This new research involved nine women with age-related macular degeneration and nine people with a rare condition called Stargardt’s macular dystrophy, both of which cause progressive damage to the retina. Macular degeneration is the leading cause of visual impairment in the UK, and until now there has been no treatment.

The participants had retina cells derived from human embryonic stem cells transplanted into the space behind the retina in the eye with the worst vision. Before surgery, they had severe visual loss. After 12 months, vision in the treated eye improved significantly.

Some papers reported how some participants were able to read a watch or use a computer after receiving treatment, though this is not mentioned in the actual study. What is described is a marked improvement in Snellen test scores, where you read a chart of letters that get progressively smaller.

Contrary to headlines, there were some side effects, but these all resolved. And over an average of 22 months’ follow-up, there were no signs of transplant rejection or major complications.

Further larger studies will doubtless now be underway to determine the optimal dosage and conditions for success.

Where did the story come from?

The study was carried out by researchers from the University of California, Wills Eye Hospital and Thomas Jefferson University, the University of Miami, the Massachusetts Eye and Ear Infirmary and Harvard Medical School, Advanced Cell Technology, and the Medical University of South Carolina.

It was funded by Advanced Cell Technology, a biotechnology company that develops and commercialises stem cell therapies.

Researchers from the company were involved in the study design, data collection, analysis, interpretation and writing the report.

This means there is potential for bias of the results. However, the other authors had full access to all of the data and responsibility for arranging publication.

The study was published in the peer-reviewed medical journal, The Lancet.

In general, the media reported on the study accurately, and The Guardian provided expert quotes about the positive effect the improvement in sight had on some of the participants.

Contrary to The Independent’s headline, however, a number of side effects were experienced as a result of the surgical procedure and immunosuppression.

Claims the participants were blind are also not entirely technically correct, as they had some degree of vision. Blindness – complete loss of vision – is actually uncommon. Most people with visual impairment have some limited degree of low vision.

What kind of research was this?

This research described two phase one/two trials that aimed to test the long-term safety and effectiveness of transplanting retinal pigment epithelium cells derived from human embryonic stem cells into the eye.

Phase one trials are the first type of study for new treatments conducted in humans. They aim to test the safety and suitability of the treatment for humans.

Phase two trials look at the effectiveness of the treatment and help determine the optimal dose. They also further assess any side effects. These trials were a combination of the two.

What did the research involve?

Nine women with age-related macular degeneration (median age 77, range 70 to 88) and nine people with Stargardt’s macular dystrophy (median age 50, range 20 to 71) were recruited to the study from four US hospitals.

The researchers took human embryonic stem cells, which are able to become any tissue type, and developed them into retinal pigment epithelium cells. These cells were injected just behind the retina of the eye with the worst vision.

Three different doses were tested: 50,000 cells, 100,000 cells or 150,000 cells. Each dose was given to three people with age-related macular degeneration and three people with Stargardt’s macular dystrophy.

All of the participants were given drugs to suppress their immune system (immunosuppressants) from one week before the transplant to 12 weeks afterwards to reduce the risk of transplant rejection.

The groups were followed up with numerous eye and physical examinations over a median period of 22 months (four people for less than 12 months, 12 people for 12 to 36 months, and two people for more than 36 months).

What were the basic results?

Before treatment, the best visual ability ranged from 20/200 (severe visual loss) to near blindness. (The 20/200 score means they could only read words from 20 metres away that a person with healthy vision would be able to read from 200 metres away).

