In Brief: Stem cells obtained from cord blood and cord tissue are being utilized in regenerative medicine for a varying host of conditions. The most recent research indicates promising results for the treatment of neurological disorders, immune deficiencies, perinatal brain injuries, as well as degenerative diseases, and skin abnormalities.

Current and Ongoing Clinical Trials Show Promise

Currently, there nearly 80 FDA approved cord blood treatments, and approximately 100 ongoing clinical trials taking place worldwide to explore the effectiveness of umbilical stem cells in the treatment of other well-known conditions. Studies have been conducted to investigate the use of autologous and allogeneic umbilical cord blood in the treatment of autism and cerebral palsy.¹

In fact, the FDA granted Duke University Medical Center expanded access in 2017 to offer treatment to patients outside of the clinical trials.

In addition, this past year has seen progress in the use of cord blood and cord tissue in the treatment of Alzheimer’s ² and skin repair in cases of burns and wounds.³ Umbilical cord blood and tissue stem cells continue to show great promise, due to their ability to facilitate cell repairment, their prolonged self-renewal capacity, and their capability to separate into other various cell forms.

An attractive characteristic of cord blood is its high concentration of hematopoietic stem cells; approximately 0.1-0.8 CD34+ cells per 100 nucleated cells. CD34+ cells derived from cord blood contain higher proliferative potential in vitro compared to those of bone marrow.⁴ This same study also concluded that the concentration of endothelial progenitor cells found in cord blood CD34+ cells is estimated to be ten times higher than that of its bone marrow counterpart. ⁴ The progenitor cells found in cord blood support durable engraftment in allogeneic and autologous transplantation.

Furthermore, extracting mesenchymal stem cells from bone marrow and an infant’s adipose tissue is an invasive and risky procedure which can lead to complications involving bleeding, infection, and chronic pain.5 In contrast, umbilical cord blood cell retrieval is a simple and painless process with minimal risks. Cord blood and tissue stem cells continue to be utilized in clinical trials as an alternative stem cell source for related and unrelated marrow transplantation. 

Listed below are categorized tables by diagnoses of clinical trial studies that will be, have been, or, are currently being conducted into the investigation of cord blood or cord tissue as a source of treatment for emerging therapy.  ^{6, 7, 8}

The Clinical Trials

Neurological Disorders

Disorder	Cord Blood (Allogeneic)	Cord Blood (Autologous)	Cord Tissue (Allogeneic)	Trial Identifier/NCT Number
Alzheimer’s disease	X		X	01297218, 03899298, 02833792, 01547689
Autism	X	X	X	02192749, 02847182, 03099239, 01638819
Cerebral Palsy	X	X	X	02866331, 01147653, 01147653 03795974
Encephalopathy		X		02256618,00593242
Global Developmental Delay	X	X		01769716, 01601158
Hearing Loss (acquired sensorineural)		X		02038972
Intraventricular Hemorrhage	X	X		02274428
Parkinson’s disease	X		X	02236065, 03684122
Spinal Cord Injury	X		X	01471613, 01046786
Stroke	X	X	X	02397018, 01438593
Traumatic Brain Injury	X	X	X	01451528, 01885663

Diabetic

Disorder	Cord Blood (Allogeneic)	Cord Blood (Autologous)	Cord Tissue (Allogeneic)	Trial Identifier/NCT Number
Diabetes, Type 1 (Auto-Immune)	X	X		01374854, 00305344, 00989547
Diabetes, Type 2	X	X	X	01954147, 03943940, 02302599
Diabetic Foot Ulcer	X		X	02389010, 03230175, 02672280

Cardiovascular

Disorder	Cord Blood (Allogeneic)	Cord Tissue (Allogeneic)	Trial Identifier/NCT Number
Acute Myocardial Infarction (Heart Attack)		X	00555828
Cardiomyopathy		X	01946048
Critical Limb Ischemia (CLI)	X	X	01216865
Heart Failure		X	00721045, 03180450
Peripheral Arterial Disease (PAD)	X		02287831
Surgery for Congenital Heart Defects			03558269, 03431480

