What can Stem Cells Cure and Uses of Stem Cells

Diseases Treated with Stem Cell Therapy

Haematological Disorders

Haematological Stem Cell Disorders

  • Aplastic Anaemia (Severe)
  • Fanconi Anaemia
  • Paroxysmal nocturnal haemoglobinuria (PNH)

Acute Leukaemias

  • Acute Lymphoblastic Leukaemia (ALL)
  • Acute Myelogenous Leukaemia (AML)
  • Acute Biphenotypic Leukaemia
  • Acute undifferentiated Leukaemia
  • Acute Myelo-monocytic Leukaemia

Chronic Leukaemia

  • Chronic Myelogenous Leukaemia
  • Chronic Lymphocytic Leukaemia
  • Juvenile chronic Myelogenous Leukaemia
  • Juvenile Myelomonocytic Leukaemia

Myeloproliferative disorders

  • Acute myelofibrosis
  • Agnogenic myeloid metaplasia
  • Polycythaemia Vera

Lymphoproliferative disorders

  • Non-Hodgkin’s lymphoma
  • Hodgkin’s disease
  • Prolymphocytic leukaemia
  • Chronic Granulomatous disease
  • Neutrophil actin deficiency
  • Reticular dysgenesis

Myelodysplastic Syndromes

  • Refractory Anaemia
  • Refractory Anaemia with ring sideroblasts
  • Refractory Anaemia with excess blasts
  • Refractory anaemia with excess blasts in transformation
  • Chronic myelo-monocytic leukaemia
  • Beta Thalassaemia Major
  • HbE Beta Thalassaemia
  • Pure Red Cell Aplasia
  • Sickle Cell Disease

Inherited Platelet abnormalities

  • Amegakaryocytosis - I
  • Congenital thrombocytopenia
  • Glanzmann thromboasthenia
  • Essential Thrombocythaemias

Histiocytic disorders

  • Familial erythrophagocytic Lymphohistiocytosis
  • Histiocytosis X
  • Haemophagocytosis

Metabolic Disorders

Liposomal Storage Disease

  • Mucopolysaccharidosis
  • Hurlers Syndrome
  • Hunters Syndrome
  • Scheie syndrome
  • Sanfillipo syndrome
  • Morquio syndrome
  • Macroteaux-Lamy syndrome
  • Sly syndrome
  • Beta Glucuronidase deficiency
  • Adrenoleukodystrophy
  • Mucolipidosis II
  • Krabbe disease
  • Gauchers disease
  • Niemann-Pick disease
  • Wolman disease
  • Metachromatic leukodystrophy

Immunological Disorders

Phagocytic disorders

  • Chediak-Higashi syndrome
  • Ataxia – telangiectasia
  • Kostmann syndrome
  • Leukocyte adhesion deficiency
  • DiGeorge syndrome
  • Bare Lymphocyte Syndrome
  • Omonn’s syndrome
  • Severe combined immune deficiency
  • SCID with adenosine deaminase deficiency
  • Absence of T & B cells SCID
  • Absence of Tcells, Normal B Cells SCID
  • Common variable immune deficiency
  • Wiscott Aldrich Syndrome
  • X-linked Lymphoproliferative disorder

Plasma cell disorders

  • Multiple Myeloma
  • Plasma cell leukaemia
  • Waldenstorm’s macroglobulinemia

Other Inherited disorders

  • Lesch-Nyhan syndrome
  • Cartilage-hair hypoplasia
  • Osteopetrosis

Other malignancies

Breast Cancer[1,2]

  • Metastatic Breast Cancer
  • Inflammatory Breast Cancer
  • Triple Negative Breast Cancer

Rational of High-dose chemotherapy with stem cell transplant

High-dose chemotherapy with stem cell transplant is a way of giving high doses of chemotherapy and replacing blood - forming cells destroyed by the cancer treatment. Stem cells (immature blood cells) are removed from the blood, bone marrow of the patient or a donor or umbilical cord blood are frozen and stored. After the chemotherapy is completed, the stored stem cells are thawed and given back to the patient through an infusion. These reinfused stem cells grow into (and restore) the body’s blood cells.
  • Ewings sarcoma[3, 4]

Neuroblastoma[5, 6]

  • Childhood Central Nervous System Germ Cell Tumors
  • Childhood Extra-cranial Germ Cell Tumors

Renal Cell Carcinoma[7, 8, 9]

  • Renal Cell Carcinoma
  • Metastatic Renal Cell Carcinoma


  1. https://www.bcbst.com/mpmanual/Hematopoietic_Stem_Cell_Transplant_for_Breast_Cancer.htm
  2. http://www.nature.com/bmt/journal/v32/n2/full/1704120a.html
  3. http://www.ncbi.nlm.nih.gov/pubmed/22439159; Hematopoietic Stem-Cell Transplantation in the Pediatric Population , Editors: Ratko TA, Belinson SE, Brown HM, Noorani HZ, Chopra RD, Marbella A, Samson DJ, Bonnell CJ, Ziegler KM, Aronson N. SourceRockville (MD): Agency for Healthcare Research and Quality (US); 2012 Feb. Report No.: 12-EHC018-EF. AHRQ Comparative Effectiveness Reviews.
  4. http://www.ncbi.nlm.nih.gov/pubmed/1627311; Bone Marrow Transplant. 2006 Jan;37(2):175-81.Autologous stem cell transplantation for high-risk Ewing's sarcoma and other pediatric solid tumors. Fraser CJ1, Weigel BJ, Perentesis JP, Dusenbery KE, DeFor TE, Baker KS, Verneris MR.
  5. http://www.cancer.gov/cancertopics/pdq/treatment/childCNS-germ-cell/healthprofessional
  6. http://www.cancer.gov/cancertopics/pdq/treatment/extracranial-germ-cell/HealthProfessional
  7. http://cancerguide.org/rcc_stemcell.html
  8. http://www.ncbi.nlm.nih.gov/pubmed/21707287, Expert Rev Anticancer Ther. 2011 Jun;11(6):901-11. doi: 10.1586/era.11.12.Allogeneic stem cell transplantation for renal cell carcinoma.Bregni M, Herr W, Blaise D; Solid Tumor Working Party of EBMT.
  9. http://www.nature.com/bmt/journal/v38/n11/full/1705523a.html.
  10. Standard Therapies; http://parentsguidecordblood.org/diseases.php