Diseases Treated by Blood Stem Cells 

This was the first website (in 2003) to group hematopoietic (blood) stem cell treatments according to whether they are standard, in trial, or experimental.

Since 2010 we also distinguish treatments which require "allogeneic" stem cells from another person (ALLO) versus those which use "autologous" stem cells from the patient (AUTO). 

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Image courtesy of Dr. Jeff Drew
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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 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 only pay for a stem cell transplant if it is a "standard therapy" for the patient's diagnosis.

The vast majority of 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 May 2010).


Recommended Resources:

Cancer.gov National Cancer Institute database on cancer
www.acor.org Association of Cancer Online Resources is a clearinghouse for both medical information and patient support groups
www.oncolink.com Oncolink is one of the best cancer guides on the internet; it is produced by the University of Pennsylvania
 
Leukemias (ALLOgeneic therapy)
( 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 (ALLOgeneic therapy)
( 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 (ALLOgeneic)
( Lymphoma is a cancer of the leukocytes that circulate in the blood and lymph vessels)
Inherited Red Cell (Erythrocyte) Abnormalities (ALLOgeneic therapy)
(Red cells contain hemoglobin and carry oxygen to the body)
Other Disorders of Blood Cell Proliferation (ALLOgeneic therapy)
Anemias (Anemias are deficiencies or malformations of red cells)
Inherited Platelet Abnormalities ( Platelets are small blood cells
needed for clotting)
Myeloproliferative Disorders
  • 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) (ALLOgeneic therapy)
Solid tumors (Not originating in the blood system) (AUTOLOGOUS therapy)
Transplants for Inherited Disorders effecting the Immune System & Other Organs (ALLOgeneic therapy)

Transplants for Inherited Metabolic Disorders (ALLOgeneic therapy)

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
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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, ClinicalTrials.gov.

Recommended Resources:

ClinicalTrials.gov 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.
www.marrow.org National Marrow Donor Program (NMDP) has a Patient Resources page on learning about clinical trials which apply stem cell transplants.
www.ninds.nih.gov National Institute of Neurological Disorders and Stroke (NINDS) offers an index covering a long list of neurological diseases.
www.rarediseases.org National Organization for Rare Disorders (NORD) has compiled an index of rare diseases, which includes alternate disease names and disorder subdivisions.
 
Auto-Immune Diseases


Repair of the nervous system


    Disorders of Cell Proliferation or Metabolism (ALLOgeneic therapy)

    Gene Therapy: Transplanting the patient's AUTOlogous stem cells after they are genetically altered

    Cellular "Cardiomyoplasty":
    The treatment of cardiac patients with stem cells, either AUTOlogous or ALLOgeneic


    Transplants for Cancerous Tumors     (ALLOgeneic therapy)
    Organ repair (AUTOlogous therapy)

    • restoring vision by growing a new cornea

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    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. 

    In experimental research, it is not always clear whether an eventual therapy, if developed, would be auotologous or allogeneic.

    Auto-Immune Diseases (AUTOlogous therapy, European review)
    Gene Therapy:     Transplanting the patient's AUTOlogous stem cells after they are genetically altered
    Nerve cell repair

    Diseases of the Central Nervous System

      Other forms of nerve damage

      Organ repair

      Kidney

      Liver

      Lungs


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      Last modified: 02.August 2010
      Copyright 2000 - 2010 Frances Verter