In the age-related macular degeneration group, after six months:

• four people could see at least 15 more letters in the treated eye during the Snellen test (equivalent to three lines on the standard visual chart)
• two people could see at least 11 to 14 more letters
• three people remained stable or could see up to 10 more letters

After 12 months:

• three people could see at least 15 more letters in the treated eye
• one person could see at least 13 more letters
• three people remained stable or could see up to 10 more letters
• two people did not have a 12-month assessment

In the Stargardt’s macular dystrophy group, after six months:

• three people could see at least 15 more letters in the treated eye
• four people remained stable or could see up to 10 more letters
• one person had a deterioration of 11 letters
• one person did not have a six-month assessment

After 12 months:

• three people could see at least 15 more letters in the treated eye
• three people remained stable or could see up to 10 more letters
• one person had a deterioration of more than 10 letters
• two people did not have a 12-month assessment

The higher dose of cells – 150,000 – gave better results in the age-related macular degeneration group. A lower dose of 50,000 gave the best results in the Stargardt’s macular dystrophy group.

Participants also had, on average, improved quality of life, as measured by a questionnaire.

Nobody had acute transplant rejection, abnormal excess growth of the transplanted stem cells, or tumour formation. In addition, they did not experience retinal detachment or changes in the blood vessels of the retina.

There were some side effects from the treatment, however, including:

• retinal cells grew in front of the retina in three people, but did not cause any problems
• four eyes developed cataracts, which were treated with surgery – one with age-related macular degeneration, and three with Stargardt’s macular dystrophy
• one person had severe inflammation of the fluid compartment inside the eye after surgery and an infection with Staphylococcus epidermidis – this took two months to resolve and sight returned to pre-operation level after three months
• another person also had inflammation of the fluid compartment three weeks after the transplant, which resolved slowly over six months
• several systemic adverse events were reported as a result of immunosuppression

How did the researchers interpret the results?

The researchers concluded that they “have shown the medium-term to long-term safety, graft survival, and possible biological activity of pluripotent stem cell progeny in individuals with atrophic age-related macular degeneration and Stargardt’s macular dystrophy.

They go on to say that, “Our results suggest that hESC-derived cells [human embryonic stem cells] could provide a potentially safe new source of cells for the treatment of a variety of unmet medical conditions caused by tissue loss or dysfunction.

“The goal should be to treat patients early in the disease, potentially increasing the likelihood of photoreceptor and central visual maintenance or rescue in amenable retinal disorders.”

Conclusion

These phase one/two studies have shown human embryonic stem cells can be developed into retinal cells in the laboratory and successfully transplanted into the eye, causing clinically significant visual improvements.

The technique does come with the usual potential surgical complications, but other major side effects were not found.

Limitations of the study include the small size, but this is normal in early trials whose primary aim is to determine safety.

Larger studies will be required to determine the optimal dose and the most appropriate candidates for the technique, as it was detrimental in one person and did not improve – or gave minimal improvement to – six other people.

There will also be ongoing ethical considerations for the technique, which currently uses cells left over from in vitro fertilisation (IVF).

Overall, these studies show a promising treatment for two of the commonest causes of visual impairment in the developed world, though it will take a few more years of trials to optimise the technique.

Analysis by Bazian. Edited by NHS Choices. Follow Behind the Headlines on Twitter. Join the Healthy Evidence forum.

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This study saw researchers transplant stem cells into rats’ brains. These cells then developed into dopamine-producing brain cells.

Parkinson’s disease is a neurological condition associated with the loss of dopamine-producing brain cells. This leads to the symptoms characteristic of the condition, such as tremor, stiff, rigid muscles, and slow movements.

Parkinson’s is currently treated with medication that attempts to compensate for the loss of these cells, but it cannot replace them.

This new research demonstrated it may be possible to use stem cell-derived dopamine nerve cells to treat the condition, giving long-term functional results.

Up to six months after the cells were grafted into the brains of the rats, brain scans and functional tests showed the transplanted cells had proliferated and matured, reinnervated the brain tissue, and were producing dopamine.

The next step would to be to try to follow on from this research with clinical trials in humans.

Where did the story come from?

The study was carried out by researchers from Lund University in Sweden and other research institutions in France.

The research and individual authors received various sources of financial support, including the European Community’s 7th Framework Programme.

The study was published in the peer-reviewed journal, Cell Stem Cell on an open access basis, so it is free to read online.

Both BBC News and ITV News gave a good representation of the research.

