Treatable Diseases
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, ranging from the stem cells in the bone marrow down to specific cell types in the blood
(Leukemia is a cancer of the blood immune system, whose cells are called leukocytes or white cells)
Acute Leukemia
Chronic Leukemia
Myelodysplastic Syndromes (Myelodysplasia is sometimes called pre-leukemia)
(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)
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)
Phagocyte Disorders (Phagocytes are immune system cells that can engulf and kill foreign organisms)
Other cancers (Not originating in the blood system)
Phagocyte Disorders (Phagocytes are immune system cells that can engulf and kill foreign organisms)
Transplants for Inherited Disorders affecting the Immune System & Other Organs
Phagocyte Disorders (Phagocytes are immune system cells that can engulf and kill foreign organisms)
Transplants for Inherited Metabolic Disorders
Mucopolysaccharidosis (MPS) Storage Disease
Leukodystrophy Disorders
Lysosomal Storage Diseases
Lysosomal Storage Diseases
Inherited Disorders - Other
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 affect a cure, but the optimum dosage and usage of the stem cells is still under investigation. Patients seeking a trial for their situation should consult the first resource below, ClinicalTrials.gov.
Transplants for diseases of the Central Nervous System
Transplants for Disorders of Cell Proliferation
Epidermolysis Bullosa Gene Therapy (ie: Transplanting genetically altered stem cells)
Gene Therapy (ie: Transplanting genetically altered stem cells)
Cellular Cardiomyoplasty (ie: Strengthening damaged heart muscle by infusing stem cells or promoting their growth)
Transplants for Cancerous Tumors
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. This category also covers experiments in the laboratory, either with cell cultures or animals.
Gene Therapy (i.e.: Transplanting genetically altered stem cells)
Diseases of the Central Nervous System
Recommended Resources:
National Cancer Institute database on cancer
Oncolink is one of the best cancer guides on the internet; it is produced by the University of Pennsylvania
NIH National Library of Medicine central resource for information about clinical trials.
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.
