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New stem cell findings – hope or hype?

People waking up on the morning of Friday 10 October were greeted with dramatic front page headlines and national TV and radio coverage about how a cure for Type 1 diabetes might be on the way.

But what exactly was involved? How does this build on what has been done already? And what do the findings mean for people with diabetes?

About the research

The researchers, led by Dr Doug Melton (himself a father of two children with Type 1 diabetes), worked in the lab with human stem cells, which can develop into many different human cell types. Using a series of complex steps, the stem cells were encouraged to become beta cells, which are the insulin-producing cells that are destroyed when Type 1 diabetes develops.

The results

Crucially, when the new beta cells were transplanted into mice, they could produce and release insulin in response to changes in blood glucose – a key indication that they were working effectively. The researchers were also able to produce hundreds of millions of the cells in one go, making them a potentially useful resource for transplants on a large scale.

Teams of researchers around the world have already used stem cells to produce immature beta cells that develop over time when transplanted into animals. But Dr Melton believes that his team is the first to ever produce fully functioning beta cells that could be used in human transplant or for studying new medications in the lab. Both types of stem cells used by the researchers – cells from embryos and reprogrammed cells from adult tissue – seemed to work equally well.

A step forward, but more hurdles to overcome

This is a significant and exciting step forward, though it is important to emphasise that the new cells have not yet been tested in humans and several extra hurdles would need to be overcome before they can be used as part of routine treatment for people with diabetes.

The first problem is the potential for the immune system to destroy the transplanted cells. This is the reason why people who haveislet cell transplantsat the moment need to take anti-rejection medication, which can have its own side effects.

This – and the fact that there is a limited supply of donor islets – means that only limited numbers of people with Type 1 have so far been able to benefit from such transplants. Many research groups, including Dr Melton’s team, are already trialling new and better ways to protect transplanted beta cells from immune attack. These includevaccinesto modify the immune system and techniques to shield transplanted cells.

Used together with the new cells, this could ultimately provide an effective cure for Type 1, and perhaps also benefit people with Type 2 who use insulin. This would be a historic breakthrough with the potential to transform the lives of people with diabetes the world over.

We do not know how long this will take, and it is important that we do not create false hope that a cure is just around the corner. But Dr Melton believes that the cells produced by his team could be used in human trials within the next few years. In the meantime, the new cells could be incredibly useful in the lab by offering scientists new ways to give existing beta cells a boost or to work out the details of why diabetes develops in the first place.

The research carried out by Dr Melton and his team was funded by JDRF, the Harvard Stem Cell Institute, the National Institutes of Health, Helmsley Charitable Trust, the JPB Foundation, and Mike and Amy Barry. Their findings were published inthe journal Cell on 9 October.


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