Written evidence submitted by the British Heart Foundation (REG0012)

Regenerative medicine in the treatment of cardiovascular disease

1. The field of regenerative medicine holds real promise for the treatment of cardiovascular disease. There are currently over half a million people in the UK diagnosed with heart failure, most often caused by damage to the heart muscle. At present there is no way to reverse this damage, leaving many people and their families living with its devastating effects.

2. Regenerative medicine has the potential to repair damaged heart muscle in people living with heart failure or those that have suffered a heart attack. This could involve introducing undifferentiated stem cells into the heart; converting stem cells into heart cells before introduction into the heart; using cells to stimulate the body’s own repair processes; or creating of new heart muscle in the laboratory to replace a damaged area. It may also be possible to use regenerative medicine to improve the ability of blood vessels to repair themselves, to create better heart valves, and to help repair defective hearts in children with congenital heart disease. 

British Heart Foundation involvement

3. The BHF has a significant interest in regenerative medicine. Between 2011 and 2014 we committed £44 million to funding for cardiovascular regenerative medicine. Our current funding in this area is approximately £20 million across 35 research grants.

4. In 2011, BHF launched the Mending Broken Hearts appeal to fund research looking for new treatments to repair the damage caused by a heart attack. Through this initiative we provided funding to establish three BHF Centres of Regenerative Medicine. The Centres are linked to the MRC-led UK Regenerative Medicine Platform (UKRMP) to help bring regenerative therapies to the clinic.

• The BHF Centre for Vascular Regeneration is focused on finding ways to grow new blood vessels to replace those damaged during a heart attack. It is directed by Professor Andrew Baker at the University of Edinburgh in collaboration with researchers from Kings College London, University of Bristol, and University of Glasgow.
• The BHF Centre for Cardiac Regeneration is developing ways to grow patches of heart tissue from stem cells that could be used to repair damaged hearts. It is directed by Professor Sian Harding at Imperial College London, in collaboration with researchers from the University of Nottingham, University of Westminster, University of Glasgow, and UKE Hamburg.
• BHF Centre for Regenerative Medicine is advancing our understanding of how the heart might be taught to repair itself after the damage of a heart attack. It is directed by Professor Paul Riley at the University of Oxford, in collaboration with researchers from the University of Bristol and University of Cambridge.

5. We have also appointed two BHF professors whose work focuses on regenerative medicine – Professor Michael Schneider at Imperial College London and Professor Paul Riley at the University of Oxford.

6. We are supporting a number of other regenerative medicine initiatives, including:
• The Institute for Developmental and Regenerative Medicine – we are providing £10 million to help establish a new research facility at the University of Oxford.
• BIRAX (British Israel Research and Academic Exchange) Regenerative Medicine Initiative – we are supporting the BIRAX regenerative medicine initiative, which brings together leading scientists from the UK and Israel. To date, we have committed £600,000.

Challenges for regenerative medicine in the treatment of cardiovascular disease

Underpinning science

7. Whilst there have already been some clinical successes in regenerative medicine in areas such as reconstructive surgery[1] and opthalmology[2], regenerative medicine for cardiovascular disease is still largely in the discovery research phase. A number of early cell therapy trials conducted in patients who had recently suffered a heart attack appeared to produce no clinical benefit[3]. This has led to a greater emphasis on the need to understand the underlying biological mechanism by which cells introduced into damaged tissue stimulate repair. There is some discussion, for example, as to whether cells introduced into the heart produce new tissue themselves or instead release factors that stimulate the heart’s own repair processes (known as a paracrine effect). Therefore, investment in the underpinning science as well as its translation remains important.

Manufacturing

8. The regulation of regenerative medicines is overseen by a number of bodies. The Human Fertilisation and Embryology Authority and the Human Tissue Authority cover the use of tissues and cells, and the Medicines and Healthcare products Regulatory Agency (MHRA) is responsible for the regulation of clinical trials, including ensuring that medicinal products used in a clinical setting comply with Good Manufacturing Practice (GMP).

9. There are additional challenges associated with the manufacturing of high-quality cell-based therapies compared with small molecule drugs. Cellular material displays inherent variation and is sensitive to changes in environment. Ensuring that high-quality cellular products can be produced consistently and in large enough quantities to be used in a clinical setting therefore remains a hurdle.

10. The manufacturing challenges presented by regenerative medicine are widely recognised by the sector and we welcome initiatives such as the Advanced Therapy Manufacturing Taskforce, and the new Cell and Gene Therapy Manufacturing Centre, which are working to overcome these challenges and build on the UK’s manufacturing capabilities.

Funding

11. Regenerative medicine receives significant support from the public sector, through government initiatives such as the Cell and Gene Therapy Catapult, the UK Regenerative Medicines Platform, and charities like the BHF. Collaboration between funders will be important to enable successful translation of new therapies and the BHF is keen to facilitate this.

12. In order for regenerative medicines to attract substantial commercial interest, companies must have the confidence that their investment will generate a return. Potential barriers to commercial investment remain, including uncertainty over the patentability of cell-based products, and the need for expensive development of GMP quality products before testing in humans. These must continue to be addressed if regenerative medicine is to have a wide-reaching impact for patients.

About the British Heart Foundation

The British Heart Foundation is the largest independent funder of cardiovascular research and the third largest charitable funder of medical research in the UK. We are working to achieve our vision of a world in which people do not die prematurely or suffer from cardiovascular disease. In the fight for every heartbeat we fund ground-breaking medical research, provide support and care to people living with cardiovascular disease and advocate for change.

Each year, thanks to the generosity of our supporters, we aim to fund around £100 million of new research across all four nations of the UK. Our funding portfolio extends from laboratory science to clinical trials and population studies.  We fund people from PhDs to professors as well as investing in large programme and project grants.

Thanks to modern treatments built on our research, huge progress has been made in saving lives. Most babies born today with heart defects survive and 7 out of 10 people survive a heart attack. But cardiovascular disease still kills 1 in 4 people and affects 7 million people in the UK, so there is so much more to do.

May 2016