News Article | May 9, 2017
Neuroblastoma is the second most common solid tumour in childhood, following brain tumours, and predominantly affects children under five years old. Every year in Europe, around 1,200 children are diagnosed with neuroblastoma, a rare cancer arising from neural crest cells, which are involved in the foetal development of the nervous system and other tissues. Because neuroblastoma can spread very quickly, almost half of children are initially diagnosed at an advanced stage of their disease and are recognised as 'high-risk' and with a poor prognosis. Approval of dinutuximab beta brings new hope to these 'high-risk' children who have previously received induction chemotherapy and achieved at least a partial response, followed by myeloablative therapy and stem cell transplantation, as well as those with history of relapsed or refractory neuroblastoma, with or without residual disease. "Today's announcement is a leap forward for the children and families affected by neuroblastoma, particularly those who have keenly followed the positive clinical trial results for dinutuximab beta and long anticipated its approval in Europe," commented Dr Juliet Gray, Associate Professor and Consultant in Paediatric Oncology at University of Southampton, UK. "As a clinician working in a highly specialised disease area with limited treatment options, I greatly welcome the availability of this targeted immunotherapy treatment that offers improved results for high-risk neuroblastoma patients used alone or in combination with existing therapies." Steve Richards, CEO of the neuroblastoma charity Solving Kids' Cancer Europe, added: "In the absence of any other targeted immunotherapy for children with high-risk neuroblastoma, the European regulatory body that approves medicines to be marketed in Europe, expedited the review of dinutuximab beta. Today's approval means that EUSA Pharma who manufactures dinutuximab beta is able to make it available for use by hospitals across Europe, improving access for thousands of children and their families to this new treatment, with proven improved survival rates. The next challenge will be for EUSA Pharma to engage with relevant access bodies throughout Europe, including NICE in the UK, to ensure timely review through the new drugs processes and secure access to this medicine for patients. The young innocent victims of this cruel and devastating disease deserve nothing less." Lee Morley, CEO of EUSA Pharma, commented, "We are delighted that the EC has recognised the urgent need to accelerate approval of dinutuximab beta in order to provide an effective and targeted treatment for this debilitating disease. EUSA Pharma in partnership with Apeiron and SIOPEN has believed strongly in the potential of this treatment throughout the clinical trial process and today's announcement is final acknowledgement of its value to address the serious unmet need of children and their families affected by high-risk neuroblastoma." Dinutuximab beta is a monoclonal chimeric antibody developed to target a specific antigen, GD2, on neuroblastoma cells. It has been investigated in clinical trials for high-risk neuroblastoma, with more than 1000 patients having received treatment to date. Dinutuximab beta has orphan drug designation in the US and EU, and EUSA plans to file the product for approval in the United States in 2017. Founded in March 2015, EUSA Pharma is a specialty pharmaceutical company with commercial operations across Europe and the USA, and a wider distribution network in approximately 40 further countries. The management team comprises highly experienced pharmaceutical professionals with a broad experience and proven track record of successfully identifying, developing and commercializing innovative medicines that advance patient care and improve their wellbeing. For more information visit: http://www.eusapharma.com.
