News Review: Science battles to keep up with patients' hopes

IMAGINE a time when, rather than joining the waiting list for a heart, liver or kidney, patients simply hand over a few cells and their desired organ is grown for them in the lab.

Researchers this week suggested that such a development is now much closer following the case of a woman who had a new windpipe created by scientists using her own stem cells.

Others more cynically suggested that the only reason the case received so much coverage was that the patient, Claudia Castillo, was an attractive young woman who looked good on TV and in print, rather than anything to do with the importance of the breakthrough.

Whatever the stance taken, it is clear that stem cells are now dominating much of the research being carried out, and patients are pinning their hopes on treatments to cure devastating conditions such as Alzheimer's, Parkinson's and motor neurone disease (MND).

But in reality, such treatments are still a long way off for the majority of patients.

Ms Castillo is one of the lucky ones to benefit from the early steps in stem cell development. She would have lost a lung if scientists had not taken part of a donor windpipe and treated it with her own stem cells to create an organ which would not be rejected when transplanted.

The Bristol-based researcher Professor Martin Birchall, who was involved in the procedure, said afterwards he could envision a time when such surgeries were common practice.

But stem cell experts warn that there is still some way to go before we are really able to master the technology.

Ben Sykes, co-ordinator of the UK National Stem Cell Network, said the UK government needed to continue to invest in research into all types of stem cells – those which have the ability to turn into different cell types. This includes adult stem cells, such as those used in Ms Castillo's operation, embryonic stem cells and induced pluripotent stem cells – adult cells manipulated to have embryonic qualities.

However, experimenting with stem cells can have unintended and disastrous consequences, such as causing cancerous tumours. "Until we understand how to differentiate these cells safely, these are really early stage technologies," Mr Sykes said.

While some treatments using adult stem cells have been used for decades – such as bone marrow transplants – other types are still at an experimental stage.

Mr Sykes said: "In the UK there are very few studies involving stem cells going into early stage clinical trials. They are still a long way from being used on the NHS as a treatment. There are more going on in the US. But there are currently no clinical trials involving embryonic stem cells."

Some scientists in the US have been trying to carry out a trial using embryonic stem cells to treat spinal injury, but have been held up by drugs watchdogs.

"Everyone is watching this particular case. It will be a tipping point in regulation," Mr Sykes said.

For charities, stem cells pose a challenge. While they clearly offer hope of new treatments, many suspect we could be years, or even decades, away from the breakthroughs which will make their use widespread.

Dr Iain Frame, the director of research at Diabetes UK, said he was cautious about the prospect of a cure for Type 1 diabetes.

He said he thought that developments would come quicker in diseases such as Parkinson's and muscular dystrophy. "As a charity we would want to have a world without diabetes and have a cure for diabetes," he said. "When you hear about stem cell research it always seems to mention muscular dystrophy, Parkinson's and diabetes.

"But there are two types of diabetes. For Type 1 diabetes, stem cells might help us towards a cure, but that is not necessarily the case with Type 2, which affects 85 per cent of people with diabetes.

"If you do not differentiate between the two, people could be thinking stem cells will be the answer for them and it is unlikely that it will be the answer for the majority."

Dr Belinda Cupid, research manager at the Motor Neurone Disease Association, had a similarly measured view about the prospects of stem cell cures.

"We don't think we are going to see a stem cell treatment for MND in the near future. But we do think that stem cells for use in MND research hold huge potential.

"They can be used as a model of the disease in a dish so we can test new treatments and also find out more about the condition which will help us make further progress."

The UK is well placed to carry out stem cell research and make the breakthroughs so desired.

Researchers in the United States also look set to benefit from a likely move by Barack Obama, the president-elect, to lift a ban on federal funding for embryonic stem cell research.

Mr Sykes said: "The lifting of the (George] Bush ban on federal funding for stem cell research is to be welcomed. It will undoubtedly take the brakes off US public-funded research and lead to a greater number of research papers coming from the US. But it would be wrong to think that stem cell research has stood still in the US because of this ban. It has been moving forward through other sources of funding. But access to federal funding should help it proceed even faster now."

Dr Josh Brickman, an American who works at the Institute for Stem Cell Research in Edinburgh and has been in the UK for ten years, said he did not think that researchers would rush back to the US because of the expected U-turn.

"The funding climate in the UK for science is better than in the US. The US has really suffered because of the wars in Iraq and Afghanistan.

"The NIH (National Institutes for Health] is funding at less than 10 per cent, which potentially means the US has lost a generation of scientists, because those who are my age who would have started their labs under the Bush administration have not been able to get funding at all."

But while stem cell research will push forward in the UK and elsewhere, there are also fears that the hype about the work is leading patients down a dangerous path. Mr Sykes said his organisation was getting several calls a week from patients looking into "stem cell tourism" – heading abroad for experimental treatments.

"A lot of patients are quite desperate and will go to great lengths to try out what is an experimental and untested treatment abroad," he said. "There are countries where this type of work is not regulated as carefully as it is in the UK.

"The potential for someone to suffer a poor outcome is significant and then they might require treatment on the NHS to help them. They may suffer serious infection or worse."

