Cancer has become a major killer of children. Why is our R & D investment far from enough?

Source: Li Zhizhong (pineapple) / Nandu public welfare observation special agreement July 1, 2016, the number of cancer patients in China has been increasing, cancer has been the second leading cause of death for children under 15 years old Cancer is a disease of the whole As the age increases, the incidence rate of cancer increases year by year Nearly 90% of cancer patients are over 50 years old Due to aging population, unhealthy lifestyle and environmental pollution, the incidence rate of cancer in China has been increasing exponentially How to prevent cancer and cancer has become the focus of more and more people But what many people don't know is that children and teenagers also get cancer, and it's not uncommon In China, there are nearly 40000 new children with cancer every year, one out of 400 children on average! At the same time, more than 10000 children die of cancer each year, and another 1 million families have child cancer survivors Due to the aggravation of environmental pollution, the change of life style, the improvement of cancer detection level and other factors, the number of children with cancer in China has been continuously increasing Cancer has been the second leading cause of death for children under 15 years old, next only to accidental injury In the United States, due to the popularity of safety measures, such as children's safety seats and other facilities, accidental injuries have been greatly reduced, and cancer has become the first killer of children By right, we should attach great importance to children's cancer Emotionally and morally, a child's illness is compassionate and compassionate, and a child is enough to devastate a happy family From the scientific and clinical point of view, children's cancer gene mutation is less, the general response to drugs is better, and drug resistance is not as serious as adult cancer, so the cure rate is high, which has been proved in leukemia At the same time, curing a child patient can save life not 5 years, 10 years, but possibly 50 years, bringing greater hope to life Therefore, no matter from the perspective of emotion, morality, science or economy, it is of infinite value to conquer children's cancer We should make great efforts to overcome it Unfortunately, on the contrary, the world is lagging behind in the investment and research of children's cancer In China, it is almost negligible Only when the government is short of funds for scientific research can it win every battle If we want to conquer any disease, we must first understand its pathogenesis, then we can find its weakness and attack it accurately In essence, children's cancer is the same as that of adults At the same time, the failure of immune system leads to malignant overgrowth of cells However, children's cancer is very different from adults in terms of gene mutation type, clinical characteristics and drug response, so it needs to be studied separately Children's cancer is not a single disease, but a very diverse one There are 12 categories and more than 100 subtypes in total, which cannot be generalized Research needs to go hand in hand and blossom in many ways, so long-term support from research funds is needed But compared with adults, funding for cancer research in children is very limited The U.S government is a global leader in this area, but it also accounted for only 4% of its cancer research spending in 2015 In contrast, the research funding for prostate cancer alone exceeds the sum of all childhood cancers The average age of prostate cancer patients is 66 years old, and most of them have achieved good results The 5-year survival rate is almost 100%, the 10-year survival rate is 98%, and the 15-year survival rate is 95% (at this time, the average age of patients is 81 years old) Although any cancer that has not been completely conquered should be paid attention to, I think such a distribution ratio is questionable In China, the funding for children's cancer research is even less, and the research is very backward In addition to leukaemia, there are very few laboratories focusing on children's cancer research This is a pity, because China has the largest number of children with cancer in the world, and it is very concentrated, so it is a great advantage to focus on scientific research American doctors usually see a few or dozens of children with cancer a year, but Chinese doctors may see so many in a day For example, the number of children with brain tumors treated in Beijing Tiantan Hospital alone is almost equal to the total number of all major hospitals in the United States One of the biggest difficulties in children's cancer research is the lack of samples China has this natural advantage, but unfortunately, many samples have been wasted, and it is regrettable that China has not made more breakthroughs in related research How to encourage more people to make use of these precious samples, and make them the key to conquer diseases, is a problem that the government should consider The president of the United States recently put forward the "anti cancer moon landing plan", which starts from six directions, including children's cancer The Chinese government should also have special research in this area There is a lack of non-governmental public welfare funds to support scientific research in the United States In addition to the government, many research funds come from non-governmental public welfare organizations Due to the lack of government funding, private funding is very important for many rare diseases, including children's cancer research, which has a huge role in promoting St Jude Children's research hospital alone receives more than $850 million in donations a year, a large part of which is used to support research The research supported by these funds produced 810 papers in 2014, most of which were about childhood cancer In addition to these large-scale institutions, there are many small foundations in the United States, which tend to focus on a small category of children's cancer, initiated by parents of children, and set up to commemorate the children who left after bravely fighting against cancer Their goal is clear They want to fund research to find new treatments for children with the same diseases in the