News Article | May 9, 2017
Researchers at the University of Zagreb say pemetrexed (Alimta) is more than just a first-line treatment for people with mesothelioma; their new case report suggests that it can also extend survival after recurrent asbestos cancer. Surviving Mesothelioma has just published an article on the case report. Click here to read the article. The peritoneal mesothelioma patient that is the subject of the newly-published case report received cytoreductive surgery and chemotherapy both directly in his abdomen (called HIPEC) and via infusion. When he eventually relapsed, his doctors turned once again to the only drug that is FDA approved to treat mesothelioma. “Although the intent behind systemic treatment was at first solely palliative, overall survival after the initial diagnosis was 50 months,” writes the report’s author, oncologist Dr. Milena Peitl in the Indian Journal of Medical and Paediatric Oncology. There is no universally-accepted second-line treatment for recurrent peritoneal mesothelioma, which accounts for fewer than 500 of the estimated 2,500 cases of mesothelioma in the US each year. “Because peritoneal mesothelioma is so rare, there are even fewer medical studies on this type of asbestos cancer than on the more common pleural mesothelioma,” says Alex Strauss, Managing Editor for Surviving Mesothelioma. “It is always encouraging to see a new study, especially one detailing a positive outcome.” To read more about the Croatian study and its potential implications for peritoneal mesothelioma patients, see Retreatment with Alimta Leads to 4+ Year Survival for Peritoneal Mesothelioma Patient, now available on the Surviving Mesothelioma website. Peitl, M, et al, “Significant clinical benefits of pemetrexed-based chemotherapy for advanced diffuse peritoneal mesothelioma: a case report”, 2017, Indian Journal of Medical and Paediatric Oncology, pp. 73-77, http://www.ijmpo.org/article.asp?issn=0971-5851;year=2017;volume=38;issue=1;spage=73;epage=77;aulast=Peitl
News Article | May 18, 2017
This finding could have a great impact on the study of pediatric tumors, as it could already constitute one of the factors that produces their growth The Neural Mother Cell Physiopathology Research Group of the University of Seville and the Seville Institute of Biomedicine (IBiS) have just published a scientific article in the review Cell Reports, in which they show that mother cells from the adult carotid body can transform into blood vessels, as well as into neurons. The work has mainly been carried out by the post-doctoral researcher Valentina Annese, as a member of the group. This discovery could have important repercussions on the advance in treatment of diseases as different as paediatric tumours and Parkinson's. "We believe that the ability to produce blood vessels from neural stems cells could directly affect the growth of certain types of tumours on the infant population," said the project's main researcher, Ricardo Pardal. The carotid body is a small structure of nerve tissue situated at the fork of the carotid artery. Its function is to act as a chemoreceptor in the blood. It monitors oxygen pressure in the blood and plays a role in the regulation of breathing. The plasticity of adult mother cells, or the somatic mother cells, to cross boundaries and to differ in unrelated cell types has been a subject of debate in the last decade. The stem cells that come from the neural crest (NCSCs) show notable plasticity during their development, but it is not known if adult NCSCs maintain this plasticity. In this sense, "we describe that the adult stem cells from the carotid body taken from the neural crest (CBSCs) are capable of experiencing endothelial differentiation, as well as their already described role in neurogenesis, contributing to both neurogenic and angiogenic processes that take place in the organ during acclimation to hypoxia. In addition, the conversion of CBSCs into blood vessels is dependent on the hypoxia-inducible factor (HIF) and is sensitive to vascular cytokines released in hypoxia, such as erythropoietin. Our data highlights a notable physiological plasticity in an adult population of stem cells specifically from tissue, and they could have an impact on the use of those cells for cellular therapy", Pardal added. These results are some of the conclusions of the ERC Starting Grant project 'Physiology of the adult carotid body stem cell niche', funded by the European Research Council, as well as the Ministry of Economics and Competiveness project, 'Physiopathology of Stem Cells Derived from the Neural Crest". For these studies, the experts have had the collaboration of the Paediatric Oncology and Pathology units of the University Hospital Virgen del Rocío. Bibliographical reference: 'Physiological Plasticity of Neural-Crest-Derived Stem Cells in the Adult Mammalian Carotid Body'. Valentina Annese, Elena Navarro-Guerrero, Ismael Rodríguez-Prieto and Ricardo Pardal. Published in Cell Reports
News Article | May 22, 2017
