from DoNoHarm: The Coalition of Americans for Research Ethics
www.stemcellresearch.org 

CURRENT APPLICATIONS OF ADULT STEM CELLS FOR HUMAN PATIENTS                 (not a complete listing) (sample of references)

ADULT STEM CELLS--HEMATOPOIETIC REPLACEMENT
CANCERS

BRAIN TUMORS—medulloblastoma and glioma
Dunkel, IJ; “High-dose chemotherapy with autologous stem cell rescue for malignant brain tumors”; Cancer Invest. 18, 492-493; 2000.
Abrey, LE et al.; “High dose chemotherapy with autologous stem cell rescue in adults with malignant primary brain tumors”; J. Neurooncol. 44, 147-153; Sept., 1999
Finlay, JL; “The role of high-dose chemotherapy and stem cell rescue in the treatment of malignant brain tumors: a reappraisal”; Pediatr. Transplant 3 Suppl. 1, 87-95; 1999

RETINOBLASTOMA
Hertzberg H et al.; “Recurrent disseminated retinoblastoma in a 7-year-old girl treated successfully by high-dose chemotherapy and CD34-selected autologous peripheral blood stem cell transplantation”; Bone Marrow Transplant 27(6), 653-655; March 2001
Dunkel IJ et al.; “Successful treatment of metastatic retinoblastoma”; Cancer 89, 2117-2121; Nov 15 2000

OVARIAN CANCER
Stiff PJ et al.; “High-dose chemotherapy and autologous stem-cell transplantation for ovarian cancer: An autologous blood and marrow transplant registry report”; Ann. Intern. Med. 133, 504-515; Oct. 3,
2000
Schilder, RJ and Shea, TC; “Multiple cycles of high-dose chemotherapy for ovarian cancer”; Semin. Oncol. 25, 349-355; June 1998

MERKEL CELL CARCINOMA
Waldmann V et al.; “Transient complete remission of metastasized merkel cell carcinoma by high-dose polychemotherapy and autologous peripheral blood stem cell transplantation”; Br. J. Dermatol. 143,
837-839; Oct 2000

TESTICULAR CANCER
Bhatia S et al.; “High-dose chemotherapy as initial salvage chemotherapy in patients with relapsed testicular cancer”; J. Clin. Oncol. 18, 3346-3351; Oct. 19, 2000
Hanazawa, K et al.; “Collection of peripheral blood stem cells with granulocyte-colony-stimulating factor alone in testicular cancer patients”; Int. J. Urol. 7, 77-82; March 2000.

LYMPHOMA
Tabata M et al.; “Peripheral blood stem cell transplantation in patients over 65 years old with malignant lymphoma--possibility of early completion of chemotherapy and improvement of performance status”; Intern Med 40, 471-474; June 2001

Josting, A; “Treatment of Primary Progressive Hodgkin’s and Aggressive Non-Hodgkin’s Lymphoma: Is There a Chance for Cure?”; J Clin Oncol 18, 332-339; 2000
Koizumi M et al.; “Successful treatment of intravascular malignant lymphomatosis with high-dose chemotherapy and autologous peripheral blood stem cell transplantation”; Bone Marrow Transplant 27, 1101-1103; May 2001

ACUTE LYMPHOBLASTIC LEUKEMIA
Ohnuma K et al.; “Cord blood transplantation from HLA-mismatched unrelated donors as a treatment for children with haematological malignancies”; Br J Haematol 112(4), 981-987; March 2001
Marco F et al.; “High Survival Rate in Infant Acute Leukemia Treated With Early High-Dose Chemotherapy and Stem-Cell Support”; J Clin Oncol 18, 3256-3261; Sept. 15 2000

ACUTE MYELOGENOUS LEUKEMIA
Ohnuma K et al.; “Cord blood transplantation from HLA-mismatched unrelated donors as a treatment for children with haematological malignancies”; Br J Haematol 112(4), 981-987; March 2001
Gorin NC et al.; “Feasibility and recent improvement of autologous stem cell transplantation for acute myelocytic leukaemia in patients over 60 years of age: importance of the source of stem cells”; Br. J. Haematol. 110, 887-893; Sept 2000
Bruserud O et al.; “New strategies in the treatment of acute myelogenous leukemia: mobilization and transplantation of autologous peripheral blood stem cells in adult patients”; Stem Cells 18, 343-351;
2000