future This kind of fund sponsorship amount is not very large, but due to the large number of drops into a trickle, the impact on society can not be ignored And these parents are often very seriously involved in the project selection and pay attention to the progress of the project, so these funds are less wasted and the utilization rate is higher In 2013 and 2014, I participated in two academic seminars on rhabdomyosarcoma in children, which were sponsored by the fund established by parents of children I was moved by a speech from my parents at the meeting: "we spent a lot of money and failed to save my son, because we found that we didn't understand the disease at all Now, I hope we can pay, and you can help, to save other people's children " I think one of the best examples of this is neuroblastoma in children There are only 700 cases of this rare childhood cancer in the United States a year, but in recent years, it has made rapid progress Whether it is the understanding of the pathogenesis, the development of new drugs, clinical trials, are all at the forefront of all solid tumors in children One of the important reasons is the push of a large number of parents in the past 20 years They not only actively raised funds and set up a foundation to support the research on the tumor, but also helped to establish cooperation and information sharing between doctors and researchers, so that new knowledge could be spread quickly and new clinical trials could be carried out as soon as possible Years of perseverance have brought about qualitative changes In China, although people's awareness of public welfare is increasing year by year, most of the public welfare for children's cancer still stays in the donation to individual patients, and few public welfare funds are used to sponsor basic scientific research or clinical translational medicine, which is indispensable to completely defeat children's cancer There are also signs of ice breaking Last year, I witnessed and participated in the establishment of the "new sunshine · help medical children leukemia research fund" - the first medical research fund initiated by parents of patients in China I believe that this is just the beginning, and we will see more funds for children's cancer research in the future Lack of drug development The ultimate goal of promoting scientific research is to develop new drugs to treat children's cancer Although the development of cancer drugs has always been the focus of attention of pharmaceutical companies, with huge investment and the most new drug production in the past few years, children's cancer has always been forgotten by pharmaceutical companies Traditional pharmaceutical companies are keen to develop drugs for adult cancer, but they are not interested in drugs specifically for children's cancer for many reasons First of all, the cost of developing new drugs is very high, the cost is up to US $100 million, while the number of children with cancer is relatively small, and there are fewer subtypes If there are only hundreds of new drugs in a year, it is difficult to make profits under the traditional mode Secondly, it is necessary to do several clinical trials for any new drug to be put on the market It is very important to quickly recruit enough patients to participate in the new drug test Due to the small number of patients, it is difficult to recruit enough patients for any new drug It is not enough to assume that less than half of the 500 patients of a disease in a year meet the clinical trial standards, and that ten new drugs can be tested, each of which can find up to 25 patients The difficulty of finding patients may not only bring uncertainty to new drug testing, but also prolong the testing time and increase the cost greatly Moreover, pharmaceutical companies like to pay attention to diseases that have no drugs to treat On the one hand, there is a large demand, on the other hand, the threshold is relatively low Once a new drug has a little effect, it may be approved, such as pancreatic cancer But many children cancer use chemotherapy drugs have certain effect, which is a good thing in itself, but put forward higher requirements for new drugs However, it must be noted that although chemotherapy drugs have good therapeutic effect on many children's cancer, and even can cure it, the cost is huge side effects 95% of children's cancer survivors will have side effects before they are 45 years old, and one third of them are serious side effects, including mental retardation, secondary cancer, etc Finally, in order to protect children's safety, the regulatory authorities, such as FDA, have higher requirements and stricter control on children's drug use More preparations and challenges are needed to carry out clinical trials for children This makes many companies retreat All in all, there are many factors that make the development of drugs for children's cancer less attractive to the market, with additional risks, but no additional returns, so it is not attractive to pharmaceutical companies The direct consequence is that in the past 30 years, only three new drugs for children's cancer have been launched, namely, etoposide for childhood leukemia in 1980, clofarabine for childhood leukemia in 2004 and unituxin for Shenmu cell tumor in 2015 In contrast, more than 200 new therapies for adult cancer are on the market in the same period No matter how much progress has been made in scientific research, without the support of new drug development, it can not be transformed into an effective treatment for patients How to encourage pharmaceutical companies to develop drugs for children's cancer is an urgent problem Apart from developing drugs for children's cancer alone, another faster and more cost-effective way is to test whether some drugs used by adults for cancer can be used for children's cancer treatment, so-called "one drug for multiple purposes" More and more evidences show that cancer classification based on gene mutation (such as EGFR mutation cancer) can better guide clinical treatment than traditional classification based on the location of disease (such as adult lung cancer) Some childhood cancers have genetic mutations similar to those in adults, and may be able to use the same drugs For example, some children's seminoma and adult lung cancer have ALK gene mutations because