Umbilical cord stem cell therapy appears to offer an effective treatment for autism.
Autism is a large and increasing health problem here in the UK. Currently over 600,000 people are diagnosed with the condition and that number is increasing every year. This has a huge impact on the quality of life of suffers and their families, as 25% of people with autism will never even learn to talk and 85% will never hold down a full time job.
What is the cost of autism to the UK?
It is estimated that the cost of autism to the UK economy is approximately £32bn per year, considerably higher than cancer at £12bn, heart disease at £8bn and strokes at £5bn. And yet as a country we only spend £4m per year on autism research, as compared to cancer at £590m, heart disease at £169m and stroke at £32m.
What is autism?
The symptoms of autism generally (but not always) become apparent in the first two years of a child’s life due to alterations in the organisation and connection of the nerve cells in the brain. This can result in a range of neurodevelopmental disorders which stereotypically show as social deficits, cognitive delays, communication difficulties and repetitive behaviours.
How is autism currently treated?
Currently autism is treated in two ways, by the prescription of psychoactive drugs or anticonvulsants and/or by educational input. Neither of these treatments have been particularly efficacious and indeed until recently there has been no known medication that relieves the core symptoms of the disorder. This is quite possibly because the cause of autism is still not fully understood.
Whilst the root cause of autism remains unclear, recent brain scans and post mortem investigations of brain tissue have pointed to the fact that immune cells called microglia are often unusually active in the brains of those suffering from autism. Microglia cells will ordinarily produce an inflammatory response designed to fight infection or repair tissue damage. However, when there is no injury or infection to respond to this same inflammation may actually harm the brain.
Stem cell therapy using cord blood may be an effective approach to treating autism!
If inflammation in the brain is one of the causes of autism, as has been mooted, then stem cells, particularly taken from cord blood or cord tissue may be able to help. It is thought that these cells could assist to reduce inflammation, improve blood and oxygen flow to the brain, repair or replace damaged neurons and stimulates improved synaptic transmission.
Recent studies support this view
2017 saw the publication of a potentially transformative study into the use of umbilical cord blood to treat children with autism at Duke University, North Carolina. This safety and limited efficacy trial demonstrated that cord blood transfusion was safe with no significant side effects and that 70% of children saw improvements in their symptoms, including behaviours, communication and socialisation, indicating for the very first time that we may be able to tackle the root causes of autism.
Not definitive but an important step in the right direction
Whilst stem cells for autism are still a way from being standard of care, this study is a huge advancement in the possible treatment of the condition. The safety trial is now being followed up by a phase II randomised clinical trial that will focus on further demonstrating the efficacy of this treatment. It is hoped that within the next few years, umbilical cord blood will become the routine therapy for autism suffers and deliver huge improvements to quality of life.
I am a serial entrepreneur specialising in life sciences and chair two exceptional companies Cells4Life, the UK's largest cord blood stem cell bank, and Stabilitech Biopharma, developer of the world's first 15-strain influenza vaccine and the first platform that will allow any vaccine to be take orally.
I graduated from Imperial with a First Class Honours degree in mathematics and physics and joined IBM during the computing revolution in the 1980s. And 4 years later, at the age of 26, founded my first company which specialised in networking and communications products distribution. It grew to revenues of over $500m in 13 years, employing over 1,200 people operating in 9 European countries with a full listing on the London Stock Exchange. I have run several other companies including a high-speed firewall company based in California; founded one of Europe's largest network training companies; and developed one of the first LAN search engines.
By far, the most rewarding part of my career has been life sciences - it combines my love of science, bleeding edge technology, managing complicated companies and directly helping people achieve better outcomes in their health.
In my personal life, I am a very keen dressage rider and my move from technology to life sciences was because of an injury to one of my horses. I have ridden all my life and had a dream of making Team GB in dressage. I bought Kaspar when he was two years old - he was, and still is, a dream horse to ride and train. He was the first horse that I managed to train to Grand Prix and in 2003 he had a very common injury, he tore a suspensory ligament. Normally, this would mean the end for his competitive career. However, my vet suggested we try stem cells - which at the time was a very rare therapy. They worked brilliantly and he returned to a full and long competition career and he did make Team GB.
Astonishingly, whilst I could treat my horse with stem cells, these therapies were not available to the public. The first cord blood stem cell treatment was performed back in 1988 and were used to treat Fanconi anaemia, today they are being used for over 85 different conditions and are the only therapy for cerebral palsy and autism. Stem cells are the building blocks of regenerative medicine and are going to fundamentally change how we are able to treat most illnesses and injuries - everyone of us needs to know about them and have the opportunity to use them as therapies are developed.
The most powerful stem cells available are potentially those contained in umbilical cord blood. My mission is to promote regenerative medicine and help make it available to everyone.