CHRONIC MYELOGENOUS LEUKEMIA
Ohnuma K et al.; “Cord blood transplantation from HLA-mismatched unrelated donors as a treatment for children with haematological malignancies”; Br J Haematol 112(4), 981-987; March 2001

JUVENILE MYELOMONOCYTIC LEUKEMIA
Ohnuma K et al.; “Cord blood transplantation from HLA-mismatched unrelated donors as a treatment for children with haematological malignancies”; Br J Haematol 112(4), 981-987; March 2001

ANGIOIMMUNOBLASTIC LYMPHADENOPATHY with DYSPROTEINEMIA
Lindahl J et al.; “High-dose chemotherapy and APSCT as a potential cure for relapsing hemolysing AILD”; Leuk Res 25(3), 267-270; March 2001

MULTIPLE MYELOMA
Laughlin MJ et al.; “Hematopoietic engraftment and survival in adult recipients of umbilical-cord blood from unrelated donors”, New England Journal of Medicine 344, 1815-1822; June 14, 2001
Vesole, DH et al.; “High-Dose Melphalan With Autotransplantation for Refractory Multiple Myeloma: Results of a Southwest Oncology Group Phase II Trial”; J Clin Oncol 17, 2173-2179; July 1999.

MYELODYSPLASIA
Ohnuma K et al.; “Cord blood transplantation from HLA-mismatched unrelated donors as a treatment for children with haematological malignancies”; Br J Haematol 112(4), 981-987; March 2001
Bensinger WI et al.; “Transplantation of bone marrow as compared with peripheral-blood cells from HLAidentical relatives in patients with hematologic cancers”; New England Journal of Medicine 344, 175-181; Jan 18 2001

BREAST CANCER
Damon LE et al.; “High-dose chemotherapy and hematopoietic stem cell rescue for breast cancer: experience in California”; Biol. Blood Marrow Transplant 6, 496-505; 2000
Paquette, RL et al., “Ex vivo expanded unselected peripheral blood: progenitor cells reduce posttransplantation neutropenia, thrombocytopenia, and anemia in patients with breast cancer”, Blood 96, 2385-2390; October, 2000.
Stiff P et al.; “Autologous transplantation of ex vivo expanded bone marrow cells grown from small aliquots after high-dose chemotherapy for breast cancer”; Blood 95, 2169-2174; March 15, 2000
Koc, ON et al.; “Rapid Hematopoietic Recovery After Coinfusion of Autologous-Blood Stem Cells and Culture-Expanded Marrow Mesenchymal Stem Cells in Advanced Breast Cancer Patients Receiving High-Dose Chemotherapy”; J Clin Oncol 18, 307-316; January 2000

NEUROBLASTOMA
Kawa, K et al.; “Long-Term Survivors of Advanced Neuroblastoma With MYCN Amplification: A Report of 19 Patients Surviving Disease-Free for More Than 66 Months”; J Clin Oncol 17:3216-3220; October 1999

NON-HODGKIN’S LYMPHOMA
Tabata M et al.; “Peripheral blood stem cell transplantation in patients over 65 years old with malignant lymphoma--possibility of early completion of chemotherapy and improvement of performance status”; Intern Med 40, 471-474; June 2001
Josting, A; “Treatment of Primary Progressive Hodgkin’s and Aggressive Non-Hodgkin’s Lymphoma: Is There a Chance for Cure?”; J Clin Oncol 18, 332-339; 2000
Kirita T et al.; “Primary non-Hodgkin’s lymphoma of the mandible treated with radiotherapy, chemotherapy, and autologous peripheral blood stem cell transplantation”; Oral Surg Oral Med Oral Pathol Oral Radiol Endod. 90, 450-455; Oct. 2000
Yao M et al.; “Ex vivo expansion of CD34-positive peripheral blood progenitor cells from patients with non-Hodgkin’s lymphoma: no evidence of concomitant expansion of contaminating bcl2/JHpositive
lymphoma cells”; Bone Marrow Transplant 26, 497-503; Sept. 2000

