Disease Treatments


· A Parents Guide to Cord Blood
· Standard Therapies
· Lymphomas
· Therapies in Clinical Trials
· Experimental Treatments
 
A Parents Guide to Cord Blood
Verter, F. (2006) Diseases Treated by Blood Stem Cells. Retrieved from A Parents Guide to Cord Blood
Banks USA Website:

www.parentsguidecordblood.com/content/usa/medical/diseases.shtml
 
 
  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:
 
www.cancer.govNational Cancer Institute database on cancer
www.acor.orgAssociation of Cancer Online Resources is a clearinghouse for both medical information and patient support groups
www.oncolink.comOncolink is one of the best cancer guides on the internet; it is produced by the University of Pennsylvania
Leukemias
(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
 
Lymphoma is a cancer of the leukocytes that circulate in the blood and lymph vessels)
 
• Hodgkin's Lymphoma
• ANon-Hodgkin's Lymphoma Burkitt's Lymphoma
   
   
Inherited Red Cell (Erythrocyte) 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
   
   
Other Disorders of Blood Cell Proliferation Anemias (Anemias are deficiencies or malformations of red cells)
• 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
   
   
Inherited Platelet Abnormalities (Platelets are small blood cells needed for clotting)
• Amegakaryocytosis / Congenital Thrombocytopenia
• Glanzmann Thrombasthenia
   
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
• Kostmann Syndrome
• Myelokathexis
   
   
Inherited Immune System Disorders - Other
• 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
   
   
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
   
   
Cancers in the bone marrow (Plasma Cell Disorders)
• Multiple Myeloma
• Plasma Cell Leukemia
• Waldenstrom's Macroglobulinemia
   
   
Other cancers (Not originating in the blood system)
• Neuroblastoma
• Retinoblastoma
   
   
 
 
  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.
   
   
Transplants for Cancerous Tumors
• Breast Cancer
• Ewing's sarcoma
• Renal cell carcinoma
   
   
Transplants for Inherited Disorders effecting the Immune System & Other Organs
• Cartilage-Hair Hypoplasia
• Gunther's Disease (Erythropoietic Porphyria)
• Hermansky-Pudlak Syndrome
• Pearson's Syndrome
• Shwachman-Diamond Syndrome
• Systemic Mastocytosis
   
   
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
• Adrenoleukodystrophy (ALD)/Adrenomyeloneuropathy (AMN)
• Krabbe Disease (Globoid Cell Leukodystrophy)
• Metachromatic Leukodystrophy
   
   
Lysosomal Storage Diseases
• Gaucher Disease
• Niemann-Pick Disease
• Sandhoff Disease
• Tay-Sachs Disease
• Tay-Sachs Disease
   
   
Inherited Disorders - Other
• Lesch-Nyhan Syndrome
• Osteopetrosis
   
   
Transplants for Disorders of Cell Proliferation Histiocytic Disorders
• Familial Erythrophagocytic Lymphohistiocytos
• Hemophagocytosis
• Langerhans Cell Histiocytosis (LCH; formerly called Histiocytosis-X
   
   
Transplants for diseases of the Central Nervous System
• Multiple Sclerosis (MS)
   
   
Gene Therapy (ie: Transplanting genetically altered stem cells)
• Glanzmann Thrombasthenia
• Severe Combined Immunodeficiency (SCID)
   
   
SCID with Adenosine Deaminase Deficiency (ADA-SCID) SCID which is X-linked
Cellular Cardiomyoplasty (ie: Strengthening damaged heart muscle by infusing stem cells or promoting their growth)
• autologous stem cell transplantation
• drug-enhanced stem cell proliferation
   
   
 
 
  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
• Arthritis, Juvenile
• Arthritis, Rheumatoid
• Crohn's Disease
• Diabetes, Type I
• Evan Syndrome
• Juvenile Dermatomyositis
• Scleroderma
• Systemic Lupus Erythematosus
   
   
Gene Therapy (ie: Transplanting genetically altered stem cells)
• Fanconi's Anemia
• Metabolic Disorders (Leukodystrophy Diseases, Storage Disorders, etc.)
• Parkinson's Disease
   
   
Nerve cell repair - Diseases of the Central Nervous System
• Amyotrophic Lateral Sclerosis (ALS, or "Lou Gehrig's disease")
• Alzheimer's Disease
• Huntington's Disease
• Parkinson's Disease
   
   
Traumatic injury
• Spinal cord injury
• Stroke recovery
   
   
Organ repair Kidney
• Combined transplant of kidney plus hematopoietic stem cells
• Growth of renal cells from hematopoietic stem cells
   
   
Liver
• Growth of liver cells from hematopoietic stem cells