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Buddhini: Hi everybody and thanks for joining a second cancer research UK & some Google plus hangout on air. This time are talking about childhood cancer and joining me is my co-host Dr Kat Arney. Pam and Richard, please could you introduce yourselves.

Pam: I’m Pam Kearns, I’m a paediatric oncologist based at Birmingham Children’s Hospital. I’m also the director of the Cancer research UK clinical trials unit at the University of Birmingham, and my interest is in developing new treatments for children with cancer through clinical trials.

Richard: My name is Richard Gilbertson. I’m based at Cambridge University where I’m director of the cancer centre and the chair of oncology. I just started five weeks ago and for 15 years I was in the States and was scientific director of the St Jude’s Children’s Hospital, which is large cancer hospital in the US.

Buddhini: What’s the difference between children’s and teens cancers and adult cancers? Are they different types of cancer? And do we need a different approach to researching and treating them?

Richard: That’s a great question. The simple answer is they’re very different, and at one level even the general public would recognise that children and adults get very different types of cancers. Whereas adults tend to get breast, stomach, pancreatic, colon cancer – those sorts of cancers don’t occur or are exceptionally rare in children. The kind of cancers that we get in children are commonly of the blood and the brain and other tissues that we think relate largely to things that go wrong in development, particularly how the cells would normally form organs go wrong. So a lot of cancers in children look like developing tissues that have just overgrown, but they are cancers just the way that adults have cancer and they need serious treatments.

The biology is very different between adult and paediatric cancers, and we think the underlying causes for those diseases are probably different as well, and we can talk more about that in detail if people would like to. I think in terms of treating, obviously they’re extremely different. The youngest age group – most of those children now are treated in children’s hospitals as part of special children’s cancer units, and that’s incredibly important to facilitate the research and the care of the broader family as well as the child with cancer. The group the panel will be able to talk more about as a practising oncologist is the young adults and teens group – the group that are in the middle between adulthood and childhood. There are particularly special group that we have to look after particularly carefully with cancer, not just the need for their treatment in terms of the disease but also the psychosocial needs. They’re going through major changes as they’re growing up to adulthood.

Kat: We’re going to come to that area a little bit later. Pam, can you explain a bit about how far we’ve come in terms of treating childhood cancers, in terms of the progress we’ve made so far and is this the same across all types of cancer that affect children and young adults?

Pam: That’s another great question. The often quoted statistic about childhood cancers is that we can cure 80 per cent of children with cancer, and we’ve described cure rate is the number of children who are still alive following diagnosis at five years, is a statistic that is used in clinical research. And that’s a fantastic statistic – if you think that when I was a child (which is a while ago) cancer was an incurable disease, basically. Over the last few decades we’ve made massive strides to get to this average number of eight out of ten children being cured. But what that number hides is two really important facts. One is that if you do the calculation that still means 300 children a year in this country don’t survive cancer, and that’s still the commonest cause of death from disease in children. And the second thing is that it’s a spectrum. As Richard said it’s not just one disease in children, it’s a lot of different diseases, more common in different age groups in the different parts of the body. For some of those diseases, a type of leukaemia called acute lymphoblastic leukaemia for example, we have very, very good treatment and survival, with cure rates well into 90 per cent. But there are other diseases – and importantly a large group of brain tumours and the bone tumours, Ewing’s sarcoma and osteosarcoma – where we haven’t made the progress we really need to. They are diseases that we are still struggling to develop new treatments – we know the drugs we’ve got at the moment aren’t giving us the cure rate is that we need.

Overall, even the groups of patients that do extremely well, that we can cure, children have to live a long time with the side effects of treatment that we give them, and so we need to be much better and making the treatments we give them now not have an impact on them in the future, the side-effects – for example the effects of their fertility, the effects on particular organs like the heart of the kidneys – can carry on for many years into the future, so we need to make the drugs better and kinder on the body, without losing their effect on the cancer. But we also need to develop new treatments for those that just aren’t responding to the drugs we got the moment.

Buddhini: That brings us very well to the next question I wanted to ask you, which is what are the big research challenges moment in developing these kinds of treatments? As you just said, the side-effects can be extremely debilitating for patients at such a young age.

