When it comes to research into children’s diseases, these are surely the best of times – and the worst.
The strides made in many areas of scientific research in recent years have produced welcome improvements in the treatment of serious childhood disease. Former killer diseases – specific forms of leukaemia, for example – are now curable and others are eminently treatable.
But even as the death rates for some childhood diseases have dropped, others remain stubbornly resistant to treatment, recur often or involve treatments with difficult side effects.
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Meanwhile, the recession has put the health service under tremendous pressure, and recent funding scandals involving a small number of charities have made it harder for all charities to raise money.
Paediatric research has always been a harder “sell” for fundraisers than the purchase of hospital equipment for bubbly, camera-friendly babies. It is also expensive to run and requires long-term, stable funding, so falling revenue threatens the foundation of such programmes.
And yet so much of the progress made in eliminating, reducing or treating childhood diseases is due to academic research carried out in proximity to children’s hospitals around the world.
In Ireland, the focal point for research into paediatric medicine is the National Children’s Research Centre (NCRC), located in a compact building at the back of Our Lady’s Children’s Hospital in Crumlin.
This year marks the 50th anniversary of the centre, set up by former Catholic Archbishop of Dublin John Charles McQuaid in one of his more visionary moments. With a low-key but solid record of achievement behind it, the centre now stands on the cusp of a new era. When Crumlin merges with the other Dublin children’s hospitals and moves to the planned new national children’s hospital at St James’s, the research facilities will move with it.
For those in the business of children’s research, it’s like being on the brink of the Promised Land – again. “We’re back where we were in 2007-2008,” says chief executive Dr Jacinta Kelly, recalling how the centre was supposed to move to the site of a new children’s hospital at the Mater.
That proposal collapsed when planning permission was refused, and a new plan that would see the hospital built at St James’s is going through the planning process. Kelly is, understandably, wary. “Either it goes ahead, and we gear up for that . . . or it doesn’t and we stay here”.
Long-term stability
While a change to modern, state-of-the-art premises would be welcome, of equal importance is the longer term stability required to underpin the work at the centre.
Eight years ago, despite the imminence of the Mater transfer, it was decided to revamp the centre, by upgrading the facilities and bestowing a national character on its work. The decision proved prescient, given how events turned out.
“We wouldn’t be here today if they hadn’t revamped the centre at that time,” Kelly acknowledges.
The NCRC is small by international standards but its collaboration with more than 90 research institutions around the world ensures it punches above its weight.
“A small centre is not going to come up with the new Herceptin [cancer drug] or blockbuster drug for asthma” but it can, by working in a network, make a significant difference, says Prof Paul McNally, a respiratory medicine specialist at Crumlin.
The best-qualified doctors are attracted to working in hospitals where research is carried out. McNally says an emphasis on academic medicine in Crumlin has had a very positive effect on the children’s hospital. “It’s about the ethos of the place, and a focus on excellence. It draws people back in.”
But according to Prof Owen Smith, doctors often find they have to pursue research interests outside normal working hours. “Your contract tells you that you should have 15-20 per cent of your time dedicated to research. But this isn’t the case. The system is set against that because it’s top- heavy with the delivery of clinical programmes and outpatient lists.”
As a result, he says, consultants involved in research end up sacrificing social interests in order to find time for their work. “The system doesn’t lend itself to the promotion of physician scientists, and the Government needs to reflect on this.”
Beacon for Europe
Smith, an outspoken supporter of the plan for a children’s hospital at St James’s, believes the new facility, by dint of its size, staffing and newness, can become “a beacon for Europe” in terms of children’s medicine.
However, the new hospital has to be an institution “embedded in research and innovation”, he says. In this respect, it can build on synergies with the adult research institutes already on the St James’s campus.
What also excites scientists and doctors is the fact that the new hospital will be a national institution providing for the care of all children in the Republic. As one of the biggest children’s hospitals in Europe, it will be a major attraction for researchers and drug companies trialling new treatments.
Kelly explains what this could mean for genetic research: “When you’re looking for a needle in the haystack, it helps when you have the whole haystack.”
Should the project get the go-ahead from An Bord Pleanála, one of the big challenges will be the financing of a new children’s research centre. The €650 million envelope allocated for the hospital does not include research facilities, so a massive fundraising effort will be needed.
Currently the centre is funded by the Children’s Medical Research Fund, which last year split €12 million in income between the centre and the hospital in Crumlin. The centre’s 2014 annual report describes the fundraising environment as challenging and says the money available for new projects was “very limited” compared with previous years.
