Diseases Treated

This website became the first in 2003 to sort diseases treated with blood-forming stem cells into the following 3 categories: (see below)

It is also important to distinguish between 2 modes of treatment:

Allogeneic - patient receives stem cells from a matching donor, either a sibling or an unrelated donor
Autologous - patient receives their own stem cells

When parents donate to a public bank, they are supporting patients around the world who are searching for an unrelated Allogeneic donor. When parents save cord blood in a family bank, they are reserving the options that the baby can use its own stem cells for an Autologous treatment or an immediate relative (sbiling or parents) can use the stem cells for an Allogeneic treatment.

The lists below include ALL therapies that use blood-forming stem cells, without distinction as to whether the stem cells were extracted from bone marrow, cord blood, or other perinatal tissues. In fact there are some differences in the cell populations from the different cell sources, but those are omitted in an effort to give a comprehensive overview of therapeutic options.

Finally, in 2011 we have added a new category to this page listing Clinical Trials with stem cells from Cord Tissue. The stem cells of interest in cord tissue are not hematopoietic (blood) stem cells, but Mesenchymal Stem Cells (MSC; more details if you download this pdf poster). Since human research with cells from cord tissue is a very new field, the treatments in that section are not sub-divided.

This image (courtesy of Dr. Jeff Drew) shows how a hematopoietic (blood) stem cell can differentiate to create the entire lineage of blood cells:

Standard Therapies

These are diseases for which transplants of blood-forming stem cells (Hematopoietic Stem Cell Transplants, HSCT) are a standard treatment. For some diseases they are the only therapy, and in other diseases they are only employed when front-line therapies have failed or the disease is very aggressive. Most of the diseases for which HSCT is a standard treatment are disorders of blood cell lineage (see figure above), ranging from the stem cells in the bone marrow down to specific cell types in the blood. In the United States, most health insurance providers will pay for a stem cell transplant if it is a "standard therapy" for the patient's diagnosis.

Almost all standard therapies are allogeneic, where the patient must find a matching donor. The only exceptions are patients who are transplanted for solid tumors or acquired anemias (last updated Sept. 2011).

Leukemias (all therapies are allogeneic)
(Leukemia is a cancer of the blood immune system, whose cells are called leukocytes or white cells)

Acute Leukemia

Acute Lymphoblastic Leukemia (ALL)
Acute Myelogenous Leukemia (AML)
Acute Biphenotypic Leukemia
Acute Undifferentiated Leukemia

Chronic Leukemia

Chronic Myelogenous Leukemia (CML)
Chronic Lymphocytic Leukemia (CLL)
Juvenile Chronic Myelogenous Leukemia (JCML)
Juvenile Myelomonocytic Leukemia (JMML)

Myelodysplastic Syndromes (Myelodysplasia is sometimes called pre-leukemia)

Refractory Anemia (RA)
Refractory Anemia with Ringed Sideroblasts (RARS)
Refractory Anemia with Excess Blasts (RAEB)
Refractory Anemia with Excess Blasts in Transformation (RAEB-T)
Chronic Myelomonocytic Leukemia (CMML)

Lymphomas (all therapies are allogeneic)
(Lymphoma is a cancer of the leukocytes that circulate in the blood and lymph vessels)

Hodgkin's Lymphoma
Non-Hodgkin's Lymphoma (Burkitt's Lymphoma)

Other Disorders of Blood Cell Proliferation (all therapies are allogeneic)

Anemias (Anemias are deficiencies or malformations of red cells)

Aplastic Anemia
Congenital Dyserythropoietic Anemia
Fanconi Anemia (Note: the first cord blood transplant in 1988 was for FA, an inherited disorder)
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Pure Red Cell Aplasia

Inherited Red Cell Abnormalities (Red cells contain hemoglobin and carry oxygen to the body)

Beta Thalassemia Major (also known as Cooley's Anemia)
Blackfan-Diamond Anemia
Pure Red Cell Aplasia
Sickle Cell Disease

Inherited Platelet Abnormalities (Platelets are blood cells needed for clotting)

Amegakaryocytosis / Congenital Thrombocytopenia
Glanzmann Thrombasthenia

Inherited Immune System Disorders - Severe Combined Immunodeficiency (SCID)

SCID with Adenosine Deaminase Deficiency (ADA-SCID)
SCID which is X-linked
SCID with absence of T & B Cells
SCID with absence of T Cells, Normal B Cells
Omenn Syndrome

Inherited Immune System Disorders - Neutropenias

Inherited Immune System Disorders - Other

Ataxia-Telangiectasia
Bare Lymphocyte Syndrome
Common Variable Immunodeficiency
DiGeorge Syndrome
Hemophagocytic Lymphohistiocytosis
Leukocyte Adhesion Deficiency
Lymphoproliferative Disorders (LPD)
Lymphoproliferative Disorder, X-linked (also known as Epstein-Barr Virus Susceptibility)
Wiskott-Aldrich Syndrome

