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.

Transplants for Cancerous Tumors

Ø   Breast Cancer

Ø   Ewing's sarcoma

Ø   Renal cell carcinoma


Inherited Disorders - Other

Ø   Lesch-Nyhan Syndrome

Ø   Osteopetrosis

Transplants for Inherited Disorders effecting theImmune System & Other Organs

Ø   Cartilage-Hair Hypoplasia

Ø   Gunther's Disease (Erythropoietic Porphyria)

Ø   Hermansky-Pudlak Syndrome

Ø   Pearson's Syndrome

Ø   Shwachman-Diamond Syndrome

Ø   Systemic Mastocytosis


Transplants for Disorders of Cell Proliferation

Histiocytic Disorders

Ø   Familial Erythrophagocytic Lymphohistiocytosis

Ø   Hemophagocytosis

Ø   Langerhans Cell Histiocytosis (LCH; formerly called Histiocytosis-X)

Transplants for Inherited Metabolic Disorders

Mucopolysaccharidoses (MPS) Storage Diseases

Ø   Mucopolysaccharidoses (MPS)

Ø   Hurler's Syndrome (MPS-IH)

Ø   Scheie Syndrome (MPS-IS)

Ø   Hunter's Syndrome (MPS-II)

Ø   Sanfilippo Syndrome (MPS-III)

Ø   Morquio Syndrome (MPS-IV)

Ø   Maroteaux-Lamy Syndrome (MPS-VI)

Ø   Sly Syndrome, Beta-Glucuronidase Deficiency (MPS-VII)

Ø   Mucolipidosis II (I-cell Disease)


Transplants for diseases of the Central Nervous System


Multiple Sclerosis (MS)

Transplants for Inherited Metabolic Disorders

Leukodystrophy Disorders

Ø   Adrenoleukodystrophy (ALD)/Adrenomyeloneuropathy (AMN)

Ø   Krabbe Disease (Globoid Cell Leukodystrophy)

Ø   Metachromatic Leukodystrophy


Gene Therapy (ie: Transplanting genetically altered stem cells)

Ø   Glanzmann Thrombasthenia

Ø   Severe Combined Immunodeficiency (SCID) 
SCID with Adenosine Deaminase Deficiency (ADA-SCID) SCID which is X-linked

Transplants for Inherited Metabolic Disorders

Lysosomal Storage Diseases

Ø   Gaucher Disease

Ø   Niemann-Pick Disease

Ø   Sandhoff Disease

Ø   Tay-Sachs Disease

Ø   Wolman Disease

Cellular Cardiomyoplasty (ie: Strengthening damaged heart muscle by infusing stem cells or promoting their growth)

Ø   autologous stem cell transplantation

Ø   drug-enhanced stem cell proliferation