Richard: I think in terms of the biology, actually paediatric cancer is quite a success story. We have made some considerable inroads in understanding the biology of children’s brain tumours and children’s leukaemia, and even some of the solid tumours that Pam described that are very tough to treat. But there are some very significant challenges in taking that understanding of those diseases into the clinic and making an impact on patients. Some of that is due to the rarity of children’s cancer – although as Pam pointed out it’s the commonest cause of disease-related death in children, it’s still a relatively uncommon condition which means that the populations of patients that are available to treat are quite small. And as we understand the biology better I think we’re poised more than ever to understand the biology of cancer better children, we take what was once regarded as a brain tumour or solid tumour, or leukaemia and divided into smaller subgroups that we now have particular mutations that are driving the disease – that’s great because we understand the disease better, but what it means is that we have an even smaller pool of patients.

That’s fine but the challenge for us now means that rather than doing local studies or national studies we have to start thinking about doing international studies. How do we collaborate better with the US, with the wider European community to make sure we’re doing these clinical trials on patients? So there’s that aspect in terms of the challenge of biology. But actually I think that paediatric cancer is a success story in terms of understanding cancer biology – I know we’re going to talk a little bit about this later, but we can actually learn a lot about insights into adult cancers studying childhood cancers.

Kat: Pam can you talk a little bit about the challenges of the research to try and find kinder treatments – how do we go about doing that to try and reduce the long-term side-effects that you talked about?

Pam: Any change in treatment has to be based on evidence and that’s why we do clinical trials. And a clinical trial is where we ask patients to participate in what is effectively an experiment, where we’re trying to introduce new treatments. There’s broadly two types of trials we do. This trials where we compare the current gold standard and we try and introduce a new treatment, and the question we’re asking is ‘is this new treatment better or kinder’, and there’s a whole class of other trials where we’re trying to bring in really novel innovative treatment, and that’s where we’re saying is this drug safe and does it work? And they are smaller trials. To examine kinder treatments we need to do these randomised trials were comparing the new treatment against the current gold standard, and the absolute crucial point is that if it’s kinder, it can’t be less effective. So the trial that you run has to answer the question is it as good, is this new treatment going to be at least as good as the old treatment, and ultimately is it kinder? These trials are quite difficult because you need quite large numbers to show that difference, and as Richard commented childhood cancer is rare. We have 1,500 new cases overall of childhood cancer in this country every year, and if you try and run a clinical trial to answer these questions you’re usually looking at comparing hundreds of people patients in each arm. So the only way we can do the studies is to work internationally, that has complexities just from the logistics of running clinical trials across national borders and the financial limitations that go along with it. If you sit down with a piece of paper work and out how to do it, it’s easy, but when you try and deliver it in practice there’s an enormous number of hurdles that we have to deal with.

Kat: I was going to ask, what’s the current picture with UK trials in children and teens – because we’ve done really well, we’ve had things like the children’s Cancer and Leukaemia Group, now the Children’s Cancer Trials Team – roughly how many trials have we got running in the UK and with your international collaborators?

Pam: Thank you for mentioning the Children’s Cancer Trials Team – this is a team that sits within our trials unit, and this is a unique team it runs the national portfolio of all the clinical trials that are run by academics. I’m not talking about trials that are run by the pharmaceutical industry. We have oversight of the whole portfolio of trials. The absolute number is that we have 14 trials open with patients going into them at the moment, with another 10 or 12 trials were following up in the long term, so no longer getting new patients we’re collecting the data going on, and then we have currently 11 trials that are in the setup phase, where we’ve got the scientific approval for them, we’ve got the funding to run them, and were going through all the regulatory processes to get the trials up and running, and then behind that there are at least six grant applications being prepared, and another dozen trials that we’re developing and designing. To put that into a more understandable perspective, today when a child is diagnosed there’s an open trial for somewhere between 65 to 70 per cent of children newly diagnosed with cancer.  So when they’re in a treatment centre the doctor would say would you like to participate in this clinical trial? And extraordinarily – and it is a testament to the altruism of children and families – we have a hugely high recruitment rate. Somewhere around about 80 per cent will agree to going into the trials – a huge number. And if you put that into context of adult cancers where it’s considerably less than one in five going to a clinical trial, where at the other end of the spectrum. But what I’m very excited about is that our portfolio of trials also includes a set of trials that are for patients not diagnosis but who have failed treatment, where it’s come back, and currently there is no known curative treatment. And were trying to develop new innovative treatments and we have a portfolio of these new trials the children disease that is difficult to treat where we’re going to develop the next generation of treatments. And that something that as a unit we’ve made great progress over the last five years.