HODGKIN’S LYMPHOMA
Peggs KS et al., “Clinical evidence of a graft-versus-Hodgkin’s-lymphoma effect after reduced-intensity allogeneic transplantion”, Lancet 365, 1934-1941, 4 June 2005
Josting, A; “Treatment of Primary Progressive Hodgkin’s and Aggressive Non-Hodgkin’s Lymphoma: Is There a Chance for Cure?”; J Clin Oncol 18, 332-339; 2000

RENAL CELL CARCINOMA
Childs R et al., “Regression of Metastatic Renal-Cell Carcinoma after Nonmyeloablative Allogeneic Peripheral-Blood Stem-Cell Transplantation”, New England Journal of Medicine 343, 750-758;
Sept. 14, 2000
Childs, RW; “Successful Treatment of Metastatic Renal Cell Carcinoma With a Nonmyeloablative Allogeneic Peripheral-Blood Progenitor-Cell Transplant: Evidence for a Graft-Versus-Tumor Effect:; J Clin Oncol 17, 2044-2049; July 1999

VARIOUS SOLID TUMORS
Nieboer P et al.; “Long-term haematological recovery following high-dose chemotherapy with autologous bone marrow transplantation or peripheral stem cell transplantation in patients with solid tumours”;
Bone Marrow Transplant 27, 959-966; May 2001
Lafay-Cousin L et al.; “High-dose thiotepa and hematopoietic stem cell transplantation in pediatric malignant mesenchymal tumors: a phase II study”; Bone Marrow Transplant 26, 627-632; Sept. 2000
Michon, J and Schleiermacher, G. “Autologous haematopoietic stem cell transplantation for paediatric solid tumors”, Baillieres Best Practice Research in Clinical Haematology 12, 247-259, March-June, 1999.
Schilder, RJ et al.; “Phase I trial of multiple cycles of high-dose chemotherapy supported by autologous peripheral-blood stem cells”; J. Clin. Oncol. 17, 2198-2207; July 1999

SOFT TISSUE SARCOMA
Blay JY et al.; “High-dose chemotherapy with autologous hematopoietic stem-cell transplantation for advanced soft tissue sarcoma in adults”; J. Clin. Oncol. 18, 3643-3650; Nov 1 2000

HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS
Matthes-Martin S et al.; “Successful stem cell transplantation following orthotopic liver transplantation from the same haploidentical family donor in a girl with hemophagocytic lymphohistiocytosis”; Blood 96, 3997-3999; Dec 1, 2000

WALDENSTROM’S MACROGLOBULINEMIA
Anagnostopoulos A et al.; “High-dose chemotherapy followed by stem cell transplantation in patients with resistant Waldenstrom's macroglobulinemia”; Bone Marrow Transplant 27, 1027-1029; May 2001

POEMS SYNDROME (OSTEOSCLEROTIC MYELOMA)
Dispenzieri A et al., Peripheral blood stem cell transplantation in 16 patients with POEMS syndrome, and a review of the literature, Blood 104, 3400-3407, 15 November 2004

ADULT STEM CELLS—IMMUNE SYSTEM REPLACEMENT
AUTOIMMUNE DISEASES

SCLEROMYXEDEMA
A.M. Feasel et al., "Complete remission of scleromyxedema following autologous stem cell transplantation," Archives of Dermatology 137, 1071-1072; Aug. 2001.

