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SPARKS’ aim is to give every child the chance of a healthy start in life. We do this through funding vital medical research that will:
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SPARKS is funding the formation of a pioneering new brain injury research group at University College London. The first stage of the project will be a £199,337, two-year grant to allow doctors to gain a better understanding of the mechanisms of brain injury in premature babies. It is the major unsolved problem of foetal medicine and neonatal paediatrics, affecting over 1,000 babies born in the UK every year. Ultimately, the team hope to be able to pioneer corrective brain surgery while the baby is still in the womb, but this will require new scanning equipment likely to cost at least £1million… for more information about this project, click here Other groundbreaking new SPARKS-funded research projects include: *A new anti-tumour therapy using cells of the immune system at The Institute of Child Health (£138,962 over 3 years). It’s part of the continuing battle to combat Neuroblastoma, the biggest cancer killer of children under 4 and the most resistant to improved treatment techniques that have reduced fatality levels in other forms of child cancers. *A revolutionary new research project—also at The Institute of Child Health-designed to improve the upper limb movement among children affected by strokes. It’s not generally known that a thousand children a year suffer strokes in the UK and it is among the least researched areas of paediatric medicine. Despite the fact that strokes rank among the top ten causes of childhood mortality. The one-year project is receiving a SPARKS grant of £44,917. *Plaster Immobilisation to restore muscle length in children with cerebral palsy. Children with spastic diplegic cerebral palsy(SDCP) develop fixed shortening of the calf muscles that seriously impairs walking, causing them to walk on their toes. This project, at Guy’s & St. Thomas’s Hospital, attempts to assess the benefits of using plaster casts to stretch these muscles. The research programme is receiving funding of £85,909 over 2 years.
Did YOU know that in the UK up to 80% of the type of groundbreaking medical research SPARKS supports has to be funded by charity and not the public purse? |
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“The construction and testing of a novel lentivirus and adeno/lentivirus hybrid vectors for gene therapy of cystic fibrosis” Cystic Fibrosis is an inherited disease which affects vital organs in the body, especially the lungs and digestive system, by clogging them with thick, sticky mucus. It is the UK’s most common life-threatening inherited disease affecting around 7,500 babies, children and young adults in the UK. As an inherited disease, for a baby to be born with cystic fibrosis, both parents must be carriers of the faulty CF gene. But even then, it is not certain that the baby will be born with cystic fibrosis. One in twenty five people in the UK are carriers of the CF gene. One in 2,500 babies will have cystic fibrosis, meaning there is a baby born with CF nearly every day. In 1989 an international team of scientists identified the gene for cystic fibrosis. It makes a protein called the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) which carries salt in and out of the cells that line the lungs and digestive system. In people with cystic fibrosis CFTR doesn’t work properly. It gives out too much chloride (salt) and not enough water. This helps to explain why the secretions of the airways are always sticky and become easily infected and why the sweat of a person with CF is excessively salty. These sticky secretions make it difficult to breathe and absorb food properly. Serious symptoms and complications of CF include infection and inflammation of the lungs, malnutrition, diabetes, liver problems, and osteoporosis. Just forty years ago, children with cystic fibrosis did not live beyond their early teens and many died as young children. Today the outlook is much brighter but cystic fibrosis (CF) still claims the lives of three young people every week. There is no known cure for CF. Scientists and doctors throughout the world are working as quickly as they can to find ways of repairing or replacing the faulty gene and are now confident that a cure will be found. Gene therapy is a major focus of research into finding a cure for cystic fibrosis. This is where a disease is treated by replacing the faulty gene of the patient with the normal gene. As gene therapy is in its infancy there are still many problems to overcome. Inadequate gene transfer to the human airway epithelia by all applied gene carriers has been identified as the main reason that there is still no curative formula for cystic fibrosis. As different carrier systems have different advantages and disadvantages this study at Imperial College of Science Technology and Medicine aims to combine two viral carrier systems, to make use of their advantages and to compensate for the disadvantages of each of them. Several different constructs will be made using this approach and will be tested for their ability to transfer genes to the airway epithelia. Adenoviral vectors are remarkably stable in the body, and are still the most effective gene transfer systems to the airway epithelia. However, they lack the potential for sustained gene expression and cause considerable immune reactions when applied repeatedly. The team at Imperial College propose to apply a novel adenovirus hybrid system for construction of new gene therapy vectors for CF. This will combine the advantages of adenovirus with the ability of lentiviruses to infect non-dividing cells and provide long-term transgene expression. Cystic Fibrosis is characterised by early irreparable organ damage; application of gene therapy to the foetus during the later stages of pregnancy may prevent this damage from occurring. This work is being carried out in collaboration with Prof. C. Rodeck’s Foetal Medicine Group at UCL/The Royal Free Hospital. It is hoped that this research will break the deadlock in clinically applicable cystic fibrosis gene therapy within the next 5-10 years.
