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Neuroblastoma (Monoclonal Antibody Therapy)

Volume 502: debated on Wednesday 16 December 2009

I thank Mr. Speaker for granting me this debate so quickly. Not many people know about this very nasty form of cancer, which affects a small number of children in this country each year. I welcome the hon. Member for Brentford and Isleworth (Ann Keen) to the Chamber as the Minister who will respond to this debate. We are very fortunate to have her here because she is renowned throughout the House for her compassion, genuineness and her ability to get things done behind the scenes. I will be asking her to employ all those qualities to solve a difficult problem and to give hope to a little boy called Zac.

Zac is a constituent of mine who suffers from a rare form of cancer called neuroblastoma. He is five years old and was diagnosed with stage 4 acute neuroblastoma in February this year. Doctors discovered a 10 cm tumour in his stomach, and smaller growths on his thigh and pelvis. Such news would have been devastating to any family, but it was made all the worse by the fact that Zac’s three-year-old cousin Chelsea had been diagnosed with the same horrible cancer 18 months earlier, when she was just two years old.

Sadly, after more than two years of bravely battling the cancer, Chelsea passed away on 9 August 2009, just one month before her fourth birthday. The treatment that Chelsea received in the UK gave her a 20 to 30 per cent. chance of survival. Treatment in America would have given her a 70 per cent. chance of survival, but it would have cost her family a quarter of a million pounds.

Michelle Tomkins, Chelsea’s aunt told me:

“Chelsea passed away because we did not have enough time to raise the money to get her to a hospital in New York offering a treatment that gives a 70 per cent. chance of survival, whereas here in the United Kingdom, it is only 30 per cent. The UK could do no more for Chelsea and this was her only hope, but we struggled so much. I can’t bear seeing them having to go through this all over again. A child’s life should not have a price on it.”

I want to emphasise two clear points that Mrs. Tomkins made. She said that the UK could do no more, and that a child’s life should not have a price on it. In this day and age, such statements should never have to be said, but sadly both are true.

Neuroblastoma is a rare cancer of the sympathetic nervous system—a nerve network that carries messages from the brain throughout the body. It is usually found in young children, and it is the most common cancer among infants. The solid tumours, which take the form of a lump or mass, may begin in nerve tissues in the neck, chest, abdomen, pelvis, or, most commonly, in the adrenal gland. They may also spread to other areas of the body, including bone and bone marrow.

The disease affects about 100 children in the UK. Fewer than 30 cases are diagnosed each year, with the majority of cases affecting children aged one to four. Neuroblastoma is the second most common solid tumour in childhood, and makes up 8 per cent. of the total number of children’s cancers. However, the odds on having two cases of neuroblastoma in the same family are one in 10 million, and sadly that has happened to Chelsea and Zac.

Detecting the cancer can be difficult. The first signs are that the child becomes tired and loses their appetite. The symptoms also depend on where the disease is in the body. For example, if the tumour is in the abdomen, the belly will swell. However, because many of the symptoms can be seen as symptoms of less serious illnesses, diagnosing it can come at a late stage when the tumour has spread to other parts of the body.

There are different ways of treating neuroblastoma, but prognosis is dependent on age, stage of disease, and the molecular biologic and cytogenetic characteristics of the tumour. Surgery is a way of curing the disease, but it is only possible if the tumour has not spread, and is not in a position of high risk. As the disease is hard to diagnose at an early stage, that is rarely a viable option. However, the next stage would be intensive chemotherapy, which can reduce the size of the tumour and prepare it for surgery, but that, too, can be dangerous to the child.

High-dose chemotherapy with stem cell rescue can also be given. If the neuroblastoma has spread to several parts of the body, or is high-risk with MYCN amplification, high-dose chemotherapy with stem cell rescue is used after the initial courses of chemotherapy. High doses of chemotherapy wipe out any remaining neuroblastoma cells, but they also wipe out the body’s bone marrow, where blood cells are made. To prevent the problems that that causes, stem cells—blood cells at their earliest stages of development—are collected from the child through a drip, before the chemotherapy is given. The stem cells are then frozen and stored. After the chemotherapy, the stem cells are given back to the child through a drip. The stem cells make their way into the bone marrow, where they grow and develop into mature blood cells over a period of 14 to 21 days.

