For the parents of children living with cancer, there is often so little hope. And for those facing the bleakest of prognoses – that, barring a miracle, the disease will cut short their young lives – the agony is almost indescribable. So the decision to enrol such a child on to a trial for largely untested treatments that may save them, but may make them suffer far more, is another kind of mental anguish altogether. Now, a ground-breaking television documentary filmed over two years follows three children and their families through such a situation. Battler: Chloe Ballouqui during her treatment. She is one of three children featured in a ground-breaking TV documentary . In the film they reveal with heroic honesty why they are willing to risk their lives as they become the first children in the world to go through a pioneering treatment called immunotherapy. Shocking as it might seem, this kind of ‘experimentation’ happens in Britain. And it is a necessary part of the process that discovers new treatments that ultimately offer hope to other young cancer patients. Sophie Ryan-Palmer, 12, Fabian Bates, nine, and Chloe Balloqui, three, are patients at Great Ormond Street Hospital, London, the leading British centre for treatment of child cancers, and are the youngsters featured in the documentary. Tragically, although for one of them, treatment is successful, two do not survive the radical procedure. Immunotherapy involves powerful drugs being given to kill off all a patient’s cancer cells and destroy their bone marrow. Loving life: Sophie Ryan-Palmer enjoying a party. The 12-year-old was two when she was diagnosed with leukaemia and entered the immunotherapy trial after her fourth relapse . This allows a transplant of new bone marrow containing immune system cells, harvested either from the patient or a donor. These cells are genetically modified in a laboratory so they are better at attacking cancer. Similar treatments have been carried out in America, and although the early results look hopeful, the risks are huge and many of the terminally ill young patients die from complications. The toxicity of the immunotherapy means children often have to be sedated and given strong painkillers such as morphine, to cope with the treatment, and many end up in intensive care. When presented with these facts, some parents decide the risks are simply to great, yet others choose to go ahead. Fabian’s father, Darrell, describes the anger and devastation after being told of his son’s diagnosis but also the optimism they had about the outcome of the trial. Chloe, pictured with her mother Karen Kallaste, has neuroblastoma . He says: ‘There were no patients who had gone through it, no evidence it would work, which was a little bit frightening. You tend to grab these things as a ray of hope.’ Some 30 children a week are diagnosed with cancer – that’s more than 1,500 a year – and 250 of them will die every year. Child leukaemia has a high survival rate, with four out of five youngsters being cured completely. But for some, standard treatment doesn’t work and the cancer comes back, which is why clinical trials such as the one for immunotherapy can be a lifeline – they offer families their last hope. Kate Law, director of clinical trials at Cancer Research UK, says they are an essential way forward. ‘There is strict regulation and a child would have undergone three or four lines of treatment such as chemotherapy before being considered for a trial,’ she explains. ‘The child has to have a life expectancy of at least six months and they have to undergo extensive clinical tests to be certain their bodies can cope with any potential treatment. ‘There is a real balance between whether we are doing more harm than good, and it is therefore essential that the doctor and the parents weigh up what their options are and whether a clinical trial is something they should pursue.’ Another parent in the documentary, Richard Balloqui, knows this dilemma all too well. His daughter Chloe has neuroblastoma, one of the most deadly of all childhood cancers, with only two in five of those diagnosed at her age surviving. As well as having a tumour on her adrenal gland, Chloe’s cancer had spread throughout her bones. During filming, the toddler has surgery to remove the tumour as well as chemotherapy so strong it could kill an adult, in preparation for the immunotherapy. At one stage her liver becomes dangerously swollen, a potential fatal side effect of the treatment. Her father admits the pain of ‘seeing your child in discomfort, seeing the side effects of what we are doing here to try to kill the cancer’. He adds: ‘You are filling them full of toxicity and you do see the effects of that, and it hits you that your child is going through something you could never imagine.’ Fabian Bates chats to the Duchess of Cambridge during a hospital visit. The child was given modified cells taken from his 14-year-old sister Cassia to battle his cancer . Sophie, the third child, was two when she was diagnosed with leukaemia and entered the immunotherapy trial after her fourth relapse. Her mother, Debbie, says: ‘When I told Sophie [the cancer] was back again, she was totally devastated and just said, ‘‘I don’t want to die, mummy, I don’t want to die.’’ ’ The youngster had two options: take drugs to fight the pain, and allow the cancer to progress to its inevitable end; or take part in the trial. ‘Straight away, without any thought about it at all, she said she wanted to go on the trial. She is a fighter,’ Debbie says. Of her relapse, Sophie says: ‘When I was told, I thought, “Not again.” But I dealt with it like I have all the other times. Of course I was upset but there is something we can do, so do it. ‘Mum said it was a clinical trial and stuff like that but I didn’t want to read the information, I just wanted to go ahead with it. It will make me better, hopefully.’ There are currently 36 child cancer medical trials open in the UK – 22 of which are funded by charity Cancer Research UK. Some 15 of these are studies into new treatments for relapse patients such as those highlighted in the documentary. Earlier this year The Mail on Sunday reported that cancer experts had hit out at EU red tape that is denying hundreds of terminally ill children cancer drugs – because a legal loophole means pharmaceutical companies have to test new treatments on adults only. If firms opt out of testing new treatments on under-18s during clinical trials, doctors are then barred from using them on youngsters because they are cleared for adult use only. Only half of 28 cancer drugs trialled between July 2007 and June 2012 were tested on children. Kate Law, of Cancer Research, says: ‘From a drug company point of view, they are in business for profit. They have to answer to their shareholders and they are therefore risk-averse to trials with children, so child cancer trials have to be funded independently through organisations like us.’ There are 21 specialist centres treating childhood cancer in the UK and every child with cancer will be seen at one of them. About 70 per cent of newly diagnosed children will be offered the chance to trial a new drug, a figure higher than that in the rest of Europe and the US. At one point during treatment, Sophie is confined to an isolation ward for eight weeks to prevent any risk of infection which her weakened immune system would be unable to fight. Her room is decorated in One Direction posters and she tells the film crew she copes with her situation due to her motto of ‘Happy, happy, happy, fun, fun, fun’. Sadly, Sophie’s tiny body was unable to withstand the first stages of the treatment, and she died in July 2013. The ITV film captures the poignant moment her pink coffin leaves the family home near Heathrow. Debbie says: ‘She always wanted to help other children so I have no regrets about her going on the trial, it was Sophie’s choice. ‘Unfortunately the bone marrow transplant never worked so she never had the chance to have the modified cells. ‘After what she had gone through for ten years of her life, her body just couldn’t do it any more. She was such a brave little girl and I was proud to be called her mummy’. Like Sophie, Fabian, who received letters from the Duchess of Cambridge after meeting her in hospital in 2011, was determined to look to the future and was given modified cells taken from his 14-year-old sister Cassia. He says: ‘The good cells fight the bad cells and destroy them, and as soon as they are destroyed I won’t be ill any more, which is a good thing because I want to stay well, not in a coffin and then, like dead. That would be horrible.’ Fabian dreamed of one day having a wife, a house and a car. Tragically, his new cells stopped working and he died at his home in London last November. The Duchess’s letters were laid to rest with him. Despite these tragedies, clinical trials have transformed outcomes for cancer patients and particularly in children, as three-quarters of those with cancer are now cured compared with about a quarter in the late 1960s. For Chloe, the fight goes on. Parents Karen and Richard found an American drug trial which aimed to prevent neuroblastoma coming back after immunotherapy. In a whirlwind six-week campaign, an appeal raised more than £100,000 to pay for further experimental treatment scheduled to begin early this year. Cruelly, final tests before travelling to the US showed that Chloe’s cancer had returned, although not in her bones this time, indicating the relapse had fortunately been caught early. Specialists at Great Ormond Street are now discussing the best ways to get the disease back into remission. Richard says: ‘We remain positive that this has been caught early and we can treat it.’
Ground-breaking TV documentary followed three children in medical trials.
They were the first in world to have pioneering treatment immunotherapy.
It uses powerful drugs to kill off cancer cells and destroy bone marrow.
Toxicity of immunotherapy means children often need to be sedated.
In the film, the children's parents reveal why they decided to take part.
The documentary, Raining In My Heart, will be shown on ITV on April 15.
Raining In My Heart will be shown on ITV on April 15.