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Identification of the Gene for Severe Spina Bifida |
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Dr Philip Stanier and Professor Andrew Copp – Institute of Child Health Spina Bifida is a birth defect that can cause considerable disability and affects 1 in every 500 – 1000 births. It occurs when the spinal cord does not form correctly during early development in the womb, leading to paralysis and loss of sensation in the legs. For many this means life in a wheelchair, bladder and bowel problems and in some cases hydrocephalus (water on the brain). The precise outcome depends on how severe the spina bifida is. The most severe type, which is being studied in this SPARKS project, accounts for 10-20% of all cases of spina bifida. Hence, this is an important health problem for children, world-wide. Currently, there are few medical ‘treatments’ available to prevent spina bifida. Although taking folic acid early in pregnancy can avoid some cases, there has been disappointingly little reduction in the frequency of spina bifida in recent years. This is despite the recommendation that all women planning a pregnancy should take folic acid. Alternative options are to detect the spina bifida before birth, and abort affected fetuses. The aim of this SPARKS funded project is to identify the genes that cause spina bifida, so that doctors can develop new genetic tests. These will show whether parents contemplating pregnancy may be at high risk for having a baby with spina bifida. Such parents could then receive intensive folic acid treatment, for instance, to reduce their risk. Until this study, no genes for severe spina bifida had been found. Professor Copp and Philip Stanier have been working, since 1992, towards isolating a gene that causes severe spina bifida. This has been a long and intensive project, and it recently came to fruition when they successfully isolated the gene. It is named Lpp1 and its identification will allow their research to go forward in two directions. 1. They can now investigate the precise reason why embryos develop spina bifida. For instance, they know that Lpp1 must normally perform an essential function allowing the embryo to develop a normal spinal cord. However, the researchers are only just beginning to understand exactly what role Lpp1 plays in this process. 2.They can establish whether patients with spina bifida all have a defective copy of the Lpp1 gene, which causes this condition. This genetic discovery is the most significant advance in spina bifida research for several years. It opens up enormous possibilities both in terms of scientific understanding and possible new clinical procedures for improved diagnosis and treatment, which should benefit children’s health in the future. |
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For further information please contact [email protected] |
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About 5000 babies are born very prematurely (less than 28 weeks gestation, or 3 months early) in the UK each year. One of the complications of being delivered so early is that the brain is still developing and is vulnerable to damage. Up to 10 % of these very premature babies may develop serious brain injury, with profound consequences for the child and their family. Funding from SPARKS has helped establish a research group of doctors and scientists, working at the Perinatal Brain Research Centre at University College Hospital London, who are focusing their attention on this vital challenge. Recent research suggests that infection, one of the main causes of premature labour, may be an important factor leading to brain damage in the baby. Infection of the membranes surrounding the pregnancy, called chorioamnionitis, can occur in anyone. It seems that this dangerous infection may be caused by bacteria which are normally present harmlessly in the mother’s body. The important thing is that in some people these bacteria lead to an inflammatory response around the pregnancy, similar in some ways to the reaction seen around a healing cut. It is thought that chemicals released from inflammatory cells, called cytokines, can stimulate premature labour, but can also cross the placenta to the unborn baby. We will explore why these molecules seem to be associated with brain injury in the baby. Such information is necessary to determine whether normal delivery or caesarean section is best for some of these very premature babies. It might also solve the following difficulty: if infection causes both premature labour and brain injury in the baby is it best to try and prevent the baby being born very early by stopping the labour, or will this just mean that the unborn baby is exposed to higher levels of cytokines, making the brain damage worse? With the help of SPARKS a group of premature babies at University College London Hospitals will be studied in great detail, including the use of magnetic resonance imaging (MRI). This sophisticated technique provides precise information about whether any brain injury has occurred. If we can determine which chemicals are particularly associated with evidence of brain injury it might be possible to design drugs to counteract them and so prevent or reduce the extent of damage. In addition, we hope to be able to explain why these normally harmless bacteria can have such a catastrophic effect on some, but not all, mothers and their babies. The generous support of SPARKS is enabling us develop a unique research centre dedicated to the protection and repair of the brain of premature babies, using specialised techniques that are at the forefront of medical science. With your help we can really make a difference to the lives of these precious and vulnerable children. |
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General Guidelines For Research Grant Applications General: SPARKS funds medical research related to the prenatal period and the early years of life. The current focus is on research aimed at providing a therapeutic intervention that will make a significant contribution to reducing mortality or relieving the condition, disease or disability in question. The Charity will only support research which is likely to have a clear clinical application in the near future. Therefore grant applications for routine basic research which is unlikely to have clinical application within ten years will not be considered. More specifically: SPARKS-funded research takes the form of project grants of up to three years in length with a clearly definable subject area and outcome, or for equipment grants for use within a specific research proposal as previously defined. Pilot projects of short duration to test a concept in preparation for a full application will also be considered. Grants are usually made to projects where the principal applicant is in a tenured position at a university or institution. Funding guideline: A guideline for a clearly justified grant application including all salary, consumable and equipment components is not likely to exceed £45,000 per year. Please note that SPARKS are unable to consider applications which are concurrently submitted to other funding bodies, and do not fund
Outline process: Prior to submitting a full application, all applicants are required to complete an outline proposal form which is available from: Dr Renny Leach Email: [email protected] Medical Research Consultancy Linden House Ashdown Road Forest Row East Sussex RH18 5BN Tel/Fax: 01342 825390 Please note that outlines take up to 5 days to review. Suitable applicants will be sent an application form (by email in Word format) and a copy of the relating terms and conditions. Closing dates: For year 2004 the deadlines for receipt of full applications will be: 19 March and 15 October 2004. Peer review: All complete applications are assessed by full peer review, firstly by independent external referees and then by the SPARKS Medical Advisory Committee. |
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