Treatable diseases

When it comes to stem cells, the next question that arises is: which are currently the treatable diseases with stem cells? Which are the standard indications contained in medical practice guidelines?

Blood forming stem cells (known also as hematopoietic stem cells) over the last decades have become an important therapeutic source for the treatment of more than 70 severe diseases, often being the frontline life saving therapy.

stem cell treatable diseases

The first human marrow transplants were performed 50’s, but only the breakthroughs in immunology from the 60’s made it possible the clinical bone marrow transplantation takes off in the 70’s. Since then bone marrow transplantation becomes the “Golden standard” for blood forming stem cells transplantation.

In the 90’s new stem cell sources (peripheral blood and umbilical cord blood) have been identified and then, accepted for transplantation.

80’s and 90’s marked the widening indications of the hematopoietic stem cell transplantations, besides acute leukemia and aplastic anemia.

Today, transplantation of blood forming stem cells becomes a standard of care for “blood cancers” (hematologic malignancies), congenital or acquired disorders of the hematopoietic system, some of the solid tumors and genetic metabolic disorders. Autoimmune diseases are currently under investigations, as novel indications of hematopoietic stem cell transplant.

More than 70 diseases are included in the list of standard indications of hematopoietic stem cell transplant:

  • Hematologic malignancies (include the various forms of leukemia, lymphoma, and myeloma)

Researches of the last years marked important steps to improve the treatment, prognosis and overall survival rate of the patient with hematologic malignancies.

Progresses in immunotherapy (e.g. monoclonal antibodies drugs, antibody-drug conjugates), more accurate methods of diagnosis and assessment of the risk (e.g. immunophenotyping,  molecular techniques and cytogenetic analysis), new classes of chemotherapy drugs and improved treatment algorithms lead to major improvements. Today 70% of children with acute leukemia and 80% of children and adults with Hodgkin lymphoma are cured.

Despite remarkable advances, there are still high risk forms of blood cancer, drug resistance blood cancers which do not respond favorably to conventional treatment algorithms or relapsed forms. For majority of these patients the only lifesaving treatment is hematopoietic stem cell transplant.

Disease
Allogeneic/ Donated stem cells transplant
Autologous/ Own stem cells transplant
Leukemias
Acute Limphoblastic Leukemia
Yes
No, in pediatric forms/Yes, only for certain forms in adults
Acute Myeloid Leukemia
Yes
No, in pediatric forms/Yes, in adults
Acute Promyelocytes Leukemia
Yes, in adults
No/Except one certain form in adults
Chronic Myeloid Leukemia
Yes
No
Lymphomas
Hodgkin lymphoma
Yes, in most forms
Yes, in most forms
Diffuse large B-cell lymphoma
Yes, in most forms
Yes, only in some forms
Follicular lymphoma
Yes, in most forms
Yes, only in some forms
Chronic lymphocytic leukemia
Yes, rare only in some forms
Yes, rare in some forms
Mantle cell lymphoma
Yes
Yes
Burkitt lymphoma
Yes
Yes, only in some forms
Lymphoplasmacytic lymphoma (Waldenstrom macroglobulinemia)
Yes, in most forms
Yes, only in some forms
T-cell lymphoma
Yes
Yes, only in some forms
Plasmablastic lymphoma
Yes, rare indication
Yes, rare indication
 
  • Related blood cancers (myeloproliferative diseases, myelodysplastic syndromes and myeloproliferative), so called pre-leukemic conditions, because  some cases may progress into acute leukemia
Disease
Allogeneic/ Donated stem cells transplant
Autologous/ Own stem cells transplant
Myelodysplastic Syndromes
Refractory anemia
Yes
No
Refractory anemia with excess blasts
Yes
No
Refractory cytopenia with multilineage dysplasia
Yes
No
Refractory cytopenia with unilineage dysplasia
Yes
No
Unclassifiable myelodysplastic syndrome
Yes
No
Myelodysplastic syndrome associated with an isolated del(5q) chromosome abnormality
Yes
No
Myeloproliferative Neoplasms
Polycythemia vera
Yes, in advanced forms
No
Essential thrombocythemia
Yes, in advanced forms
No
Primary myelofibrosis
Yes
No
Chronic myelogenous leukemia 
Yes, in certain forms
No
Chronic neutrophilic leukemia
Yes, in rare cases
No
Chronic eosinophilic leukemia
Yes, in rare cases
No
Myelodysplastic / Myeloproliferative Neoplasms
Chronic myelomonocytic leukemia
Yes
No
Juvenile myelomonocytic leukemia
Yes
No
Atypical chronic myelogenous leukemia
Yes
No
 
  • Disorders of blood cell proliferation, non-malignant but severe (congenital or acquired immunological deficits)
Disease
Allogeneic/ Donated stem cells transplant
Autologous/ Own stem cells transplant
Aplastic anemia
Yes
Yes, limited experience with own cord blood unit
Fanconi anemia
Yes
No
Congenital dyserythropoietic anemia
No
Paroxysmal nocturnal hemoglobinuria
Yes
No
Sickel cell disease
No
Beta thalassemia major
Yes
No
Diamond Blackfan anemia
No
Pure red cell  anemia
No
Congenital amegakaryocytic thrombocytopenia
Yes
No
Glanzmann thrombasthenia
Yes
No
 
