It’s about acceleration

Professor Pam Kearns talks frankly about clinical trials and children’s cancer.


If you search on Facebook, you can find a picture of Pam Kearns with ‘the girl whose life she helped save.’ In the photograph, doctor and patient share a relaxed smile and an obvious affinity. Pam was Paige’s clinician after the 11 year-old fell ill with Wilm’s Tumour, a rare form of kidney cancer. However, the role she plays in helping children like Paige is not just that of providing treatment. She is Director of Cancer Research UK’s Clinical Trials Unit and a leading figure in the search for new ways to combat paediatric illnesses like Wilm’s.

Paige’s story is a heartening one but if you go to hear Pam speak, she will show you another side of the picture, flashing up on a Powerpoint slide the words of different child’s father:

‘It is inconceivable in this day and age that, globally, there is no drug that can cure my daughter’


Any child diagnosed with cancer faces an uncertain future and to find out more about why this is so and what can be done about it, I went to visit Pam at the Clinical Trials Unit in Birmingham on a sweltering morning this June.

Pam and I have known each other for 5 years through involvement in various projects to do with childhood cancer, but I had never visited her on her ‘home turf’ and I was curious to know how the ‘nerve centre’ of childhood cancer research in Britain operates.

She welcomed me into a surprisingly modest office she shares with a sleek, carbon-frame Cannondale, the bike she uses for a 20 mile daily commute and the sportive challenge she promises she will get round to soon.

I start at the beginning and ask her what exactly the Clinical Trials Unit does.

‘Lots of people don’t know what a Trials Unit is and that includes many clinicians. There are about 30 Trials Units within universities in the UK, of which about a dozen or so are dedicated to cancer and 8 are core-funded by Cancer Research UK.

‘The aim of a Trials Unit is to be the place where an investigator comes with an idea about improving treatment, whether it’s giving radiotherapy in a different way, introducing a new drug, a new type of surgery, any clinical idea that changes the patient pathway. We work with them to see if it’s a viable idea and if it is, we help design the trial, collect the data – everything from concept to completion; the only thing we don’t do is recruit the patients.’

Who pays the piper?

I ask her who funds the research.

‘By far, the majority of paediatric cancer trials are charity funded. Cancer Research UK (CRUK) is our biggest funder; they fund the core team (at the Clinical Trials Unit) and if a trial is good enough, they’ll fund the trial too. We sometimes manage to get into collaborative partnerships with the pharmaceutical industry where they will give us a grant and a drug to support the trial.’

She gives the example of a major investigation into leukaemia.


Figure 1: 2008 Spend on Children’s Cancers (NCRI 2014)


‘ If you look at the AML trial which we’ve just launched, that has a lot of questions about how to use standard chemotherapy, but it’s also got a drug called mylotarg, which industry are still trying to get a licence for. The majority of the funding is from CRUK but there’s also a large grant from Pfizer – plus the drug. So that’s a true partnership, where you’ve got charitable funding delivering the academic questions plus Pharma contributing to answers we are interested in – important to patients, to families, but because they are also interesting to Pharma, they are investing money in it.’

She goes on to explain, though, that ‘we don’t necessarily need the industry’s involvement if, for example, we’re asking questions that are about generic drugs.’

When she refers to generic drugs, she means ones that are already commercially available. She explains that in past decades, many of the advances in children’s cancers were made by simply repurposing generic drugs originally licensed for use with adults. Even in the present day, a lot of research simply involves revisiting generics already well-established with children, refining their doses and tweaking their combinations. This process, however, she argues, has run its course.

Big Pharma and Small Biotechs

‘We’ve hit a plateau of improvement in many diseases,’ she explains. ‘To make the step changes we need in the future, I don’t think we are going to achieve that by just using generic drugs. We need new approaches, new drugs with different mechanisms of action that we can add to our armoury. That’s when we need the industry to give us access to their new drugs and innovations.’

The revolution in genetic science in recent years has stimulated a new wave of commercial drug development. These are the so-called novel agents that target illness at a molecular level and which hold great potential for shifting children’s cancers off the ‘plateau.’

Going ‘off-label’

A drug is usually licensed to treat a specific type of disease or cancer. If doctors feel that a particular drug may help a patient with a condition not included in the drug license they can prescribe that drug for the patient. But the doctor has to take full responsibility for the outcome. This is called ‘off label prescribing’. Many clinical trials for cancer use medicines ‘off label’ in this way. The trials want to find out whether drugs licensed for a particular type of cancer could help to treat other types of cancer.

The problem is that the pharmaceutical industry is adult-orientated. Its focus is on the blockbuster markets created by the common cancers that afflict older people, rather than on the messy patchwork of rare conditions that are children’s cancers. Pam’s concern is with the delays involved in waiting for new agents to be commercially developed for adults before we investigate their potential in children.