Myeloproliferative Disorders

Acute Myelofibrosis
Agnogenic Myeloid Metaplasia (Myelofibrosis)
Polycythemia Vera
Essential Thrombocythemia

Phagocyte Disorders (Phagocytes are immune system cells that can engulf and kill foreign organisms)

Chediak-Higashi Syndrome
Chronic Granulomatous Disease
Neutrophil Actin Deficiency
Reticular Dysgenesis

Bone marrow cancers (Plasma Cell Disorders)

Transplants for Inherited Disorders effecting the Immune System & Other Organs
(all therapies are Allogeneic)

Cartilage-Hair Hypoplasia
Gunther's Disease (Erythropoietic Porphyria)
Hermansky-Pudlak Syndrome
Pearson's Syndrome
Shwachman-Diamond Syndrome
Systemic Mastocytosis

Transplants for Inherited Metabolic Disorders
(all therapies are Allogeneic - but beware that for some of these disorders, only certain manifestations are transplanted, and it is best to consult a hematologist with cord blood transplantation experience for advice)

Mucopolysaccharidoses (MPS) Storage Diseases

Mucopolysaccharidoses (MPS)
Hurler's Syndrome (MPS-IH)
Scheie Syndrome (MPS-IS)
Hunter's Syndrome (MPS-II)
Sanfilippo Syndrome (MPS-III)
Morquio Syndrome (MPS-IV)
Maroteaux-Lamy Syndrome (MPS-VI)
Sly Syndrome, Beta-Glucuronidase Deficiency (MPS-VII)
Mucolipidosis II (I-cell Disease)

Leukodystrophy Disorders

Adrenoleukodystrophy (ALD)/Adrenomyeloneuropathy (AMN)
Krabbe Disease (Globoid Cell Leukodystrophy)
Metachromatic Leukodystrophy
Pelizaeus-Merzbacher Disease

Lysosomal Storage Diseases

Inherited Metabolic Disorders - Other

Solid tumors not originating in the blood or immune system (Autologous therapy)

Therapies in Clinical Trials

These are diseases for which stem cell treatments appear to be beneficial, but have not been adopted as standard therapy. For some of these diseases, stem cell transplants only slow the progression of the disease, but do not produce a cure. For other diseases, stem cell treatments may help effect a cure, but further research is needed to determine the best candidate patients for stem cell therapy, the optimum stem cell dosage, the optimum method of cell delivery, etc.

In the United States, patients with these diagnoses can only get access to stem cell therapy if they are enrolled in a clinical trial. Patients seeking a trial for their situation should consult ClinicalTrials.gov. The phases of clinical trials are defined as follows:

Phase 1: Safety study to see if the procedure or drug is well-tolerated.
Phase 2: Larger study to measure effectiveness of the new treatment against a control group.
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.
Phase 4: Post-marketing studies to learn even more about risks, benefits, and optimal use.

The therapies in clinical trials are a mix of Allogeneic and Autologous modes of treatment.

Auto-Immune

Graft-versus-Host Disease (GvHD): Phase 3 Allogeneic Trial - pediatric: PROCHYMAL[tm] mesenchymal cells; Phase 3 Allogeneic Trial - adult: PROCHYMAL[tm] mesenchymal cells; Kidney transplant plus stem cells from donor's allogeneic bone marrow

Diabetes, Type 1 (formerly called Juvenile Diabetes): Published Phase 1 Trial: autologous cord blood stem cells (follow up trial of expanded Treg cells); Enrolling Phase 1 Trial: autologous cord blood stem cells; Enrolling Phase 2 Trial: autologous cord blood stem cells; Enrolling Phase 2 Trial: allogeneic cord blood stem cells in the "Stem Cell Educator", see publication; Approved Phase 2 Trial: Allogeneic PROCHYMAL[tm] mesenchymal cells (granted FDA Orphan Drug Status)

Crohn's Disease: Phase 2 Allogeneic Trial: placental stem cells, (this was 1st placenta cell trial approved by the FDA); Phase 2 Autologous Trial: stem cell transplants; Phase 3 Allogeneic Trial: PROCHYMAL[tm] mesenchymal cells; Phase 3 Autologous Trial: ASTIC; Ulcerative Colitis Phase 2 Allogeneic Trial: MultiStem adult stem cells prepared by Athersys

Rheumatoid Arthritis: Phase 2 Allogeneic Trial: placental stem cells prepared by Celgene

Diseases Treated

Standard Therapies

Therapies in Clinical Trials

Experimental Treatments

Clinical Trials with stem cells from Cord Tissue