used to the benefit of over a million people.

Adult stem cell transplants are being used to treat dozens of conditions in patients.

[i] Gratwohl A et al., One million haematopoietic stem-cell transplants: a retrospective observational study, Lancet Haematology 2, e91-e100, March 2015. doi:10.1016/S2352-3026(15)00028-9.

[ii]Niederwieser D, Baldomero H, Szer J, Gratwohl M, et al., Hematopoietic stem cell transplantation activity worldwide in 2012 and a SWOT analysis of the Worldwide Network for Blood and Marrow Transplantation Group including the global survey. Bone Marrow Transplant. 51(6):778-785, 2016. doi:10.1038/bmt.2016.18.

[iii] General FAQ. U.S. Department of Health and Human Services, Health Resources and Services Administration. http://bloodcell.transplant.hrsa.gov/about/general_faqs/index.html, accessed August 3, 2016.

[iv] Ballen KK, Gluckman E, Broxmeyer HE, Umbilical cord blood transplantation: the first 25 years and beyond, Blood. 122:491-498, 2013. doi:10.1182/blood-2013-02-453175.

[v] Gratwohl A et al., One million haematopoietic stem-cell transplants: a retrospective observational study, Lancet Haematology 2, e91-e100, March 2015. doi:10.1016/S2352-3026(15)00028-9.

[vi] Search term: http://www.clinicaltrials.gov/ct2/results?term=adult+stem+cell+transplants&type=Intr, accessed August 2, 2016.

[vii] Patel AN et al., Ixmyelocel-T for patients with ischaemic heart failure: a prospective randomised double-blind trial. Lancet 387:2412-2421, 2016. doi: 10.1016/S0140-6736(16)30137-4.

[viii] Steinberg GK, Kondziolka D, Wechsker LR et al., Clinical Outcomes of Transplanted Modified Bone MarrowDerived Mesenchymal Stem Cells in Stroke: A Phase 1/2a Study. Stroke. 447: 1817-1824, 2016. doi: 10.1161/STROKEAHA.116.012995; Cox CS, Hetz RA, Liao GP, et al., Treatment of Severe Adult Traumatic Brain Injury Using Bone Marrow Mononuclear Cells. Stem Cells. 35:1065-1079, 2017. doi: 10.1002/stem.2538; Hess, DC et al., Safety and Efficacy of Multipotent Adult Progenitor Cells in Acute Ischaemic Stroke (MASTERS): A Randomised, Double-Blind, Placebo-Controlled, Phase 2 Trials. Lancet. 16 (5):360-368, 2017. doi: 10.1016/S1474-4422(17)30046-7; Savitz SI, Misra V, Kasam M, et al., Intravenous Autologous Bone Marrow Mononuclear Cells for Ischemic Stroke. Annals of Neurology. 70:59-69, 2011. doi: 10.1002/ana.22458.

[ix]See Bernaudin F et al., Long-term results of related myeloablative stem-cell transplantation to cure sickle cell disease. Blood. 110:2749-2756,2007. doi: 10.1182/blood-2007-03-079665. Hematopoietic stem cell transplantation (HSCT) is the only curative therapy for sickle cell disease.

[x] Lima C, Pratas-Vital J, Escada P, Hasse-Ferreira A, Capucho C, Peduzzi JD. Olfactory Mucosal Autografts and Rehabilitation for Chronic Traumatic Spinal Cord Injury. Neurorehabil Neural Repair January 24:10-22, 2010. doi: 10.1177/1545968309347685; Lima C, Pratas-Vital J, Escada P, Hasse-Ferreira A, Capucho C, Peduzzi JD. Olfactory Mucosa Autografts in Human Spinal Cord Injury: A Pilot Clinical Study. The Journal of Spinal Cord Medicine. 29(3):191-203, 2006.

[xi] Burt RK et al., Association of Nonmyeloablative Hematopoietic Stem Cell Transplantation With Neurological Disability in Patients With Relapsing-Remitting Multiple Sclerosis, JAMA. 313(3):275-284, 2015. doi: 10.1001/jama.2014.17986.

