On the brink of the stem cell revolution
From Parkinson’s to multiple sclerosis, 2018 promises to be a game-changing year for the transplant technology, Prof Brendon Noble tells Guy Kelly
It has now been decades since stem cell technology emerged as the next great breakthrough in modern medicine, with the bold potential for one day curing everything from heart disease to cancer. Today, that optimism doesn’t appear to have diminished. It’s easy to recall the excitement. In the late Nineties, when stem cell research was still relatively unexplored but gathering pace, the hope surrounding future uses for such treatments appeared near limitless. Once greater advances had been made, it was often argued that doctors could one day inject patients with cells that had the ability to transform into any other type of cell, making it possible to grow whole new organs. In theory, any damaged area of the human body could be reparable.
It is fair to say that progress hasn’t been as quick as many had hoped. But search for the term “stem cells” on Google, and the cacophony of recent news headlines suggest that things may finally be looking up.
In just the past few weeks, a “game-changing” international trial of a stem cell transplants showed hugely encouraging signs for multiple sclerosis sufferers; in a different British trial, two patients with age-related macular degeneration experienced greatly improved sight after stem cells were used to regrow cells in the eye; and a Canadian woman reportedly became the first adult cured of sickle cell anaemia thanks to a stem cell transplant.
There’s more, too. Stem cells have long been used in bone marrow transplants for leukaemia patients, and to grow skin to graft on to burns victims. But more pioneering treatments involving stem cells have also been mooted, for conditions as wide-ranging as Parkinson’s, osteoarthritis, erectile dysfunction and the premature menopause.
It all begs the question: are we now on the brink of the stem cell revolution?
“There is a lot of hype around stem cells,” says Prof Brendon Noble, chief science officer and director of strategy at the UK Stem Cell Foundation. “However, they are now meeting unmet clinical needs. What’s going on all over the world in this area is amazing.”
For the past dozen years, Prof Noble has helped oversee the distribution of around £20 million in research grants from the charity, meaning he is better placed than most to say where the medical breakthroughs will be emerging. “When [a stem cell treatment] works perfectly, this technology is not just going to treat, it’s going to cure. It is so gamechanging. In medical advances through history you can get nothing for decades – as with antibiotics, anaesthetics and organ transplants. This is absolutely one of those that will change everything.”
Prof Noble says 2018 will be a momentous year for stem cells, particularly given the success of the recent macular degeneration and MS trials.
The latter – led from Chicago and included hospitals in Brazil, Sweden and Sheffield – saw 100 patients have their immune system “rebooted” with an injection of stem cells derived from their bone marrow. Three years after the transplant, MS had progressed in 6 per cent of patients who underwent the treatment, compared to 60 per cent in a control group taking standard medicine.
“We know it has taken a while for major breakthroughs, but in retrospect it has progressed as I suspected it would. In medicine, it takes 10 to 12 years for big steps forward, because you need industry engagement,” he says.
“It’s taken a long time, but now there’s this [MS] trial it looks very positive, especially alongside the
macular degeneration news, the recent approval of a therapy to treat complications of Crohn’s disease, the enormous activity around cell based cancer therapies and a range of treatments for back pain and rheumatoid arthritis in clinical trial. I would say the treatment of tendon injuries is also looking hopeful, and we have potential diabetes therapies are being developed in collaboration with large drugs companies.”
Much of what’s holding back great leaps forward in this country is regulation. Despite being one of the world leaders in stem cell research, the UK and the EU have some of the strictest laws. They’re reasonable, in Prof Noble’s view, particularly given what can go wrong if risky new procedures are undertaken without the relevant trials, but so much red tape has forced many British patients to seek new treatments in overseas clinics, where regulations are slacker. “We’ve now got a new phenomenon of ‘stem cell tourism’,” he says. “People are going all over the world – Thailand, Russia, Mexico – and paying a lot of money for therapies that haven’t been tested. It’s dangerous. Some people use all their savings to go, then try and come back to the NHS if it goes wrong, but that often excludes them from genuine trials here.”
Examples of “cowboy clinics” specialising in pioneering treatments have risen dramatically over the past decade, and while cases of grave errors have been found – last year,
The New York Times reported that three women lost their sight after stem cells were injected into their eyes at a loosely-regulated clinic in Florida, while others have died shortly after treatments – there are many success stories.
In 2017, the veteran BBC correspondent Caroline Wyatt, who has MS, travelled to Mexico in order to undergo a hematopoietic stem cell transplant (HSCT), a combination of chemotherapy followed by a stem cell transplant, having been denied the same treatment in Britain. The trip required raising around £60,000 for flights, accommodation, nursing care and the treatment itself, which had no guaranteed success. In a blog last month, Wyatt described feeling “quite often worse than I did before HSCT”, but that “there are sunnier days when I feel a little
better than I did immediately before the treatment, and then my hopes soar”.
Mindy Watt, a fiftysomething from Ashford in Kent, was diagnosed with MS in 2014. Taking to Google one night, she entered the desperate question, “Is there a cure for MS?”, which led her to a series of blogs run by other sufferers, before she found a doctor in Moscow offering HSCT. So she signed up.
“I was slightly concerned about it being Russia, because you have certain preconceptions, but once I looked into it and spoke to other British patients who had been, you realise that really they’ve always been way ahead of the rest of Europe in a lot of research areas, because they’ve been in isolation,” says Watt, who made the trip in 2015.
When she left to have HSCT, Watt could barely walk 300ft without a support frame. She endured complications afterwards in the form of pulmonary embolisms (a rare side effect of the treatment), but has since “progressively improved”, and enjoys a far greater quality of life than before.
“If I don’t go too berserk, I can be up and about all day. Overdoing it would be trying to pretend I’m normal, and I know I am not cured.”
She now campaigns and has a blog, presented under the motto “Face the fear and do it anyway”, protesting the fact UK patients have to go overseas.
“Most doctors and neurologists in this country don’t even understand HSCT, so we’re all forced abroad. It’s absolutely criminal, and countries like Russia are so far ahead because they don’t have so many regulations suffocating you. Even if you do try to fight the NHS, you’re better off spending that money to have it done abroad – it’d certainly be quicker.”
To Prof Noble, unregulated treatments are viewed as a threat to a delicate area of research. He calls it “tailgating” – waiting for scientists to make slight breakthroughs, then irresponsibly offering those therapies before tests have been completed.
“This technology is so powerful that society’s lack of understanding of the science has allowed unscrupulous clinics in,” he says. “I know people are desperate and it’s awful for the patients, but going too fast has the power to destroy this emerging industry.”
Over the past 12 years, he says, there’s been a lot of progress, but what of the next decade? At the question, his enthusiasm perks up again.
“Well, we could see combinations of cell therapies and genetic engineering, and these therapies coming out in clinics. Then, the really exciting thing is that health conditions could go completely off the radar. Will we get to a point where diabetes is no longer a discussion? Or osteoarthritis? With one treatment in trials, some cancers could be tracked down and destroyed, too, meaning those illnesses become minor issues.
“That’s why we need to fund this research more – think of the potential savings for the public purse.”
He takes a breath.
“At the moment, we like to think of stem cell technology as a bit like a schoolchild who has huge potential to become a doctor or a lawyer. They’re untapped potential: they have to be pushed down a specific path and given the right support. Without the correct push,” he adds, “they might not be effective.”
‘Going too fast has the power to destroy this emerging industry’