SCLERODERMA
Burt RK et al., “Induction of tolerance in autoimmune diseases by hematopoietic stem cell transplantation: getting closer to a cure?”, Blood 99, 768-784, 1 February 2002
Burt, RK and Traynor, AE; “Hematopoietic Stem Cell Transplantation: A New Therapy for Autoimmune Disease”; Stem Cells17, 366-372; 1999

MULTIPLE SCLEROSIS
Saccardi R et al., Autologous HSCT for severe progressive multiple sclerosis in a multicenter trial: impact on disease activity and quality of life, Blood 105, 2601-2607, 15 March 2005
Burt RK et al., “Induction of tolerance in autoimmune diseases by hematopoietic stem cell transplantation: getting closer to a cure?”, Blood 99, 768-784, 1 February 2002
Mancardi GL et al.; “Autologous hematopoietic stem cell transplantation suppresses Gd-enhanced MRI activity in MS”; Neurology 57, 62-68; July 10, 2001
Rabusin M et al.; “Immunoablation followed by autologous hematopoietic stem cell infusion for the treatment of severe autoimmune disease”; Haematologica 85(11 Suppl), 81-85; Nov. 2000
Burt, RK and Traynor, AE; “Hematopoietic Stem Cell Transplantation: A New Therapy for Autoimmune Disease”; Stem Cells17, 366-372; 1999
Burt RK et al.; “Hematopoietic stem cell transplantation of multiple sclerosis, rheumatoid arthritis, and systemic lupus erythematosus”; Cancer Treat. Res. 101, 157-184; 1999

CROHN’S DISEASE
Kreisel W et al., Complete remission of Crohn’s disease after high-dose cyclophosphamide and autologous stem cell transplantation, Bone Marrow Transplantation 32, 337-340, 2003
Burt RK et al., “High-dose immune suppression and autologous hematopoietic stem cell transplantation in refractory Crohn disease”, Blood 101, 2064-2066, March 2003
Rabusin M et al.; “Immunoablation followed by autologous hematopoietic stem cell infusion for the treatment of severe autoimmune disease”; Haematologica 85(11 Suppl), 81-85; Nov. 2000
Hawkey CJ et al.; “Stem cell transplantation for inflammatory bowel disease: practical and ethical issues”; Gut 46, 869-872; June 2000

RHEUMATOID ARTHRITIS
Burt RK et al., “Induction of tolerance in autoimmune diseases by hematopoietic stem cell transplantation: getting closer to a cure?”, Blood 99, 768-784, 1 February 2002
Burt RK et al., “Induction of remission of severe and refractory rheumatoid arthritis by allogeneic mixed chimerism”, Arthritis & Rheumatism 50, 2466-2470, August 2004
Verburg RJ et al.; “High-dose chemotherapy and autologous hematopoietic stem cell transplantation in patients with rheumatoid arthritis: results of an open study to assess feasibility, safety, and
efficacy”; Arthritis Rheum 44(4), 754-760; April 2001
Rabusin M et al.; “Immunoablation followed by autologous hematopoietic stem cell infusion for the treatment of severe autoimmune disease”; Haematologica 85(11 Suppl), 81-85; Nov. 2000
Burt, RK and Traynor, AE; “Hematopoietic Stem Cell Transplantation: A New Therapy for Autoimmune Disease”; Stem Cells17, 366-372; 1999
Burt RK et al.; “Hematopoietic stem cell transplantation of multiple sclerosis, rheumatoid arthritis, and systemic lupus erythematosus”; Cancer Treat. Res. 101, 157-184; 1999
Burt, RK et al., “Autologous hematopoietic stem cell transplantation in refractory rheumatoid arthritis: sustained response in two of four patients”, Arthritis & Rheumatology 42, 2281-2285, November,
1999.

JUVENILE ARTHRITIS
Rabusin M et al.; “Immunoablation followed by autologous hematopoietic stem cell infusion for the treatment of severe autoimmune disease”; Haematologica 85(11 Suppl), 81-85; Nov. 2000
Burt, RK and Traynor, AE; “Hematopoietic Stem Cell Transplantation: A New Therapy for Autoimmune Disease”; Stem Cells17, 366-372; 1999

