The United Kingdom has long been at the forefront of medical research, but why are we falling behind when it comes to cell-based therapy?
The UK is not alone. The most pioneering and promising research is now conducted in countries like China, South Korea, Japan, and even Panama, whilst global research leaders like the US, Germany, France and Britain trail behind them.
Cell therapy is now widely recognised as future of medical science. Stem cells extracted from a person’s fat, umbilical cord or bone marrow have already been used to treat disorders ranging from blood cancers to previously untreatable conditions like cerebral palsy, autism and muscular dystrophy.
However the onerous pharmaceutical-centric regulatory pathways in the European Union and US mean that is often too difficult or too expensive to develop cell therapies in these countries.
This begs the question, do cell therapies really need to go through the same level of rigorous testing as drugs? Or would patients benefit if we moved to a Japanese style system where a time limited licence for use can be issued following completion of successful safety and small efficacy trials?
With Brexit fast approaching the UK has the perfect opportunity to evaluate this model and steal a march on its US and European competitors in the cell therapy arena.
Why is it so difficult to get cell therapies approved in the UK?
The development of new medical treatments in the UK is regulated by the Medicines and Healthcare products Regulatory Agency (MHRA), who enforce European Union legislation. This legislation requires that a new treatment undergo several clinical trial stages in order to receive approval and become standard-of-care. These are a Phase I trial to demonstrate safety, a Phase II on a small population of patients to demonstrate efficacy, and a much larger Phase III trial on thousands of patients to confirm efficacy in a far more substantial cohort. Each one of these Phases must be cleared before a medicine can become routine in the UK or Europe.
The difficulty with this stringent process is that it cost hundreds of millions of pounds to complete, putting the development of new medicines from bench to bedside out of reach to almost all but big pharma. Of course, if you are developing a new drug, this process is necessary. Trialling a drug requires the introduction of a foreign chemical into the human body, without any real knowledge of how it will be received and what the side-effects will be. Absolute caution is paramount to ensuring the well-being of patients.
However, should cell therapies really be regarded in the same way? By their very nature, cell therapies are far more predicable than drugs. They consist of introducing human cellular material into the human body. This cellular material is used to perform its natural role, with little modification. Scientist can be fairly certain of what it will do, and the risk of serious adverse effect is significantly reduced. For this reason, should we be looking for another way? Should we seek to introduce a system that enables the accelerated development of cellular therapies?
When you test a new treatment on human patients for the very first time, absolute care is always required, however, once safety has been demonstrated, patients may derive a net benefit from receiving potentially transformative treatments sooner rather than later.
Following in the footsteps of Japan?
Japan has taken steps to make the regulatory path for cell therapies simpler and less onerous, providing almost immediate access to the patient.
Recognising that cell based therapies have a very different safety profile to drugs, the Japanese government passed legislation in 2014 that only required a cell based therapy to prove safety and some efficacy and it would then be awarded a 7-year temporary marketing authorisation.
With the temporary marketing authorisation, the company developing the therapy can charge a fee for the treatment, whilst gathering considerable safety and efficacy data from the patients it treats, which is essentially the Phase III clinical trial. The company is required to provide continuous updates to the authorities, particularly with regards to any serious adverse events.
This new regulatory pathway has made the development of cellular therapies accessible to small, innovative organisations who would otherwise have been unable to afford the cost of a Phase III trial. Whilst the first seven year temporary marketing period has yet to expire since the change of legislation, the impact is clear. Since 2014 more than 3,600 clinical trials have commenced in Japan in this field and Japan has become the go to place to develop new cell-based treatments.
And what of the UK?
Great Britain has long been a world leader in the medical field, however existing legislation leaves us in danger of falling behind. With Brexit on the horizon, the UK has a unique opportunity follow the Japanese example, and become the leader in cellular therapy in the western world.
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.