making muscle wasting history

TREAT-NMD is aware that new scientific discoveries and innovations can lead to raised expectations and enthusiasm and this may in turn lead to hype, which can be seen as increased publicity and unfounded claims. To help inform patients and the public, TREAT-NMD has put together a new resource to give both patients and researchers guidance on stem cell research and how this can lead to stem cell tourism. With the ever-increasing media attention that stem cells have received over the years, it is no wonder that people may get confused about what is actually safe and scientifically valid and what is not.
There have been some promising results in animal model studies using stem cells for neuromuscular disorders (NMDs), however there has not yet been any clinical trials in humans. It will probably be a long time yet until the work on stem cells currently ongoing in the lab can be translated into reality for NMDs in the clinic. In recent years, “stem cell tourism” has become more and more popular with patients who have NMDs. Unregulated clinics all over the world are charging substantial amounts of money for stem cell “therapies”, which have not been shown to be effective, and most importantly safe. These clinics advertise their “treatments” on their websites without giving any scientific evidence to back up their claims, with only anecdotal evidence of their limited successes. Therefore, there is great concern from scientists that public confidence in their work could be undermined by these unregulated clinics selling bogus treatments.

See the TREAT-NMD website.

Hope versus Hype: an online guide

In recent years the hype surrounding stem cells and the hope they might bring to patients with incurable diseases has grown at an alarming rate. We need to remember that stem cell treatments which make claims that are not based on valid science and good clinical practice can be unethical and potentially harmful.

Clinics providing such stem cell “therapies” and claiming to cure or improve the health of people with neuromuscular disorders (NMDs) without providing scientific proof of these claims, only encourage false hope in patients. Clinics which charge large amounts of money for these therapies may be fraudulent, as they use the hype surrounding stem cells to mislead people for financial gain.

There are lots of trials using stem cells for various diseases, but not currently for NMDs, where promising results from animal studies have not yet been moved into many trials. There is much scientific work to be done before they can be truly shown to be effective and safe in treating NMDs and ultimately hopefully offered as part of clinical practice.

Use the links below to explore the issue of stem cells for yourself.

Guidance and articles

UK stem cell network position statement on stem cell tourism
Short statement for those interested in stem cell therapy outside the UK.

International society for stem cell research video
An introductory video to the society’s handbook for patients (provided below).

International society for stem cell research patient handbook
Issues to think about if you are considering unproven stem cell therapy. Includes an FAQ.

Stem cell researchers face down stem cell tourism
Article from the journal Nature with background to unproven stem cell therapy and some patient and carer views.

Will hope triumph over hype?
Article from the Times online newspaper (UK) highlighting some patient views on unproven stem cell therapy, using the example of multiple sclerosis.

Muscular dystrophy

DMD research overview
A leading geneticist provides a summary of the current therapeutic approaches for Duchenne muscular dystrophy.

Warning against unproven myoblast transfer therapy
A letter from the Secretary General of the World Alliance of Neuromuscular Disorder Associations.

Myoblast therapies lack scientific validation
A personal viewpoint from Chris Rosa director of the Office of Special Services for Students with Disabilities at Queens College of the City University of New York.

Scientific articles annotated bibliography

Subscription only articles

(links are provided below but you probably won’t be able to get free access to these articles unless you have a subscription to the journal or are accessing them from a library or institution that has a subscription)

The murky ethics of stem cell tourism
MacReady N. (2009) Special Report, Lancet Oncology, 10(4) 317.
The author says that “desperately ill people and their loved ones will snatch at any glimmer of hope” which is what gives the stem cell clinics such appeal to the public. The motive of these practitioners is yet to be understood and they must perform an ethical balancing act.

Medical innovation versus stem cell tourism
Lindvall O, and Hyun I. (2009) Perspective, Science, 324, 1664.
The issues surrounding stem cell tourism are complex as they invoke questions concerning the acceptable levels of medical innovation. The issues and conditions under which “unproven” therapies may be offered to patients outside of regular clinical trials are discussed. It is stated that patients should continue to be counselled against medical travel for unproven stem cell-based therapies until our knowledge changes.