‘If there was a generic drug, a drug that had come off its licence now,’ she explains, ‘ and that was being used for adult cancer and we wanted to use it in children because we had scientific evidence that it could make a difference, we could go ahead and do that. 

‘But what we’re saying is, “Look, drug companies with your massive pipelines of new drugs, you’re developing targeted drugs that would be useful to children now not when that drug has been developed for adult illnesses.”

‘It’s about acceleration. We could wait until X or whatever targeted drug has been licensed for adults and then repurpose it for children and just carry on the way we have before. But we’re delaying access to children by sometimes 30 years. So the whole purpose of this is getting into the drugs pipeline really early, before the drugs are on licence in adults and let’s see if they’re interesting in children.’

I ask her why we are so dependent on the private sector.  Could not research centres in universities or hospitals simply develop the drugs themselves? The answer, of course, is about money.

‘It costs a huge amount of money and there’s high attrition rate [ in drug development] and academic departments would struggle to do that. I’m not saying they don’t exist. There are drug discovery units, for example at the Royal Marsden’s Institute for Cancer Research but they can’t turn on the high throughput screening in the way industry does. If an academic department developed a drug, there is no reason why we would not work with them, but at some point they will hand on the further clinical development to a small biotech and if it’s good, it will be bought up by Pharma. It’s the money that’s required to develop the drug.’

She gives an example from Birmingham.

‘My colleague, Frank Mussai, he’s doing some really interesting work looking at a particular pathway relevant to childhood cancers, and he’s found a biotech company based in Asia who produce the drug that targets this pathway. They’re not rich and they haven’t got the expertise to do the clinical trials, so they’ve been giving him small amounts of money to do his lab research. They’ve been giving him access to the drug to develop the laboratory research. Today we have finalised a grant application to the charity sector to do a trial in paediatric cancers .

‘Now if that drug were ever to become a blockbuster, then we would get some of that investment back so that we could develop the next set drugs. But that would be a rare event.’

From Brussels to Birmingham

The conversation inevitably shifts to the measure the European Union introduced to stimulate commercial interest in children’s drug development, the Paediatric Medicines Regulation. Both Pam and I are involved in campaigning to reform the Regulation and are aware of its disappointing record to date. However, she emphasises that it has had an effect.

‘I can remember the days, ‘she says, ‘of going to big cancer conferences and talking to a Pharma company at a stand about drug that was relevant to my research and they were really interested in talking to me as a medic until I said I was a paediatrician and they were like, “How quickly can I get this woman away from me?”

‘And that attitude has changed and it’s changed because of the Paediatric Regulation. There is a benefit; it might not have translated into new drugs for children but a positive attitude change has happened. ‘

I ask her about a similar campaign she was involved with, around another piece of Brussels legislation, the Clinical Trials Regulation.

‘It feels like it was a huge chunk of my life,’ she sighs. ‘But it was only two years’ serious campaigning  ’

The important point, though, was that the campaign was broadly successful and layers of unnecessary bureaucracy that hold trials back are scheduled to be peeled away.

What did you learn, I ask her, from the experience of that campaign?

‘That you had to work with the European Parliament and the relevant rapporteur,’ she says. ‘But rather late in the game we realised if you don’t have the European Council on board, the changes you want could be voted out. I think we were quite late in engaging with the member states. For the UK, we were in a better position because the MHRA (Medicines & Healthcare Products Regulatory Agency) were taking the role  on behalf of the Health Minister and they were sympathetic to the changes we wanted, so we were pushing at an open door. But that wasn’t the case across Europe. We met with health attaches from one of the member states and they were still concerned with clinical research in children being unethical. So they were miles away from where we wanted them to be and they were the people sitting around the table at the vote.

‘This is what we need to remember with the Paediatric Medicines Regulation. We know the arguments inside out but when you sit down with someone for the first time, they’ve only got this vague idea what you’re talking about so getting that sound bite, that message across is really important.

That’s what I learnt.’

Ethics and Equipoise

I pick up on the worries about the ethics of involving children in research   and ask her to explain about randomised trials.

Phase 1, 2 and 3 – What’s the difference?

Clinical trials are often divided into three stages or phases. Earlier phase 1 trials focus on how much of the drug it is safe to give and whether it actually shrinks the cancer. If a phase 1 is successful, the therapy will be tested more extensively in phase 2 and 3 trials. A new treatment usually needs to go through phase 3 before it is accepted as standard.

Phase 2 and 3 trials are often randomised. This means that people taking part in the trials are divided into different groups at random, with each group being given a different treatment or arm. Trials that compare three or more treatments are called multi-arm trials.