Imagine that treatment, Mr. Cook, for your own child. It would be bad enough for an adult, but for a toddler it must be almost unbearable. Radiotherapy is another avenue that sufferers can pursue, but it has high risks and can harm normal cells. Those options are bad enough for any child and any family, but they lead to a survival rate of only 20 to 30 per cent., which is simply not good enough.

Another treatment is available in America that can improve that sad statistic of 20 to 30 per cent. to a respectable 70 per cent.-plus survival rate. The treatment is called 3F8 monoclonal antibody therapy. Normally a person’s immune system makes antibodies to attack germs such as bacteria or viruses, but unfortunately it will not attack neuroblastoma because the tumour is part of our own bodies. However, an antibody that attaches to neuroblastoma can be made in a laboratory, then given intravenously to a patient. That antibody will circulate in the bloodstream until it finds and attaches itself to a neuroblastoma cell, then the patient’s own immune system will attack and kill that neuroblastoma cell.

On Thursday 10 December 2009, I tabled a parliamentary question asking the Secretary of State for Health

“if he will make it his policy that monoclonal antibody therapy is available to NHS patients diagnosed with neuroblastoma.”

In response, the Under-Secretary of State for Health said:

“The National Institute for Health and Clinical Excellence (NICE) has not issued any guidance on monoclonal antibody therapy…In the absence of NICE guidance, it is a matter for local national health service organisations to decide whether to fund a particular drug or treatment. The NHS Constitution gives patients the right to expect local funding decisions on the availability of drugs and treatments to be made rationally following consideration of the available evidence.”—[Official Report, 10 December 2009; Vol. 502, c. 551W.]

It is therefore quite clear that there is nothing to prevent the NHS from providing my constituent with monoclonal antibody therapy. In fact, the NHS constitution would lead my constituents to expect such treatment. It is also clear that this is an exceptional case. For one family to have suffered two cases of this dreadful killing cancer is more than unfortunate.

Little Chelsea’s final wish to her mummy and daddy

“was for them to help other poorly children.”

Recently, I met Chelsea’s mother and I was amazed at her courage and fortitude after what has happened to her. This is what she said to me:

“If we help with making these children smile, our angel will be smiling too.”

That is exactly what needs to be done. Little Zac needs this monoclonal antibody therapy.

I have spoken to Zac’s consultant, Dr. Johan Visser, and he believes that the monoclonal antibody therapy is especially helpful to children who have responded well to the traditional treatment of surgery and chemotherapy. Six weeks ago, Zac had a very large tumour successfully removed and subsequently he has had more chemotherapy, to mop up any remaining cancerous cells. However, to give Zac the very best chance of a full recovery, he needs monoclonal antibody therapy in March or early April next year, so time is of the essence.

Michelle Tomkins, Zac’s aunt, eloquently states:

“My cousin’s family needs to raise this extortionate amount of money to stop another little child in their family dying.”

A life, especially a child’s life, should not have a price tag, but sadly £250,000 is needed—that sum needs to be raised to send Zac across to the United States to receive treatment. Zac’s family have set up an appeal, which people can donate to online at Zac’s appeal has raised a fantastic £8,000. Among other things, the appeal has sold Zac wristbands and calendars, and even arranged a charity football game between two local teams. It has also received media attention, with the help of my excellent local newspaper, the Northamptonshire Evening Telegraph. Like my eight-year-old son, Zac is a Manchester United fan and he was the mascot for the match on 21 November 2009 between Manchester United and Everton. Nevertheless, after all these valiant efforts Zac’s appeal is still short of £242,000, so it has raised only 3 per cent. of what is needed.

However, it is not all doom and gloom. There is another solution. This disease affects approximately 24 children a year in the United Kingdom, so treating it is not a huge cost to the taxpayer. There is even better news. The Children’s Cancer and Leukaemia Group, or CCLG, has announced a clinical trial using monoclonal antibody therapy. However, as it is a trial, all the children need to have had the same therapy for the same period of time. Unfortunately, Zac misses this trial as he started his treatment six weeks too soon.

