Stem cells will be grafted into the brains of patients during the new trial.ALAMYPosted on 01/20/2009 11:12:15 PM PST by neverdem
Stroke patients to be treated with tailor-made brain cells.
Stem cells will be grafted into the brains of patients during the new trial.ALAMYUK researchers have been given the go-ahead for a clinical trial to assess the use of stem-cell transplants for stroke. Twelve people will take part in the preliminary safety study, the first time that brain-derived stem cells have been used to treat stroke patients.
The trial, due to start later this year, will see different doses of cultured human neural stem cells grafted into the brains of patients who have had a stroke — often caused by a blood clot blocking one of the vessels leading to the brain. The study will assess how safe the procedure is, but will also monitor changes in mobility and brain function.
"I'm cautious but hopeful," says neurologist Keith Muir of the University of Glasgow, UK, who will coordinate the trial. "We've tried a lot of things in stroke over the years, most of which haven't been helpful."
Stroke is the leading cause of death and the single biggest cause of adult disability in the developed world. Around half of all stroke survivors are left with permanent disabilities. Although clot-busting treatments can help restore blood flow if given soon after the stroke, most treatment focuses on rehabilitation such as physiotherapy.
So researchers are searching for other therapies. Stem-cell transplants are an attractive option because the primitive cells have the potential to form many other cell types, and may be able to repair or replace damaged brain cells.
Human stem cells have been grafted into the brains of stroke patients before, but the results were inconclusive. Because those cells came from an embryonic tumour, scientists were concerned that they might become cancerous in the patients' brains.
The cells in this study, developed by UK biotechnology firm ReNeuron, have been tailor-made for transplantation. Originally isolated from a human fetus, the cells have been modified by the addition of a gene that promotes cell growth. This helps them to divide in culture in the lab so they can be grown up into the vast numbers required for the trial.
Crucially, the gene's activity can be switched on and off by adding or removing a chemical in the culture dish. When the chemical is removed, the cells stop dividing and are ready for transplantation without the risk of tumour formation. The UK Medicines and Healthcare products Regulatory Agency has given the green light for the use of these cells in the study.
"It's exciting to see things that we've worked on for many years coming to trial," says Steve Dunnett from Cardiff University, UK, who studies cell transplantation.
ReNeuron researchers have already tested the therapy in stroke-damaged rat brains, in which the cells prompted new blood vessels and neurons to form. The animals also regained control of movement in their front paws.
The cells will be transplanted into patients around six months after their stroke, by which time any remaining brain damage is thought to be permanent. "But it's hard to imagine that once you've got an area of dead tissue, transplanting cells into it will make much difference," says Roger Barker from the University of Cambridge, UK, who studies transplantation therapies for neurodegenerative diseases. It may be that the cells will work better if transplanted earlier, he adds.
I hope they grow bones in their brains.
regenerative medicine ping
It may have been a spontaneous abortion, aka miscarriage. You can't tell from these detail free stories.
Other press releases say it was cells from a cell line from aborted fetus.
I find press releases usually wanting. Chemical or surgical abortions are morally and ethically tainted. Spontaneous abortions are considered normal, but questionable as far as being compatible with life.
“ReNeuron Requests Human Stem Cell Trials
Posted on: Wednesday, 6 December 2006, 21:00 CST
UK biotechnology company ReNeuron has applied to the FDA for authorization to begin trials that will use the stem cells of fetuses to treat stroke patients and potentially reverse their brain damage.
ReNeuron has successfully extracted stem cells from the developing brain of a 12 week old aborted fetus. The company plans to use the stem cells, known as ReN001, in a human trial and implant the cells into the brains of 12 stroke patients.”
redorbit.com/news/health/758221/reneuron_requests_human_stem_cell_trials/index.html This was a few years ago and now they have the green light.
What stopped it in the U.S. was law, not the moral and ethical fastidiousness of the researchers.
Otherwise, I fear something like this (Warning: Gross!)
Cheers!
Some info. that says yes.