What kind of research was this?

In this laboratory study, researchers aimed to produce dopamine neurones (nerve cells) from human embryonic stem cells and graft these into a rat model of Parkinson’s disease. They wanted to see if this had the potential to be used as a treatment for the disease.

Parkinson’s is a neurological disease with an unknown cause, which sees a loss of the nerve cells in the brain that produce the chemical dopamine.

Loss of dopamine causes the three classic Parkinson’s symptoms of tremor, stiff, rigid muscles and slow movements, as well as a range of other effects, including dementia and depression. There is no cure, and current drugs aim to try to control symptoms by treating this dopamine imbalance.

Human embryonic stem cells have the potential to develop into any type of cell in the body. Using these stem cells to replace dopamine nerve cells seems a promising area for research, and this study is the first step in investigating whether this type of treatment could one day be possible.

What did the research involve?

The researchers developed dopamine nerve cells from human embryonic stem cells (hESC) in the laboratory.

They then needed to see whether these cells would survive and function in the long term when grafted into brain tissue.

They transplanted these hESC-derived dopamine neurones into a rat model of Parkinson’s disease, where the rats’ brains were injected with a toxin to stop dopamine production.

The researchers followed the rats for six months after the cells were transplanted into their brains, carrying out various brain scans and tissue examinations to see how the cells had developed and were functioning.

They then carried out a behavioural test in the rats to see whether the transplanted cells had caused a recovery of their motor function (movement).

What were the basic results?

One to five months after the hESC-derived dopamine neurones had been grafted into the brains of the rats, MRI scans showed the transplanted cells had increased in volume, indicating that they were proliferating and maturing.

Further imaging was carried out using PET scans to detect a radiolabeled chemical marker that targets dopamine receptors.

Before grafting, the brains of the Parkinson’s rats demonstrated a high level of binding of this chemical to the dopamine receptors, indicating that dopamine was lacking and that this marker was taking dopamine’s place in the receptors.

Five months after grafting, binding of this chemical was reduced to normal levels, which indicated there was an active release of dopamine from the transplanted cells and dopamine was therefore now binding to these receptors.

Examination of the rats’ brain tissue confirmed these imaging findings, showing that the tissue was rich in dopamine neurones and that the transplanted cells had reinnervated the brain tissue.

The behavioural test also gave positive results, indicating that the transplanted hESC-derived dopamine neurones led to functional motor recovery in the rats.

How did the researchers interpret the results?

The researchers concluded they have “performed a comprehensive preclinical validation of hESC-derived [dopamine] neurons that fully supports their functional efficacy and capacity for long-distance, target-specific reinnervation, predictive of their therapeutic potential”.

Conclusion

This is promising early-stage research that demonstrates it is possible to manufacture dopamine-producing nerve cells from human embryonic stem cells in the laboratory.

The cells were then transplanted into a rat model of Parkinson’s disease (the rats were given a toxin that destroyed their dopamine-producing cells).

Up to six months after the cell transplant, brain scans and functional tests showed that the transplanted cells had proliferated and matured, reinnervated the brain tissue, and were producing dopamine.

The next step is to follow on from this research with the first clinical trials in humans. The researchers say they hope they will be ready for the first clinical trial in about three years’ time.

But there are several technical obstacles that need to be overcome first. Although the results indicate the transplanted cells were functioning well in the rat model at five months, as the researchers say, it is important to verify that these functional effects are robust and stable over significantly longer time periods.

Also, the rat brain is much smaller than the human brain. It would therefore need to be demonstrated that the transplanted cells have the capacity to grow nerve fibres that can reinnervate distances relevant to the size of the human brain.

This research holds promise for a future stem cell treatment that could restore the dopamine-producing nerve cells lost in people with Parkinson’s disease. The next stages in this research are awaited eagerly.

Analysis by Bazian. Edited by NHS Choices. Follow Behind the Headlines on Twitter. Join the Healthy Evidence forum.

