Scientific Proof

Umbilical Cord Blood in Medicine

Umbilical cord blood is a valuable resource for the treatment of various blood disorders. This is due to the blood stem cells contained in umbilical cord blood. These cells can generate all types of blood cells when needed and are therefore used, for example, to restore blood formation and the immune system after severe chemotherapy or radiation therapy¹.

So far, umbilical cord blood has been used in the treatment of approximately 80 diseases, including various types of cancer, blood disorders, and immune system disorders².

Due to the special properties of stem cells, umbilical cord blood is also a foundation for the development of new, innovative therapies.

Diseases for Which Umbilical Cord Blood Can Be Used²

Adrenoleukodystrophy

Acute biphenotypic leukemia

Acute lymphoblastic leukemia (ALL)

Acute myelofibrosis

Acute myeloid leukemia (AML)

Acute lymphogranulomatosis

Common variable immunodeficiency

Severe aplastic anemia

Beta-thalassemia

Breast cancer

Chediak-Higashi syndrome

Chronic granulomatous disease (CGD)

Chronic lymphocytic leukemia (CLL)

Chronic myeloid leukemia (CML)

Chronic myelomonocytic leukemia (CMML)

DiGeorge syndrome

Ewing sarcoma

Familial erythrophagocytic lymphohistiocytosis

Fanconi anemia

Gaucher disease

Glanzmann’s thrombasthenia

Hemophagocytosis

Histiocytic disorders

Histiocytosis-X

Hunter syndrome (MPS-II)

Juvenile chronic myeloid leukemia (JCML)

Juvenile myelomonocytic leukemia (Naegeleg leukemia) (JMML)

Cartilage-hair hypoplasia

Kostmann syndrome

Lesch-Nyhan syndrome

Leukocyte adhesion deficiency

Maroteaux-Lamy syndrome (MPS-VI)

Megakaryocytosis (inherited thrombocythemia)

Metachromatic leukodystrophy

Krabbe disease

Morquio syndrome (MPS-IV)

Mucolipidosis II

Mucopolysaccharidoses (MPS)

Multiple myeloma

Myelofibrosis

Neuroblastoma

Neutrophil actin dysfunction

Niemann-Pick disease

Kidney cancer

Non-Hodgkin lymphoma

Omenn syndrome

Osteopetrosis (marble bone disease)

Paroxysmal nocturnal hemoglobinuria

Plasmacytic leukemia

Polycythemia vera

Prolymphocytic 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)

Pure red cell aplasia

Reticular dysgenesis

Sanfilippo syndrome (MPS-III)

Scheie syndrome (MPS-IS)

Poorly differentiated acute leukemia

Severe combined immunodeficiency (SCID)

SCID adenosine deaminase deficiency

SCID T- and B-lymphocyte negative

SCID T-lymphocyte negative and B-lymphocyte positive

Sickle cell anemia

Sly syndrome, beta-glucuronidase deficiency (MPS-VII)

Bare lymphocyte syndrome

Telangiectasia

Waldenström’s macroglobulinemia

Essential thrombophlebitis

Wiskott-Aldrich syndrome

Wolman disease

X-linked lymphoproliferative disorder

PLEASE NOTE: For the diseases listed above, umbilical cord blood has been proven to be used in treatment. However, the decision to use stored umbilical cord blood is made individually by the attending physician based on the specific disease situation and available treatment options.

Diseases Under Research for Umbilical Cord Blood Treatment

New therapies for blood formation and immune system restoration³

Immunocell therapies⁴

Brain damage⁵

Heart defects⁶

Lung diseases⁷

Autoimmune diseases⁸

Metabolic diseases⁹

COVID¹⁰

Degenerative muscle diseases¹¹

Endometrial diseases¹²

Solid tumors¹³

References

Bundesärztekammer. 2019. Dtsch Arztebl International. doi:10.3238/arztebl.2019.rl_haematop_sz02.
Dessels C;Alessandrini M;Pepper M.S. 2018. Stem Cells Transl Med. doi:10.1002/sctm.17-0244.
ClinicalTrials.gov. NCT03441958: A Phase I-II Open-label Study of Non-myeloablative-allogeneic Transplant of ECT-001 (UM171/ Fed-batch Culture System) Expanded Cord Blood in Patients With High-risk Multiple Myeloma. https://clinicaltrials.gov/ct2/show/NCT03441958. Accessed September 10, 2024.
ClinicalTrials.gov. NCT02727803: Personalized NK Cell Therapy in Cord Blood Transplantation. https://clinicaltrials.gov/ct2/show/NCT02727803. Accessed September 27, 2024.
ClinicalTrials.gov. NCT02881970: Neonatal Hypoxic Ischemic Encephalopathy : Safety and Feasibility Study of a Curative Treatment With Autologous Cord Blood Stem Cells (NEOSTEM). https://clinicaltrials.gov/ct2/show/NCT02881970. Accessed September 27, 2024.
ClinicalTrials.gov. NCT04907526: Intramyocardial Injection of Autologous UCB-MNC During Fontan Surgery for SRV Dependent CHD. https://clinicaltrials.gov/ct2/show/NCT04907526?recrs=abdf&cond=heart&intr=cord+blood&draw=2&rank=3. Accessed September 27, 2027.
ClinicalTrials.gov. NCT01632475: Long Term Follow-Up Study of the Safety and Exploratory Efficacy of Pneumostem® in Premature Infants With Bronchopulmonary Dysplasia. https://clinicaltrials.gov/ct2/show/NCT01632475. Accessed September 27, 2024.
ClinicalTrials.gov. NCT03835312: Sequential Transplantation of Umbilical Cord Blood Stem Cells and Islet Cells in Children and Adolescents With Monogenic Immunodeficiency Type 1 Diabetes Mellitus. https://clinicaltrials.gov/ct2/show/NCT03835312. Accessed September 27, 2024.
ClinicalTrials.gov. NCT02254863: Augmentation of Umbilical Cord Blood Transplantation for Inherited Metabolic Diseases With Intrathecal Administration of Human Umbilical Cord Blood-Derived Oligodendrocyte-Like Cells. https://clinicaltrials.gov/ct2/show/NCT02254863. Accessed September 27, 2024.
ClinicalTrials.gov. NCT05682560: Human Umbilical Cord Blood (RegeneCyte) Infusion in Patients With Post-COVID Syndrome. [Phase 2, USA]. https://www.clinicaltrials.gov/study/NCT05682560. Updated August 3, 2023. Accessed September 27, 2024.
ClinicalTrials.gov. NCT05695521: Regulatory T Cells for Amyotrophic Lateral Sclerosis (REGALS). [Phase 1, USA]. https://www.clinicaltrials.gov/study/NCT05695521. Updated August 3, 2023. Accessed September 27, 2024.
ClinicalTrials.gov. NCT05095597: Umbilical Cord Plasma for Treating Endometrial Pathologies (Thin Endometrium / Asherman’s Syndrome/ Endometria Atrophy) (hSCU-PRP). [Phase 2, Spanien]. https://www.clinicaltrials.gov/study/NCT05095597. Updated August 3, 2023. Accessed September 10, 2024.
ClinicalTrials.gov. NCT05303727: Allogeneic Hematopoietic Stem Cell Transplantation for 4/M Neuroblastoma. [Phase 2, China]. https://www.clinicaltrials.gov/study/NCT05303727. Updated August 3, 2023. Accessed September 10, 2024.