Monitoring and regulating offshore stem cell clinics
Kiatpongsan D, and Sipp D. (2009) Policy Forum, Science, 323, 1564.
This article deals with the regulations involved with stem cell-based therapies. The authors state that a global ban on unapproved treatments seems unlikely to succeed; instead each government must take great care when granting funds and recognise those that fall short on ethical and professional standards. To ensure the potential of stem cell research has the chance to continue developing it must remain unhampered by fraudulent claims of success.

The allure of stem cell therapy for muscular dystrophy
Grounds M D, and Davis K E. (2007) Neuromuscular Disorders, 17(3) 206.
Claims of success using stem cell treatment in animal models of human muscle diseases require careful evaluation and are not necessarily easily extrapolated to the clinical situation. Studies on dystrophic dog models using mesangioblasts have claimed to show a reduction in the disease; however they failed to take into account the benefits of the immunosuppressive drugs when interpreting their results.

Therapeutic possibilities in muscular dystrophy: the hope versus the hype
Dubowitz V. (2002) Editorial, Neuromuscular Disorders, 12(2) 113.
This article looks at the science and the publicity surrounding the major developments. There has been an imbalance created between the hope and aspirations of the families and the clinicians and the hype that has been generated by the media. Much of the confusion seems to have come from the misuse of terminology.

Stem cells: roadmap to the clinic
Daley G. Q. (2010) Review Series, Journal of Clinical Investigation, 120(1) 8.
Short review article on the state of the field of stem cell research and some of the major advances. Provides many references to other papers.

Stem cell transplants: the power of peer-to-peer
Creasy G, and Scott C T. (2009) Correspondence, Nature Biotechnology, 27(1) 21.
Letter to the editor about how misinformation can be transmitted using the internet and online communities. It is the authors’ view that engaging reliable peer-to-peer networks early can uncover misinformation.

Tracking the rise of stem cell tourism
Ryan K A, Sanders A N, Wang D D, and Levine A D. (2010) Regenerative Medicine, 5(1) 27.
This paper has attempted to offer insight into the motivations of the patients who are willing to, or have had, unproven stem cell treatment. There is little published data on these treatments and so blogs written by patients or their caretakers were analyzed instead. The data collected showed that patients travelled to 11 clinics in 8 countries suggesting that the use of unproven stem cell therapies is growing and attracting a wider range of patients.

Selling the stem cell dream
Enserink M. (2006) News focus, Science, 313.
This news article outlines the claims of success by some scientists and clinics, which are challenged by some experts, saying instead that “scepticism and caution are in order”. Unregulated clinics around the world offering stem cell therapies recruit patients on the premise of providing what regular medicine cannot.

Stem cell clinics online: the direct-to-consumer portrayal of stem cell medicine
Lau D, Ogbogu U, Taylor B, Stafinski T, Menon D, and Caulfield T. (2008) Correspondence, Cell Stem Cell, 3, 591.
Research article outlining how unregulated stem cell clinics around the world use direct-to-consumer advertising as a means of reaching patients. The article questions the way that stem cell therapies are portrayed, the sorts of therapies being offered and whether there is any clinical evidence to support the use of these therapies. The claims made on these websites have been found to be “optimistic and unsubstantiated” by peer-reviewed literature and there are concerns that some, if not all, of these clinics are exploiting desperate patients.

Free articles

Donor-derived brain tumour following neural stem cell transplantation in an Ataxia Telangiectasia patient
A boy with ataxia telangiectasia (AT) was treated with injections of human fetal neural stem cells. Four years after the first treatment he was diagnosed with a multifocal brain tumour. Studies revealed that the tumour was of non-host origin suggesting it was derived from the transplanted neural stem cells.

Stem cell hype and the dangers of stem cell tourism
Free article from the Stem Cell Network website outlining the danger that hype and unrealistic expectations can have for people going abroad to receive unproven stem cell treatment.

General stem cell information

A stem cell story – video (English)
A 15 minute film describing what stems cells are, how they work and their potential for therapy. Also available in Deutsch, Français, Nederlands, Italiano and Svensk.

Stem cell education resources
Introductions to some of the different aspects of stem cells and stem cell research.