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Regenerative medicine: from the lab to the clinic, and back

by Guest Author on 27 Sep 2018

Regenerative medicine is a fast-moving, interdisciplinary field, looking for ways to repair or replace parts of the body that are diseased or damaged. Now there’s an established and growing UK research community, we’re changing the way we fund this type of research. Two researchers explain why our continued support for this field – from the early discovery stage to translation into the clinic – will help deliver life-changing treatments for currently incurable conditions.

Adult stem cells from the tissue lining the human knee joint, grown in a dish. These cells can repair
damaged cartilage and are being trialled in the clinic. Individual stem cells are labelled with different fluorescent colours. Image credit: Nathan White, University of Aberdeen.

From the lab…

Biologist Professor Robin Franklin, Wellcome Trust-MRC Cambridge Stem Cell Institute

What’ve been the biggest developments in regenerative medicine over the last 10 years?

Robin Franklin

We now realise that if we understand the biology of healing and why it fails, there’s an opportunity to fix it with medicine-based approaches. Instead of transplanting a cell – or a group of cells – to replace lost tissue, we’ll also be able to give drugs that promote the natural healing process, by kickstarting adult stem cells already present in and around the damaged tissue.

In my field we’re trialling drugs for multiple sclerosis – a disease that affects some 100,000 people in the UK. It destroys the cells that make the insulating sheath around nerve fibres, called myelin, and is currently incurable. We’re looking for drugs to help regrow the cells that make myelin (oligodendrocytes) and so repair the damage.

Where do you see your field in five years’ time?

My prediction is that drugs will become the dominant form of regenerative medicine over the next 10 to 20 years. We’ll replace cell-based therapies with treatments that are more cost-effective, easier to give to patients and don’t require surgery. In my own field, I think we’re going to see many more clinical trials testing potential new treatments for patients, with better ways of assessing if they work or not.

What are the main challenges?

As a biologist studying the basic mechanisms of regeneration, there are experimental challenges. Because the healing process is similar in adult humans and mammals, it’s helpful to study injury and disease in animals. Tissue healing becomes less effective with ageing – one of the most important reasons why regeneration fails. So an important challenge for the future is to look at healing in aged adult animals.

What would you say to someone interested in applying for funding?

There are distinctive phases in the pipeline, from the basic biology through to clinical trials, supported by several types of MRC funding. Successful treatments will depend on a deep knowledge of regenerative biology. While there’s been an enormous amount of hype in this area over the last 10 years, this remains one of the most exciting areas of experimental biology and medicine.

… to the clinic, and back

Clinician Professor Cosimo De Bari, Aberdeen Centre for Arthritis and Musculoskeletal Health, University of Aberdeen

What’ve been the most significant developments over the last 10 years?

Cosimo De Bari

By studying adult stem cells in their tissues, we understand the biology of healing better and this informs the development of regenerative treatments. For example, by knowing more about the stem cells in bone marrow we’ve improved bone marrow transplantation, which is benefiting patients.

In my field, we’ve developed the first advanced therapy medicine – approved in Europe in 2009, for cartilage repair. It involves removing healthy cartilage cells from a patient’s knee, growing them in the lab, then implanting the cells back into their knee to repair damaged cartilage. This therapy has already benefited thousands of people worldwide.

Where do you see your field in five years’ time?

I predict a move towards medicines that target stem cells in the body to stimulate the bodies’ own ability to repair. We’ll also see new treatments for subtypes of the same disease. By identifying who will benefit from specific treatments, we’ll give the most appropriate treatment to the right people at the right time.

And the main challenge?    

When we prescribe drugs, we know how they’re going to react. With living cells, which produce their own chemicals, it’s hard to predict exactly how they’ll behave when transplanted into a patient. So fine-tuning, or ‘standardising’, cell therapies is a big challenge.

Clinical studies that not only treat patients, but at the same time study the patients’ cells and tissues to understand how the therapy works, can help address this challenge. A recent example is the successful treatment of a seven-year-old boy with a devastating genetic skin disease. He received a transplantation of new skin, grown from his own cells, corrected for the mutation causing the disease. By studying his new skin, scientists showed that stem cells were responsible for the regeneration.

Any thoughts for someone interested in applying for funding?

Support for all phases of regenerative medicine is giving us a better understanding of regenerative biology. This will help improve and standardise current treatments, and stimulate new ones. It’s a two-way process of learning – from discovery science to clinical application, and back.

The final round of applications for the Regenerative Medicine Research Committee closes on 14 November, after which it will no longer act as an independent funding scheme.

After this date, regenerative medicine researchers should apply for funding to the most relevant of our four MRC research boards or to our Developmental Pathway Funding Scheme. Their choice will depend on the stage of their research along the pathway, from basic biomedical research to translational impact.

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