The most common type of malignant childhood brain cancer has been identified as seven separate conditions each needing a different treatment, new research has revealed. A study by Newcastle and Northumbria universities has found that childhood medulloblastoma can be separated into seven different subgroups which all have their own biological and clinical characteristics. It is hoped that this finding, published today in The Lancet Oncology, will give young patients access to better treatment programmes tailored to their needs and new drug options in the future. Up until now there had only been four recognised molecular subtypes. Experts say this new understanding could lead to increased survival rates as each responds differently to treatment so can be targeted individually. It is anticipated this discovery will help personalise treatment so that young patients get the best care possible, tailored to the specific biology of their cancer. Gentler therapies could be used for children with a good prognosis, while reserving the most intense treatments for those with high-risk tumours, as well as developing new treatments. Steve Clifford, Professor of Molecular Paediatric Oncology at the Wolfson Childhood Cancer Research Centre, Newcastle University, has led the research. He said: "Medulloblastoma is the most common type of malignant paediatric brain tumour and it is a devastating condition that causes approximately 10 percent of all childhood cancer deaths. "Our research has provided critical new insights into the cancer's molecular basis and it is a significant step forwards in enhancing our understanding of this life-threatening disease. "This new discovery allows us to undertake studies to see how we could use these insights in diagnosis and to personalise treatments according to the biological features of each patient's tumour." Medulloblastoma is the most common brain tumour affecting young people. It is estimated that around 650 patients are diagnosed each year in the EU. There is an urgent need for greater understanding into the condition in order to assign patients to the most appropriate treatment programme. Children with this cancer are currently given a combination of surgery, chemotherapy and radiotherapy, but this course of action has numerous debilitating side effects, such as lower IQ and social problems. The study looked at tumour samples from more than 700 children with medulloblastoma, together with clinical and disease details. Analysis of each tumour assessed its genetic characteristics and identified unique biomarkers which the experts then compared with clinical information, allowing them to establish that each subgroup has distinct clinical features and survival rates. Dr Ed Schwalbe, Senior Lecturer in Bioinformatics and Biostatistics at Northumbria University, Newcastle, was the study's first author. He said: "Excitingly, these new subtypes are characterised by better survival rates for some children but sadly also worse outcomes for some. "This research could therefore lead to new clinical trials, to better help clinicians decide on optimal treatments for their patients, focusing on reducing treatment side effects in patients with a good prognosis, and continuing to aggressively treat patients with a worse prognosis." Cancer Research UK and The Brain Tumour Charity provided funding for the study. Dr Catherine Pickworth, Cancer Research UK's science information officer, said: "Improving the way we treat cancer is a priority, especially as many children who survive cancer will live with long-term side effects of their treatment. "It's vital that we make treatment both better and kinder, and this study is a positive step forward in achieving this. "By showing that different subgroups have different outcomes we can move towards giving treatment by subgroup, allowing doctors to use kinder treatments where possible and only resort to the more powerful ones where absolutely necessary. "The next steps will be to look at which treatments help each subgroup the best, so we can tailor treatment for every child with medulloblastoma." Findings of the study will be used by the Newcastle University team, at the Wolfson Childhood Cancer Research Centre, for further research into childhood medulloblastoma. They are looking to develop international clinical trials which test whether implementing these findings can improve diagnosis, treatments and outcomes. They also want to further understand the biology of each subgroup so that scientists can begin to target them with specific therapies. Neil Dickson, Vice Chair of The Brain Tumour Charity, said: "These findings are extremely encouraging and will enable more accurate diagnosis and appropriate treatment for those affected by medulloblastoma. "This research was one of the first major projects made possible by our fundraising and investment as a charity. "We are delighted that the research we are funding at Newcastle is having a positive impact for children and families facing this traumatic diagnosis and treatment." Novel molecular subgroups for clinical classification and outcome prediction in childhood medulloblastoma: a cohort study Once the research paper has gone live it can be accessed at this web address: http://www.thelancet.com/journals/lanonc/article/PIIS1470-2045(17)30243-7/fulltext?elsca1=tlxpr
Ghafoor T.,Bristol Royal Hospital for Children |
Zaidi A.,Paediatric Oncology |
Al Nassir I.,Shaukat Khanum Memorial Cancer Hospital and Research Center
Journal of the Pakistan Medical Association | Year: 2010
Granulocytic sarcoma is an extramedullary tumour of primitive granulocytic cells. It can develop at any anatomic site and is often a forerunner to the development of acute myelogenous leukaemia. Granulocytic sarcoma of the small intestine presents with abdominal pain and obstruction. We report a case of a 17-years-old boy who presented with epigastric pain. His endoscopy revealed multiple polypoid lesions throughout the duodenum and small bowel. Histopathology and flowcytometery confirmed the diagnosis of granulocytic sarcoma associated with acute myelogenous leukaemia. To our knowledge there have been only two previous case reports of multiple granulocytic sarcomas in the small intestine, both of these were adult patients. This is the first patient in the paediatric age group with multiple granulocytic sarcomas of the small intestine.