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Sadly the baby´s brain had been starved of oxygen for a period during the pregnancy and her brave parents made the heartbreaking decision to donate her organs.

NHS Blood and Transplant´s organ donation team, the hospital nursing staff, and the hospital´s psychologist, all supported the family in their decision to donate their daughter´s kidneys and liver cells to be used for the benefit of other sick patients.

Newborns that die in neonatal units could potentially be organ donors, and therefore save the lives of other sick patients, but current guidelines make it very difficult to diagnose brain stem death in babies under two months old. New guidelines from the Royal College of Paediatrics and Child Health are expected shortly.

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Leading heart and lung surgeons at the Freeman Hospital will be showcasing their life-saving transplant techniques next week, when they host their first International Teaching Course for Heart and Lung (Cardiopulmonary) Transplantation.

Newcastle’s Freeman Hospital – an internationally-renowned Centre of Excellence for all forms of organ transplant surgery – will welcome over 20 Surgeons from around the world when they attend the course at the Newcastle’s Surgical Training Centre on 2 and 3 September 2014. Some are travelling from as far afield as Brazil and India to meet our transplant experts.

It aims to teach complex techniques in the field of Cardiopulmonary Transplantation and the use of Ventricular Assisted Devices or VADs which are used to keep people alive while they wait for a heart transplant. This unique course gives delegates the chance to witness complex procedures first-hand, and carry out some transplantation procedures themselves using cutting edge simulation techniques.

Prof Stephen Clark, Consultant Cardiothoracic Surgeon and Co-Course Chairman, explains: “We are delighted to be holding our first International Heart and Lung Transplant Course here in Newcastle. The course has attracted clinicians from all over the world which confirms the international reputation we enjoy in this field.

“The programme we offer has garnered a great deal of interest because it offers the best of both worlds for Surgeons. The opportunity to observe the latest techniques carried out by leaders at the cutting edge of Cardiopulmonary Transplantation, and then to gain practical experience with one to one guidance and direction. This includes Cardiac and Lung Transplantation, implantation of VADs, ex vivo lung perfusion (reconditioning of donor lungs) and ECMO (a treatment that uses a pump to circulate blood through an artificial lung back into the bloodstream).

“This is our first course of this calibre and we hope to continue to invite our international colleagues annually.”

Sir Leonard Fenwick, Chief Executive for the Newcastle upon Tyne Hospitals says: “We are extremely proud to hold this trailblazing course – the first of its kind in the UK – which offers a superb opportunity to showcase our transplantation expertise to colleagues on an international level. This course follows on from our balloon release celebrations at the Freeman Hospital on Monday, to acknowledge the major milestone of performing over 100 adult congenital heart transplants here in Newcastle. No other centre in the UK has the same expertise in this challenging field of transplantation which means we essentially provide a national service for these patients.”

ENDS

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The Freeman Hospital is celebrating 100 adult congenital heart transplants with a balloon release, conference and celebratory lunch, alongside patients and their families. All of these patients were born with complex heart disease and never expected to live to adulthood.

The Freeman Hospital has an international reputation for pioneering expertise in transplant surgery and infant cardiology treatment with best in class outcomes. The hospital’s Cardiothoracic Centre is home to the largest adult congenital heart transplant centre in the world, and is the only centre in the UK to provide comprehensive cardiac care, from birth through childhood and onto adulthood.

Many of the patients attending the celebration have received heart and heart/lung transplants at the Freeman Hospital spanning the last 27 years. They will attend to show their support and thanks to their donors and the work of the Specialist Team.

Ranked one of the leading and most progressive centres in the UK, the Freeman Hospital provides specialist treatment for all types of heart and respiratory related conditions in adults and children. The first adult heart transplant was successfully performed in 1985, followed by the UK’s first successful baby heart transplant in 1987. The first adult congenital heart transplant also took place in 1988.

