* Zoe Derrick was ‘cured’ of MS after getting stem cell treatment in Mexico
* But when Gregg Burgess-Salisbury, from Berkshire, had stem cell treatment two years ago it didn’t work, and he is still confined to a wheelchair
* Steve Storey, from Sheffield, was paralysed with MS but had successful therapy
* Experts warn the risky and invasive treatment is a lottery for patients
* Stem cells could cure diabetes and repair cartilage, liver, brain and soft tissue
Zoe Derrick was diagnosed with multiple sclerosis after the birth of her second son, Freddie, in January 2012.
‘At first, I thought breastfeeding was the reason I was so, so tired all the time,’ she says. ‘It was so bad that Paul, my husband, was having to help me up the stairs.
‘I kept tripping on the pavement when I was pushing the pram, then I trapped my hand in the car door. It was very bruised and swollen, but I couldn’t feel a thing. I should have been in agony.’
An MRI scan that night revealed patches of damage all over her brain. Zoe, with her medical training as an NHS midwife, knew what it meant.
‘I wondered how I could be alive, let alone speak.’
Multiple sclerosis (MS) is a neurological condition in which the immune system destroys the vital protective sheaths around nerves, causing damage that can have a devastating and paralysing effect on functions including movement, sight and speech.
Over the next two years, Zoe failed to respond to three drugs she was prescribed and became ‘weaker and weaker’; she had lost her peripheral vision and soon couldn’t walk.
‘If I wanted to go anywhere, I had to use a mobility scooter,’ she says. ‘I cut down work to alternate days but even then I was off sick a lot, I was just exhausted.
‘I couldn’t cook a meal, bathe my baby or do anything. My balance and speech were affected. I felt and looked drunk all the time.’
Zoe was given another drug, ‘but the side-effects were unbearable: the most excruciating burning sensation throughout my body. I was only 36 but I felt that my life was ending.’
She desperately looked for other options and last year came across stem cell treatment. Stem cells are capable of turning into all kinds of other cells the body needs.
The treatment for MS involves completely destroying the malfunctioning immune system using chemotherapy, then healthy stem cells, harvested from the patient’s bone marrow, are returned to the body to re-grow a healthy immune system.
Cancer specialists have already successfully treated blood cancers using stem cells from bone marrow and, in 2012, specialists at the Royal Hallamshire Hospital in Sheffield and King’s College Hospital in London, began testing the technique on a handful of MS patients with good results.
‘Stem cell treatment looked like the only option,’ says Zoe, ‘but I was turned down for the trial because they said my MS wasn’t actively advancing.’
Zoe decided to go abroad for the treatment. Her NHS consultant ‘pleaded with me not to, saying it would be a highway to death because it was untested and unregulated, and treatment would be available here in five years. I said I didn’t have five years’.
As a young mother, Zoe should surely have been given access to the best available healthcare. Instead, she had to spend £38,000 to go to Mexico for the treatment and now faces selling her home to repay the loan.
ONLY A FEW HAVE BENEFITED
It is more than 35 years since the biologist Professor Sir Martin Evans first identified stem cells in mice at Cardiff University in 1981.
He won a Nobel prize for his work, and stem cells have been hailed ever since for their ability to provide a ‘master repair kit’ for all of us, with the promise of curing MS, spinal injuries, heart failure and even age-related macular degeneration, the most common cause of blindness.
Breakthroughs continue to be made. Last month, for instance, researchers at Bristol University announced they had found a way to use stem cells to make limitless quantities of red blood cells, which could mean human blood donors will no longer be needed.
Ultimately, stem cells could treat diabetes (by regenerating insulin-producing cells) and be used to repair cartilage (to treat arthritis), the liver, the brain and virtually any other sort of soft tissue, according to Professor Brendon Noble, a spokesman for the UK Stem Cell Foundation.
Yet so far, only a small number of patients in the UK have been given the treatment.
Global progress on research was initially limited because ethical concerns about using stem cells from early embryos led President George W. Bush to declare a moratorium on stem cell work in 2001, which continued until 2009.
Q: What are stem cells?
A: They are special ‘master’ cells that can turn into any type of cell the body needs.
There are two types: embryonic stem cells, which can convert into any cell, and adult stem cells, which turn into different cell types based on their tissue of origin. The latter have been found in the bone marrow, brain, skin, teeth, gut and other organs.
They may remain dormant for years, dividing and creating new cells only when they are activated by injury or disease.
Q: How do they work?
A: Transplanted stem cells can help repair or regenerate damaged tissue.
The exact form of treatment depends on the condition. With MS, for instance, faulty immune cells attacking nerve coverings must first be destroyed with chemotherapy.
