Cord blood stem cell research



Clinical trials where children use their own (autologous) cord blood stem cells

  • Cerebral palsy
  • Autism
  • Neonatal oxygen deprivation
  • Acquired hearing loss
  • Encephalopathy (neonatal)
  • Hypoplastic left heart syndrome
  • Stroke (pre/peri-natal)
  • Traumatic brain injury
  • Type 1 Diabetes

Clinical trials where children use donor (allogeneic) cord blood stem cells

  • Alzheimer’s disease
  • Bronchopulmonary dysplasia
  • Cartilage repair
  • Cerebral palsy
  • Critical limb ischemia
  • Global development delay
  • Graft versus host diseases
  • HIV
  • Fertility
  • Intraventricular haemorrhage
  • Liver cisshosis
  • Neurodegenerative disorders
  • Stroke
  • Sweat gland regeneration
  • Type 1 Diabetes

Clinical trials: Autism & cerebral palsy

Cerebral palsy

Joanne Kurztberg: “Children who received a high dose of cord blood cells had statistically significant changes in their motor function, more than 30% above where they would have been predicted to be without cord blood therapy. These children made new connections in their brain and that’s why their function was better.”

1 in 326 children are affected

Autism spectrum disorder

Joanne Kurztberg: “Some children, who were not speaking very much, had big increases in their vocabulary and their functional speech. Many children were able to attend to play and have meaningful communication in a way that they weren’t before. Some children had less repetitive behaviours than they did when they came onto the study.”

1 in 68 children are affected

Cord blood therapies have shown highly promising results to treat diseases for which there are currently no available therapies. Results using autologous (own) cells in babies and children with HIE, cerebral palsy and autism spectrum disorder are safe and results are promising. The safety of partially or fully matched sibling cord blood infusions has also been demonstrated preliminarily

Featured advanced cell therapy trial: Expanded access cord blood therapy for autism and cerebral palsy

The Duke University Medical Center has received permission from the FDA to offer cord blood therapy for conditions like autism spectrum disorder and cerebral palsy under an expanded access clinical trial. This protocol establishes an umbrella clinical trial, registered on 31 October 2017. This trial enables children who have these neurological disorders to receive therapy with their own cord blood or cord blood from a sibling, regardless of whether they qualify for a targeted clinical trial.

The registration of this clinical trial opens the door for many children who are af icted with an acquired neurological disorder to travel to Duke University for cord blood therapy, provided they have a suf ciently matching cord blood unit in a family bank. Sibling therapy only requires a partial match, not a perfect match.

Michael Chez (Sutter Neurological Institute) speaks about the recent progress in clinical trials using cord blood to potentially treat autism. He discusses his own research in this area and gives an overview of other research currently underway at Duke by Dr Joanne Kurtzberg.

Cord tissue stem cell research (MSCs)



  • Autism (Phase 2)
  • Cerebral palsy (Phase 2)
  • Hearing loss (acquired sensori-neural) (Phase 2)
  • Hypoxic Ischemic Encephalopathy (HIE) (Phase 1)
  • Spinal cord injury (Phase 2)


  • Critical Limb Ischemia (Phase 3)
  • Ischemic stroke (Phase 2 placenta) (Phase 3)
  • Myocardial Infarction (Phase 3)
  • Cardiomyopathy (Phase 3)


  • Crohn’s Disease (Phase 3)
  • Diabetes, Type 1 (Phase 2)
  • Graft-versus-Host Disease (GvHD) (Phase 3)
  • Kidney plus stem cell transplant (Phase 2)
  • Systemic Lupus (SLE) (Phase 2)
  • Multiple Sclerosis (Phase 1)
  • Rheumatoid Arthritis (Phase 2)
  • Scleroderma (Phase 2)
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