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29 Years of Cord Blood Stem Cell Treatments

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October marked 29 years since Matthew Farrow, a 5-year old boy with Fanconi anemia received treatment using his newborn sister’s umbilical cord blood in the world’s first cord blood transplant.

Since then, cord blood stem cells have been used in more than 40,0001 transplants in the treatment of over 80 life threatening conditions2 and Matthew, now in his thirties, is a father, and leads a healthy, normal life.

In an industry that is focussed on advancing the future therapeutic potential of cord blood stem cells, taking time to look in the rear-vision mirror is not a regular practice. Doing so however, provides a window into the encouraging trajectory of this rapidly advancing field.

For example, in 2005, the industry had surpassed 6,000 cord blood transplants, today we can report over 40,000 transplants. Moreover, in the last 10 years alone, the number of treatable conditions using a cord blood stem cell transplant has grown to over 80, up from 33 in 20082.

Today, a number of studies continue to evaluate how cord blood may provide new therapies for a broad number of diseases which currently have no effective treatment.
 

CLINICAL TRIALS
Clinical trials are underway for conditions such as autism, cerebral palsy, type 1 diabetes, paediatric stroke and traumatic brain injury, with promising developments being reported on a regular basis.


Autism Spectrum Disorder
A one-year study, conducted at the world leading Duke University, assessed 25 children with autism spectrum disorder (ASD) who received treatment with their own (autologous) cord blood stored from birth. This landmark clinical trial has shown that cord blood infusion in autism spectrum disorder can deliver behavioural improvements, with more than two-thirds of the children in the study demonstrating significant improvements across a wide range of behavioural measures. 

Australian Bureau of Statistics data highlights that there were 164,000 Australians with Autism in 2015 with prevalence rates of 1.1% and 0.3% for males and females respectively.
 

Cerebral Palsy
Cerebral palsy (CP) is another prevalent condition being researched for treatment using a cord blood stem cell infusion. With one Australian child born with CP every 15 hours, it is the most common physical disability in children.

An Australian first clinical trial which aims to find out whether stem cells from a siblings umbilical cord blood can aid the treatment of CP is currently underway. The study, taking place at The Royal Children’s Hospital in Melbourne, is providing exciting steps forward for a condition which affects the posture and movement of approximately 34,000 people in Australia.

A baby’s cord blood stem cells are a perfect match and are more likely to be a match for their siblings. This is why private cord blood banking for families with a child who has a condition such as CP may prove valuable.

These promising clinical trials are but two examples of the advancements in this field, and signal a promising future for the regenerative medicine applications utilising cord blood stem cells.
 

CORD BLOOD EXPANSION
Cord blood stem cells have shown advantages over other potential stem cell sources in a number of therapeutic scenarios. However, because of the amount of cord blood that can be collected at birth (on average 80ml - 100 ml), relatively lower absolute numbers of haematopoietic stem cells (HSCs) within a single cord blood unit has been a limiting factor in the use of cord blood. This is particularly the case for adult recipients3 where larger cell doses are required.

Cord blood scientific research has focused on the ability to expand the number of cells derived from a cord blood unit. An expanded unit can further boost utility in both current and future medical uses4; i.e., both in transplantation and in regenerative medicine.

Today, cord blood stem cell expansion technologies have developed significantly to suggest it is now a matter of when, not if, expanded cord blood cells will be approved for clinical use.
 

LOOKING TO THE FUTURE
Dr. Joanne Kurtzberg, MD, a pioneer in cord blood cellular therapies, predicted the use of cord blood cells "will emerge as one of the major great advances in novel therapeutics in medicine over the next decade."

In Australia, awareness and education remains a primary barrier for expectant parents choosing to store their baby’s stem cells.

As cord blood and cord tissue stem cells can only be collected at birth for potential future use, expectant parents interested in learning more are encouraged to do so well prior to their expected due date.

As education and awareness improve concurrently with developments in stem cell technologies, cord blood, which Dr. Kurtzberg aptly refers to being “18 years young”, has an exciting future ahead.

 

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1. Update on umbilical cord blood transplantation, Ballen K, (2017) https://www.ncbi.nlm.nih.gov/pubmed/28928957 2. Bioinformant Industry report: Capitalising on opportunities in cord blood industry growth, www.Bioinformant.com, March 2013 3. Baron F,  Ruggeri A, Nagler A.; Methods of ex vivo expansion of human cord blood cells: challenges, successes and clinical implications; Expert Rev Hematol.   2016 Mar;9(3):297-314 4Tiwari  A, Moeneclaey G, Jenkin G, Kirkland MA.; Exploring Life Saving Potential of Umbilical Cord Blood Derived Hematopoietic Stem   Cells; Insights in Stem Cells 2016