Updated: 4/11/2007
PEER-REVIEWED REFERENCES SHOWING APPLICATIONS OF
ADULT STEM CELLS THAT PRODUCE
THERAPEUTIC BENEFIT FOR HUMAN PATIENTS
(not a complete listing, sample 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
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
Updated: 4/11/2007
NON-HODGKIN’S LYMPHOMA
Buadi FK et al., Autologous hematopoietic stem cell transplantation for older patients with relapsed non-
Hodgkin’s lymphoma, Bone Marrow Transplant 37, 1017-1022, June 2006
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
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
ACUTE LYMPHOBLASTIC LEUKEMIA
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
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
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
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
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
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
CHRONIC MYELOMONOCYTIC LEUKEMIA
Updated: 4/11/2007
Elliott MA et al., Allogeneic stem cell transplantation and donor lymphocyte infusions for chronic
myelomonocytic leukemia, Bone Marrow Transplantation 37, 1003-1008, 2006
Updated: 4/11/2007
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
Aviles A et al., Biological modifiers as cytoreductive therapy before stem cell transplant in previously
untreated patients with multiple myeloma, Annals of Oncology 16, 219-221, 2005
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
RENAL CELL CARCINOMA
Barkholt L et al., Allogeneic haematopoietic stem cell transplantation for metastatic renal carcinoma in
Europe, Annals of Oncology published online 28 April 2006
Arya M et al., Allogeneic hematopoietic stem-cell transplantation: the next generation of therapy for
metastatic renal cell cancer, Nat Clin Pract Oncol. 1, 32-38, Nov 2004
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
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
Updated: 4/11/2007
EWING’S SARCOMA
Drabko K et al., Megachemotherapy followed by autologous stem cell transplantation in children with
Ewing’s sarcoma, Pediatric Transplantation 9, 618-621, 2005
VARIOUS SOLID TUMORS
Pedrazolli P et al., High dose chemotherapy with autologous hematopoietic stem cell support for solid
tumors other than breast cancer in adults, Annals of Oncology published online 17 March 2006
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
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
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
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
MYELOFIBROSIS
Cornetta K et al., Umbilical cord blood transplantation in adults: results of the prospective Cord Blood
Transplantation (COBLT), Biol Blood Marrow Transplant 11, 149-160, February 2005
Cervantes F, Modern management of myelofibrosis, Br J Haematol 128, 583-592, March 2005
Kroger N et al., Pilot study of reduced-intensity conditioning followed by allogeneic stem cell
transplantation from related and unrelated donors in patients with myelofibrosis, Br J Haematol
128, 690-697, March 2005
Thiele J et al., Dynamics of bone marrow changes in patients with chronic idiopathic myelofibrosis
following allogeneic stem cell transplantation, Histol Histopathol 20, 87-89, July 2005
Rondelli D et al., Allogeneic hematopoietic stem-cell transplantation with reduced-intensity conditioning in
intermediate- or high-risk patients with myelofibrosis with myeloid metaplasia, Blood 105, 4115-
4119, 15 May 2005
Benesova P et al., [Complete regression of bone marrow fibrosis following allogeneic peripheral blood
stem cell transplantation in a patient with idiopathic myelofibrosis] [Article in Czech], Cesk Patol
40, 167-171, October 2004
Updated: 4/11/2007
ADULT STEM CELLS—IMMUNE SYSTEM REPLACEMENT
AUTOIMMUNE DISEASES
DIABETES TYPE I (JUVENILE DIABETES)
Voltarelli JC et al., Autologous Nonmyeloablative Hematopoietic Stem Cell Transplantation in Newly
Diagnosed Type 1 Diabetes Mellitus, Journal of the American Medical Association 297, 1568-1576, 11
April 2007
SYSTEMIC LUPUS
Burt RK et al., Nonmyeloablative hematopoietic stem cell transplantation for systemic lupus
erythematosus, Journal of the American Medical Association 295, 527-535, February 1, 2006
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
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
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
Passweg, JR et al., Haematopoetic stem cell transplantation for refractory autoimmune cytopenia, British
Journal of Haematology 125, 749-755, June 2004
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
SCLEROMYXEDEMA
A.M. Feasel et al., “Complete remission of scleromyxedema following autologous stem cell
transplantation,” Archives of Dermatology 137, 1071-1072; Aug. 2001.
