Stem cell therapy – challenges from bench to bedside

by Mohammad Firdaus Abdul Aziz

“The lack of clinical evidence to support stem cell therapy puts patients at risk of receiving at best ineffective, and at worst unsafe or even harmful treatments, raising calls for a campaign to raise awareness and educate patients about these dangers. This issue has recently become urgent.” – Jawad et al. (2012)


Human embryonic stem cell (gold) growing on a layer of supporting cells (fibroblasts). Source: Annie Cavanagh, Wellcome Images.

Human embryonic stem cell (gold) growing on a layer of supporting cells (fibroblasts). Source: Annie Cavanagh, Wellcome Images.

Stem cell therapy is a treatment using stem cells that are induced to differentiate into a specific cell type, which is required to repair damaged or destroyed cells or tissues. Stem cell scientists have articulated that this therapy may be used to treat autoimmune diseases such as Parkinson’s disease, multiple sclerosis, type 1 diabetes, rheumatoid arthritis, systemic sclerosis; organ and tissue repair such as heart diseases, macular degeneration, spinal cord injuries; and reproductive applications for germ cells generation. The great therapeutic potential of stem cell-based therapy has led to its recognition across the globe, as a modern medical intervention. Many patients with degenerative diseases believe that their incurable conditions can be treated using this new procedure.

However, it is important to comprehend that similar to other emerging technologies, the development of stem cells is a long and challenging process. Scientists all over the world are working hard to establish sound and scientifically proven basic research, before translating it into an effective medical procedure. Even the process of translation also involves an extensive process, which has to go through well-designed pre-clinical and clinical studies. The latter then requires different stages of strictly regulated trials to ensure patients’ safety. In other words, despite the efforts to translate research from the bench to bedside, patients’ safety remains the main concern among scientists. It is one of the core prerequisites that are required by ethics committees before any therapy can be accepted as effective and safe to be introduced in hospitals or clinics.

Ethical challenges of stem cell therapy

Despite its great potential, stem cells are surrounded by various ethical issues, ranging from the protracted debate over the use of human embryos for research, consent disclosure, privacy and confidentiality, to the concern over the protection for patients’ safety. This is a challenge shared by stem cell scientists globally: advancing the field while safeguarding ethical conducts. The prolonged debate over the surrounding ethical issues are not the only challenges faced by the scientists, as the field is also facing the risk of its credibility being tarnished by scammers who are more interested in making profit out of this promising field.

Unapproved and unproven stem cell treatments

Given the fact that stem cell-based therapy is extraordinarily promising and patients are desperate to see if it works, many clinics all over the world are taking advantage over this situation by offering stem cell treatments for a wide range of diseases and conditions [1,2]. Whilst many leading countries in this field such as the UK, Australia, United States, and Singapore spend a huge amount of money in research activities to develop scientifically proven and safe cell treatments to protect patients, scammers, on the other hand, are more interested in lining their pockets first and serving the patients’ need or safety second by commercially exploiting them.

similar to other emerging technologies, the development of stem cells is a long and challenging process.

These providers claim that their treatments can cure a multitude of unrelated diseases by using a single cell type. The treatments are not backed by scientific evidence, are unproven to be safe and effective, and thus are unapproved by authorised regulatory bodies. In addition, commercial entities are also opportunistically exploiting the situation to their own advantage by manipulating and misusing the term ‘stem cells’ as part of their products’ branding. It has become a trend in many countries, including Malaysia, whereby ‘stem cells’ appears to be the magical ingredient in many health-related products encompassing supplementary pills and cosmetic products, e.g., facial treatments.

Stem cell tourism, where patients travel to another country to get stem cell treatments, is another problematic phenomenon that imposes a great challenge for scientists and regulators to ensure ethical conducts, which is vital in order to safeguard patients’ safety.

Illustration by Kong Yink Heay

Illustration by Kong Yink Heay

Lack of public awareness

Examples of existing dubious commercialisation of stem cells products in Malaysia are multi-level marketing companies that are rigorously promoting their so-called stem cells products to the public who mostly lack the knowledge about stem cells; and clinics that offer stem cells injection to treat various diseases, i.e., knee arthritis, which have not been scientifically proven to be safe and effective.

It is always the case that general public are easily influenced by ‘word of mouth’ testimonials that are commonly exploited by scammers as their marketing strategy to build trust among their potential customers. It can be said as one of the most effective means of marketing, particularly when it involves spokespersons that are normally chosen among the high profile celebrities.

In addition, a plethora of exaggerated facts on stem cells in media such as portrayals in the movies, TV adverts, magazines and on the internet have led to misperceptions among the members of public. A very large number of people who are interested in stem cells technology often do not procure the right information from scientific reading materials but depend largely on the information from the internet, which often portrays stem cell myths and almost magical medical outcome from its usage via medical intervention. This has led to the increase of unrealistic expectations of stem cells among the general public. Hence, it has resulted in the exploitation that is now becoming a regular practice, and has proliferated quickly all over the world because there is a demand from the misled and misinformed public.