Such treatments – some of which involve injecting stem cells straight into the brain and cost up to £20,000 – are on offer in countries including Mexico, China and India.

Dr Cupid said: "There are people who realise that those with MND are looking for anything that could help them and are using stem cells to offer treatments that don't have any scientific basis. They just see the opportunity to make money out of desperate people."

While patients continue to pray for another stem cell breakthrough which will help them, the research shows no signs of letting up.

Next week, Aberdeen University plays host to a lecture on the possible use of stem cell therapies to grow "spare parts", such as bone or cartilage.

With such an attractive carrot being dangled in front of them, it is hardly surprising that patients' hopes are rising too fast for science to keep up.

THE EXPERT'S INSIGHT: Next hope is for breakthrough is cure for diabetes

Dr Josh Brickman Senior Fellow, Medical Research Council

PREDICTING a major breakthrough in fundamental biology is by definition impossible.

However, the next real breakthrough in the application of stem cell research is likely to be cell replacement therapy for Type 1 diabetes. As there already exists a protocol for the treatment of diabetes by transplanting insulin-producing cells from dead donors, the big hurdle that remains is learning how to produce large amounts of this type of cell from stem cells.

While you have to be careful that the cells you put back into patients don't cause more harm than good, for example cancer, I believe that we are close to being able to cure diabetes. That will be a tremendous breakthrough.

Scotland is a centre of excellence for stem cell work. There is a strong emphasis on basic science that has been very significant in the development of embryonic stem cell technologies. A lot of major innovations in the field have come from here. But it would be foolish to say that Scotland is on the brink of solving any major disease through stem cell research tomorrow. The same applies to any country.

However, it is important not to lose patience. Currently, there is a very strong push from governments to translate science into the clinic, and to some extent this is leaving behind basic science.

This is worrying as it is very difficult to predict where that breakthrough will come from, and the discovery of embryonic stem cells themselves was a result of this sort of "blue skies" research. It is also essential to understand the nature of any cell to be used for a clinical application.

But the UK has developed an environment in which stem cell research can go forward. We are hopeful that we can find new cures and new treatments, but only if we do not lose sight of the importance of basic science.

THE INSIDER'S VIEW: Exciting times as studies produce results

Dr Tilo Kunath Stem Cell Researcher, Edinburgh University

IT IS very exciting to be working in stem cell research at the moment. Part of the reason for this excitement is that many of the studies being carried out now are producing very promising results.

For diseases such as Parkinson's and diabetes there is real hope that we could find new treatments through stem cell research. There is also hope for diseases such as motor neurone disease for which there is currently no effective treatment or cure.

When I go to stem cell meetings there is a lot of excitement about the hard core science and what that has discovered. What is more challenging is the translational work which will result in the treatments to be used in patients. These kind of developments are still some time off. Scientists are always urging caution. A lot of hype has been built up by the media and some scientists who are not as familiar with the basic stem cell science.

It is good to have a buzz and excitement, but it is also good to have reality checks. This is not going to be the cure-all for degenerative diseases in the next five or ten years.

There are a lot of hurdles still to overcome. Stem cell treatments will be very personalised and that means they will be expensive.

But we are now seeing important discoveries, such as the development of embryonic-type stem cells made from adult cells. This will really help push forward the progress that has already been made.

IN QUOTES

"What we're seeing today is just the beginning … I reckon in 20 years' time it will be the commonest operation surgeons will be doing. I think it will completely transform the way we think about surgery, health and disease"

– Professor Martin Birchall, of the University of Bristol, one of the team who helped make the stem cell windpipe transplant possible

"I was scared at the beginning because I was the first patient but had confidence and trusted the doctors. I am now enjoying life and am very happy that my illness has been cured"

– Claudia Castillo, whose new windpipe was created using stem cells harvested from her bone marrow

"Regenerative medicine is a new and exciting field of medicine that could completely revolutionise the way we treat patients with injuries such as broken bones, sports injuries and osteoarthritis, a very common degenerative joint disease"

– Professor Cosimo De Bari, professor of translational medicine, University of Aberdeen

"While we must devote enormous energy to conquering disease, it is equally important that we pay attention to the moral concerns raised by the new frontier of human embryo stem cell research. Even the most noble ends do not justify any means"

– US president George Bush, from a speech made in August 2001

"The best that can be said about embryonic stem cell research is that it is scientific exploration into the potential benefits of killing human beings"

– Tom DeLay, a Republican former majority leader of the US House of Representatives

"Researchers and biotech executives foresee the day when catastrophic diseases can be reversed… Stem cells provide hope that this dream will become a reality"

– George Wolff, author of The Biotech Investor's Bible

KEY WORDS

REGENERATIVE MEDICINE

Medicine with the aim of restoring function to damaged body organs and tissues.

STEM CELL

The building blocks of life – a cell with the ability to turn into different types of cell, such as those in the heart, brain or spinal cord.

TRANSLATIONAL RESEARCH

Taking basic science from "bench to bedside", turning discoveries made in the lab into treatments for patients.

BREAKTHROUGH

A major advance in knowledge; in medical terms it usually refers to significant developments in our understanding or treatment of disease.

CURE

To restore to health and make well again.