SCLERODERMA
Updated: 4/11/2007
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
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
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
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
I M de Kleer et al., Autologous stem cell transplantation for refractory juvenile idiopathic arthritis: analysis
of clinical effects, mortality, and transplant related morbidity, Ann Rheum Dis 63, 1318-1326, 2004
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
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
Updated: 4/11/2007
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
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
ALOPECIA UNIVERSAL
Seifert B et al., Complete rfemission of alopecia universalis after allogeneic hematopoietic stem cell
transplantion, Blood 105, 426-427, 1 January 2005
BUERGER’S DISEASE
Kim D-I et al., Angiogenesis facilitated by autologous whole bone marrow stem cell transplantation for
Buerger’s disease, Stem Cells 24, 1194-1200, 2006
IMMUNODEFICIENCIES
SEVERE COMBINED IMMUNODEFICIENCY SYNDROME
Grunebaum E et al., Bone marrow transplantation for severe combined immune deficiency, Journal of the
American Medical Association 295, 508-518, 1 February 2006
Cavazzana-Calvo M et al.; “Gene therapy of human severe combined immunodeficiency (SCID)-X1
disease”; Science 288, 669-672; April 28, 2000
(NOTE: gene therapy using bone marrow adult stem cells as gene vehicle)
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 Xlinked
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.
Updated: 4/11/2007
Reference
Amrolia, P. et al., “Nonmyeloablative stem cell transplantation for congenital immunodeficiencies”, Blood
96, 1239-1246, Aug. 15, 2000.
Updated: 4/11/2007
ANEMIAS and OTHER BLOOD CONDITIONS
SICKLE CELL ANEMIA
Klein A et al., Hematopoietic stem cell transplantation for severe sickle cell disease, Rev Med Brux.
2005;26 Spec no:Sp23-5
Adamkiewicz TV et al., Transplantation of unrelated placental blood cells in children with high-risk sickle
cell disease, Bone Marrow Transplant. 34, 405-411, Sept 2004
Wu CJ et al., Molecular assessment of erythroid lineage chimerism following nonmyeloablative allogeneic
stem cell transplantation, Exp Hematol. 31, 924-933, Oct 2003
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
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
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
AMEGAKARYOCYTIC THROMBOCYTOPENIA
Yesilipek et al.; “Peripheral stem cell transplantation in a child with amegakaryocytic thrombocytopenia”;
Bone Marrow Transplant 26, 571-572; Sept. 2000
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
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
Updated: 4/11/2007
Updated: 4/11/2007
FANCONI’S ANEMIA
Bitan M et al., Fludarabine-based reduced intensity conditioning for stem cell transplantation of fanconi
anemia patients from fully matched related and unrelated donors, Biol Blood Marrow Transplant.
12, 712-718, July 2006
Tan PL et al., Successful engraftment without radiation after fludarabine-based regimen in Fanconi anemia
patients undergoing genotypically identical donor hematopoietic cell transplantation, Pediatr Blood
Cancer, 46, 630-636, May 1, 2006
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
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
Updated: 4/11/2007
ADULT STEM CELLS—REPAIR/REPLACEMENT
OF SOLID TISSUES
METABOLIC DISORDERS
HURLER’S SYNDROME
Cox-Brinkman J et al., Haematopoietic cell transplantation (HCT) in combination with enzyme
replacement therapy (ERT) in patients with Hurler syndrome, Bone Marrow Transplantation 38,
17-21, 2006
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.
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.
