An Update on Clinical Trials Using Cord Blood and Cord Tissue

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.

Last Updated on: 09/14/2023 by Diane Paradise