Clinical Trials of Diseases tread by Cord Blood

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

• Diabetes, Type I
• Lupus

Transplants for diseases of the Central Nervous System

• Cerebral Palsy
• Multiple Sclerosis (MS)

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
• Wolman Disease

Inherited Disorders - Other

• Lesch-Nyhan Syndrome
• Osteopetrosis

Transplants for Disorders of Cell Proliferation

Histiocytic Disorders:

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

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 cell proliferation

Transplants for Cancerous Tumors

• Breast Cancer
• Ewing’s sarcoma
• Renal cell carcinoma

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