SYSTEMIC LUPUS
Burt RK et al., “Induction of tolerance in autoimmune diseases by hematopoietic stem cell transplantation: getting closer to a cure?”, Blood 99, 768-784, 1 February 2002
Wulffraat NM et al.; “Prolonged remission without treatment after autologous stem cell transplantation for refractory childhood systemic lupus erythematosus”; Arthritis Rheum 44(3), 728-731; March 2001
Rosen O et al.; “Autologous stem-cell transplantation in refractory autoimmune diseases after in vivo immunoablation and ex vivo depletion of mononuclear cells”; Arthritis res. 2, 327-336; 2000
Traynor AE et al.; “Treatment of severe systemic lupus erythematosus with high-dose chemotherapy and haemopoietic stem-cell transplantation: a phase I study”; Lancet 356, 701-707; August 26, 2000
Burt, RK and Traynor, AE; “Hematopoietic Stem Cell Transplantation: A New Therapy for Autoimmune Disease”; Stem Cells17, 366-372; 1999
Burt RK et al.; “Hematopoietic stem cell transplantation of multiple sclerosis, rheumatoid arthritis, and systemic lupus erythematosus”; Cancer Treat. Res. 101, 157-184; 1999
Traynor A and Burt RK; “Haematopoietic stem cell transplantation for active systemic lupus erythematosus”; Rheumatology 38, 767-772; August 1999
Martini A et al.; “Marked and sustained improvement 2 years after autologous stem cell transplant in a girl with system sclerosis”; Rheumatology 38, 773; August 1999

POLYCHONDRITIS
Rosen O et al.; “Autologous stem-cell transplantation in refractory autoimmune diseases after in vivo immunoablation and ex vivo depletion of mononuclear cells”; Arthritis res. 2, 327-336; 2000

SYSTEMIC VASCULITIS
Rabusin M et al.; “Immunoablation followed by autologous hematopoietic stem cell infusion for the treatment of severe autoimmune disease”; Haematologica 85(11 Suppl), 81-85; Nov. 2000

SJOGREN’S SYNDROME
Rabusin M et al.; “Immunoablation followed by autologous hematopoietic stem cell infusion for the treatment of severe autoimmune disease”; Haematologica 85(11 Suppl), 81-85; Nov. 2000

BEHCET’S DISEASE
Rabusin M et al.; “Immunoablation followed by autologous hematopoietic stem cell infusion for the treatment of severe autoimmune disease”; Haematologica 85(11 Suppl), 81-85; Nov. 2000

MYASTHENIA
Rabusin M et al.; “Immunoablation followed by autologous hematopoietic stem cell infusion for the treatment of severe autoimmune disease”; Haematologica 85(11 Suppl), 81-85; Nov. 2000

AUTOIMMUNE CYTOPENIA
Rabusin M et al.; “Immunoablation followed by autologous hematopoietic stem cell infusion for the treatment of severe autoimmune disease”; Haematologica 85(11 Suppl), 81-85; Nov. 2000
Papadaki HA et al.; “Assessment of bone marrow stem cell reserve and function and stromal cell function in patients with autoimmune cytopenias”; Blood 96, 3272-3275; Nov 1 2000

ALOPECIA UNIVERSAL
Seifert B et al., Complete rfemission of alopecia universalis after allogeneic hematopoietic stem cell
transplantion, Blood 105, 426-427, 1 January 2005

IMMUNODEFICIENCIES
X-LINKED LYMPHOPROLIFERATIVE SYNDROME and
X-LINKED HYPERIMMUNOGLOBULIN M SYNDROME
Banked unrelated umbilical cord blood was used to reconstitute the immune system in 2 brothers with X-linked lymphoproliferative syndrome and 1 boy with X-linked hyperimmunoglobulin-M syndrome.
Two years after transplantation, all 3 patients have normal immune systems.

These reports support the wider use of banked partially matched cord blood for transplantation in primary immunodeficiencies.
Reference:
Ziegner UH et al.; “Unrelated umbilical cord stem cell transplantation for X-linked immunodeficiencies”; J Pediatr 138(4), 570-573; April 2001
Eight children with severe immunodeficiencies treated by adult bone marrow stem cell transplants. Six of 8 showed relatively normal immune systems after 1 year.
Reference
Amrolia, P. et al., “Nonmyeloablative stem cell transplantation for congenital immunodeficiencies”, Blood 96, 1239-1246, Aug. 15, 2000.

