Diseases Treated by Blood Stem Cells 

This page groups hematopoietic (blood) stem cell treatments according to whether they are standard, in trial, or experimental.

Hematopoietic stem cells are capable of evolving into all the specific cell types in the blood and immune system. They can be found in people of all ages. The three sources of hematopoietic stem cells which are routinely used for medical treatments are:
  1. the bone marrow of an adult person
  2. the peripheral blood of an adult person
  3. the umbilical cord blood of a newborn baby
When a patient requires a Hemaotpoietic Stem Cell Transplant (HSCT), the treating physician will decide which source of stem cells to use. This will depend on several factors, including but not limited to: the degree of match between donor and patient (sometimes the donor and patient are one and the same person), the expected speed of engraftment, and the amount of time available to search for a perfectly matching donor.

Categories below:
Image courtesy of Dr. Jeff Drew

Standard Therapies

These are diseases for which 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 below), 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 only pay for a stem cell transplant if it is a "standard therapy" for the patient's diagnosis. As an example, for illustrative purposes only, here is a list of diagnoses for which the large insurance carrier Blue Cross/Blue Shield will cover allogeneic transplants.

Recommended Resources: National Cancer Institute database on cancer Association of Cancer Online Resources is a clearinghouse for both medical information and patient support groups Oncolink is one of the best cancer guides on the internet; it is produced by the University of Pennsylvania
(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)


( Lymphoma is a cancer of the leukocytes that circulate in the blood and lymph vessels)
Inherited Red Cell (Erythrocyte) Abnormalities

(Red cells contain hemoglobin and carry oxygen to the body)
Other Disorders of Blood Cell Proliferation

Anemias (Anemias are deficiencies or malformations of red cells)
Inherited Platelet Abnormalities (Platelets are small blood cells
needed for clotting)
  • Acute Myelofibrosis
  • Agnogenic Myeloid Metaplasia (Myelofibrosis)
  • Polycythemia Vera
  • Essential Thrombocythemia

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
Phagocyte Disorders ( Phagocytes are immune system cells
that can engulf and kill foreign organisms)
Cancers in the bone marrow (Plasma Cell Disorders)

Other cancers
(Not originating in the blood system)

Therapies in Clinical Trials

These are diseases for which stem cell treatments have been shown 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 effect a cure, but the optimum dosage and usage of the stem cells is still under investigation. In the United States, patients with these diagnoses can usually only get access to a stem cell treatments if they are enrolled in a clinical trial. Patients seeking a trial for their situation should consult the first resource below,

Recommended Resources: NIH National Library of Medicine central resource for information about clinical trials.  Search for a trial that is enrolling patients for a specific condition.  Unfortunately, many clinical trials are not incuded in this list. National Marrow Donor Program (NMDP) has a Patient Resources page on learning about clinical trials which apply stem cell transplants. National Institute of Neurological Disorders and Stroke (NINDS) offers an index covering a long list of neurological diseases. National Organization for Rare Disorders (NORD) has compiled an index of rare diseases, which includes alternate disease names and disorder subdivisions.
Transplants for Cancerous Tumors
Transplants for Inherited Disorders effecting the Immune System & Other Organs

Transplants for Inherited Metabolic Disorders

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
Lysosomal Storage Diseases
Inherited Disorders - Other
Transplants for Disorders of Cell Proliferation

Histiocytic Disorders
  • Familial Erythrophagocytic Lymphohistiocytosis
  • Hemophagocytosis
  • Langerhans Cell Histiocytosis (LCH; formerly called Histiocytosis-X)

Transplants for diseases of the Central Nervous System

Gene Therapy
(ie: Transplanting genetically altered stem cells)
Cellular Cardiomyoplasty
(ie: Strengthening damaged heart muscle by infusing stem cells or promoting their growth)

Experimental Treatments

These are diseases for which stem cell treatments have not been proven to have any efficacy in human beings. In a "Phase I" clinical trial, the purpose of the study is to find out if the therapy makes any difference in the course of the disease, as compared to a control group. Some doctors may also be giving stem cells to patients in experiments outside of clinical trials, but in the United States such experiments are subject to limitations imposed by FDA regulations. This category also covers experiments in the laboratory, either with cell cultures or animals.

Auto-Immune Diseases
Gene Therapy (ie: Transplanting genetically altered stem cells)

Nerve cell repair

Diseases of the Central Nervous System
Traumatic injury
Organ repair


Next: Private Cord Blood Banks in the USA