Buddhini: Richard you mentioned something about how we fit in internationally, now that there’s an increasing need to do more international collaboration. Where does the UK fit in and what are the logistics of doing international clinical trials are child cancers?

Richard: I think the UK’s always had a leadership role in the international scene in paediatric cancer and that’s not change. When you go back through the history of leukaemia studies, many of which were driven by the MRC in the UK, that has always been the case. And as someone who spent the last 15 years of the US there’s a real respect for the science and the clinicians within the UK. I think increasingly also there is a leadership role on the national scale and international scale. So as Pam has alluded to and I described earlier, because of the rarity of paediatric cancer were going to have to look beyond our borders. Every country is going have to do that. In the US we realised that with the rarest of populations of children with cancer we simply can’t complete a clinical trial in a time that is acceptable just by operating with our country. That presents huge challenge. It is an enormous challenge as palms already said to work across borders with the medical regulatory bodies et cetera but we’re going to do it. That’s the bottom line. There’s a huge amount of will to do it, and has to be done to save the lives of children and there’s no question that that will happen.

I think the UK is particularly well positioned because of the expertise within its universities. One of the reasons I came back here is that the UK has some of the best seats of learning, quite honestly, in the world – some of the best scientists in the world. I spent last Friday with a bunch of engineers, and all they do is tune light to pick up different proteins in cells, and they were all round the table thinking about childhood cancer. That’s an amazing opportunity that you just don’t see anywhere else in the world. I think the UK has an innovative aspect and an imaginative aspect of science that will really bring forward ideas that will really have an impact on the lives of children in the future.

Kat: You’ve just come from St Jude’s in the US, are you going to be maintaining those links? It would be nice to think about the research collaborations, what can we learn from the colleagues you’ve left behind in the US?

Richard: Absolutely. I have two collaborative grants in the US, one of them with St Jude’s and another with another college particularly to develop drugs for paediatric brain tumours. I think in terms of teaching the adult community something, these aren’t just taking drugs that are being used for other purposes, actually one of the studies is about completely new compounds, chemicals that have never been used as drugs before that will now be used to treat a particularly rare type of brain tumour called choroid plexus carcinoma that occurs in babies that is a very difficult disease to treat, so we have new opportunities to develop brand-new drugs. They themselves may be useful in adult cancer, so paediatric cancer has a lot to teach adult cancer, both about the biology and the way we treat it as well.

Kat: Moving on from very young children with cancer, Pam could you tell us about some of the challenges of working with teens and young adults with cancer, some of their particular needs?

Pam: That’s an enormous subject. The particular problems that teenagers face, the types of cancers they get often go across quite a broad spectrum, and so they’re either sitting at the older end of the paediatric type of cancer, or at the younger end of an adult cancer. And then they have some cancers for which they are the biggest age group for developing them, so in particular the bone tumours like Ewing sarcoma and osteosarcoma. They are small numbers overall.

The trials that we do at the moment, there’s been a traditional split – for no genuinely logical reason to be honest – that trials finish at 16 or 18 because that’s a chilhood trial, and adult trials go down to age 18 because then you are an adult, and never crossover in between. And that has changed, now that is changing, and we have to keep pushing that change. So for example we were on trials now to Ewing sarcoma where they are international – and international isn’t just transatlantic, we have a massive European network, we work very closely with our European colleagues, even with the language barrier we are able to work incredibly well running these trials – so we run a Ewing sarcoma trial where the age goes up to the full spectrum of when Ewing sarcoma will present. Young children, teenagers into young adults and older adulthood. And that’s how we do all the trials now. So we can do that now, will do the research project, will develop the new treatments across the age group.

