• Medical Professionals


Umbilical Cord Stem Cell Banking

Comprehensive list of diseases treated:

Standard therapies (cord blood) – these are diseases in which stem cell transplants are a standard treatment. In some cases they are the only therapy, and in others they are employed when first line treatments have failed, or if the disease is particularly aggressive.


Diagnosis Allogeneic Autologous
Acute Lymphoblastic Leukaemia (ALL) X
Acute Myelogenous Leukaemia (AML) X
Acute Biphenotypic Leukaemia X
Acute Undifferentiated Leukaemia X
Chronic Lymphocytic Leukaemia (CLL) X
Chronic Myelogenous Leukaemia (CML) X
Juvenile Chronic Myelogenous Leukaemia (JCML) X
Juvenile Myelomonocytic Leukaemia (JMML) X

Myelodysplastic Syndromes

Diagnosis Allogeneic Autologous
Refractory Anaemia X
Sideroblastic anaemia X
Refractory Anaemia with Excess Blasts X
Refractory Anaemia with Excess Blasts in Transformation X
Chronic Myelomonocytic Leukaemia (CMML) X


Diagnosis Allogeneic Autologous
Hodgkin’s Lymphoma X
Non-Hodgkin’s Lymphoma X

Other Disorders of Blood Cell Proliferation

Diagnosis Allogeneic Autologous
Aplastic Anaemia X X
Fanconi’s Anaemia X see gene therapy trials
Congenital Dyserythropoietic Anaemia X
Paroxysmal Nocturnal Haemoglobinuria (PNH) X
Inherited Red Cell Abnormalities
Sickle Cell Disease X
Beta Thalassemia Major X
Diamond-Blackfan Anaemia X
Pure Red Cell Aplasia X
Inherited Platelet Abnormalities
Amegakaryocytosis / Congenital Thrombocytopenia X
Glanzmann Thrombasthenia X
Inherited Immune System Disorders: Severe Combined Immunodeficiency
SCID with Adenosine Deaminase Deficiency (ADA-SCID) X
SCID which is X-linked X
SCID with absence of T & B Cells X
SCID with absence of T Cells, Normal B Cells X
Omenn Syndrome X
Inherited Immune System Disorders: Neutropenias
Infantile Genetic Agranulocytosis (Kostmann Syndrome) X
Myelokathexis X
Inherited Immune System Disorders: Other
Ataxia-Telangiectasia X
Bare Lymphocyte Syndrome X
Common Variable Immunodeficiency X
DiGeorge Syndrome X
Haemophagocytic Lymphohistiocytosis X
Leukocyte Adhesion Deficiency X
Lymphoproliferative Disorders X
Lymphoproliferative Disorder, X-linked (Susceptibility to Epstein-Barr virus) X
Wiskott-Aldrich Syndrome X
Myeloproliferative Disorders
Acute Myelofibrosis X
Agnogenic Myeloid Metaplasia (Myelofibrosis) X
Polycythemia Vera X
Essential Thrombocythaemia X
Phagocyte Disorders
Chediak-Higashi Syndrome X
Chronic Granulomatous Disease X
Neutrophil Actin Deficiency X
Reticular Dysgenesis X
Plasma cell disorders
Multiple Myeloma X X
Plasma Cell Leukaemia X X
Waldenstrom’s Macroglobulinaemia X X


Transplants for Inherited Disorders of the Immune System & Other Organs

Diagnosis Allogeneic Autologous
Cartilage-Hair Hypoplasia X
Erythropoietic Porphyria X
Hermansky-Pudlak Syndrome X
Pearson’s Syndrome X
Shwachman-Diamond Syndrome X
Systemic Mastocytosis X

Transplants for Inherited Metabolic Disorders

Diagnosis Allogeneic Autologous
Mucopolysaccharidosis (MPS) Storage Diseases
Hurler Syndrome (MPS-IH) X
Scheie Syndrome (MPS-IS) X
Hunter Syndrome (MPS-II) X
Sanfilippo Syndrome (MPS-III) X
Morquio Syndrome (MPS-IV) X
Maroteaux-Lamy Syndrome (MPS-VI) X
Sly Syndrome (MPS-VII) (beta-glucuronidase deficiency) X
Mucolipidosis II (I-cell Disease) X
Leukodystrophy Disorders
Adrenoleukodystrophy (ALD) X
Krabbe Disease(Globoid Cell Leukodystrophy) X
Metachromatic Leukodystrophy X
Pelizaeus-Merzbacher Disease X
Lysosomal Storage Diseases
Niemann-Pick Disease X
Sandhoff Disease X
Wolman Disease X
Other Inherited Metabolic Disorders
Lesch-Nyhan Syndrome X
Osteopetrosis X


Solid tumors not originating in the blood or immune system

Diagnosis Allogeneic Autologous
Neuroblastoma X
Medulloblastoma X
Retinoblastoma X

Copyright Frances Verter PhD, PGCB, 2014

Therapies in Clinical Trials (cord blood):

The phases of U.S. clinical trials in humans are defined as follows:

  1. Phase 1: Safety study to see if the procedure or drug is well-tolerated.
  2. Phase 2: Larger study to measure effectiveness of the new treatment against a control group.
  3. Phase 3: Even larger study to compare the effect of various parameters such as dose and administration, and to monitor side effects prior to market release.
  4. Phase 4: Post-marketing studies to learn even more about risks, benefits, and optimal use.