There are patients who contend through their testimonials that they view the unproven treatment as their last chance to find a cure and often argue that they feel much better after receiving the treatment and would repeat it again [3]. It is important to know that patients usually experience ‘the placebo effect’, which is a beneficial effect assumed by patients merely due to the patients’ own belief that whichever treatment they received works. Also, testimonials do not justify and prove the efficacy of a medical treatment. Whilst some may be only experiencing placebo effect and no immediate harm occurs, others have been reported to fall casualty to this misinformation.

… dedicated research institutions are tirelessly continuing their research work and endeavour to develop safe and effective therapies.

A German clinical center, XCell, offered stem cell based treatments involving injections of autologous bone marrow-defined cell to patients. The center was closed due to the death of its 18-month old Romanian patient after being injected with cells in the brain. However, since there is a demand, it is now moved to and operating in Lebanon and India using a new designation, Cell4health [4,5]. In Italy, a 72-year old man who suffered from Parkinson’s disease has allegedly died following an autologous stem cell injection by an Italian physician in a clinic in the Republic of San Marino, which has prompted the government to regulate this area [6].

Approved and proven stem cell-based therapies

Since its inception in 1978 [7], only a few therapies that use stem cells are widely accepted and extensively practiced, i.e., blood stem cell transplantation used to treat blood diseases, some degenerative diseases. Injuries involving bone, skin and corneas can now be treated using tissue grafts generated from adult stem cells derived from these organs. In addition, while many potential treatments are now still being tested on animal models, a few have successfully been brought into clinical trials. ReNeuron, a British company, has announced its success in getting approval for conducting phase 1 clinical trial for stroke using neural stem cells. In USA, its Food and Drug Administration (FDA) has approved the first embryonic stem cell-based treatment for acute spinal cord injury to move into Phase 1 clinical trials [8, 9].

Even though stem cells science has a great therapeutic potential, its complexity has inevitably resulted in the slow pace of translation into regenerative medicine. Nevertheless, many world-renowned stem cell scientists and dedicated reputable research institutions are tirelessly continuing their research work and endeavour to develop safe and effective therapies so that they can be made available to the patients in the near future. It is worth noting that public trust is a vital factor that would influence government’s decision in using federal funding to develop this area. If there is a backlash from the general public, it might affect funding, which is crucially needed for advancing this area [10].

Conclusion

As has been pointed out, stem cell treatment is indeed the way forward in medicine. However, more research is needed to develop an efficient treatment procedure. Scientists are aware that they need to bring safe and effective treatments to patients quickly but it is inevitable that the scientific process of improving the safety and efficacy of new treatments takes time. In the mean time, the general public must be aware of the fabricated treatments offered by unscrupulous private clinics and other commercial entities that are preying on potential patients through their false advertising. Public must be aware of the casualties that have been caused by unproven and fabricated stem cell treatments. It is important for the public to educate themselves with the right information and making sure that any stem cell treatment that they wish to undergo is proven and approved by relevant authorities.

References

[1] Stem Cell Clinic ‘that preyed on the vulnerable’ (The Telegraph, 19 February 2014) available at http://goo.gl/8k4c9l
[2] Stem Cell Treatments Overtake Science (The New York Times, 9 September 2013) available at http://goo.gl/PPbdM8
[3] ChinaStemCellNews available at http://stemcellschina.com
[4] Abbott A. (2011). Nature Newsblog. http://goo.gl/Xvw6e1
[5] Stafford N. (2009). Germany tightens law on stem cell treatments. BMJ 339:b2967.
[6] Scientists criticize Italy for allowing unproven stem cell therapy (Reuters, 28 March 2013) available at http://goo.gl/GeGJZh
[7] History of Stem Cell Research (ExploreStemCells) available at http://goo.gl/oqcIun
[8] Stem cell research & therapy: types of stem cells and their current uses (EuroStemCell) available at http://goo.gl/rakR1Y
[9] Scientists raise alarm as Italian Government rules on unproven stem cell therapy (EuroStemCell) available at http://goo.gl/1FXbMO
[10] Hyun I. (2013). Cell Stem Cell, 12:505-507.
[11] Jawad, S., Al-Yassin, A., Herridge, D., et al. (2012). British Journal of General Practice, 62:269–270.

About the Author

Mohammad Firdaus Abdul Aziz is a DPhil candidate at the Centre of Health, Law and Emerging Technologies, University of Oxford, UK. He graduated with an MA in Biotechnological Law and Ethics (University of Sheffield, UK) and a BSc in Genetics and Molecular Biology (University of Malaya, Malaysia). His current research interest is on the interdisciplinary development of human stem cell research, particularly in embryonic stem cell research. He focuses on the scientific progress, the ethical issues involved, and the governance of stem cell research in different jurisdictions, such as in the UK, Singapore, Canada, Australia, South Korea, and Malaysia. His research aims to develop policy recommendations for Malaysia’s future regulatory system that can facilitate the nation to advance stem cell research whilst ensuring ethical compliance. Firdaus aspires to facilitate active involvements and build strong connection between Malaysian government bodies and Scientific Malaysian. Find out more about Firdaus by visiting his Scientific Malaysian profile at http://www.scientificmalaysian.com/members/firdaus