With the move to St James’s still in doubt – the oral hearing into the project will hear determined opposition from a variety of parties – there seems little point in fundraising yet.
Even if this goes well, there is little prospect of raising the kinds of sums that US children’s hospitals collect. Smith notes that one leading US children’s cancer centre – St Jude’s in Memphis – raises $1.5 million a day in charitable donations.
The National Children’s Research: Centre What does it do?
The National Children’s Research Centre (NCRC) is the largest paediatric research institute in Ireland, with a remit for all children’s research groups in the State. It funds research in a variety of different areas, such as:
Eczema/allergies
Eczema is the most common inflammatory disease of childhood, with the vast majority of cases starting in the first five years of life. As many parents can attest, it has a significant impact on the quality of life of affected children, and is associated with food allergies, asthma and hay fever.
With so much still to be understood about the condition, it’s also a natural fit for paediatric research. Research at the NCRC focuses on the relationship between the barrier provided by skin and immune abnormalities.
One project, conducted with UCC, which measures the amount of water leaking from newborn babies’ skin, found a correlation between high leakage and the presence of eczema at one year of age.
This suggests a way of being able to tell at birth which baby is going to develop eczema, according to principal investigator Prof Alan Irvine.
The focus is now on developing simple solutions that can control the problem, such as moisturising aggressively and avoiding soaps. Another aim, he says, is to develop nationally applicable standards for the treatment of childhood eczema.
Related research into peanut allergy shows the risk of developing the allergy increases when exposure occurs through the skin rather than orally. Work from King’s College London suggests that a solution may lie in feeding children peanuts so their gut develops the required tolerance, rather than avoiding the item altogether.
Childhood cancer
Cancer is rarer among children than among adults, but nonetheless distressing for being so.
Every case of childhood cancer in the Republic is diagnosed in Our Lady’s Children’s Hospital in Crumlin, where the aim is to get all children with the disease onto a clinical trial offering the most up-to-date treatments.
At present, up to 65 per cent of children with cancer enter a trial, according to haematologist Prof Owen Smith. This is far higher than the less than 5 per cent of adults who get access to a trial.
Smith is confident Crumlin will be formally recognised as the State’s ninth cancer centre when a national strategy for the disease is drawn up shortly.
He would like to see a more co-ordinated approach to adolescent medicine than exists at present, and hopes the move to a national children’s hospital alongside the adult facilities in St James’s will improve services.
Crumlin can admit children only up to 16 years, he points out, even though 80 per cent of the concerns with 16-21- year-olds involve cancers that predominate among children.
One line of cancer research at the NCRC is examining how to use genetics to pick out young patients who might react adversely to treatment, so that alternative treatments can be tried. For example, 8 per cent of children with one form of leukaemia develop severe bone disease from the steroids administered as part of the therapy. Another leukaemia research project is trialling a treatment of vitamins and arsenic instead of chemotherapy. It has proved successful but is linked to heart disease among some patients.
Cystic fibrosis
Ireland has one of the highest incidences of cystic fibrosis (CF) in the world. The silver lining to this cloud is that this makes the country especially attractive to international researchers questing for a cure for the disease.
A direct consequence of this research interest has been the early availability of innovative treatments to Irish patients, leading to an improvement in life quality and expectancy.
CF researcher Prof Paul McNally cites the example of one wonder drug, Kalydeco, which was given to Irish patients with specific CF mutations in trials up to three years before it was made generally available.
However, McNally believes huge improvements can be effected simply by making use of the body of research already available, possibly by using it in a different way. Improving the organisation and delivery of care for patients can make a huge difference to outcomes, though it isn’t very “sexy”, he says.
“Sometimes it’s the simple things, done well, that make a huge difference.” With all newborns now screened for CF and lung sample collected regularly thereafter, a vast amount of data is available to researchers who are seeking to detect early signs of deterioration of lung function among affected children.
“We need detailed longitudinal data so that if more information becomes available, we can go back and check it. A national programme will bring together research, clinical trials and clinical care.
The Shield CF study seeks to combine all lab research and patient data into one centrally controlled programme operating eventually through all six of Ireland’s specialist paediatric CF centres.
Maintaining this data in a biobank is an expensive, long-term project but, as McNally says: “We cannot afford not to do it.”
CF remains a degenerative condition. McNally says sufferers and their families are, understandably, prone to negativity at times. “At least by being involved in research though you get a sense that you’re part of something that’s going somewhere.
