For people with incurable conditions, the uncertain promise of unproven treatments can outweigh the risks. As ‘stem cell tourism’ increases, one research team aims to ensure patients stay well informed – and connected to mainstream support networks.
When she was released from hospital three months after suffering a spinal injury, Natalie (not her real name) had to confront the prospect of spending the rest of her life in a wheelchair.
“I got out fairly depressed, couldn’t believe I was going to spend … life sitting in this chair, after having a fairly active life up until then, and also I was running around after three kids, not being able to do that anymore,” Natalie told researchers from Monash University and the Australian Stem Cell Centre in a 2010 study called Hopeful Journeys.
From the day of her accident, Natalie and her family had been seeking sources of assistance and “researching any new therapies … We were looking into stem cell therapies“. In 2007 she travelled to India for a series of three experimental treatments in which embryonic stem cells were delivered intravenously and via intra-muscular injection and lumbar puncture.
The treatment did not reverse Natalie’s injury, but she did report improvements to bowel and bladder function. “All I can say is that it’s definitely been worth it,” she said. “It certainly made a difference and … it’s given me hope.”
Natalie is one of hundreds of people living with incurable conditions who each year travel abroad for experimental stem cell therapies not available under clinical trial regulations in their home countries.
From affluent nations such as Australia, the UK and the US, they are lured by the hope of biomedical miracles: the re-fusing of a severed spinal cord; improvement to a child’s cerebral palsy; ways to slow the assault of degenerative conditions such as multiple sclerosis (MS) or motor neuron disease. For many patients whose mainstream treatment has reached its limits, the hope of even small improvements can seem enough to justify the high cost of travel and treatment.
But stem cell therapies have so far been proven to work for only a limited number of conditions, including some related to blood and the immune system, and some injuries to, or diseases of, the cornea, skin or bone. Few of the clinics in countries such as India, China or Russia provide objective measures of success for the therapies they promote, relying instead on testimonials, websites and the simple power of hope.
‘Stem cell tourism’, as it is dubbed, attracts heated opinions. Some argue that treatments that have not met high regulatory standards for human trials are rightfully outlawed, to protect the community. Meanwhile, many patients and advocacy groups respond that potentially life-saving treatments at home are being tied up in red tape and should be available to consenting patients.
Monash Professor of Sociology Alan Petersen, investigator on Hopeful Journeys, says discussions in mainstream healthcare focus heavily on the risks. Often cited are cases such as that of an 18-month-old Romanian boy who died in 2010 from internal bleeding after stem cells were injected into his brain at a German clinic.
“So there is the view that it’s the new snake oil, that patients should be forewarned,” says Professor Petersen, who leads a major interdisciplinary research project funded by the Australian Research Council. “But the reality is much more complex.”
Professor Petersen and fellow members of the research team are trying to tease apart the emotionally charged issues that influence patients to travel for treatment. The work builds on Hopeful Journeys, which was funded by Australia’s National Enabling Technologies Strategy and involved interviews with 16 stem cell tourists.
The ultimate aim is to offer doctors and other health practitioners greater insight into the decisions and experiences of gravely ill patients, helping to keep carer–patient communication open.
“The last thing we want is for stem cell tourism to go underground,” explains project collaborator Associate Professor Megan Munsie, who is director of the Education, Ethics, Law and Community Awareness Unit at Stem Cells Australia, a multi-institution body funded by the Australian Government.
She says general practitioners are increasingly being asked about stem cell therapies but do not feel adequately equipped to discuss options with their patients.
Associate Professor Munsie considers it important that doctors stay engaged with patients who consider travel for treatment, even if they disagree with the patient’s ultimate decision. Such engagement could allow doctors to stay abreast of the experimental therapy, factoring its impact into the patient’s ongoing treatment. They might also offer pre and post-treatment testing, the results of which could help the patient decide whether to undertake subsequent trips.
The team will interview 80 patients who have considered travelling for stem cell therapy, including some who decided not to make the trip. Although patients with any condition are invited to participate, the team is focusing on cerebral palsy, MS and spinal cord injuries, conditions for which there are high rates of travel. The study will also consider the perspectives of patient advocacy groups, doctors and stem cell scientists, and will include interviews with clinicians and participants at Chinese centres.