 ‘When you are doing a randomised trial, there should be no prior knowledge or belief that one arm is better than another. This is what we call equipoise. As a clinician, when I sit down with a family and I ask a child to participate, I can explain the background to both the arms but I should be able to say, hand on heart, I don’t know which one is better. If I did know, it would be unethical to ask someone to take part. ‘

She refers to the major randomised trial into Ewing’s Sarcoma, the illness my own daughter had.

‘It is like Euro Ewing’s 2012. Do you want to participate in a comparison of the known gold standard in Europe against the known gold standard in America? We cannot differentiate between the two and now we are asking the question which is more effective and which has fewer side effects.

‘Equipoise is about randomised trials. However, there is no equipoise at phase 1.

‘In a phase 1 trial, the ethical question is that you are asking a parent for their son or daughter to participate in a trial that may not have benefit for their child.

‘Now you can  ask an adult to take part in a phase 1 and it’s that adult’s informed decision on the basis of altruism and, undoubtedly, a little bit of hope that they’re going to get the “miracle response.”

‘The Declaration of Helsinki states, however, that you cannot do clinical research in children when there is not at least the potential for benefit, which means you cannot do the type of  ‘first in man’ studies you can in adults, starting from very low doses. There needs to be evidence from laboratory research or clinical trials in adults that suggests the treatment could have a benefit at the doses given to a child.

Talking to families

I ask her how she puts the option of a phase 1 trial to parents and child.

‘If I can give this in very personal terms , when I sit down with a family to talk about a phase 1 study, particularly a family I have never met before, the first thing I do is establish their understanding of where a child is in (I hate the expression ) the “cancer journey.”

‘So do they understand their child has no curative option? And that it’s effectively palliative at that stage? Because, without that understanding, the rest of the discussion about the phase I trial is almost dishonest. The eligibility criteria for most phase 1 studies is that there is no known better curative option. I then explain to them the background to the trial but I also say what the other options are and those might be purely supportive.

‘It might involve  using a drug like etoposide. We don’t have a lot of published data to prove this  has benefit but substantial clinical experience tells us that it has some success in holding back the progression of disease in some children.

‘I’ll explain where the trial is in terms of the drugs development, what the implications are of being in that particular trial. Does that mean more time in hospital, more blood tests, more investigations, backwards and forwards from half way across the country to visit me?

‘I outline all that.

‘I strongly dissuade families from going, “Where do sign?” ‘I always say, “Go away and think about it, talk about it.” And I talk to the child about it, depending on their age.

‘Morally, ethically and within the Declaration of Helsinki, you never ask a child to participate in a trial where there is no evidence of benefit. But the chance of the benefit has to be understood to be quite small and then it comes down to the motivation of the family and the child and whether they want, at least, to try something.

‘Now my experience is that each family is different. You get some families that respond saying, “I don’t really want to have anything more to do with hospitals – that means we can have some life together for a while.” They’ll do that. They won’t participate in phase 1 clinical trials – and that’s fine.

‘Then you get the different kind of family that says, “Whatever there is. They want to know if a phase 1 trial fails, what’s the one after that and what’s the one after that. They want to keep going. They want to be involved in something that brings their child to the hospital and might have the potential for a miraculous benefit.

‘There’s another kind of family. Often it’s done with the involvement of the child in the decision making; for example, slightly older children. They really know there isn’t much chance of benefit but they feel they want to be doing something, even if it is for someone else. That’s extraordinary, in my mind, but parents have said to me that at least we tried to do something even if that something is for somebody else.

‘I am forever in awe of all three sets of families. None of them is making a right or wrong decision. My job as a medic is to listen and answer questions and hope they go away feeling they have made the right decision for them and their child.

 ‘It’s a hard area and that’s why it shouldn’t be done in every centre in the country. It must be done by people who have that experience of talking to families about these issues.

‘Most of our experience is in breaking bad news to parents, because the majority of children treated are very young. It is harder to gain the experience of breaking this type of bad news to  the age group of children who are just starting to be old enough to understand.

I remember doing it for the first time when I was a junior doctor and I can remember, to this day, this teenager whose disease had become incurable quizzing me really hard about what was going to happen and I wanted to run away from the question’

‘It would have been really easy to say, “It’ll be fine” and patronise this teenager. But I stuck in there and that is something we all need to do.

In passing, I mention the difficult exchanges we had with our consultant over the only trial my daughter was offered in the whole period of her relapse. I relate the mobile call just before Christmas in which we turned down a phase 1 study of aurora kinase, a drug I have heard nothing of since in relation to Ewing’s.

‘That’s because the clinical development was abandoned,’ says Pam, ‘because it didn’t work. They completed the study but it wasn’t effective in anybody.’