A new immune system is then built using stem cells from bone marrow.
Q: What can they treat?
A: Scientists are looking at treatments for everything from age-related sight loss to diabetes and spinal injuries.
In the UK, stem cells have so far shown beneficial results in treating blood cancers such as leukaemia, heart failure and MS, all as part of clinical trials.
Q: Can you get it on the NHS?
A: It is only available through trials here, so many patients opt to travel to Mexico, Brazil, the Philippines and Russia.
But there are concerns these clinics are offering treatments that haven’t been proven.
Stem cells were first harvested from terminated pregnancies. These days, the patient’s own stem cells are generally used.
Britain, which imposed no such limitation, should have forged ahead, but didn’t. Despite decades of research, progress here has been painfully slow.
Lack of funding is a problem, say experts, who blame the NHS for failing to appreciate the promise of long-term cures that could hugely reduce expensive ongoing drug treatments.
There is a lack of commercial investment for most projects because there is no money to be made from a technique that involves curing people by giving them back their own cells.
But another problem, which may reflect this lack of funding, is the missing evidence for the technique’s success.
Yes, there have been some apparently spectacular results — yet there have also been failures, some worrying.
PATIENTS FORCED TO GO ABROAD
What all this means for desperate patients who fail to qualify for the few NHS trials in existence, is that they are driven to travel overseas for very expensive and possibly risky treatments in clinics that have popped up everywhere from the Philippines to Brazil and Russia.
Zoe had her treatment at a private hospital in Mexico last July.
‘It has cost every penny of our savings, plus gifts from family and a £15,000 loan, which we will have to sell the house to repay,’ she says.
‘My family have bankrupted themselves paying for this for me, but it was worth it. My vision came back almost immediately, then my balance and speech started to return. My walking began to improve in October and I’m still getting better.’
Such individual success stories are good publicity for stem cell treatment, yet it would be wrong to assume all our ills will soon be cured by it. The picture varies with the medical condition.
The most promising start has been made with MS, a condition that affects more than 100,000 people in Britain.
Mass-market stem cells could be key
Rather than using the patient’s own stem cells, developing standard, ‘off-the-shelf’ versions presents the best hope for treating large numbers of patients at significantly less cost.
In Greece, a group of 11 patients all dying of heart failure had a miraculous recovery after they were treated with the one-size-fits-all stem cells, and a plan is under way to treat 100 more patients at the Royal Brompton Hospital in London this autumn.
Meanwhile, Celixir, a company led by Professor Sir Martin Evans, the medical pioneer who won a Nobel prize for his work on stem cells, is now developing off-the-shelf stem cells made from donated blood.
‘We think you have to make stem cell treatment disease-specific, not person-specific, to produce an off-the-shelf treatment,’ says Ajan Reginald, the company’s chief executive.
The value of stem cell treatment for MS is now ‘established’, according to the latest European Society for Blood and Marrow Transplantation annual report.
One of the few NHS patients who has benefited from the treatment is 43-year-old Steve Storey, a former management consultant and marathon runner from Sheffield. Though he is still confined to a wheelchair, Steve, a father of two, says the treatment has given him a new lease of life.
‘Within 18 months of MS being diagnosed in 2013, I was pretty much paralysed and needed 24-hour care,’ he says. ‘The next stage was respiratory failure. I didn’t think my life expectancy was more than a year or two.’
Nine days after his transplant in October 2014, he could move a previously paralysed toe. Within four weeks, he could stand up, and ten months later, he competed in a one-mile open-water swimming race in the Lake District.
‘I’m continuing to improve. I’m still in a wheelchair but I don’t need 24-hour care,’ he says. ‘Who knows what the future holds?’
WHEN IT’S GONE HORRIBLY WRONG
Professor John Snowden, who is leading the work at the Royal Hallamshire, has said there is ‘an increasing evidence base’ to support the use of stem cells in treating MS particularly, but also connective tissue diseases and Crohn’s disease.
This all suggests that more MS patients should be offered the treatment — but Paolo Muraro, a senior neuroscientist at Imperial College London, says that between only 50 and 75 MS patients in Britain have so far received stem cell transplants.
Across Europe, more than 1,000 patients have been treated.
‘It is vastly underfunded,’ Dr Muraro told Good Health. ‘Thousands could benefit from this, but the NHS is not proactive in adopting innovations.
‘I read that $600 million [£469 million] was invested in developing the new MS drug ocrelizumab — stem cell therapy gets a fraction of that investment.
‘One stem cell transplant operation costs about £30,000, which is the same as a year’s supply of one of these drugs.
‘If we received the money for more trials that show it is safe and effective, we could save many years of treatment costs.’
