"Pluripotent stem cells represent a vital step in translating genomic understanding into medical treatments."
Professor Sir Mike Stratton
The Sanger Institute will play a key role in the new £4.5 million 'Hub' for pluripotent stem cell research that is being established with the help of the funding as part of the UK Regenerative Medicine Platform (UKRMP), funded by the Biotechnology and Biological Sciences Research Council (BBSRC), Engineering and Physical Sciences Research Council (EPSRC) and the Medical Research Council (MRC). The capabilities and pipelines of the Sanger Institute will provide genomic and epigenomic analysis of pluripotent cells used in treatments as a quality control measure.
"We will sequence the DNA of the pluripotent cells to check that there aren't unwanted genetic changes that could pose potential dangers,"said Dr Kosuke Yusa from the Sanger Institute. "We have an important role to play in ensuring the veracity of cells using our high-throughput-next-generation sequencing technologies."
A further £20 million of capital funding from the Medical Research Council will provide new state-of-the-art facilities and equipment to support the work of the UKRMP and the wider regenerative medicine research community.
At the moment, experimental cell therapies are made in small numbers, usually by laboratory researchers. To ultimately be able to treat the thousands of patients who could benefit from regenerative medicine, scientists need to be able to reliably manufacture millions of cells under uniform and controlled conditions.
The aim of the Hub is to lay the initial foundations for scaling up the production of cell-based therapies from a 'cottage industry' to a robust system that will be suitable for clinical practice. It will develop a set of protocols for manufacturing cell therapies that meet the requirements of doctors, regulators and industry. This will entail making sure cells do not undergo unwanted genetic changes, improving differentiation so that scientist can reliably turn 'blank' cells into the type of cell they want and ensuring the right quality control systems are in place so that manufactured cell therapies are safe and suitable for use in human treatments.
Initially they will focus on two disease areas - Parkinson's disease and deafness - where efforts to develop cell therapies are already well underway. The researchers will work closely with commercial companies from the start to ensure that the procedures they develop are commercially viable.
"Pluripotent stem cells represent a vital step in translating genomic understanding into medical treatments,"said Professor Sir Mike Stratton, Director of the Sanger Institute. "We are delighted to bring our expertise to this exciting new initiative, which fits very well with our already established programmes in cellular differentiation."
The Hub will be led by the Universities of Sheffield, Loughborough and Cambridge and builds on existing capabilities within MRC and EPSRC Centres and the UK Stem Cell Bank. It will collaborate with the Wellcome Trust Sanger Institute and Babraham Institute and will complement the work of the existing UKRMP research Hubs.
"The pluripotency hub brings together for the first time in the UK, researchers with the range of expertise necessary to develop the processes needed to take these cells from laboratory-based research to the commercial manufacture of safe, effective and reproducible products for use in regenerative medicine."said Professor Peter Andrews, from the Centre for Stem Cell Biology at the University of Sheffield.