Feasibility Report: GEM Cell Treatment


In our proposal, genetically modified microglia (the residential cells of the central nervous system that form the immune defence) would be introduced through surgical or through other means into the brain. This way, the designed microglia would be at the ideal location to remove the Alzheimer-inducing plaques. This treatment would involve many areas of controversy and debate on a philosophical, practical, economical and legal level.


Our treatment is novel and unprecedented, hence, difficult to define and evaluate the issues revolving around our treatment. We define our treatment as somewhere between the boundary of allotransplantation and autotransplantation. Autotransplantation is the transfer of cells or tissue from one part of the body to another in the same individual. Allotransplantation is the transfer of cells or tissue between individuals of the same species. Because microglia will naturally have to be compatible with the host, the microglia must essentially be recognized as ‘self’ within the host. A MHC (major histocompatibility complex) class I protein, that is unique to an individual, are found on nearly every cell and acts as a marker to identify the cell as “self”. Cells and other antigens are scanned by the immune system, (phagocytes, lymphocytes etc.) such that foreign antigens (anything recognizable as harmful or non-self) would generate an immune response leading to the antigen’s removal by the immune system. However, because of genetic modification, the microglia will no longer have the same genetic identity as other cells in the host. Since our inserted cells are essentially both “self” and “non-self”, our treatment has principles in both allotransplantation and autotransplantation.

Public Perception

Before going onto analysis on the different levels of debate, let us discuss what the public perception on these issues.

As discussed earlier, since our treatment is unprecedented, there are not any papers discussing the public’s opinion on it. However, since our treatment is a combination with components in genetic modification, cell transplantation, and neural engineering, the public acceptance of these components could be evaluated to reflect the public perception on our treatment.

Genetic modification has been a hot topic for debate ever since it was introduced in commercial sale of food in 1994. Currently, the sale of genetically modified foods is highly regulated in the world, especially in Europe. This is mainly due to the concern of creating allergic reactions on consumption or damaging the environment through cross fertilizing other plants. GM foods remain among the least supported biotechnologies. Genetic modification in medicine, on the other hand, receives far more positive views compared to GM in foods. This was shown by the survey conducted over a period of 10 years by the Department of Innovation, Industry, Science and Research in Australia [1].

Individuals seem mostly willing for tissue or cell allotransplantation or even xenotransplantation (60% of the public voted positive and 87% of patients voted positive) [2]. This reflects the attitude, which most individuals have, that as long as the treatment provides benefits with minimal harms, he or she would allow the treatment to exist. After discussing with members of the community from a non-scientific background in our Speed Debate Event, we came to the conclusion that they continue to place their faith in scientists as long as research is transparent and risk assessment is extensive. Risks and side effects are the main concern in genetic engineering in medicine. Hence, when informed about the risks involved such as the potential of acquiring zoonotic diseases from xenotransplantation or immune rejection from allotransplantation, proportions for acceptance decreased [3].

The public appears very open towards accepting foreign/modified cells. However, opinions are easily swayed by information. Therefore, transparency and extensive research on the benefits of genetic engineering in medicine must be conducted to generate trust among the community that genetic engineering in medicine will become a safe and reliable asset in the future.

In our Speed Debate Event, an event targeting a non-science based audience, individuals were encouraged to discuss with one another their opinions on synthetic biology and neural engineering. We collected statistics and observed trends in how confident participants in their knowledge in the field of synthetic biology, and their comfort level with neural engineering before and after the event. We observed that 69% of participants felt more confident in their understanding in synthetic biology at the end of the event. This shows that after healthy discussion, individuals feel more aware and knowledgeable in synthetic biology. Subsequently, we also observed dramatic shifts in an individual’s opinion after the event on neuro engineering and synthetic biology, both in favor and against. This shows that discussion could sway the public’s opinion towards any direction.