SEVERE COMBINED IMMUNODEFICIENCY SYNDROME-X1 (ASC gene therapy)
Cavazzana-Calvo M et al.; “Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease”; Science 288, 669-672; April 28, 2000

ANEMIAS

SICKLE CELL ANEMIA
Gore L. et al.; “Successful cord blood transplantation for sickle cell anemia from a sibling who is human leukocyte antigen-identical: implications for comprehensive care”, J Pediatr Hematol Oncol 22(5):437-440; Sep-Oct 2000
Steen RG et al.; “Improved cerebrovascular patency following therapy in patients with sickle cell disease: initial results in 4 patients who received HLA-identical hematopoietic stem cell allografts”; Ann
Neurol 49(2), 222-229; Feb. 2001
Wethers DL; “Sickle cell disease in childhood: Part II. Diagnosis and treatment of major complications and recent advances in treatment”; Am. Fam. Physician 62, 1309-1314; Sept. 15, 2000

SIDEROBLASTIC ANEMIA
Ayas M et al.; “Congenital sideroblastic anaemia successfully treated using allogeneic stem cell transplantation”; Br J Haematol 113, 938-939; June 2001
Gonzalez MI et al.; “Allogeneic peripheral stem cell transplantation in a case of hereditary sideroblastic anaemia”; British Journal of Haematology 109, 658-660; 2000

RED CELL APLASIA
Rabusin M et al.; “Immunoablation followed by autologous hematopoietic stem cell infusion for the treatment of severe autoimmune disease”; Haematologica 85(11 Suppl), 81-85; Nov. 2000

APLASTIC ANEMIA
Gurman G et al.; “Allogeneic peripheral blood stem cell transplantation for severe aplastic anemia”; Ther Apher 5(1), 54-57; Feb. 2001
Kook H et al.; “Rubella-associated aplastic anemia treated by syngeneic stem cell transplantations”; Am. J. Hematol. 64, 303-305; August 2000

AMEGAKARYOCYTIC THROMBOCYTOPENIA
Yesilipek et al.; “Peripheral stem cell transplantation in a child with amegakaryocytic thrombocytopenia”; Bone Marrow Transplant 26, 571-572; Sept. 2000

CHRONIC EPSTEIN-BARR INFECTION
Fujii N et al.; “Allogeneic peripheral blood stem cell transplantation for the treatment of chronic active epstein-barr virus infection”; Bone Marrow Transplant 26, 805-808; Oct. 2000
Okamura T et al.; “Blood stem-cell transplantation for chronic active Epstein-Barr virus with lymphoproliferation”; Lancet 356, 223-224; July 2000

FANCONI’S ANEMIA
Kohli-Kumar M et al., “Haemopoietic stem/progenitor cell transplant in Fanconi anaemia using HLAmatched sibling umbilical cord blood cells”, British Journal of Haematology 85, 419-422, October 1993

DIAMOND BLACKFAN ANEMIA
Ostronoff M et al., “Successful nonmyeloablative bone marrow transplantation in a corticosteroid-resistant infant with Diamond-Blackfan anemia”, Bone Marrow Transplant. 34, 371-372, August 2004

THALASSEMIA
Tan PH et al., “Unrelated peripheral blood and cord blood hematopoietic stem cell transplants for thalassemia major”, Am J Hematol 75, 209-212, April 2004

PRIMARY AMYLOIDOSIS
Sezer O et al.; “Novel approaches to the treatment of primary amyloidosis”; Exper Opin. Investig. Drugs 9,
2343-2350; Oct 2000

ADULT STEM CELLS—REPLACEMENT OF SOLID TISSUES
OTHER METABOLIC DISORDERS

OSTEOGENESIS IMPERFECTA
Horwitz EM et al., “Isolated allogeneic bone marrow-derived mesenchymal cells engraft and stimulate growth in children with osteogenesis imperfecta: Implications for cell therapy of bone”,
Proceedings of the National Academy of Sciences USA 99, 8932-8937; 25 June 2002.
Horwitz EM et al., “Clinical responses to bone marrow transplantation in children with severe osteogenesis imperfecta”, Blood 97, 1227-1231; 1 March 2001.
Horwitz, EM et al.; “Transplantability and therapeutic effects of bone marrow-derived mesenchymal cells in children with osteogenesis imperfecta”; Nat. Med. 5, 309-313; March 1999.

