The Priority Challenges are indicative of the MRC’s strategic assessment of the need to tackle pressing health challenges and opportunities to exploit newly developed scientific expertise.
The four priority challenges are:
The MRC’s commitment to funding the breadth and depth of biomedical research is continued within this sub-set of challenges as it is across the MRC funding portfolio. The following case studies and quantitative data demonstrate the impact of the MRC’s investment into these priority challenges.
What is regenerative medicine?
Regenerative medicine is a branch of science that seeks to repair or replace damaged and diseased human cells and tissues. It is not a new discipline; using bone marrow transplantation to regenerate the blood cell compartment became clinically established in the 1970s. However, recent developments in the field, including advances in our understanding of stem cell biology, mean that it is now poised to move beyond its historic focus on blood, bone, cartilage, and skin repair, to help address the broader health needs of the population. Regenerative medicine is therefore considered a multidisciplinary field, spanning tissue engineering, developmental and stem cell biology, gene therapy, cellular therapeutics, biomaterials (scaffolds and matrices), nanoscience, bioengineering, and chemical biology.
Regenerative medicine offers huge potential to support the UK economy; the sector in the UK is expected to create 15,000 jobs by 2020 and generate £5 billion of revenue by 2020. Statistics published in 2016 by the Cell and Gene Therapy Catapult show that investment in the cell and gene therapy industry in 2015 was more than £400m at year end compared to £35m in 2012.
Research using stem cells is at the heart of regenerative medicine. These are cells that can continue to divide almost indefinitely. Some, known as pluripotent stem cells, can develop into any of the cell types in the body. This extraordinary flexibility means they have the potential to treat many different diseases and conditions that currently have no cure, like type 1 diabetes, blindness, Parkinson’s disease, heart disease, and arthritis. Stem cells also offer the opportunity to develop gene therapies for treating rare genetic diseases. As a result, the MRC has made Regenerative Medicine a strategic priority.
Patients regain sight after being first to receive retinal tissue engineered from stem cells
Scientists at University College London and Moorfields Eye Hospital have successfully restored sight in people with wet age-related macular degeneration (AMD) using stem cell technology developed with MRC funding. The team implanted a specially engineered patch of cells derived from stem cells, and is the first instance of intentionally programmed cells being successfully used in this way. This ground-breaking clinical study has evolved from nearly 15 years of MRC’s strategic funding towards establishing a framework and platform to accelerate regenerative therapies to clinic.
Gene therapy successfully treats ‘Bubble Boy disease’ in children
MRC-funded scientists at the UCL Institute of Child Health have developed a successful treatment for children with ‘Bubble boy disease’, a rare immune disorder. These children are born with defective immune systems, and it is also known as the ‘Bubble boy disease’ because affected children are extremely vulnerable to infectious diseases and some of them have become famous for living in a sterile environment. In 2015, this work led to the launch of Orchard Therapeutics, a spin-out company which aims to further develop gene therapy to benefit patients with serious and life-threatening diseases.
Successful restoration of ovarian function in a young woman with cancer
Scientists at the MRC Centre for Reproductive Health in the University of Edinburgh developed a new technique for restoring ovarian function in 2016. This technique led to the first UK woman giving birth following a transplant of her frozen ovary tissue.
Stem-cell derived liver models allow scientists to test treatments for drug overdoses
Scientists at the MRC Centre for Regenerative Medicine at the University of Edinburgh showed in 2016 a novel microRNA a tiny molecule that can switch genes on or off -- could reduce drug-induced liver toxicity in their stem-cell derived liver model. This could lead to developing new treatments for paracetamol overdoses.