Compassionate Use

We talk about the children who are never offered the option of a trial – because the trials are full, because the children do not meet the eligibility criteria or, as is more often the case, because the trials are simply not running.

I ask her about compassionate use, which allows patients access to new medicines, even hough they are not enrolled on a trial.

‘Compassionate use exists in America. We’ve got it here too. It’s called “Named Patient Access.”

‘As an example, it is possible for a child with leukaemia to get access to inotuzamab, even though there’s no trial open for that drug at the moment. We could open the trial of this drug when we get through all the regulatory paperwork. Compassionate use of drugs cannot substitute for proper drug development because we’ll never know if we just use it compassionately whether the drug should be properly developed and marketed and available to everybody.

I push her on whether we can get more children off phase 1 and onto phase 2 studies, where the emphasis is less on the safety of the drug and more on its efficacy. She talks about a new trial.

‘That’s the aim of E-smart. It will have multi-arms and, as new drugs become available and they’ve got their safety data, we can slot them into the trial. When the child has that molecular pathway identified, it doesn’t matter whether it’s Rhabdomyosarcoma or Ewing’s, does it have the target for that drug, can they enter the trial? And we’ll just keep that trial open and growing as long as we can get access to the drugs and that’s the sort of trial we need more of.’

Reference: NCRI (2014) Funding of children’s cancer research. Accessed 19 June 2016 at:

Article written by Chris Copland, Unite2Cure

New drugs for childhood cancers: could biotechs end the drought?

An article in Cancerworld magazine by journalist Sophie Fessl, summarizes well the issues Unite2Cure is raising.


The chances of surviving a childhood cancer have changed very little over the past two decades. Sophie Fessl talked to parents, doctors, regulators and researchers about what has to be done to address this disastrous impasse. “It feels odd to say this, but Elliot is one of the lucky ones.” The comment comes from Nicole Scobie (photo, above, with Elliot), and refers to her son, who was only four years old when he was diagnosed with a stage IV Wilm’s tumour. His left kidney was engulfed by a huge cancerous mass and his lungs were full of metastases. The heart-stopping diagnosis was just the start of a rollercoaster of emotions, hospital stays and exhaustion. But this is a story with hope: Elliot responded well to chemotherapy. He went into remission after 10 months of treatment, and has remained so for the past four years.


“At least for his cancer, there is a treatment that works,” says Nicole. Not all the children she and her son befriended during their long stay at the Lausanne University Hospital’s children’s cancer ward can consider themselves as ‘lucky’. Elliot became close friends with Zoe, a little girl battling an aggressive neuroblastoma. But while Elliot’s prospects looked good, for Zoe, the odds were stacked against her. In the end, there was nothing her medical team could do: Zoe died in her mother’s arms aged four.

The difference between Elliot and Zoe? Elliot had a type of cancer that has been successfully treated for decades, being one of the first childhood cancers – alongside acute lymphoblastic leukaemia – to benefit from the chemotherapies pioneered by Sydney Farber back in the 1950s. His is part of the celebrated success story that saw cancer survival rates among children increase from 10% to 80% over 50 years.

Zoe, by contrast, had a type of childhood cancer that remains fatal in the majority of cases. Her story is shared by 6,000 children and young people under 24 who are still dying of cancer each year in Europe. For parents like Nicole Scobie, who’ve seen their child’s life in the balance, that is a heartbreaking statistic. But there’s a worse one – the mortality rate from childhood cancers has barely changed over the past 16 years.

Last year, Scobie was one of a large group of parents and advocacy organisations that got together to found Unite2Cure, an advocacy organisation that aims to kickstart progress again.

Read the full article online here or download in PDF format here

An Update from the Unite2Cure Team

Where do we go from here?

Here is an update on the state of play regarding the campaign on the Paediatric Medicines Regulation (PMR).


A Resolution to be put before the European Parliament has been drafted by members of the European Society for Paediatric Oncology, Unite2Cure and Cancer Research UK. The Resolution has now been been approved for debate in the Parliament and we expect this to take place in Strasbourg by the end of October. It is being sponsored by Francoise Grosstete of France and Elena Gentile of Italy. It will give a clear message to the Health Commissioner to act boldly in the forthcoming Review of the PMR.


Prior to this, there will be a special meeting of MEPs Against Cancer (MAC) Group in the European Parliament on 7 September, which will call for revision of the PMR. We hope to organise a ‘stunt’ (eg. a human ‘gold ribbon) to coincide with one of the above meetings.


Unite2Cure ‘ambassadors’ are working to lobby national politicians to exert influence on their representatives in the Europe, including members of the Council. In France, there was a lobby dinner for the Senate on 28 June. An extraordinary session on the PMR wiil be held at the European Council on13 September, arranged by French Health Minister. In the UK, a meeting is being sought with the Life Sciences Minister and appointments have already been made with the UK Research Attache and Health Attache. ‘Ambassadors’ are working in Germany and Italy on similar lines.