  • Inherited disorders of the immune system and other organs, include life-threatening conditions that are all inherited as autosomal recessive traits. Today are available screening tests of newborns (e.g. T-cell Receptor Excision Circles (TREC) which makes possible the early identification of SCID, before infections occur.
Disease
Allogeneic/ Donated stem cells transplant
Autologous/ Own stem cells transplant
Severe Combined Immune Deficiency
Deficiency of the Common Gamma Chain of the T-Cell Receptor (X-SCID)
No
Adenosine Deaminase Deficiency
Yes
No/Gene therapy is available on an experimental basis
Reticular dysgenesis
No
Deficiency of the Alpha Chain of the IL-7 Receptor /absence of T cells
Yes
No
Deficiency of Janus Kinase 3/ Absence of T and NK cells
Yes
No
Deficiencies of CD3 Chains
No
Deficiency of CD45
Yes
No
Other Combined immunodeficiency 
Leaky SCID
Yes
No
Omenn syndrome
Yes
No
Bare lymphocyte syndrome
Yes
No
Combined immunodeficiency with associated features
DiGeorge syndrome
Yes
No
Cartilage hair hypoplasia
Yes
No
CID with skeletal dysplasia
Yes
No
Inherited neutropenias and Genetic syndromes often associated with neutropenia & affected organs 
Severe Congenital Neutropenia
Yes
No
Kostmann's Disease 
Yes
No
WHIM syndrome
Yes
No
Shwachman-Diamond syndrome
Yes
No
Wiscott-Aldrich syndrome
Yes
No/ Gene therapy is a promising alternative to allogeneic transplant
Antibody deficiencies
Severe CVID
Yes
No
MDS with hypogammaglobulinaemia
Yes
No
Primary hemophagocytic lymphohistiocytosis
Familial hemophagocytic lymphohistiocytosis 
Yes
No
Chediak-Higashi syndrome
Yes
No
Griscelli syndrome type II
Yes
No
Hermansky Pudlak syndrome
Yes
No
X-linked lymphoproliferative disorder type I
Yes
No
X-linked lymphoproliferative disorder type II
Yes
No
Other inherited immune deficiency & organs 
Chronic granulomatous disease
Yes
No
Pearson's syndrome
Yes
No
 
  • Plasma cell disorders, occur as a result of the excessive proliferation of a single plasma cell line (clonal proliferation) responsible for the secretion of a single type of antibody
Disease
Allogeneic/ Donated stem cells transplant
Autologous/ Own stem cells transplant
Multiple myeloma
Yes
Yes
Plasma cell leukemia
Yes
Yes
Waldenstrom macroglobulinemia
Yes
Yes
Primary amyloidosis
No
Yes
POEMS syndrome
No
Yes, rare indication
 
  • Inherited metabolic disorders, include diseases with very low incidence in the population but usually with high morbidity and a predicted prognosis
Disease
Allogeneic/ Donated stem cells transplant
Autologous/ Own stem cells transplant
Mucopolysaccharidosis
Hurler syndrome and Scheie syndrome(MPS type 1)
Yes
No
Huntersyndrome (MPS type 2)
Yes
No
San Filippo syndrome (MPS type 3)
Yes
No
Morquio syndrome (MPS type 4)
Yes
No
Maroteaux -Lamy syndrome(MPS type 6)
Yes
No
Sly syndrome (MPS type 7)
Yes
No
Mucolipidosis - leukodistrophy disorders 
Adrenoleukodystrophy
Yes
No
Krabbe's disease
Yes
No
Metachromatic Leukodystrophy
Yes
No
Pelizaeus Merzbacher disease
Yes
No
Mucolipidosis - lysosomal storage diseases
Niemann-Pick disease
Yes
No
Sandhoff disease
Yes
No
Wolman disease
Yes
No
Other inherited metabolic diseases
Lesch-Nyhan syndrome
Yes
No
Osteopetrosis
Yes
No
 
  • Certain solid tumor
Disease
Allogeneic/ Donated stem cells transplant
Autologous/ Own stem cells transplant
Germ cell tumor, relapse or refractory
No in adults/Yes, under development treatment in pediatric 
Yes
Ewing's Sarcoma high risk
No in adults/Yes, under development treatment in pediatric
Yes
Soft tissue sarcoma, high risk or metastatic
Yes, developmental treatment in pediatric
Yes,under development treatment in pediatric
Neuroblastoma, high risk or relapse
Yes, under development treatment in pediatric
Yes
Wilm's tumor, relapse
No
Yes
Osteosarcoma, high risk
No
Yes
Medulloblastoma, high risk
No
Yes
Other malignant brain tumor
No
Yes, in pediatric
Breast cancer, adjuvant high risk or metastatic
No
Yes, treatment under development 
Renal cancer, metastatic
Yes, treatment under development
No
 

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