Auto-Immune Disorders

Disorder	Cord Blood (Allogeneic)	Cord Tissue (Allogeneic)	Trial Identifier/NCT Number
Alopecia Areata	X		03676400, 01673789
Amyotrophic Lateral Sclerosis (ALS)	X	X	02236065, 01494480
Eczema (Atopic Dermatitis)	X		03269773
Graft-versus-Host Disease (GvHD)	X	X	00003270, 00749164, 01549665, 00003661
Lupus	X	X	01741857, 03219801
Multiple Scelerosis	X	X	02034188, 02587715, 03326505
Psoriasis		X	03745417, 03424629
Rheumatoid Arthritis	X	X	03828344, 01985464, 03798028
Scleroderma	X		00114530
Ulcerative Colitis		X	02442037

Orthopedic

Disorder	Cord Blood (Allogeneic)	Cord Tissue (Allogeneic)	Trial Identifier/NCT Number
Ankylosing Spondylitis		X	01420432
Cartilage Injury	X	X	01041001
Cleft Palate Repair		X	01932164
Non-Union Fractures		X	02307435
Osteoarthritis	X	X	02580695
Osteochondral Lesion	X		03905824
Spinal Fusion Surgery		X	02481440

Other Conditions

Disorder	Cord Blood (Allogeneic)	Cord Tissue (Allogeneic)	Trial Identifier/NCT Number
Acute Respiratory Distress Syndrome (ARDS)	X		02444455
Bronco Pulmonary Dysplasia (BPD)		X	02381366
Erectile Dysfunction		X	02648386
Eye Diseases	X	X	03609125
Fistula		X	02961725
HIV	X		02140944
Kidney Failure		X	03899298
Liver Cirrhosis	X	X	01342250
Liver Failure	X	X	02812121
Premature Ovarian Failure	X	X	01742533
Uterine Scars		X	03181087
Wounds		X	02672280

Genetic & Metabolic Disorders

 

Disorder	Cord Blood (Allogeneic)	Cord Tissue (Allogeneic)	Trial Identifier/NCT Number
Aging Frailty	X	X	02418013, 02065245
Duchenne Muscular Dystrophy		X	02235844, 02484560
Epidermolysis Bullosa	X		02323789, 01033552, 00881556
Hereditary Ataxia		X	01489267
Lysosomal Storage Diseases	X		01238328
Metabolic Syndrome		X	03059355
Tay-Sachs Disease	X		02254863, 01626092

SOURCES:
1. Expanded Access Protocol: Umbilical Cord Blood Infusions for Children With Brain Injuries - Full-Text View. (n.d.). Retrieved June 6, 2019, from https://clinicaltrials.gov/ct2/show/NCT03327467
2. Duncan, Thomas, and Michael Valenzuela. “Alzheimer’s Disease, Dementia, and Stem Cell Therapy.” US National Library of Medicine National Institute of Health, Stem Cell Research & Therapy, 2017, www.ncbi.nlm.nih.gov/pmc/articles/PMC5427593/.
3. Arno, A., Smith, A. H., Blit, P. H., Shehab, M. A., Gauglitz, G. G., & Jeschke, M. G. (2011). “Stem Cell Therapy: A New Treatment for Burns?.” Pharmaceuticals (Basel, Switzerland), 4(10), 1355–1380. doi:10.3390/ph4101355
4. Riordan, N. H., Chan, K., Marleau, A. M., & Ichim, T. E. (2007). ”Cord blood in regenerative medicine: do we need immune suppression?.” Journal of translational medicine, 5, 8. doi:10.1186/1479-5876-5-8
5. Harris, D. (2014) Stem Cell Banking for Regenerative and Personalized Medicine. Biomedicines, 2(1), 50–79. MDPI AG. Retrieved June 6, 2019, http://dx.doi.org/10.3390/biomedicines2010050
6. Verter, F., Bersenev, A., & Silva Couto, P. (2018). “Diseases Treated.” Retrieved June 6, 2019,from https://parentsguidecordblood.org/en/diseases#trial.
7. Trounson, A., & Mcdonald, C., (2015). “Stem Cell Therapies in Clinical Trials: Progress and Challenges.” Cell Stem Cell, 17(1), 11-22. doi:10.1016/j.stem.2015.06.007 Retrieved June 6, 2019 from https://www.sciencedirect.com/science/article/pii/ S1934590915002672.
8. U.S. National Library of Medicine. Find A Study. Retrieved June 6, 2019 from https://clinicaltrials.gov/ct2/home.