A public consultation on the PMR will be launched by the Commission in the autumn, in which we have been invited to participate.



What part can you play?



When we have a date for the debate to be held in the Parliament, we will be asking supporters to mobilise their networks in a letter writing campaign to MEPs.


If you want to get involved as am ‘ambassador’ in your country, contact


A Note on BREXIT


The ‘leave’ vote in the recent referendum in the UK is a matter of great concern for those who wish to see collaboration on cancer research in Europe and to use its democratic institutions to this end. However, article 50 has not been invoked and even if Britain does begin withdrawal from the European Union, this process is likely to take many years. Given the level of uncertainty, British MEP, Glenis Wilmott, is allowing French and Italian representatives take the lead, though she is still fully engaged in the process.


Unite2Cure is, therefore, continuing with ‘business as usual’ and is optimistic of the results we can achieve through 2016 and 2017. We have everything to play for. The continued collaboration of supporters within the EU and beyond will be greatly appreciated.




A Response from the Commissioner to the Golden Letters

In May, the Unite2Cure team headed to Brussels to meet with the Health Commisioner Vytenis Andriukaitis. Patricia Blanc, Chris Copland, and Anne Goeres, our delegates from Unite2cure informed the Health Commissioner as to the importance of the Paediatric Regulation, explained where problems have been identified with this regulation in relation to childhood cancer and why change is necessary .

This very productive meeting was also the opportunity to give the Commissioner 200 golden letters from parents and organizations across Europe: together we are stronger!
Yesterday, we received the following letter by post, in reply from the Commissioner.

Unite2Cure Meets with the EU Health Commissioner

UNITE2CURE at the European Commission to meet with Health Commisioner Vytenis Andriukaitis: Changes are needed to  the Pediatric Medecine Regulation

Patricia Blanc, Chris Copland, Anne Goeres, our delegates from Unite2cure informed the Health Commissioner as to the importance of the Paediatric Regulation, explained where problems have been identified with this regulation in relation to Childhood cancer and why change is necessary .
This very productive meeting was also the opportunity to give the Commissioner 200 golden letters from parents and organizations across Europe: together we are stronger!
We also had a working session on the PMR and next steps with the other participants of the delegation:
Pr Gilles Vassal and Pr Pamela Kearns, Olga  Kozhaeva from SIOPE, Catherine Guignard from CRUK , MEP Glenis Willmott, and her parliamentary assistant Emily Hunter and Remy Petitot parliamentary assistant of MEP Françoise Grossetête.
More information to follow.


Unite2Cure at the Childhood Cancer International European Conference


An excellent three-day event in Belgrade organized by Childhood Cancer International (CCI) Europe ended on a positive and motivated note for the several Unite2Cure members who attended. The general mood was that the message had been understood: the time for action is now!

The Unite2Cure movement was presented in a one hour interactive session by Chris Copland, Anne Goeres and Nicole Scobie.

“We wanted them to really hear what we were saying, really understand the message.” explains Nicole Scobie, from Zoé4life, Switzerland. “It was an early morning session after a late night out the evening before, and we knew people would be tired. We felt if we started out with a long power point on this difficult subject (paediatric medicines regulations), we would lose the audience. We decided to use humour and small working groups to get people talking and thinking about these issues.”

“Not everyone in the audience is a native English speaker,” adds Anne Goeres, from Fondatioun Kriibskrank Kanner, Luxembourg. “We realized that we needed to make sure we used simple, clear terms that everyone could understand.”

When Chris Copland, a teacher from the UK and Unite2Cure founding member began talking to the attentive audience about “loopholes” in the law, Anne quickly broke in to point out that she did not know this term. Chris was ready with his cable, to actually build a real “loophole” and show everyone how there is a hole in the law which allows pharmaceutical companies to avoid their obligations regarding child cancer.

Although the presentation was deliberately entertaining, the message was crystal clear:  we must take action now if we are to have our voice heard. The review of the Paediatric Medicines Regulation is due in 2017 – this means we must act now.

Anne, Chris and Nicole ended their presentation with a call for action:

-become a Unite2Cure ambassador in your country

-mobilize your MEP and your minister of Health

-sign a “golden letter” to the commissioner of Health to the European Union.


The golden letters had been prepared ahead of time, with a space for each participant to add in his or her own message within Unite2Cure’s call for change to the PMR. Each letter was then sealed in a golden envelope, which were collected and will be presented to the Commissioner at Unite2Cure’s meeting with him in May.