Neural engineering is a fairly young field joining engineering and neuroscience. It involves interactions between the nervous system and artificial devices to improve impaired functions. Presently, devices such as neural prostheses or brain-computer interfaces are being developed for uses in medicine. The intrinsic interaction between neurons and glia in the brain define our minds: our thoughts, our behaviour, our memories and so on. Naturally, the main controversy involves disturbing the integrity of a person’s mind and identity [4]. Furthermore, a person’s autonomy becomes questionable as it becomes blurred whether a person is actually making his or her own decisions when foreign machinery or cells are introduced. It also leads onto a “slippery slope” – will neuro-engineering be used in enhancement or behaviour-correction treatments? Will we force individuals to conform to the norm society defines? The development of this technology will grant those who control it the power to shape people and society. The main questions will begin to revolve around whether neuro-engineering would potentially become a power of abuse and of oppression.

“There are no licensed drugs that can slow the progression of Alzheimer’s, and currently approved medications only treat symptoms” [5]. Current drug treatments have been heavily developed but with little efficacy with slowing the progression of Alzheimer’s. BACE inhibitors are currently at the research stage. It is proposed to disturb the pathway that produces beta amyloid aggregation. However, there have been issues with interference with myelination [6]. This May (2013), Gammagard, the only remaining drug in late-stage development for Alzheimer’s disease, was scrapped.

Patients and caretakers are becoming more and more desperate for a cure. There seems to be no hope and approaching Alzheimer’s and other Dementia through conventional pharmacological means have failed. Therefore, a new approach is needed – a synthetic biology approach [7].

Practical Concerns

In this section the economical aspects will be discussed. For simplicity, we will take a utilitarian perspective. Will there be a net general benefit in society if this treatment were created and used?

Firstly, on the economical level, the main question would be how cost efficient our treatment would be. Normally, keyhole surgery in the brain would cost £100,000, so we would be using this as an estimate for costs.

However, since we did not conduct phase two clinical trials (testing efficacy and safety on patients), we cannot get an estimate in how many years or days of quality life our treatment grants the patient. For this treatment to be economically feasible it has to fill two criteria: firstly it has to generate net societal benefit, i.e. the life it improves is worth the cost, and secondly it has to be more cost-effective than current treatments.

Firstly, would there be a net benefit in society? Alzheimer’s disease (AD) effects 10% of the population over 65 years of age and 50% of those over 85 years of age [8]. It currently affects approximately 800,000 people in the UK alone and forms a cost of around £23 billion per year [9]. The question in this segment revolves around whether there is any ‘societal’ interest in prolonging the life of senior citizens. From a cold pragmatic perspective, older adults have little working value and are a burden on society in terms of healthcare and benefits. Therefore, is there more societal interest in spending valuable resources and time somewhere else, like in tackling diseases that mainly affect young children. Normally in an A&E situation, younger patients are prioritized before older patients if there are limited resources. Doctors make this decision because there would be more “return” to society if they would treat the younger patient. However, one could still argue that senior citizens do hold value in society, not only their value as human beings of course, but their wisdom and memories. Furthermore, depending on the effectiveness of the treatment, resources currently dedicated to treating Alzheimer’s Disease, such as caretakers, time, tools etc. would be freed up to be reallocated elsewhere.

Secondly, we will discuss the cost-effectiveness of other treatments. Something is deemed cost-effective when the costs it imposes on the provider or buyer is outweighed by the benefit it provides. The cost-effectiveness of treatments for Alzheimer’s disease is a controversial topic. Dementia is the third most expensive illness in the US, therefore a cost-effective treatment is highly desirable and necessary [8]. Other than the value in prolonging an individual’s life there is also the issue of estimating the quantify the value in improving an individual’s quality of life. A common symptom, aneosognosia (unawareness of defects) makes patients unreliable reporters of their quality of life. Hence, it proves difficult to evaluate the full cost-effectiveness of current treatments.

Studies have shown that cost of care for patients with Alzheimer’s disease was $1097 less for each patient in the subject group using the most prominent drug treatments for AD, donepezil (a reversible acetylcholinesterase inhibitor), than no drugs [10]. However, in a similar study it was shown that donepezil treatment costed annually around £498 more than the placebo group [11]. Perhaps due to the lack of quantitative evaluation of improvements and symptoms being of subjective nature, it is difficult and controversial to evaluate any drug targeting the brain. Even though it was objectively shown to improve cognition and memory, the authors concluded, “donepezil is not cost-effective...More effective treatments than Cholinesterase inhibitors are needed for Alzheimer's disease.” [12]. Similarly, other drugs have failed in showing favorable cost-effectiveness, mainly due to lack of significant evidence for reduction of symptoms. Hence, if our treatment were to be proven to be cost-effective, our treatment would dominate by default, as there are no cost-effective treatments to date.