OSTEOPETROSIS
Driessen GJ et al., Long-term outcome of haematopoietic stem cell transplantation in autosomal recessive osteopetrosis: an EBMT report, Bone Marrow Transplantation 32, 657-663, October 2003
Schulzetal, HLA-haploidentical blood progenitor cell transplantation in osteopetrosis, Blood 99, 3458-3460, 1 May 2002

CEREBRAL X-LINKED ADRENOLEUKODYSTROPHY
Peters C et al., Cerebral X-linked adrenoleukodystrophy: the international hematopoietic cell transplantation experience from 1982 to 1999, Blood 104, 881-888, 1 August 2004

SANDHOFF DISEASE
Karen Augé, “Stem cells infuse kin with hope”, Denver Post Tuesday, August 24, 2004

HURLER’S SYNDROME
Staba SL et al., Cord-blood transplants from unrelated donors in patients with Hurler’s syndrome”, New England Journal of Medicine 350, 1960-1969, 6 May 2004
Koc ON et al., Allogeneic mesenchymal stem cell infusion for treatment of metachromatic leukodystrophy (MLD) and Hurler syndrome (MPS-IH), Bone Marrow Transplant 215-222; Aug 2002.

KRABBE LEUKODYSTROPHY
Escolar ML et al., “Transplantation of umbilical cord-blood in babies with infantile Krabbe’s disease”, New England Journal of Medicine 352, 2069-2081, 19 May 2005
Krivit W et al., “Hematopoietic Stem-Cell Transplantation in Globoid-Cell Leukodystrophy”, New England Journal of Medicine 338, 1119-1127, Apr 16, 1998

OCULAR

CORNEAL REGENERATION
Nishida K et al., Corneal reconstruction with tissue-engineered cell sheets composed of autologous oral mucosal epithelium, New England Journal of Medicine 351, 1187-1196, 16 September 2004
Anderson DF et al.; “Amniotic Membrane Transplantation After the Primary Surgical Management of Band Keratopathy”; Cornea 20(4), 354-361; May 2001
Anderson DF et al.; “Amniotic membrane transplantation for partial limbal stem cell deficiency”; Br J Ophthalmol 85(5), 567-575; May 2001
Henderson TR et al.; “The long term outcome of limbal allografts: the search for surviving cells”; Br J Ophthalmol 85(5), 604-609; May 2001
Daya SM, Ilari FA; “Living related conjuctival limbal allograft for the treatment of stem cell deficiency”; Opthalmology 180, 126-133; January 2001
Schwab IR et al.; “Successful transplantation of bioengineered tissue replacements in patients with ocular surface disease”; Cornea 19, 421-426; July 2000.
Tsai et al.; “Reconstruction of damaged corneas by transplantation of autologous limbal epithelial cells.”; New England Journal of Medicine 343, 86-93, 2000.
Tsubota K et al.; “Treatment of severe ocular-surface disorders with corneal epithelial stem-cell transplantation”; New England Journal of Medicine 340, 1697-1703; June 3, 1999
Ocular corneal regeneration

WOUNDS & INJURIES

LIMB GANGRENE
Tateishi-Yuyama E et al.; “Therapeutic angiogenesis for patients with limb ischaemia by autologous transplantation of bone-marrow cells: a pilot study and a randomised controlled trial”; Lancet 360,
427-435; 10 August 2002.

SURFACE WOUND HEALING
Badiavas EV, “Participation of Bone Marrow Derived Cells in Cutaneous Wound Healing”, Journal Of Cellular Physiology 196, 245-250; 2003.