If you would like to send a “golden letter”, please click here, download the letter, fill in your information, and email it to:

If you would like more information on becoming a Unite2Cure ambassador in your country, or in supporting the movement, send us an email at





Time for Change

There have been many incredible advances in the treatment of adult cancers in the past 10 years, but childhood cancer is losing out for many reasons, including the opportunity for industry to apply for waivers in the drug development process. Investigating new agents in children and adolescents is particularly difficult as their numbers are few, studies are costly and outcomes are not guaranteed. This leads to long delays for companies who answer to shareholders, and so sadly a waiver to avoid having to test efficacy in children is the attractive option for many. The bottom line is that children are often sidestepped in the drug development process because of financial challenges.

It is a well-known fact that most children diagnosed with cancer are treated with chemotherapy drugs which are over 30 years old. If we continue to do this, then current survival rates for the most resistant cancers are unlikely to change, the prospect for cure at relapse remains low, and the toxicities (both acute and long-term) will remain unchanged. By ignoring these issues, we are failing our children. Cancer is the number one cause for death by disease in children and adolescents, but in the adult world it is no longer recognised as the automatic death sentence that it once was. Childhood cancer is genetically a much simpler disease, with lower molecular mutation rates. Children and adolescents also have substantially less co-morbidities given that they have exposed their bodies to fewer toxins in their short lives. All these elements point towards the fact that childhood cancer should be addressed in a much more positive and productive way, which is likely to lead to much greater curative rates than are currently being achieved.

Developing new drugs specifically for children will address many of these issues, but another key element to consider is prioritisation. Although 35,000 children and adolescents are diagnosed with cancer each year in Europe, when this number is attributed to individual diseases and disease sub-categories, the numbers of children and young people in each classification are few. This makes prioritising drug development another key issue, as only the most promising compounds and technologies should be brought to the fore for testing when it comes to these very small populations. Children are precious, and should be treated as such in every regard, including childhood cancer research.

Many parents and charity advocates are extremely knowledgeable on this topic, and have spent years campaigning to bring these issues to light. Unite2Cure is uniting these highly informed people and groups to gather momentum to bring one voice to the European Commission in asking for change to regulation to address these core issues. We recognise that difficulties exist for other stakeholders, but we believe many of these can be overcome by making small but strategic changes to the European Paediatric Medicines Regulation.

Parents who have lost their children to cancer, parents who live in fear of relapse and nurse their children through long term side effects, and charities supporting these desperate families all recognise the need for change and see an opportunity for it to happen. Academics understand the limitations of the current regulation and research landscape. Paediatric oncologists state that they don’t want to be the generation who simply administered drugs to maintain this current curative plateau. Surely those highly skilled, highly paid individuals working within drug development grasp these issues and are keen to change the future of many children diagnosed with this devastating disease?

If only it was considered a good return on investment, then this might be the case.

Show your support by signing our petition.

Leona Knox, Unite2Cure

A call for concrete action

Unite2Cure is delighted to be a partner in a multi-stakeholder call for concrete action to improve the availability of effective innovative cancer medicines for children and adolescents with cancer.  The call was launched at the event ‘Development of Paediatric Cancer Medicines – Speeding up Innovation, Saving Lives’. Although cancer remains the first cause of death by disease beyond one year of age; very few new medicines reach children and adolescents with cancer in Europe, even after the implementation of the 2007 EU Paediatric Medicine Regulation.

The proposals were presented at a yearly event by The European Society for Paediactric Oncology (SIOPE)  to mark International Childhood Cancer Day (15th February 2016).  The aim of the proposals is to suggest solutions to the current lack of drug development for children and teenagers with cancer.

©SIOPE & Joke Emmerechts photography

Chris Copland, one of Unite2Cure’s founding members, and Consumer Representative, National Cancer Research Institute (UK)


We are proud that Christopher Copland (UK), one of the founding members of Unite2Cure, spoke at the event of the urgent need to accelerate drug development for these young people.  He called for more attractive rewards for the pharmaceutical industry to develop innovative drugs for children’s malignancies; following the example of the “Creating Hope Act” system in the USA.

Also there was Unite2Cure partner Anne Goeres (Lu) from the Foundation Kriibskrank Kanner.  She stressed the need to join forces with the rare diseases’ community in advocating better treatments.

©SIOPE & Joke Emmerechts photography

Anne Goeres, one of Unite2Cure’s founding members, Fondatioun Kriibskrank Kanner (Lu)



Member of the European Parliament Glenis Willmott (S&D, UK), a strenuous supporter of the causes of the childhood cancer community over the past years, hosted and introduced the event. Martin Schrappe (DE), President of SIOPE, presented the ‘SIOPE-ITCC-CDDF Multistakeholder Platform’, created in 2013 by representatives of parent/patient advocates, academia, industry and regulators. The Platform works on proposals to increase innovative drug development that are regularly presented to decision-makers during a series of Paediatric Oncology Conferences, the last of which led to three stakeholders’ joint proposals to improve the effectiveness of the Regulation’s implementation.