Zac is one of a handful of children who have missed this clinical trial who would have benefited from treatment with monoclonal antibody therapy. In future, all children who are diagnosed with neuroblastoma will go into such trials, but there is a small group of about half a dozen children who are not in the existing trial and who will not take part in future trials. They are the ones stuck in the middle.

The point or crux of this debate, and the reason why I am here today, is to get that treatment for a handful of children who need it, but who just miss out on the trial. I believe that those half a dozen or so children need to be treated here in our country and treated now. Dr. Visser has stated that he is happy to administer the treatment to Zac if funding is provided. The Government spend £100 billion on the NHS a year. Funding for those half a dozen or so children would be just a drop in the ocean.

I know that the Minister has close links with the Prime Minister and we all know of her compassion and that she can get things done. The biggest Christmas present that she could give to Zac and these other children is to go from this debate today to the Prime Minister and get the funding for them sorted out.

I am pleased to serve under your chairmanship today, Mr. Cook.

Of course, I want to thank the hon. Member for Wellingborough (Mr. Bone) for securing this extremely important debate. I also congratulate him on the manner in which he has conducted it. I have also had the opportunity to chat with him in his office and that has been incredibly helpful.

I am sure that all Members and all of us present today will sympathise with Zac and his family, and we appreciate how heartbreaking it must be for a parent to discover that his or her child has cancer. In my many years as a nurse, including my involvement with children’s nursing, I have had what I suppose I would call the privilege of being with children when they have died, and also with their families.

It is incredibly difficult for a family to cope with a child who is very seriously ill. It has knock-on effects on that family and the extended family, because of the obvious stress that it causes and because of the energy that is taken out of mum and dad, and, indeed, out of siblings if there are any. So I fully understand where the hon. Gentleman is coming from with this debate.

Thankfully, cancer in children is rare, with about 1,700 new cases being diagnosed in the UK in 2007. We estimate that about 2 per cent. of those cases were neuroblastoma cases. There have been remarkable improvements in the survival rates for most childhood malignancies in the past 30 years. Between 1970 and 2000, the overall five-year childhood cancer survival rate in Britain has increased from 28 per cent. to 77 per cent. and, in the case of neuroblastoma in the same period, the childhood cancer survival rate has increased from 19 to 59 per cent. I do not want my contribution to this debate to consist of stating lots of percentages. However, it is important that we put on record the progress that has been made in treating childhood cancer. Improving the care of children with cancer is one of our country’s success stories and we need to thank the clinicians, researchers and other scientists for the fact that some of the best children’s cancer services in the world are in the UK.

However, I am very aware that survival rates for children diagnosed with neuroblastoma are not as high as for some other cancers and, of course, even one child’s death is too many. We are not complacent and we are determined to continue to improve survival rates for all childhood cancers. We are doing that by helping GPs to identify children who are most likely to have cancer and who therefore need an urgent assessment; by ensuring that children diagnosed with cancer, including neuroblastoma, receive treatment as quickly as possible, and by organising services to ensure optimal outcomes for children and improved experiences, so that a little one’s journey through that clinical work, which the hon. Gentleman explained can be very difficult and painful, can be improved. We also need to improve our treatment process and research into children’s cancer, which is essential, and we are doing that as quickly as possible.

I would like to say a few words on each of the issues. As with all cancers, the earlier that childhood cancer is diagnosed, the better the prognosis for the child. The National Institute for Health and Clinical Excellence, or NICE as we commonly refer to it in debates, published updated referral guidelines for suspected cancers in 2005. Those guidelines are aimed at helping primary care health professionals to identify those patients who are most likely to have cancer and who therefore require urgent assessment by specialists. The guidelines include a section on cancers in children and young people.

Also, as set out in the second annual report of the cancer reform strategy, promotion of early diagnosis for all cancers is a priority for 2010. We have pledged to all patients in England access within one week to tests that can confirm or exclude cancer, and we will start rolling that out over five years from 2011-12. Quicker access to diagnostic tests, alongside work on early detection, will save 10,000 lives a year. Through the national awareness and early diagnosis initiative, we have committed £6 million to help GPs diagnose cancer earlier and to make the public aware of the symptoms so that they get them checked out early.

