Biobanking Science: Perinatal Cell Therapies

Stem cells collected from perinatal tissues and stored in biobanks may be able to treat a wide range of diseases.

Biobanking is an essential part of developing new cell therapies.
Perinatal tissues can be stored in biobanks for future use as cell therapies.
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Biobanking is an Integral Part of Cell Therapy

Cell therapy is a rapidly growing medical field with the potential to treat a wide range of human diseases. Cell therapies comprise living cells which presents a unique biobanking challenge. The biological function of these cells must be maintained during collection, handling and biobanking procedures. The first cell therapy was umbilical cord blood transplantation in 1988. Since then, more than 35,000 patients have received cord blood transplants to treat congenital and acquired disorders such as leukemia, aplastic anemia and immunodeficiencies1. There are 161 public cord blood biobanks around the world which have stored over 730,000 donated cord blood units1. In addition, around 215 private biobanks store over 4 million umbilical cord blood units for individual patients and families.

The Perinatal Cell Therapy Field is Growing

More recently, perinatal cells, including cord blood, are being used to develop a range of advanced cell therapies, particularly for regenerative medicine. Perinatal stem cells can be isolated from the placenta, umbilical cord blood and blood vessels, chorion, amniotic fluid and amniotic sac. These tissues are sources of hematopoietic stem cells, mesenchymal stem cells and epithelial stem cells, all of which can be biobanked for future use.

A recent paper reviewed 281 clinical trials of advanced perinatal cell therapies that ran between 2005 and 20152. These trials had a wide range of indications including auto-immune, cancer, cardiovascular, neurological and orthopedic disorders. Across all disease areas and cell types, the studies were early stage – 83% were observational or Phase I trials. The advanced perinatal cell therapy trials ran in many different countries around the world including the US, Europe, India, Iran, Australia, New Zealand, South Korea, Japan and China.

However, almost 80% of these trials occurred in only 3 countries – 36% in China, 30% in the US and 12% in South Korea. The majority of the Chinese trials used mesenchymal stem cells isolated from cord tissue. In fact, 77% of all cord tissue clinical trials occurred in China. China hosted 90% of the liver disease trials, 75% of spinal cord trials and 67% of diabetes trials. In contrast, the US and South Korea led trials using cord blood – 41% of cord blood trials were in the US and 28% were in South Korea. The US hosted 59% of hematology/oncology trials and 52% of metabolic and genetic disorder trials. South Korea hosted 50% of degenerative neurological trials and 45% of premature birth trials.

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The most common source of perinatal cells was cord blood. Biobanked cord blood is a well-known source of hemopoietic stem cells. However, in the advanced perinatal cell therapy trials, the most commonly used cells were mesenchymal stem cells not hematopoietic stem cells. Most of these trials sourced mesenchymal stem cells from cord blood, cord tissue or the placenta. In almost half of the trials, the cell therapy was administered directly into the circulation via intravenous, intraarterial or intraosseous infusions. In 18% of the trials, the stem cells were administered locally.

Conclusions

While umbilical cord blood has been biobanked and used to treat patients for over 30 years, other types of perinatal stem cells are now being tested in patients. These cells have a wide range of indications including cancer, spinal cord injuries and hypoxic brain injuries. To date, the majority of clinical trials testing advanced perinatal cell therapies in humans have been early stage. However, as the field grows, we will hopefully see more therapies move through trials and into the clinic. Biobanks around the world will be an integral part of this process as cell therapy cells must maintain their biological function to be effective therapeutics.

 

References:

  1. Ballen et al. Umbilical cord blood donation: public or private? Bone Marrow Transplantation. 2015
  2. Couto, PS et al. The first decade of advanced cell therapy clinical trials using perinatal cells (2005-2015). Regenerative Medicine. 2017.