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Research

In this section we aim to keep you up to date with the latest developments in research. FAN sends a representative to Neuroblastoma conferences and symposiums where researchers and clinicians inform those present on the work they are doing. When we cannot send a representative, we obtain the information available via other sources, usually another Neuroblastoma charity representative.

FAN is in its infancy, and our main objectives to date have been to raise substantial amounts of funding to support children currently diagnosed. This will always be our main objective, however without funding much needed research into Neuroblastoma, this would be a never ending task. Our objective is to Cure Neuroblastoma and this will only be done by funding vital research projects and institutions seeking better treatments until a cure can be found.

There are many worth while projects to fund. FAN currently is unable to accept grant applications for funding, but we are hoping to help form a panel with other Neuroblastoma charities to do this in future. For now, we have looked at current projects being funded by other sources and where best we can help fund cutting edge therapy. As FAN's main objective is to help children currently diagnosed, our funding for research will be awarded to projects that have a high possibility of helping our children in the very near, or near future. With this in mind we have pledged our support for the coming year to Professor Andy Pearson and his team at the ICR who are working on the problematic and often fatal Myc-n amplification gene and also phase 1 trials for relapsed Neuroblastoma.

Here is some further information from the team.

It is still early days in our studies of drugs targeting the MYCN gene which is a very important contributor to poor prognosis in neuroblastoma. We are currently working on 10 drug candidates, which may be suitable for early clinical (phase 1) studies in paediatric solid tumours, greatly increasing our chances of success. To put this into context, only 4 agents, of any type, have been taken forward in this way in paediatric oncology in the last 20 years.

The Institute of Cancer Research's 5-year goal is to successfully adapt current adult cancer drugs for use in children and the 10-year goal is to have a specifically targeted childhood cancer drug for young patients that can be used worldwide.

We have committed to support a drug development programme targeting MYCN and to initiating phase 1 clinical study of an ICR developed lead candidate drug. Three of the potential compounds we have look at are very promising. One has entered clinical trials at the Royal Marsden Hospital in late 2009, one has only been used in 40 adults around the globe and we are hoping to trial in children in early 2011, whilst the other promising target is at the pre-clinical stage and yet to be tried with patients. However, these could lead to one of the first academically conceived and developed drugs, specifically targeted to a paediatric cancer protein.

Our current work in childhood cancer research

The Institute of Cancer Research (ICR) is pioneering research in child cancer genetics and drug development to produce treatments which will not have the severe side effects associated with current treatments. At present children with cancer are treated with drugs adapted from those developed to treat adult cancers. There is a very significant unmet need for drug development for paediatric cancer. To date no drugs have been made specifically to treat child cancers and there is no other centre in the world with a major programme and facilities in this area.

The ICR, together with The Royal Marsden NHS Foundation Trust, has more than 60 researchers and clinicians, divided into seven teams, dedicated to the fight against childhood cancer. Our expertise covers all aspects of childhood cancer research from basic biology, through to the development of new drugs to their use in clinical trials. We are currently focusing on those childhood cancers which have a particularly poor survival rate, such as high-risk Neuroblastoma.

Dr Louis Chesler of the ICR, together with Professor Andy Pearson, is working specifically on developing new treatments for Neuroblastoma. Neuroblastoma is a prototypical example of a difficult-to-treat solid tumour. It usually affects children less than seven years of age and little progress has been made in the past decade to improve the cure rate. Treatment involves multiple elements, is extremely intense and is prone to toxicity. Chemotherapy and surgery are used first, followed by additional surgery, bone marrow transplant, irradiation, further chemotherapy and finally, differentiation therapy. Even at this intensity, treatment failures and resistance are common outcomes.

Dr Chesler and his team are focusing on drug development targeted at MYCN, a gene that is often involved in causing cancer but which is also necessary for normal growth and development of brain, nerve and muscle tissues. MYCN causes high-risk forms of Neuroblastoma. It is Dr Chesler's hope that within five years he and his team will have developed treatments that target MYCN and its oncoprotein.

Several academic collaborations have been established, both within the ICR and at external institutions, in the areas of Neuroblastoma modelling and drug development. We have also initiated a major new effort with Dana Farber Cancer Institute (USA) and University of Pennsylvania/Children's Hospital of Philadelphia (USA) to model and investigate the ALK gene in Neuroblastoma and develop new drugs targeted at its protein product.

The ICR's work benefits immeasurably from having its laboratories on the same site as cancer patients who are being treated at the Royal Marsden. This 'bench to bedside' factor is essential as we bring our laboratory research to clinical trials and then, ultimately, alter international practice in paediatric oncology. Paediatric Cancer Treatment Hospitals everywhere will be able to adopt our new treatments as soon as they have been tested in clinical trials

Cancer research is expensive and we still need funding to meet our goals. However, it is hoped that this research will benefit the outlook not only for children diagnosed with cancer but the families faced with providing emotional support and care for their loved-ones in the most difficult of circumstances.

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