Our mission is to raise £500K over 3 years to help children and their families impacted by rhabdomyosarcoma.
We want these funds to help advance the research in to rhabdomyosarcoma in order to find better treatments to lengthen and improve the quality of life of these children and their families. Ultimately, we would like to see treatments in place that can cure rhabdomyosarcoma.
How Will We Do It?
By collaborating with leading institutions like the Institute of Cancer Research, Great Ormond Street Hospital and the Royal Marsden we can help to realise this goal. We have pledged to support the clinical research that they undertake both in the laboratory and in the hospital. This involves providing funds for a new rhabdomyosarcoma clinical trial due to launch in mid 2019 which is being led out of the UK and co-ordinated with other European Centres. For the ICR we provide funds for the salaries of key staff required to undertake the necessary research to help make the clinical trial a success - this involves a Clinical Fellow in the laboratory and a Higher Scientific Officer. These individuals coordinate and analyse samples of tumours and look at gene sequencing data to determine how the tumour is wired. They then experiment in the laboratory with drugs that target these molecular defects and could be used to treat rhabdomyosarcoma patients. We also fund consumables/lab reagents that help the team with cell culture, drug sensitivity screening and preclinical testing including animal models. For GOSH we have pledged to fund the role of a Research Nurse working on the clinical trial. This role will help support the collection and processing of blood and other samples for the study including molecular pathology, circulating tumour DNA and PK (pharmacokinetics).
By supporting patients and families currently being treated for rhabdomyosarcoma by improving the quality of hospital stays. Creating opportunities for patients to use drugs that can potentially ease the treatment process. For example, by providing funds to acquire drugs such as neulasta, which involves a single injection, to help prevent infections as blood counts drop as a result of treatment which can result in long stays in isolation in hospital receiving antibiotics. Neulasta is a drug which boosts white blood cell recovery and involves a single injection of GcSF (granulocyte colonies stimulating factor)
By raising awareness of childhood cancers like rhabdomyosarcoma. It is surprising to learn how little investment is made in to research for childhood cancers compared to adult cancers. Research and development budgets in pharmaceutical companies covers 60% for adult cancer drugs and close to zero for children. This is a complex area involving issues relating to politics, legislation and profitability. We aim to work with other childhood cancer charities and influential cancer research centres in order to lobby for changes that can help to maximise the opportunities to make progress understanding and treating rhabdomyosarcoma.