Stem Cell Research and the Collaborative Regulation of Innovation

Sarah Devaney

• Publisher: Taylor & Francis
• Year: 2013
• Print ISBN: 9780415521307
• eBook ISBN: 9781136014482
• Edition: 1
• Topic: Diritto
• Subtopic: Etica e responsabilità professionale nel diritto

Hopes are high that stem cell (SC) research will lead to treatments and cures for some of the most serious diseases affecting humankind today. SC science has been used in a treatment setting in the replacement of patients' windpipes and in restoring sight to patients who were blind in one eye and in future it is hoped that when the body is injured it will be able to be stimulated to produce those types of SCs necessary to repair the particular damage caused. In the meantime, research into specific treatments for a wide range of serious conditions is being undertaken including Alzheimer's disease, cancer, and diabetes.

The book considers the regulatory governance of stem cell research, setting out a readily understandable account of the science and the challenges it poses for regulators as the research is increasingly being clinically applied. It provides a critical account of those elements of a regulatory system which will be required for any jurisdiction aiming to facilitate innovative and productive SC research while maintaining appropriate ethical and legal controls. The book addresses the specific failings in the current regulatory approach to SC research in the UK and goes on to look at the regulatory approaches in the US.

The book systematically analyses the roles and responsibilities of the three key participants who collaborate in this process: regulators, scientists and tissue providers, arguing that a regulatory system which fails to recognise and facilitate the vital role which each of these three groups plays runs the risk of impairing the chances of the hopes for SC research being realised. The book places a particular emphasis on ensuring that those who contribute their bodily tissues to this endeavour are treated fairly, involving a recognition that their tissues are their property.

1 Stem Cell Research and the Regulation of a Moving Target

In this chapter, an overview of the potential of SC science to overcome serious diseases will be provided together with an account of the current stage of development of the science. This will be placed in the context of the increasing move of SC research towards clinical applications at national and international levels. The challenges which this ever-developing sphere poses for regulation will be considered.

Hopes for stem cell science

SC therapies are already being successfully utilised in the clinical setting, albeit to a limited extent. For example, bone marrow transplants have been carried out for decades, while other forms of SCs are successfully used in skin grafts for the treatment of burns and for assisting regeneration in certain types of corneal injuries.¹ The results of clinical trials using patients’ own SCs for the treatment of multiple sclerosis appear promising² and reports of the potential future therapeutic applications of SCs are never far from the headlines.

One of the primary hopes for SC technology is that it will be able to be used to repair or regenerate damaged or diseased cells. Human tissue can be used to enable scientists to learn more about diseases and disease processes,³ leading to the development of regenerative or reparative medical applications. One option is to transplant tissues produced from SCs from a donor individual to the patient (allogeneic transplants) or, preferably, they could be developed from cells drawn from the patient’s own body (autologous transplants), overcoming problems of a lack of histocompatibility which can cause immune reactions leading the body to reject foreign cells.⁴ The latter approach has been successfully utilised, for example, by an international collaboration of practitioners in Spain, Italy and the UK in the replacement of patients’ windpipes, by growing SCs from their bone marrow onto a cartilage tracheal frame obtained from a deceased donor,⁵ as well as in restoring sight to patients who were blind in one eye by growing and applying replacement corneal tissue from SCs taken from their undamaged eyes.⁶ It is anticipated that in the future, when the body is injured, it will be able to be stimulated to produce those types of SCs necessary to repair the particular damage caused.⁷ Grounds for this hope continue to emerge, for example through the discovery that the adult breast contains genetically stable pluripotent cells which transform into other cell types in mice.⁸ Research into specific treatments for a wide range of serious illnesses is currently being undertaken from Alzheimer’s disease⁹ to leukaemia¹⁰ and diabetes¹¹ to Parkinson’s disease.¹² Treatments being explored go as far as gamete creation to overcome infertility¹³ and whole organ creation in relation to a raft of other diseases.¹⁴ Ever more innovative methods of using SCs for these purposes are continually being developed in the hopes that patient- or disease-specific therapies might result.¹⁵

In addition to clinical applications, SCs have the potential to be used to screen drugs and test their toxicity, and to assist in drug delivery.¹⁶ Using SCs to test the body’s response to pharmaceuticals could enable individual patient profiles to be created which predict responses to particular medicines.¹⁷ Significant investment into this use of SCs has been made for instance in the UK where a public-private consortium, Stem Cells for Safer Medicines (SC4SM), was established to co-ordinate efforts to explore ‘the potential of differentiating human SCs into normal human cells, such as those in the liver (hepatocytes) and heart muscle (cardiomyocytes) for use in the early, high throughput, toxicology screening of potential new medicines’.¹⁸ This consortium, established as a result of the recommendations of the UK Stem Cell Initiative (UKSCI),¹⁹ represented the first significant investment by ‘big pharma’²⁰ in the SC arena in the UK, with privately funded members including GlaxoSmithKline, AstraZeneca and Roche. Public funding within the consortium was co-ordinated by the Technology Strategy Board, with contributions from the Department of Health, the Department for Innovation, Universities and Skills (DIUS), the Scottish Government, the Medical Research Council (MRC) and the Biotechnology and Biological Sciences Research Council (BBSRC). In the long term it is hoped that this collaboration will lead to the development of ‘a bank of differentiated human cell lines to be used in early drug discovery to provide early identification and elimination of potential toxicity issues before clinical testing’, thus reducing risks to safety in clinical trials.²¹ This approach could also overcome the problems of animal tests not predicting the sorts of harms drugs can pose once their use is translated to human application²² and may obviate the need for disputes about the morality of undertaking experimentation on animals for this purpose.²³

The science of stem cells

SCs have the ability to transform (or “differentiate”) either into additional SCs indefinitely or into a cell type with a specific function. In the 5- to 7-day-old embryo (known as a blastocyst) SCs are categorised as ‘pluripotent’, meaning that they can give rise to all the different types of cell in the human organism.²⁴ At a later stage in development, fetal tissue can act as a source of both pluripotent embryonic germline cells from the developing gonadal tissues, as well as multipotent SCs which give rise to specific tissue types such as neural tissue.²⁵ Beyond this stage again, multipotent SCs found in the umbilical cord (which contains haematopoietic SCs, the precursors to mature blood cells) and adult can develop into specific tissue types such as blood, muscle and nerves.²⁶

Even after the process of differentiation has occurred, it has been proven that, with appropriate triggers, bodily cells can dedifferentiate, that is ‘regress to a higher level of potency’.²⁷ It has also been observed that they may be able to “transdifferentiate”, transforming into a different cell type. For example, mesenchymal SCs, present in tissues such as bone marrow, have transdifferentiated into skin, liver, brain and bone cells,²⁸ while neural SCs have given rise to blood cells.²⁹ Medical treatments will be elevated beyond current capabilities if scientists can harness the mechanism which triggers differentiation as this will allow them to direct SCs to develop into specific types of cells and tissues according to need.³⁰ In the future, this may mean that medicine could overcome injuries of the utmost severity,³¹ that the shortage of organs available for donation³² could be circumvented through the growth of parts or whole replacement organs, and that patient-specific treatments and cures for some of the most serious illnesses affecting humanity today might become available.

The state of the art

It is clear that hopes are high for wide-ranging and revolutionary future clinical applications of SC therapies. However, much remains unknown about the nature of SCs themselves and, as many of the technical processes used in SC research are in relative infancy, daunting technical challenges must still be overcome. As a result, continued and appropriate regulatory focus is vital in the development of this science to ensure that regulators have an accurate knowledge of the state of the art. An outline of two of the most important techniques in SC research is provided below, together with indications of their implications for regulation.

Somatic cell nuclear transfer

Somatic cell nuclear transfer (SCNT) or ‘therapeutic cloning’ may overcome immune rejection problems by allowing scientists to create SCs which are ‘genetically (and therefore immunologically) identical to the recipient’.³³ This process, used to create the first cloned mammal, Dolly the sheep,³⁴ involves removing the nucleus from a normal body cell and injecting it into an enucleated egg. Under the right conditions, the resulting entity behaves like a fertilised egg to develop into an embryo which could be used in research, for example to replicate and study the cells of those with inherited diseases in an attempt to develop disease-specific therapies.³⁵

SCNT techniques remain very inefficient however, requiring large numbers of eggs to provide few results.³⁶ Despite the fact that the therapeutic application of this technique has been vaunted since Wilmut’s cloning of Dolly in 1997, and that the production of SCs from human embryos was first reported in 1998,³⁷ it was not until 2005 that scientists at the University of Newcastle produced a cloned human embryo.³⁸ However, this survived for only a few days and no SCs were extracted.³⁹ Primate embryonic SCs from rhesus macaque monkeys were created using SCNT in November 2007,⁴⁰ but the efficiency of the process continued to be extremely low at 0.7 per cent.⁴¹

Eventually in January 2008 one cloned human embryo was created and developed to blastocyst stage using SCNT.⁴² SC lines were not, however, created from it. In this work, 29 oocytes had been collected from 20 to 24 year olds and used within 2 hours of extraction. This indicated that while efficiency in the technique was improving, greater advances are still to be made in reducing the number of ova required and in proce...

Table of contents

• Cover
• Half Title
• Title Page
• Copyright Page
• Dedication
• Table of Contents
• Acknowledgements
• List of Abbreviations
• Introduction
• 1. Stem Cell Research and the Regulation of a Moving Target
• Part I Collaboration in the Regulation Endeavour
• Part II Regulating Tissue Provider Collaboration
• Conclusion
• Bibliography
• Index