JAWBONE REPLACEMENT
Warnke PH et al., Growth and transplantation of a custom vascularised bone graft in a man, Lancet 364, 766-770, 28 August 2004

SKULL BONE REPAIR
Lendeckel S et al., Autologous stem cells (adipose) and fibrin glue used to treat widespread traumatic calvarial defects: case report, Journal of Cranio-Maxillofacial Surgery 32, 370-373, 2004

HEART DAMAGE
Wollert KC et al., “Intracoronary autologous bone-marrow cell transfer after myocardial infarction: the BOOST randomised controlled clinical trial”, Lancet 364, 141-148, 10 July 2004
Britten MB et al., “Infarct remodeling after intracoronary progenitor cell treatment in patients with acute myocardial infarction”; Circulation 108, 2212-2218; Nov 2003
Perin EC et al.; “Transendocardial, autologous bone marrow cell transplantation for severe, chronic ischemic heart failure”; Circulation 107, r75-r83; published online May 2003
Stamm C et al.; “Autologous bone-marrow stem-cell transplantation for myocardial regeneration”; The Lancet 361, 45-46; 4 January 2003
Tse H-F et al.; “Angiogenesis in ischaemic myocardium by intramyocardial autologous bone marrow mononuclear cell implantation”; The Lancet 361, 47-49; 4 January 2003
Strauer BE et al.; “Repair of infarcted myocardium by autologous intracoronary mononuclear bone marrow cell transplantation in humans”; Circulation 106, 1913-1918; 8 October 2002
Strauer BE et al.; “Myocardial regeneration after intracoronary transplantation of human autologous stem cells following acute myocardial infarction”; Dtsch Med Wochenschr 126, 932-938; Aug 24, 2001
Menasché P et al. “Myoblast transplantation for heart failure.” Lancet 357, 279-280; Jan 27, 2001
Menasché P et al. [“Autologous skeletal myoblast transplantation for cardiac insufficiency. First clinical case.”] [article in French] Arch Mal Coeur Vaiss 94(3), 180-182; March 2001

NEURAL DEGENERATIVE DISEASES & INJURIES
STROKE
Stilley CS et al., Changes in cognitive function after neuronal cell transplantation for basal ganglia stroke, Neurology 63, 1320-1322, October 2004
Meltzer CC et al.; “Serial [18F]Fluorodeoxyglucose Positron Emission Tomography after Human Neuronal Implantation for Stroke”; Neurosurgery 49, 586-592; 2001.
Kondziolka D et al.; “Transplantation of cultured human neuronal cells for patients with stroke”; Neurology 55, 565-569; August 2000

PARKINSON’S DISEASE
Lévesque M and Neuman T, “Autologous transplantation of adult human neural stem cells and differentiated dopaminergic neurons for Parkinson disease: 1-year postoperative clinical and functional metabolic result”, American Association of Neurological Surgeons annual meeting,
Abstract #702; 8 April 2002
Love S et al., Glial cell line-derived neurotrophic factor induces neuronal sprouting in human brain, Nature Medicine 11, 703-704, July 2005
Gill SS et al.; “Direct brain infusion of glial cell line-derived neurotrophic factor in Parkinson disease”; Nature Medicine 9, 589-595; May 2003 (published online 31 March 2003)
See also July 14, 2004 Senate testimony by Dr. Michel Lévesque:
http://commerce.senate.gov/hearings/testimony.cfm?id=1268&wit_id=3670
and Mr. Dennis Turner:
http://commerce.senate.gov/hearings/testimony.cfm?id=1268&wit_id=3676

SPINAL CORD INJURY
See July 14, 2004 Senate testimony by Dr. Jean Peduzzi-Nelson:
http://commerce.senate.gov/hearings/testimony.cfm?id=1268&wit_id=3671
and a more extensive testimony at:
http://www.stemcellresearch.org/testimony/peduzzi-nelson.htm
and Ms. Laura Dominguez:
http://commerce.senate.gov/hearings/testimony.cfm?id=1268&wit_id=3673
and Ms. Susan Fajt:
http://commerce.senate.gov/hearings/testimony.cfm?id=1268&wit_id=3674