The first patient to receive a lifesaving heart transplant for adult congenital disease, David Yates, from Newcastle explains: “26 and a half years ago, a 25 year old man died and his family unselfishly thought of others, rather than their own grief. They donated their son’s heart. I was the fortunate beneficiary – I received a second chance of life – I now had a future to hope for.”

Many, many more success stories followed (now 116 transplant operations) as a consequence of the highly skilled and dedicated staff.

Sir Leonard Fenwick, Chief Executive says: “We are so proud to reflect upon the commitment shown, year on year, by so many dedicated and highly skilled healthcare professionals who have pursued a plethora of innovative approaches to new technologies and treatments in this very challenging area of medicine.”

The post 100th Adult Congenital Heart Transplant Celebration appeared first on Transplant TV.

36 adults and 12 children, who have had life saving heart and/or lung transplants at the world renowned Freeman Hospital in Newcastle-upon-Tyne, are preparing to compete in the British Transplant Games in Bolton from the 7^th – 10th August.

This is the largest team of heart/lung transplant recipients to compete in the games since they began in 1978.

The British Transplant Games encourages those who have received a transplant to lead active and healthy lives while showcasing the benefits of transplantation. The aim is to raise awareness of the need for more people to sign on to the NHS Organ Donor Register. In the UK, 10,000 people are waiting for a life saving transplant, and 3 die each day waiting.

Over 700 transplant athletes from 55 transplant units across the UK will take part. Aged from 2 to 82 they will compete in 20 different sports including archery, athletics, golf, squash, snooker, tennis, cycling and swimming. Competition will be fierce, but the athletes have already won the race for life – winning a medal will simply be a bonus! There are guest competitors from Australia, Canada, Ireland and Italy.

Selection for Team GB&NI takes place after these games, and the successful athletes will compete at the World Transplant Games in Argentina next year.

Lynne Holt, Clinical Transplant Coordinator at the Newcastle Freeman Hospital  and Trustee of Transplant Sport (TS) says: ‘These are truly the friendly Games, demonstrating the success of transplantation at the Freeman Hospital, the joy of being alive and the opportunity to give thanks to the Donor families. We encourage everyone to discuss their wishes about organ donation with their families, without which, none of the athletes would be alive to compete.’

1,200 supporters are expected to cheer on the athletes over 4 days at the Westfield Health British Transplant Games. The event is managed by M LS Contracts, in partnership with the charity Transplant Sport UK.

To find out more about the Westfield Health British Transplant Games please visit www.transplantsport.org.uk .

To Join the NHS Organ Donor Register and help save lives TEXT: SAVE to 62323, call 0300 123 2323 or visit www.organdonation.nhs.uk

Ends

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Newcastle Hospitals is delighted to announce that the Freeman Hospital’s Institute of Transplantation will be officially opened at a special ceremony this week, by two of the world’s true pioneers in the field of transplantation:

Sir Roy Calne, who performed the first liver transplant in Europe in 1968 followed by the world’s first liver, heart and lung transplant in 1987; and Professor John Wallwork, CBE, who performed Europe’s first successful heart-lung transplant in 1984.

Also in attendance will be the Rt Hon Mr Nick Brown, MP for Newcastle upon Tyne East, who ‘cut the turf’ for the Institute’s construction site nearly four years ago in October 2009. At the time he said: “If you need an organ transplant, there is no better place to live than Tyneside. I am really proud of the work done at the Freeman Hospital. The new facility is going to be world class and will underpin Newcastle’s standing in the medical world.”

The Opening Ceremony is part of a major Symposium with a number of eminent clinicians visiting from all over the UK leading on lectures covering the history of transplantation, current clinical activity and research in Newcastle, and the future of transplant surgery.

Sir Leonard Fenwick, Chief Executive explains: “We are extremely proud of the Institute of Transplantation here in Newcastle – the first centre of its kind dedicated solely to the provision and advancement of solid organ transplant surgery for those whose lives depend on it. To have such distinguished guests to help us celebrate the official opening is a great honour for the NHS here in the North East of England.”

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