But then you get the practical delivery, and Richard alluded to this before. At what age is a child no longer sitting in the Children’s Hospital and needs to go into an adult hospital? And the Teenage Cancer Trust have done an enormous amount of good work in developing teenage cancer units, but they vary in models. I have to say talking to my European colleagues and I’d be interested to know what happens in the US, we have advanced this more than any other country I know where we have these units embedded either at the top end of the Children’s Hospital, or an adult hospital and in some trusts where they take both age groups. But it still requires expert staffing, it’s not good enough to just have a paediatric team going into a teenage unit and then looking after a 24-year-old and vice versa, an adult team coming in are looking after 16-year-olds. It’s a whole new specialty to address the needs of what it’s like to be a teenager diagnosed with cancer at a time which is difficult to the average teenager to cope with, with all the things that change around you as a teenager and your growing independence, and then you have a big disease with a life-threatening diagnosis and you have to deal with that on top of it. All the psychosocial needs, development needs, the educational needs need to be tailored to age group, and we need to do that alongside making sure they have access to treatment and there’s no artificial age barrier that’s stopping them to get into a trial. I work with a number of patient and parent groups and there’s some particular parents that talk to me about their child that didn’t survive cancer and they were frustrated because trials were open in adult units for the disease their child had, but because the child was 17 and a half and the age of going into the trial was 18 they weren’t allowed to get access to those drugs, and we have to stop that happening in the future because there is no difference between a 17 and a half-year-old and an 18 and a half-year-old pharmacologically, so there’s no reason why that can’t be given to them. So we’re doing a lot of work with all the relevant stakeholders, parents, the regulators, pharmaceutical companies to stop this happening. So let’s make sure that a new drug that is relevant to disease in young people let’s make sure that it’s accessible to young people.

Buddhini: Absolutely, bureaucracy like that shouldn’t stand in the way of saving lives like that. That’s such a horrible story. And as if puberty wasn’t bad enough already! One of the things that we asked early on in the Hangout is what are the differences between childhood cancers and adult cancers, and Richard you said they’re very different. But what can we learn from those differences between kids cancers, teenage cancers and adult cancers? Can we apply those learnings across the different cancers?

Richard: Yes you can. So basically if we think about cancer, although cancer arises in a very different context or tissue types between adults and children – as I mentioned where adults get breast, pancreatic, stomach cancer, kiddies get leukaemia, brain tumours, neuroblastoma which is tumour of the peripheral nerves in kiddies, we see that happening. But the things that go wrong in cells that drive cancer, the processes that control cell growth, cell death, how a cell grows up and does the job it’s supposed to – those things quite commonly go wrong in both adult and childhood cancer. So if we understand the biology of how a cell should divide, and that might be an insight we get from a child’s cancer, it can and has been applied to adults.

There is a very specific area that is particularly exciting where were learning from adults to children, and that is in repurposing drugs. So technological advances often drive new changes in the way we treat cancer. One particular technological advance that’s happening in most recent years is the ability to do very high throughput drug screens – that’s taking cell populations, the cells that come from a child’s cancer and literally test them against millions of compounds. With done that at St Jude’s, that’s happening at other places in the UK, and we can include that all the drugs we use to treat adult cancer – about 300 cancer drugs that are on the market. Now that would be completely possible to do clinical trials – because of the rarity of childhood cancer – with all of those drugs in every single childhood cancer, but we can test whether childhood cancers are sensitive to chemotherapies that are currently used in adults. My own lab did this recently to take a drug that is very commonly used in breast cancer, we repurposed it and now using it to treat childhood brain tumour. We would never have selected that before.

In terms of biology we can take very real insights into the basic aspects of developing tissues, how that goes wrong to make a childhood cancer. And that will often unpick and identify biological processes that are going wrong to control tissues that actually also occur in adulthood. Now adult cancers tend have many more genetic alterations than we see in children’s cancers, so often there’s a lot more noise going on in an adult cancer. So if you can see a much clearer, sharper picture of what’s going on childhood cancer, you can then tune in and look at that in adult cancers. There are examples of where that’s been done.

Pam: If I could just come back on that as well, I think that kind of work is very, very informative, and as you say it helps us have a better idea of selecting which drugs we should be taking into the clinic, but there is a practical problem that we’re facing at the moment and that is being able to access the drugs that are being developed by industry, mostly, into paediatric trials, and problem is the rarity of childhood cancer. If a company is developing a drug for disease like breast cancer or lung cancer, in fact most adult cancers, it has a market. If it proves it works it has a market and it will get the financial benefit from all the investment – and it is a huge investment in developing a drug. In childhood cancer we’ve been talking about the numbers and how small they are, and so stacking up it’s not a profitable way to do things.