Neurologic Disorder:

Diagnosis Allogeneic Autologous
Autism Phase 2
Phase 1
Cerebral Palsy Phase 2 Phase 2
Phase 2
Phase 1
Phase 2
Hearing Loss (acquired sensorineural) Phase 2
Hypoxic Ischemic Encephalopathy (HIE) Phase 1
Spinal Cord Injury case report
Phase 2


Auto-Immune Disorders

Diagnosis Allogeneic Autologous
Amyotrophic Lateral Sclerosis (ALS) case report
Phase 1
Phase 1
Crohn’s Disease Phase 2 placenta
Phase 3 Prochymal
Phase 2 Athersys
Phase 3 ASTIC
Diabetes, Type 1 Phase 2 Phase 1
Graft-versus-Host Disease (GvHD) Phase 3 Prochymal kids
Phase 3 Prochymal adult
Kidney plus stem cell transplant Phase 2
Lupus Phase 1 review of trials
Phase 2
Multiple Sclerosis review of trials
Phase 1, news
Rheumatoid Arthritis Phase 2 placenta
Scleroderma Phase 1 case report
Phase 2 SCOT
Phase 2 CD34+


Cardiovascular diseases:

Diagnosis Allogeneic Autologous
Congenital Defects
Support for Misc. Open Cardiac Surgery Phase 1
Hypoplastic Left Heart Syndrome (HLHS) Phase 1
Grow Vascular Graft Phase 1, story
Critical Limb Ischaemia Phase 2 Phase 2
Phase 2
Phase 3 BMAC
Compartment Syndrome (Battlefield Trauma) Phase 1
Ischemic Stroke Phase 2 placenta Phase 3
Ischemic Heart Disease Meta-Analysis
Cardiac Repair
Myocardial Infarction Cell Trials blog
Phase 3 BAMI
Cardiomyopathy case report Phase 1 Texas Heart Inst.
Phase 3


Gene Therapy for Inherited Disorders
Note this list only includes gene therapy that is delivered with haematopoeitic stem cells. There are many more gene therapy clinical trials, some for the same diseases that have a different route of delivery (muscle injections, intercerebral injections, etc.)

Diagnosis Allogeneic Autologous
Adrenoleukodystrophy Phase 2 bluebird bio
Chronic Granulomatous Disease (X-linked) Phase 1
Fanconi Anemia Phase 1,
HIV Phase 1 Calimmune
Phase 1 Hutch
Metachromatic Leukodystrophy Phase 1 Milan
Severe Combined Immunodeficiency Phase 1 X-linked SCID Boston
Phase 1 X-linked SCID St. Jude
Phase 1 X-linked SCID Paris
Phase 1 ADA-SCID Duke
Sickle Cell planned trial
Thalassemia case report
Phase 1 St. Jude
Phase 1 MSK
Phase 1 bluebird bio
Wiskott-Aldrich Syndrome Phase 1
Phase 1 Milan



Diagnosis Allogeneic Autologous
Alveolar Cleft Palate Repair Gintuit FDA approval (NOT blood-forming stem cells) publication
Phase 1
Knee Cartilage Repair Cartistem KFDA approval Phase 2 IMPACT



Diagnosis Allogeneic Autologous
Bronchopulmonary Dysplasia (BPD) Phase 2 Pneumostem
Epidermolysis Bullosa paper, story
HIV Phase 1 Hutch
Berlin patient PubMed & NPR
Lysosomal Storage Diseases in utero cord blood transplant

Copyright Frances Verter, PhD, PGCB, 2014


Clinical Trials with stem cells from Cord Tissue

In January 2014 a review article found that 70% of clinical trials using Mesenchymal Stem Cells (MSC) from umbilical cord tissue are in China. This list is by no means exhaustive and new clinical trials are constantly being registered.

Diagnosis Allogeneic Autologous
Alzheimer’s Disease Phase 2 Neurostem
Aplastic Anemia publication
Ataxia (Hereditary) publication
Autism publication
Cardiomyopathy Phase 2
Cerebral Palsy case report
Diabetes, Type 1 publication
Graft-versus-Host Disease (GvHD) publication
Hypoxic Ischemic Encephalopathy (HIE) Phase 1
Liver Cirrhosis publication
Lupus publication
Multiple Sclerosis case report
Rheumatoid Arthritis publication
Spinal Cord Injury publication
Phase 3
Traumatic Brain Injury publication

Copyright Frances Verter, PhD, PGCB, 2014
Please visit www.clinicaltrials.gov for more information to search clinical trials using stem cells for specific disorders.