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
OSTEOPETROSIS
Tsuji Y et al., Successful nonmyeloablative cord blood transplantation for an infant with malignant
infantile osteopetrosis, J Pediatr Hematol Oncol. 27, 495-498, Sept 2005
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
Schulz et al., 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
OCULAR
CORNEAL REGENERATION
Inatomi T et al., Midterm results on ocular surface reconstruction using cultivated autologous oral mucosal
epithelial transplantation, American Journal of Ophthalmology 141, 267-275, February 2006
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
Updated: 4/11/2007
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
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 and Falanga V, “Treatment of chronic wounds with bone marrow-derived cells”, Archives of
Dermatology 139, 510-516, 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
ACUTE HEART DAMAGE
Joseph J et al., Safety and effectiveness of granulocyte-colony stimulating factor in mobilizing stem cells
and improving cytokine profile in advanced chronic heart failure, American Journal of Cardiology
97, 681-684, 1 March 2006
Blocklet D et al., Myocardial homing of nonmobilized peripheral-blood CD34+ cells after intracoronary
injection, Stem Cells 24, 333-336, February 2006
Janssens S et al., Autologous bone marrow-derived stem-cell transfer in patients with ST-segment elevation
myocardial infarction: double-blind, randomised controlled trial, Lancet 367, 113-121, 14 January
2006
Patel AN et al., Surgical treatment for congestive heart failure with autologous adult stem cell
transplantation: a prospective randomized study, Journal Thoracic Cardiovascular Surgery 130,
1631-1638, December 2005
Updated: 4/11/2007
Ince H et al., Preservation from left ventricular remodeling by front-integrated revascularization and stem
cell liberation in evolving acute myocardial infarction by use of granulocyte-colony-stimulating
factor (FIRSTLINE-AMI), Circulation 112, 3097-3106, 15 November 2005
Ince H et al., Prevention of left ventricular remodeling with granulocyte colony-stimulating after acute
myocardial infarction, Circulation 112, I-73-I-80, 30 August 2005
Bartunek J et al., Intracoronary injection of CD133-positive enriched bone marrow progenitor cells
promotes cardiac recovery after recent myocardial infarction, Circulation 112, I-178-I-183, 30
August 2005
Dohmann HFR et al., Transendocardial autologous bone marrow mononuclear cell injection in ischemic
heart failure, Circulation 112, 121-126, 26 July 2005
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
CHRONIC CORONARY ARTERY DISEASE
Strauer BE et al., Regeneration of human infarcted heart muscle by intracoronary autologous bone marrow
cell transplantation in chronic coronary artery disease, Journal of the American College of
Cardiology 46, 1651-1658, 1 November 2005
NEURAL DEGENERATIVE DISEASES & INJURIES
STROKE
Shyu W-C et al., Granulocyte colony-stimulating factor for acute ischemic stroke: a randomized controlled
trial, Canadian Medical Association Journal 174, 927-933, 28 March 2006
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
Updated: 4/11/2007
PARKINSON’S DISEASE
USING DIRECT STIMULATION OF PATIENTS’ ENDOGENOUS ADULT NEURAL
STEM CELLS:
Love S et al., Glial cell line-derived neurotrophic factor induces neuronal sprouting in human brain, Nature
Medicine 11, 703-704, July 2005
Slevin JT et al., Improvement of bilateral motor functions in patients with Parkinson disease through the
unilateral intraputaminal infusion of glial cell line-derived neurotrophic factor, Journal of
Neurosurgery 102, 216-222, February 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)
SPINAL CORD INJURY
Lima C et al., Olfactory mucosa autografts in human spinal cord injury: A pilot clinical study, Journal of
Spinal Cord Medicine 29, 191-203, July 2006
LIVER DISEASE
CHRONIC LIVER DISEASE
Gordon MY et al., Characterisation and clinical application of human CD34+ stem/progenitor cell
populations mobilised into the blood by G-CSF, Stem Cells 24, 1822-1830, July 2006; published
online March 30, 2006
LIVER CIRRHOSIS
Terai S et al., Improved liver function in liver cirrhosis patients after autologous bone marrow cell fusion
therapy, Stem Cells published online 15 June 2006; DOI: 10.1634/stemcells.2005-0542
BLADDER DISEASE
END-STAGE BLADDER DISEASE
Atala A et al., Tissue-engineered autologous bladders for patients needing cytoplasty, The Lancet 367,
1241-1246, 15 April 2006
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