In Europe and in the US there are rules now, regulations that have been called into place – in Europe it’s for the paediatric regulation – to mandate drug companies to do studies in children, so we don’t have the situation of drugs that have been developed in the adult market and then we just have to try them out as best we can. That they are formally part of the development plan. But at the moment it’s not translating into getting lots of drugs into the clinic for children because there are still ways of getting round it. If you have a drug that is been developed for lung cancer and you want to license it, you can say lung cancer doesn’t happen in children. And under the regulations you don’t need to do the children’s trials. But Richard very clearly stated that the mechanisms that cause the cancer – the lung cancer – could easily be applicable in paediatric cancers and we have some good examples of this, where the target the drug has been developed to hit in the lung cancer is also relevant for paediatric cancer. So we’re working very hard with all the people involved, the regulators and industry, to say can we find a way that will mean that you will allow us access to a drug on the basis of its mechanism and not the disease it was developed for. And if we can get that change in attitude and a change in the pathway, there we’ll have a much more intelligent way of developing new drugs for children. I can see progress already in that direction and I would expect over the next five years that we’re going to be in a position of being able to look at the massive portfolio of drugs that are coming out of industry at the moment and say yes now we can have access to those and do much cleverer interactive trials that are targeting the patients not just diseases.

Kat: I wanted to mention as well the cancer treatment isn’t just about drugs – we don’t have time to go into the ins and outs of different types of radiotherapy. We are doing trials of things like more precise radiotherapy in the UK as well?

Richard: Absolutely. It’s a full spectrum including surgery as well – we mustn’t forget surgery which has a major role to play paediatric cancers. What’s exciting among the paediatric community and I think Pam alluded to this very well, is that the paediatric community – partly because of the rarity of the diseases – forces us to work together. So we have a very long history of close interactions between different disciplines – surgeons, radiotherapists, oncologists, nurses and pathologists – forming very tight groups of people who work together closely every day not just to deliver care but also to do the research that will deliver new treatments for the future across the spectrum of disciplines.

Kat: Maybe each one of you if you could just tell us a very brief story about a patient you’ve met that have an impact on you, maybe sums up why it’s so important to carry on with this research or perhaps even the hope that you have for the future. Richard, can you go first?

Richard: I remember lots of patients, but the one patient I remember most is the first child I ever met with cancer, before I’d even thought of doing childhood cancer as the career. She was in a bed in a hospital I was working at a medical student. We asked what was happening, and the consultant he was a fantastic consultant said, we’ve done everything we can for her, there’s nothing else we can do, we just have to let her die in peace. And that really haunted me for a long time, and I read a book later by a guy who’s called Don Pinkel who is a very famous oncologist in the states, and having had that experience with that patient I read that book and he said that he had the same experience but he said that children don’t die in peace with cancer, and it’s not acceptable to let them die in peace, and that’s really one of the things that really spurred me on to a career in childhood cancer research.

Kat: Fantastic story. Pam have you got a story like to tell us that sums things up for you?

Pam: Like Richard, you spend your whole career meeting lots of amazing families and so it’s really hard to pick out one because they will have an impact on you in one way or another, whether their child has survived or situations where they don’t survive – you carry that along with you in your career, but there are a couple of things I wanted to say. One is a bit like Richard, not my first patient with cancer but it was the last patient I saw just prior to going into the laboratory to start laboratory-based research when I started my career in research. I was in the clinic and I was talking to the family and saying ‘I won’t be seeing you in clinic next week because I’m going off to the laboratory to become a researcher’. We chatted a little bit about what I was going to do and this mum took my hand looked me straight in the eye and said, and will you be finding a cure for my child? And it was so heartfelt and it made me realise that every single piece of work we do as researchers we have to remember that it isn’t just academic interest, it is actually to make a difference to patients in the clinic. And is one other very short story and I’ve heard it several times, but it really brought it home to me in a passionate plea from a father recently whose child has a tumour that we have no known cure for her. We were talking about new drug development trials and he said he could not believe in this day and age with all the knowledge that we have that globally we do not have a cure for his child. And I thought, you know, that is just not acceptable and we really do need it, all the work we’re doing we really do need to be in a position, and I don’t how long it’s going to take but we never have to have that conversation about there’s nothing else we can do. As Richard said, it’s not good enough and we need to keep working on that.

Buddhini: I think that something that comes across very clearly in the words that the two of you said, that this is simply not acceptable, and you’re not going to put up with it. It is what it is – that’s not your attitude and I think that’s really inspiring and I hope people watching will find a similarly inspiring. I found it very inspiring to hear from the two of you like that. Thanks everyone for watching and hope you enjoy this. With regards to the next Hangout we’re doing, the topic is open for discussion, so if anyone has any cool ideas of any topic you like about let us know. We have access through Cancer research UK to some of the best researchers in the UK, so we can make that happen if you have a particular topic you’d like to learn about. That will be in two months time, so thank you for joining us and goodbye.