The project team also includes postdoctoral researcher Dr Claire Tanner and PhD student Jane Kotey, both from Monash, and Professor Steven Wainwright, deputy director at the Centre for Biomedicine and Society at Brunel University, UK.
Last year, Associate Professor Munsie started organising information sessions for patients and their advocacy groups after witnessing an increase in the number of people with spinal cord injuries travelling to a particular clinic in India. She believes many were influenced by a high-profile clinical trial for spinal cord injury in the US, with those unable to participate there deciding to give it a try in India.
“There were a couple of prominent Australians who appeared on radio and television programs on their return, and there was this air of stem cell therapy being on the checklist of therapies to try.”
Associate Professor Munsie is concerned that more invasive methods for administering stem cells are becoming normal in foreign clinics. These methods, which include the injection of cells into the brain or into the spine via a lumbar puncture, are risky because little may be known about the behaviour of the implanted stem cells, and because they could also result in infection or bleeding.
The Hopeful Journeys pilot study, conducted by Professor Petersen, Associate Professor Munsie, Dr Kate Seear and Rebecca Skinner, showed that many patients did not feel supported by mainstream medicine when deciding to travel.
“It came across quite clearly that doctors and some health professionals are not giving a strong view as to the efficacy or otherwise of the treatments,” Professor Petersen says. “They are often stepping back, taking a wait-and-see approach.”
Almost all patient and carer participants in Hopeful Journeys reported benefits from treatment, although these would not satisfy established frameworks in mainstream medicine for measuring restoration of function. There were reports of healthy colour returning to skin, improved muscular control and – in Natalie’s case – improved bladder and bowel function. In one case, a patient was thrilled just to have travelled overseas, an activity he had long considered beyond his abilities.
Associate Professor Munsie says the positive post-treatment experiences of patients are very real, not something that can be dismissed as ‘imaginary’. But the question is how to judge these experiences “through the lens of medical evidence. And, in most cases, it is the objective evaluation that is lacking.”
The International Society for Stem Cell Research warns patients on its website about the power of the placebo effect, whereby people experience improvements from a treatment that in fact has no curative impact. Another complicating factor in measuring these small reported benefits is that patients often pursue other treatments at the same time, the most beneficial of which is physiotherapy.
Although most participants in Hopeful Journeys felt there was nothing to lose, some ultimately experienced great financial and familial strain. One person, who travelled to India to have a child’s condition treated, said the family became destitute during a protracted stay in Delhi, with the clinic stepping in to cover living and treatment costs. This experience underscores another point made by Associate Professor Munsie: “The practitioners are well-meaning. But that is different from providing an accepted standard or proven treatment.”
Professor Petersen says that although strong emotions are at play, patients who travel for stem cell therapies also undertake extensive research. In a world where anything can be bought over the internet, they are part of the “political economy of hope” – a phenomenon bolstered by enthusiastic media coverage of laboratory breakthroughs, although the potential treatments are often still many decades away from human trials.
The role of media, particularly electronic media, and their contribution to this political economy of hope is another study interest. Professor Petersen says the role of public relations has been largely overlooked: “For the providers who are advertising their services online, hope is all they have for promoting their product.”
No magic bullet
Associate Professor Megan Munsie of Stem Cells Australia says speculation about the regenerative properties of stem cells often fails to take into account their diversity.
“There are many types of stem cells and many possible applications,” Associate Professor Munsie says. “Just because a stem cell treatment works, or looks like it’s working, in one condition, you can’t necessarily extrapolate and say it will work in another. If it looks positive for macular degeneration, for example, it doesn’t mean it will fix another disease, even in the eye.”
Stem cell therapies are intricate puzzles, not only because of the challenge of identifying how stem cells trigger restorative mechanisms in particular tissue, but also because of the complexities in application. How should stem cells be implanted, and at which point in their development? How will they affect surrounding tissue? How can the behaviour of implanted
cells be controlled so they do not form tumours?
These are all areas of uncertainty and the focus of ongoing research, and contribute to the cautious approach of mainstream medicine.