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Research

Repair & replacement

Regenerative medicine has the potential to provide treatments for patients with debilitating and incurable conditions by providing cells or tissues for transplantation or by stimulating self-repair.

Diseases that may be targeted include chronic health problems associated with ageing such as bone or joint repair, age-related blindness and cardiovascular problems.

The UK has the potential to build a world-leading regenerative medicine industry that will benefit UK health and wealth.

Objective

To translate the burgeoning knowledge in regenerative medicine into new treatment strategies.

Making an impact: World first clinical trial of stem cell-engineered larynx (voice box)

In 2008 MRC-funded researchers at University College London carried out the first transplant of a human trachea (wind pipe) reconstructed using stem cells. By 2013 the group were ready to build on this success by developing the first clinical trials of a stem cell-derived larynx transplant in a project known as “RegenVOX”.

The RegenVOX procedure involves preparing a reconstructed larynx made from the patient’s own stem cells and a donor larynx. The team uses chemicals to remove the cells from the donor larynx, leaving behind a scaffold onto which the patient’s stem cells are grafted. This means that the new larynx will not be rejected by the immune system so patients do not need immunosuppressant medication.

The team is also able to turn the patient's stem cells into cartilage-producing cells to give natural strength to the transplant, and into replacement mucous membrane cells to line the inside, just like a normal larynx.

Now

  • We support centres of excellence in regenerative medicine in Edinburgh and jointly with the Wellcome Trust in Cambridge.
  • We are supporting early-phase clinical trials of adult stem cell therapies for a variety of diseases.
  • MRC-funded researchers are conducting the second ever trial using human embryonic stem cells lines to treat an age-related form of blindness called macular degeneration. Pfizer is investing in this trial, which is an important endorsement of the quality of UK research.
  • The UK Stem Cell Bank makes quality-controlled and ethically-sourced human embryonic stem cell lines available to researchers and stores the world’s first clinical grade human embryonic stem cell line.
  • Through the UK Regenerative Medicine Platform, a cross-council initiative, we are supporting research hubs in key areas necessary to drive forward stem cell therapies: safety and efficacy; ‘acellular’ approaches using biomaterials and drug delivery; engineering and exploiting the microenvironment of stem cells (the stem cell ‘niche’); and scaling up the production of cells.

Future

  • We aim to harness the potential of stem cell biology and cell 'reprogramming' technologies to provide renewable supplies of defined cell and tissue products for transplantation.
  • We aim to promote self-repair by stimulating the body’s own regenerative processes.
  • We aim to establish proof of concept in regenerative medicine therapies in a number of disease areas.
  • We aim to progress clinical trials of therapies that also feed discoveries back to the laboratory to better understand stem cell biology and improve the design of future studies.
  • We aim to establish supportive technologies to help target the delivery of regenerative medicine therapies within the body and improve efficacy and safety.

How

The MRC will lead a coherent regenerative medicine programme in partnership with other research councils, Innovate UK (formerly the Technology Strategy Board), the NIHR and UK health departments, medical research charities and industry to ensure the UK maintains its leading global position.

  • We will continue to invest in discovery research in stem cell biology.
  • We will support approaches involving embryonic stem cells, induced pluripotent stem cells and adult stem cells, as well as research into biomaterials and gene therapy technologies to advance strategies for repair and replacement.
  • We will accelerate progress in regenerative medicine by bringing mathematical, physical and engineering sciences into the field.
  • We will target funding towards translational projects that require an interdisciplinary approach and a critical mass of researchers to get therapies to the point of clinical testing.
  • We will connect the best academic science with clinical research and commercial development, for example by supporting MRC centres and the UK Regenerative Medicine Platform.
  • We will ensure that strategic national investments in stem cell banking, cell characterisation and manufacture, including the Cell Therapy Catapult, are connected and complement each other.
  • We will work with regulators to ensure that the requirements for clinical development are effectively communicated and to inform a regulatory framework that is fit-for-purpose for this emerging field.

Making an impact: Vitrosafe Ltd.

Founded by MRC-funded scientists in 2008, Vitrosafe Ltd has developed an enclosed bioprocessing system for reproductive technologies in collaboration with Walker Safety Cabinets and the Centre For Life, Newcastle upon Tyne Hospitals NHS Trust. The Vitrosafe system is designed to meet good manufacturing practice (GMP) standards during the handling of embryos andstem cells in the laboratory, and has been shown to increase the survival of embryos. Vitrosafe Ltd. has successfully exported around £1m worth of systems to Canada, Europe and Thailand, and the founders have won a number of business and innovation awards.