BIRMINGHAM CHILDREN'S HOSPITAL RESEARCH FOUNDATION
Current Projects (as at October 2013)
BCHRF Awards 2006 (June & October)
Applicant(s)
1.
Dr H Duncan, Dr H Rowlands,
Dr O Bagshaw
2.
Dr M S Murphy, Dr M Cooper,
Dr N Shaw, Dr C Cummins
Project Title & Lay Summary
Amount
Requested
*Type of
Award
Ref No.
Project Title: Use of trans-oesophageal Doppler to monitor cardiac output in Paediatric
Intensive Care
Lay Summary: Children in Intensive Care often have poor heart function needing fluid or
drugs into a vein to support the heart. Sometimes it is difficult to know how much drug or fluid is
needed. The trans-oesophageal Doppler uses ultrasound to measure how well blood is
pumped from the heart. This information enables us to estimate heart function and decide on
the amount of drugs or fluid needed.
It has been used for several years in adult and children but little is known about normal
measurements for children and how well the measurements match what estimates from
examination of children. We will assess if there is a difference between doctor’s estimates of
heart function and measurements taken from the probe.
Project Title: Adverse Effects of Glucocorticoid Therapy on Bone in Childhood Crohn’s
Disease (CD)
Lay Summary: Crohn’s disease is a life-long condition of recurrent intestinal inflammation. If
not effectively treated it causes severe symptoms and children become malnourished and fail
to grow. It can also cause thinning of the bones (osteoporosis) with a risk of fractures. When
active it is either treated with steroid tablets or a special nutrition treatment in which for 6 weeks
the child must live on an exclusively liquid diet (LDT). Steroids are very effective but may have
side-effects, unfortunately including (like Crohn’s itself) a risk of osteoporosis. LDT is a
challenging and difficult treatment for children and their families. It is not yet agreed which of
these treatments is best, and so we are planning a national study to answer this question. The
present one-year study will be the first-phase of that study. It will focus particularly on the
effects of Crohn’s disease and of its treatment on bone. Using bone scans we will examine the
effects on bone density. Using specialized urine and blood tests we will explore the underlying
biochemical mechanisms.
£14,214.00
RP
BCHRF133
£48,696.00
RP
BCHRF136
Project Title & Lay Summary
Amount
Awarded
*Type of
Award
Ref No.
Project Title: A longitudinal study of bone density and geometry in boys with Duchenne
Muscular Dystrophy (DMD) and the impact of corticosteroids.
£35,974.00
RP
BCHRF161
BCHRF Awards 2007
Applicant(s)
1. Dr NJ Shaw, Dr H Roper, Miss
N Crabtree
Lay Summary: Duchenne muscular dystrophy is a disorder which mainly affects boys and
causes progressive muscle weakness such that they are unable to walk by their mid-teens.
Currently corticosteroids are used to improve muscle strength to enable the boys to remain
mobile for longer. There are concerns however, about possible side effects of steroids,
particularly the fact that they can cause thinning of bones which may lead to a risk of fracture.
This longitudinal study will examine bone density and bone geometry in boys with DMD before
1 of 12
Version 2, October 2013
BIRMINGHAM CHILDREN'S HOSPITAL RESEARCH FOUNDATION
Current Projects (as at October 2013)
Applicant(s)
Project Title & Lay Summary
Amount
Awarded
*Type of
Award
Ref No.
£40,981.99
RP
BCHRF164
Project Title & Lay Summary
Amount
Requested
*Type of
Award
Ref No.
Project Title: Developing a research clinic for the study of obesity in children
Lay Summary: Childhood obesity is increasing in the UK, with up to a third of Birmingham
children now affected; we know this is a risk factor for heart disease in adulthood, and fat
children tend to become fat adults. We do not however understand the complications of fatness
in childhood, or its relationship with diet and exercise. We plan to set up a research clinic to
study obesity in childhood; its associated complications; and what are the predictors for future
development of heart disease. We hope to use this as a platform for designing intervention
studies to treat obesity in the future. We also plan to offer children participation in an exercise
study based on dancing, to improve their cardiorespiratory fitness.
Project Title: A new treatment of children with refractory epilepsy using repetitive transcranial
magnetic stimulation (rTMS): pilot study to assess feasibility, acceptability and anti-epileptic
effects.
Lay Summary: Some children with severe epilepsy have frequent fits which cannot be
controlled by anti-epileptic drugs, or by surgical treatment. Adult patients with epilepsy have
been shown to suffer fewer fits after treatment with repetitive transcranial magnetic stimulation
(rTMS). TMS uses a high intensity magnetic field above the scalp to induce tiny electrical
currents in underlying brain, which may block the electrical discharges responsible for epileptic
seizures. The technique is safe and painless. We aim to assess whether this treatment can be
used for children, and whether it is likely to be effective as an additional treatment for children
with poorly controlled fits.
Project Title: Towards a better understanding of hyperglycaemia in critically ill children.
Lay Summary: Every year more around 800 children die in intensive care units across the UK.
£52,793.00
RP
BCHRF169
£48,238.00
ER
BCHRF191
£29,204.00
RP
BCHRF200
and after they commence treatment with steroids. This will provide important information that
will separately identify the effect of the underlying condition on bone density and the potential
detrimental effect of corticosteroids on the skeleton.
2. Mr T Jones, Mr D Barron, Mr R
Willetts, Mr W Brawn
Project Title: Techniques to Reduce Gaseous Microemboli during Cardiopulmonary Bypass.
Lay Summary: Over the last 20 years significant advances in the care of children undergoing
heart surgery have resulted in a mortality rate of less than 5% but despite these advances
brain damage during surgery continues to occur with 25% or 1 in 4 children having long-term
developmental or neurobehavioral problems. Emboli such as small gas bubbles have been
demonstrated in the blood stream during surgery and are known to cause brain damage. The
aim of this laboratory research is to look at the heart lung machine used during heart surgery
and to study different techniques to see if we can reduce the number of gaseous microemboli
in the patient’s circulation. The results of this research will be of immediate patient benefit.
BCHRF Awards 2008
Applicant(s)
1. Prof T Barrett, Drs J Kirk, M
Taylor, M Desai, C Hendriksz,
U Baumann, R Moy, A
MacDonald, C Cummins
2. Dr LM Notghi , Mr P Bill,
Professor S Seri, Dr S Philip,
Dr R Gupta
3. Dr K Morris, Prof S Gough, Dr
D Macrae, Prof D Elborune,
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Version 2, October 2013
BIRMINGHAM CHILDREN'S HOSPITAL RESEARCH FOUNDATION
Current Projects (as at October 2013)
Applicant(s)
Prof F Gao, Mr P Narendran
4. Dr M S Murphy, Dr N
Crabtree, Dr N Shaw
Project Title & