Stem cell research

We believe that stem cell research may offer new opportunities to develop innovative and safer medicines and could help to ensure new and better treatments for patients. We continue to work with experts in the field to explore the full potential of this type of research.

As a company whose success is built on leading-edge science, we continuously monitor and assess new research capabilities to identify opportunities that could help us enhance the drug discovery process and deliver better medicines for patients worldwide. We believe that stem cell research may present such opportunities.

Stem cells have the potential to differentiate into different types of normal human cells. Stable lines of human cells, such as hepatocytes (liver cells) and cardiac myocytes (heart muscle cells), can be derived from stem cells, then grown and maintained in tissue culture. We are investigating their use in improving prediction of the safety, metabolism and efficacy of emerging candidate drugs. If successful, these studies may allow testing at an earlier stage in the discovery process, and would help us to overcome the current limitations that a restricted supply of human tissues presents. Significant scientific progress has been made in the development of such stem cell-based research models, with some promising results. However, more work is needed to understand the full potential of this type of research.

Some promising research in this field uses human embryonic stem cells (hESC), which are derived from human embryos created during in vitro fertilisation procedures, but which become surplus to reproductive requirements. Our global Bioethics Policy and Standard on hESC demands compliance with all relevant external legislation, regulations and guidelines and outlines our own code of practice in using hESC (see below for more). We have active projects with the aim of earlier prediction of drug toxicity in heart, liver and nerve cells, involving hESC-derived lines from public cell banks meeting our stringent ethical criteria.

Induced pluripotent stem cells (iPSC) can be obtained safely from small tissue samples from adult volunteers and do not involve embryos at all. These may provide a scientifically viable alternative to hESC. We have established a dedicated iPSC unit to facilitate the application of iPSC as a tool to derive more native-like human cells in vitro. Combined with internal activities, we are also evaluating the functional usefulness of iPSC-derived hepatocytes and cardiac myocytes obtained from commercial providers.

We do not have all the necessary skills and technologies in-house and so we are working with external partners who have expertise and an ethical commitment consistent with our own. These include the UK public-private partnership, Stem Cells for Safer Medicines, aimed at predicting potential toxicity side effects much earlier in the drug discovery process and, the European Framework Research VI programme, ”Reducing animal experimentation in pre-clinical predictive drug testing by human hepatic in vitro models derived from embryonic stem cells.” We are also partnering with world-leading academic groups at the Harvard Stem cell Institute and the Joslin Diabetes Center to explore the utility of iPSC in diabetes research. We are also partnering with a 2013 European Innovative Medicines Initiative (IMI) on Human Induced Pluripotent Stem (hiPS) Cells.

Separately, we are increasingly exploring the potential to treat disease by modulation of stem cells within target organs, using either small molecules or biological therapies - an exciting new area often referred to as regenerative medicine. Here, we are embarking on several external partnerships combining the best ideas and latest innovation in academic research with our ability to search for new drugs. We are looking for the potential of small molecules or biologics to modulate stem cells present in patients’ tissues to repair or improve the function of diseased tissue. Our collaborations with the Institute of Ophthamology at University College London ( announced in September 2010) and Evotec AG/ Develogen AG ( announced in December 2010), represent our most important investments in regenerative medicine to date and are focused on exploring regenerative therapies for diabetic retinopathy and diabetes respectively. We anticipate additional collaborations in other therapeutic areas.

Ensuring high standards

Our commitment to ensuring high ethical standards in this area of research is reflected in our Human Embryonic Stem Cell Research Policy framework, as set out in our Bioethics Policy, which demands compliance both with external legislation, regulations and guidelines, and with our own codes of practice.

The framework applies to all internal work and external research on AstraZeneca’s behalf and includes essential criteria that must be met before any such research is undertaken. Similar to those that govern inclusion in public stem cell registries such as the UK Registry and the US National Institute of Health Registry, these criteria require that the stem cells must have been derived from a fertilised egg that was created for reproductive purposes; that the fertilised egg must no longer be needed for these purposes, and that fully informed consent (with no financial inducements) must have been obtained for the donation of the fertilised egg for scientific research. The framework is designed to ensure that all research effort in this area remains consistent with our strategy of developing more effective, safer medicines for serious disease.