Unite2Cure is a parent-led movement which aims to mobilise the parent/child/NGO voice to push urgently for better treatments and better access to treatments for young people with cancer.  The  current status quo means that:

– 6,000 children and teenagers die of cancer in Europe each year

– less than 1 in 10 of children with relapsed terminal cancer has access to  innovative new treatments.

– whilst progress has been made; many high risk cancers have not seen any therapeutic improvements in the past two decades

– childhood/teen cancers are largely neglected by industry

– current legislation allows waivers; even when science suggests a potential use

– EMA figures show that basically no drugs are developed for the high risk cancers.

If you care – join our discussion; sign our petition, or talk to us   or

Time to accelerate


Last week several parents, survivors and patient advocates from Europe and the U.S. united in Brussels for the CDDF-ITCC-SIOPE 4th Paediatric Oncology Conference. The title of this year’s meeting was: Accelerating the Development of New Oncology Drugs for Children and Adolescents.

Debbie Binner of Create for Chloë presented the Unite2Cure movement along with Chris Copland, Consumer Representative, National Cancer Research Institute and Nicole Scobie of  Zoé4life. The presentation was very well received, and as Debbie explained later, the conference ended with a general sense of possibility. “Believe me the first CDDF conference was very very different to this one.  Us parents were fighting to be heard – and now there is such a sense of integration and collaboration and a real sense that we are making some progress.  Although obviously there is a long long way to go.”


A few key moments:


Patricia Blanc presents

Patricia Blanc of Imagine for Margo‘s presentation on new incentives for pharmaceutical companies to invest in paediatric research was brilliant. Her voice is powerful and the work of her Working Group on this project has moved forward with an action plan. Nancy Goodman of KidsVCancer gave an excellent and comprehensive update on the successful US Creating Hope Act regulation.

Angela Polanco of Bethany’s Wish participated actively in a round table discussion on the new incentives topic, and explained that parents want to be involved from the very beginning in the process of approving paediatric investigation plans and participating in the development of clinical trials.


Chris Copland spoke up several times, passionately advocating for change to the Paediatric Medicines Regulation. He and Anne will be speaking on behalf of Unite2Cure today, January 27th in front of the European Parliament at an event organized by SIOPE in honour of International Childhood Cancer Awareness Day. This influential awareness raising meeting entitled ‘Speeding up Innovation, Saving Lives’ will be hosted by  MEP Ms Glenis Willmott. The goal is to address the development of paediatric cancer medicines and an enabling legislative and regulatory environment. In plain words: we are asking for very specific changes to the regulations NOW.


Leona Knox, of Solving Kids Cancer-Europe spoke with a powerful message, summarizing her feelings about the 2 day conference. “I am one of the parents here who won’t be going home to my son tonight. Oscar died, not because he had cancer, but because he ran out of treatment options. We parents have come here on our own time, and have worked so hard to make sure we understand what you have presented.” She told the audience of mostly pharmaceutical company representatives, pediatric oncologists and regulators. “Our goal is to save kids’ lives. We are working so hard to come up with solutions, and I just want to ask you all, to please help us fix this, whatever it takes.”

Both Samantha Schoolar of Bethany’s Wish and Jaap den Hartogh of VOKK spoke very clearly about the long term side effects survivors of childhood cancer experience and the need to speed up better treatments as well as research into late effects. “A real cure means being part of society once more,” explained Jaap, who called for new, better, and less toxic medicines as well as access to long-term follow up care for every survivor in Europe.


It was inspiring to hear Peter Adamson, the Chair of the Children’s Oncology Group stand up to remind the audience that the title of this conference was “Accelerating the Development of New Oncology Drugs for Children and Adolescents”, and that, having attended these conferences for the past several years, he did not want to just have the same conversations as every year. It was time to actually accelerate.


Gilles Vassal, past president of SIOPE and Director of Clinical and Translational Research at the Institut de Cancérologie Gustave-Roussy made a clear call to “change the mindset“.


Change the mindset!

The summary of the decisions and plans as a result of the conference are, briefly:

-a strengthening of the  CDDF-ENCCA – ITCC-SIOPE platform with terms of reference, a website and a new name (to be announced!).

-position papers will be created by Working Group 1 on the issues of adolescent inclusion in adult phase one trials and on mechanism of action as a determinant for paediatric investigation plan requirements.

-a position paper is being drafted on Working Group 2’s proposals for new incentives

-a white paper will be put together by Working Group 3 regarding long term follow up for children receiving new drugs. This group met immediately after the conference to continue preparing this document.

