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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).
CLICK ON LINK TO JUMP TO SECTION:
- Standard Therapies (these are all allogeneic: patient must find a matching donor)
- Therapies in Clinical Trials (these are a mix of alloegeneic and autologous)
- Experimental Treatments (these are mostly autologous: patient uses her own cells)
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:
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
Chronic Leukemia
Myelodysplastic Syndromes (ALLOgeneic therapy)
( Myelodysplasia is sometimes called pre-leukemia)
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
Inherited Immune System Disorders -
Severe Combined Immunodeficiency (SCID)
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)
( Leukemia is a cancer of the blood immune system, whose cells are called leukocytes or white cells)
- Acute Lymphoblastic Leukemia (ALL)
- Acute Myelogenous Leukemia (AML)
- Acute Biphenotypic Leukemia
- Acute Undifferentiated 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)
- Hodgkin's Lymphoma
- Non-Hodgkin's Lymphoma Burkitt's Lymphoma
Inherited Red Cell (Erythrocyte) Abnormalities (ALLOgeneic therapy)
(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
Other Disorders of Blood Cell Proliferation (ALLOgeneic therapy)
- severe Aplastic Anemia
- Congenital Dyserythropoietic Anemia
- Fanconi Anemia (Note: the first cord blood transplant in 1988 was for this disease)
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Pure Red Cell Aplasia
- Amegakaryocytosis / Congenital Thrombocytopenia
- Glanzmann Thrombasthenia
- Acute Myelofibrosis
- Agnogenic Myeloid Metaplasia (Myelofibrosis)
- Polycythemia Vera
- Essential Thrombocythemia
- 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
- Ataxia-Telangiectasia
- Bare Lymphocyte Syndrome
- Common Variable Immunodeficiency
- DiGeorge Syndrome
- Leukocyte Adhesion Deficiency
- Lymphoproliferative Disorders (LPD)
- Lymphoproliferative Disorder, X-linked (also known as Epstein-Barr Virus Susceptibility)
- Wiskott-Aldrich Syndrome
- Chediak-Higashi Syndrome
- Chronic Granulomatous Disease
- Neutrophil Actin Deficiency
- Reticular Dysgenesis
Cancers in the bone marrow (Plasma Cell Disorders) (ALLOgeneic therapy)
- Multiple Myeloma
- Plasma Cell Leukemia
- Waldenstrom's Macroglobulinemia
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
Leukodystrophy Disorders
Lysosomal Storage Diseases
Inherited Disorders - Other
- Cartilage-Hair Hypoplasia
- Gunther's Disease (Erythropoietic Porphyria)
- Hermansky-Pudlak Syndrome
- Pearson's Syndrome
- Shwachman-Diamond Syndrome
- Systemic Mastocytosis
Transplants for Inherited Metabolic Disorders (ALLOgeneic therapy)
- 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)
- Adrenoleukodystrophy (ALD)/Adrenomyeloneuropathy (AMN)
- Krabbe Disease (Globoid Cell Leukodystrophy)
- Metachromatic Leukodystrophy
- Pelizaeus-Merzbacher Disease
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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:
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
- Crohn's Disease: Phase 1 ALLO Trial: placental stem cells, Phase 2 AUTO Trial: stem cell transplants, Phase 3 ALLO Trial: PROCHYMAL[tm] mesenchymal cells, Phase 3 AUTO Trial: ASTIC
- Diabetes, Type 1 (formerly called Juvenile Diabetes): AUTO Trial: cord blood stem cells, ALLO Trial: PROCHYMAL[tm] mesenchymal cells
- Lupus
Repair of the nervous system
- Cerebral Palsy, Hypoxia, Encephalopathy (AUTOlogous)
- Multiple Sclerosis (MS): European study review, Canadian news (AUTOlogous)
- Spinal Cord Injury (SCI) (ALLOgeneic)
- Traumatic Brain Injury (AUTOlogous)
Disorders of Cell Proliferation or Metabolism
(ALLOgeneic therapy)
- Cystic Fibrosis: Australia research web page & article
- Histiocytic Disorders:
- Familial Erythrophagocytic Lymphohistiocytosis
- Hemophagocytosis
- Langerhans Cell Histiocytosis (LCH; formerly called Histiocytosis-X)
- Epidermolysis Bullosa
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)
- Glanzmann Thrombasthenia
- Severe Combined Immunodeficiency (SCID)
SCID with Adenosine Deaminase Deficiency (ADA-SCID)
SCID which is X-linked
Cellular "Cardiomyoplasty":
The treatment of cardiac patients with stem cells, either AUTOlogous or ALLOgeneic
- Myocardial Infarction: 2003 CNN report on autologous bone marrow
- Myocardial Infarction: 2009 Phase I double blind allogeneic cells
- Angina: 2009 Phase II double blind autologous cells
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
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
- Fanconi's Anemia
- Metabolic Disorders (Leukodystrophy Diseases, Storage Disorders, etc.)
- Parkinson's Disease
Nerve cell repair
Diseases of the Central Nervous System
- Duchenne Muscular Dystrophyuchenne Muscular Dystrophy
- Amyotrophic Lateral Sclerosis (ALS, or "Lou Gehrig's disease")
- Alzheimer's Disease
- Huntington's Disease
- Parkinson's Disease
- Hearing loss (AUTOlogous therapy) medical journal, newsletter1, newsletter2
- Stroke recovery (AUTOlogous therapy)
Organ repair
Kidney
Liver
Lungs
Kidney
- Combined transplant of kidney plus hematopoietic stem cells
- Growth of renal cells from adult stem cells
Liver
- Growth of liver cells from cord blood stem cells
- Repair of liver cirrhosis with cord blood stem cells
Lungs
next step...
Last modified: 02.August 2010
Copyright 2000 - 2010 Frances Verter
Copyright 2000 - 2010 Frances Verter