[xii] Cai J et al., Umbilical Cord Mesenchymal Stromal Cell With Autologous Bone Marrow Cell Transplantation in Established Type 1 Diabetes: A Pilot Randomized Controlled Open-Label Clinical Study to Assess Safety and Impact on Insulin Secretion, Diabetes Care. 39, 149, 2016. doi: 10.2337/dc15-0171; Gaipov, A et al., Autologous Bone-Marrow-Derived Stem Cells Transplantation in Type 1 Diabetes Mellitus. Nephrol. Dial. Transplant. 31 (suppl 1):i217, 2016. doi: 10.1093/ndt/gfw169.03; DAddio F et al., Autologous Nonmyeloablative Hematopoietic Stem Cell Transplantation in New-Onset Type 1 Diabetes: A Multicenter Analysis, Diabetes.63(9),3041-3046, 2014.doi:10.2337/db14-0295; Zhao et al., Reversal of type 1 diabetes via islet cell regeneration following immune modulation by cord blood-derived multipotent stem cells. BMC Medicine. 10(3), 2012. doi: 10.1186/1741-7015-10-3; Voltarelli JC and Couri CEB, Stem cell transplantation for type 1 diabetes mellitus, Diabetology & Metabolic Syndrome 1, 4, 2009. doi: 10.1186/1758-5996-1-4; Couri CEB et al., C-Peptide Levels and Insulin Independence Following Autologous Nonmyeloablative Hematopoietic Stem Cell Transplantation in Newly Diagnosed Type 1 Diabetes Mellitus, JAMA 301, 1573-1579, 2009; Voltarelli JC et al., Autologous Nonmyeloablative Hematopoietic Stem Cell Transplantation in Newly Diagnosed Type 1 Diabetes Mellitus, JAMA. 297, 1568-1576, 2007.

[xiii] Weiss JN, Levy S, Malkin A. Stem Cell Ophthalmology Treatment Study (SCOTS) for retinal and optic nerve diseases: a preliminary report. Neural Regen Res 2015; 10:982-8; Weiss JN, Levy S, Benes SC. Stem Cell Ophthalmology Treatment Study (SCOTS) for retinal and optic nerve diseases: a case report of improvement in relapsing auto-immune optic neuropathy. Neural Regen Res 2015; 10:1507-15.

[xiv] Burt RK et al., Nonmyeloablative Hematopoietic Stem Cell Transplantation for Systemic Lupus Erythematosus, JAMA 295, 527-535, 2006, doi: 10.1001/jama.295.5.527; Illei GG et al., Current state and future directions of autologous hematopoietic stem cell transplantation in systemic lupus erythematosus, Annals of the Rheumatic Diseases 70, 2071-2074, 2011 doi: 10.1136/ard.2010.148049; Szodoray P et al., Immunological reconstitution after autologous stem cell transplantation in patients with refractory systemic autoimmune diseases, Scandinavian Journal of Rheumatology 41, 110-115, 2012, doi: 10.3109/03009742.2011.606788; Alchi B et al., Autologous haematopoietic stem cell transplantation for systemic lupus erythematosus: data from the European Group for Blood and Marrow Transplantation registry, Lupus 22, 245-253, 2013, doi: 10.1177/0961203312470729; Alexander T et al., Autologous hematopoietic stem cell transplantation in systemic lupus erythematosus, Z. Rheumatologie 75, 770-779, 2016, doi: 10.1007/s00393-016-0190-3

[xv]Search term selection: multiple myeloma/plasma cell disease (PCD) http://bloodcell.transplant.hrsa.gov/RESEARCH/Transplant_Data/US_Tx_Data/Data_by_Disease/national.aspx, accessed August 3, 2016.

[xvi] Press release: The Lancet Hematology: Experts warn of stem cell underuse as transplants reach 1 million worldwide (Feb 26, 2016) http://www.eurekalert.org/pub_releases/2015-02/tl-tlh022515.php, accessed August 2, 2016.

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