However, the distinct needs of children and young people with cancer have been recognised increasingly over recent years, which is why we asked NICE to produce service guidance for the NHS on improving outcomes for children and young people with cancer. The guidance was published in August 2005 and is aimed at those responsible for planning, commissioning and organising services for children and young people with cancer.

The guidance recommends principal treatment centres for each cancer type, with associated referral pathways to centres that might be outside the patient’s area of residence; access and support to clinical trials where the patient is eligible; care delivered by multi-disciplinary teams, including all relevant specialist staff, and care offered in age-appropriate facilities. The cancer reform strategy made a commitment to implement the guidance by the end of 2011.

The hon. Gentleman highlighted that research into the disease has brought good news that will give children diagnosed with neuroblastoma the chance to receive monoclonal antibody treatment in this country as part of a European trial announced last week. I will say more about the research later, but it is with sadness that I learned that Zac will not meet the eligibility criteria for the trial.

The hon. Gentleman stated that Zac’s consultant is happy to administer treatment in this country if funding is provided. As with other unlicensed drugs, it is down to clinicians to decide whether the drug is the best treatment for an individual patient and to clarify the potential risks with the patient. Because the drug is not licensed and the treatment has not been appraised by NICE, the primary care trust must take the decision on whether to fund the treatment. I have done my best to investigate what has been applied for in Zac’s PCT. I am led to believe that no formal application has been made, so I urge that one be made. I will take a close interest in the decision.

I am grateful to the Minister for her serious and helpful response to the debate. I am sure that she is right that a formal application has not been made, as I do not think that the family realised that it was possible to do so. My only concern is that, if the treatment costs £250,000 and the local primary care trust must fund it, it will be a bit unfortunate. Not many children are affected by the disease. Could the cost not be spread out over the whole NHS?

The hon. Gentleman makes a valid point. I know that other such drugs, called orphan drugs, are considered by the strategic health authority for the area or nationally. I would have to take advice about whether that could happen in this particular case, but I do not believe that the dialogue on Zac and the other children affected will be finished during this debate; I would expect us to carry on discussing the cases concerned.

In the past week, the launch of a major new clinical trial into the use of immunotherapy to treat children with neuroblastoma was announced. Cancer Research UK is funding the research costs in this country of the Europe-wide trial. The Government will provide infrastructure support via our national cancer research network. I understand that the trial will run in all 20 childhood cancer clinical trial centres across the UK starting this month. For the first time, that form of immunotherapy will be available in the UK to eligible UK patients. We will continue our long tradition of research excellence in helping build on earlier trials and devise even better ways of using the therapy.

The hon. Gentleman said that Zac missed eligibility by some weeks. I urge the clinicians involved to reconsider. Trials are trials, and they must have criteria, but it became evident to me while researching my reply that it might be possible to look at the matter. It would be wrong to raise hope; I am just asking for clarification of when the trial will commence. Of course, because not every child with neuroblastoma will benefit from immunotherapy, eligibility criteria, as in all well-designed trials, must be precisely defined. There will be a careful and necessary selection process. The trial will offer new hope to the children who benefit—it could be as many as 40 a year—and their families.

Children who take part in the Cancer Research UK trial will join the many other children and adults involved in clinical trials of cancer treatments. It is a tribute to all those working in the NHS, the cancer charities and the commercial sector that the percentage of cancer patients who enter trials each year is higher in England than anywhere else in the world.

The existence of the National Cancer Research Network, which the Government set up in 2001 and continue to fund, has been key to that achievement. Each cancer service network receives funds for an integral cancer research network. The £18 million we put into the NCRN this year provides dedicated research nurses, data managers, medical staff sessions and other services such as radiology and pathology.

As we all recognise, the new international trial is an exciting development, and it is right that we should focus on it, but I also take note of the six other children whom the hon. Gentleman mentioned. At all times, we urge clinicians—as I urge them today—to apply to the PCT where the child lives for funding. If that has been done and an appeal process is in place, I urge the PCTs to consider it.

This is our last debate of the year. It is so important to Zac and his family and to the other children. I thank the hon. Gentleman for securing this debate. I wish him and his family well this Christmas, as I know that his son is serving in Afghanistan. I wish him all the best during this distressing time.

Question put and agreed to.

Sitting adjourned.