There would be a concern of accessibility in our treatment due to its high costing nature. To demand a good, one must be willing and able to purchase it. Assuming our treatment works but remains expensive, if we think in terms of demand and supply, if there is demand for this treatment at whatever the set cost, it should be supplied. As suggested by the famous metaphor “The Invisible Hand”, individuals are competent enough to analyse the cost-benefit of a good or service, therefore the feasibility of the market for our treatment. So, if anyone would like to pay the cost to prolong their life or the life of a close family member, they honestly believe that the treatment is worth the cost; hence this demand should be supplied. A goal would naturally be to lower the price so this treatment is more accessible, to resolve the issue of this treatment only benefiting the rich. This can be achieved through further research in improving the real capital or through subsidies. Subsidies is a financial grant to support the spender, if this treatment were subsidized or provided publicly and freely by the public sector such as the NHS, there would be less or no financial burden on the patient. Of course, the burden would be shifted towards the state and government expenditure on other sectors would diminish.

After a discussion with a medical doctor, we concluded that these forms of treatment ought to be researched. He believes synthetic biology is a new way forward and holds a future in medicine. Funding wouldn’t be an issue, he said, as long as the treatment proves to be effective. Even if it were expensive, people would demand it because Alzheimer’s is a tragic disease and a cure is desperately sought for. The issue with accessibility would be resolved in time, as price would gradually be pushed down due to technological advancements.

Our proposed treatment is highly innovative, controversial but still desperate sought for. As discussed in this report, public perceptions should be positive towards a genetic engineering route to address diseases such as Alzheimer’s Disease.

Furthermore, as for the marketability of our treatment, this report has shown that various factors (mainly the lack of alternative treatments) point towards our remedy being demanded and supplied due to economic forces. This would ultimately set the platform for more research and development conducted on the use of genetically engineered organisms in medicine; this would lead to advancements in technology and availability of these treatments.


[1] Puls, Margaret. "Public Prefer GM in Medicine, Not Food: Report." N.p., n.d. Web. 03 Oct. 2013.

[2] Hagelin, J. "Public Opinion Surveys about Xenotransplantation." Wiley Online Library. N.p., n.d. Web. Sept. 2013.

[3] Persson, Marie. "Xenotransplantation Public Perceptions: Rather Cells than Organs." Wiley Online Library. N.p., n.d. Web. Sept. 2013.


[5] Brey, P. "Biomedical Engineering Ethics." University of Twente. N.p., n.d. Web. Sept. 2013.

[6] Carroll, John. "Merck's Positive Alzheimer's Study Spotlights BACE Race." Fierce Biotech. N.p., Sept. 2013. Web.

[7] McShane, Rupert. "Drug Treatments for Alzheimer's Disease." Alzheimers Society. N.p., n.d. Web. Sept. 2013.

[8] Geldmacher, David. "Cost-effectiveness of Drug Therapies for Alzheimer’s Disease: A Brief Review." NCBI. N.p., n.d. Web. Sept. 2013.

[9] "Statistics." Alzheimers Society. N.p., n.d. Web. Sept. 2013.

[10] Courtney, C. "Are There Long-term Benefits of Donepezil in Alzheimer's Disease?" Canadian Medical Association Journal. N.p., n.d. Web. Sept. 2013.

[11] "An Economic Evaluation of Donepezil in Mild to Moderate Alzheimer's Disease: Results of a 1-Year, Double-Blind, Randomized Trial - Karger Publishers." Karger. N.p., n.d. Web. Sept. 2013.

[12] Courtney, C. "Long-term Donepezil Treatment in 565 Patients with Alzheimer's Disease (AD2000): Randomised Double-blind Trial." National Center for Biotechnology Information. U.S. National Library of Medicine, n.d. Web. Sept. 2013.