Or, as Zoe puts it: ‘Stem cell treatment is a one-off cost. Why would you want to go on shoving expensive drugs into people when it doesn’t stop the progression of the disease?’
But stem cell transplants can go wrong. Last year, the New England Journal of Medicine reported the horrific case of a stroke patient from San Diego, California, who ended up with a ‘strange, sticky fibrous mass’ growing aggressively in his spine after having injections of foetal stem-cell tissue in Argentina, China and Mexico.
Another report, in 2009, described a child from the U.S. who developed a brain tumour after his parents had taken him to Russia for foetal cell injections.
And treatment may not work. Gregg Burgess-Salisbury has spent more than a decade fighting the advance of MS after being diagnosed within days of his 30th birthday. Gregg, a former IT engineer from Twyford, Berkshire, was offered stem cell treatment two years ago, but it didn’t work and he is still confined to a wheelchair, hoping a different form of the treatment, or something else, will transform his life.
‘It’s disappointing, but you have to be stoical about it,’ he says.
‘I am no worse now than I was before, and I believe the disease would certainly have progressed if I hadn’t had the treatment. I would still recommend that any MS sufferer goes ahead with it.’
Similarly, stem cell treatment for conditions such as liver disease has brought mixed results.
In 2011, Professor Anil Dhawan, of King’s College Hospital, used the treatment on 18 children with acute liver failure. One child, Iyaad Syed, then two months old, was snatched from the jaws of death and six years later is healthy.
‘About half the children did really well,’ says Professor Dhawan.
The use of stem cells to treat paralysis still seems a long-term hope. A breakthrough was achieved in 2014, though, when nasal stem cells engineered in Britain were used in an operation in Poland to allow a 40-year-old man to walk after his spinal column was severed in a knife attack.
David Nicholls, whose charity, the Nicholls Spinal Injury Foundation, part-funded the operation, said it would help only 2 to 3 per cent of patients whose spinal columns were wholly severed.
Mr Nicholls, a leading hotel chef, set up the charity when his son, Daniel, then 18, was left paralysed after he dived into shallow water during a gap-year trip to Australia in 2003.
His father has raised more than £1 million for stem cell research.
‘When I started on this, people said I was wasting my time — paralysis would never be treatable. Look how far we’ve come,’ he says. ‘Even a 10 per cent improvement in nerve function would make a huge difference to Dan.’
Predictions of stem-cell treatments for age-related macular degeneration, a common cause of blindness, have also proved premature. There is still no such treatment available.
Current hopes are focused on Cell Cure Neurosciences, of Israel, which has developed an injectable type of stem cell derived from human embryonic stem cells.
LIMITED TO HEARTS AND MS
Recently, scientists in the U.S. published a claim that stem cells had been programmed to grow new cartilage on a worn-out hip joint, and that this new cartilage would release anti-inflammatory molecules to ward off the return of arthritis.
The proposal was dismissed by Professor Justin Cobb, an orthopaedic surgeon at Imperial College London.
‘The simple problem is that with at least three times your bodyweight going down through your hip joint, even if you got the cells in they would immediately be completely mashed,’ he says.
Indeed, apart from MS, the only condition in which regenerative cells have so far produced results in UK patients is heart disease.
In studies at St Bartholomew’s Hospital in London, 250 patients have been enrolled in stem cell treatment for heart failure, a condition that affects 900,000 people in Britain.
Gordon Foster, 59, a former welder from Bridlington in East Yorkshire, suffered from heart failure for many years and has no doubt that he owes his life to stem cell therapy.
‘My consultant put me forward for a trial at Barts five years ago,’ he says. ‘I started really going downhill afterwards and found out I had been given a placebo.
‘They contacted the hospital and I was given stem cell treatment last September on compassionate grounds, because I had already volunteered for the trial.’
Gordon is convinced that the treatment has worked.
‘I wouldn’t have made it to this year without these brilliant people. I was in constant pain and going upstairs on my hands and knees because I couldn’t get a breath. Now my family haven’t got that worried look on their faces and I’m getting better and better.’
Yet the lead clinician, cardiologist Anthony Mathur, admits the programme is going nowhere without more charitable funding. ‘Unless there is an obvious route to profit, no one is interested in funding the studies,’ he says. ‘We need another £6 million to £7 million.’
Professor Noble told Good Health: ‘I think we are on the cusp of something huge. There is evidence that stem cells can treat spinal injury, eyesight loss, all our biggest health problems.
‘Medicine is like that. You get nothing for years, then you get penicillin or organ transplants.
‘We just have to make sure we get it right, because if we get it wrong, it will set us back years.’