Gilles Vassal emphasized that parents and survivors must continue to advocate for change for all of these plans to happen.

He ended the conference with one sentence, that summarizes the event, “We are even stronger if we work together.”

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The view over Brussels on the flight home





A response to our article

On November 23rd, Unite2Cure posted an article entitled We need to move the lines, which was our message to the European Commission. Written by Chris Copland on behalf of Unite2Cure, the letter was addressed to Ms Juelicher, who is Head of Unit, Medicinal Products – Authorisations, European Medicines Agency, as well as to Commissioner Andriukaitis, Commissioner for Health and Food Safety, European Commission.

In it we describe how we found the Commission’s position, that the concerns raised by Mrs Wilmott and Mme Grossetete during the  ‘Exchange of Views with the European Commission and EMA on the Policy on the Conditions for a Paediatric Investigation Plan/Waiver.’  on November 10, should be put aside until 2017, a sadly complacent one.

On December 17th we received a reply. Unfortunately, it does not address our concerns but is simply a copy of a letter sent to Mrs Wilmott and Mme Grossetete. We are disappointed in this response.

Response from Ms. Jülicher

Reply to Ms Grossetête and Ms Willmott,

We have replied to their letter as follows and hope to hear more soon.


Dear Ms Julicher

Thank you for your response to our message. We were, however, disappointed that you simply referred to your open letter to Ms Wilmott, MEP and did not provide us with a direct reply.  Although the letter was on associated themes, it did not address specific points we had made, for example about orphan illnesses. We, however, would like to respond here to the points you made, in particular your assessment of whether the Paediatric Regulation is functioning as effectively as it might.

As you point out, what makes the Paediatric Regulation different is that it does not just offer incentives, it places a requirement on companies to conduct studies into children. The effectiveness of this approach has been demonstrated in recent years but, unfortunately, it has not achieved its potential with regard to the most deadly of young people’s illnesses cancer.

You assert that the Regulation allows companies to propose paediatric plans for cancer on their own initiative, and it is true that on occasion this has happened. However, the track record, over decades, of companies voluntarily engaging with paediatric research is not an encouraging one (see Raymond and Herold). On the other hand, their willingness to avoid requirements by resorting to the loophole of the waiver system has been clearly demonstrated. As has been frequently related, during the first five years of the Regulation, over half of the adult drugs with a significant potential for children’s cancers had waivers requested and granted (see Vassal below).

You paint a positive picture by referring to the recent statistics supplied to you by the EMA. As we have not seen this data, we cannot respond to your interpretation in detail. However, we note that you place emphasis on the number of programmes that ‘target a condition where most patients are in the paediatric age range.’ You do not clarify whether, in these cases, a PIP has proceeded because there is a common condition for both adult and child or because companies have simply opted not to apply for a waiver. Either way, this ignores the simple fact that ‘’PDCO has to grant a waiver if the disease only occurs in adults.’ (EMA)

This is the central weakness of the legislation with regard to cancer, a disease which manifests itself differently in adults and children but still derives from a common biology. As Gilles Vassal has reported, 90% of the drugs successfully given to young people with cancer over the past 40 years have been used with different tumour types in adults. Ultimately, the legislation can only be made consistent and genuinely productive by basing the requirement to produce a Paediatric Investigation Plan on the biology that underlies adult cancers rather than simply the tumour type.

We acknowledge that some companies may conduct a PIP they are not required to and which may involve pursuing the mechanism of action. This may be the result of altruism or, more likely, because of the incentives the legislation offers. However, this is unlikely to generate research in anything more than a haphazard fashion. As parents, we expect industry to conduct paediatric research systematically and as a matter of course – our children deserve no less. This is why the Paediatric Regulation’s emphasis on requirements, which you yourself accept is what makes it different, is so important. The point is such obligations must be applied consistently and correspond with our current understanding of biological science.

It is of course heartening to read of the oncology PIPs currently proceeding, but, after ten years, these are still in relatively small numbers when one considers the scale of the problem to be overcome – 6,000 deaths of young people in Europe every year from cancer.

The urgency of the situation is distilled in the words of one parent:

‘It is inconceivable in this day and age that, globally, there is no drug that can cure my daughter’

This is the view of the nearly 1500 people who have signed our petition and of the many prominent professionals and associations (SIOPE included) that have come out in our support. If one reads the literature, there is a clear consensus in the professional community of the changes that are necessary to put the regulation on track and of the urgency of doing so. As members of the CDDF ITCC ENCCA SIOPE Paediatric Platform concluded at our conference this time last year: Waiting is not an option.

We look forward to a productive engagement with ENVI in the New Year. In the meantime, may we wish you and your staff a Happy Christmas.


Christopher Copland

on behalf of Unite2Cure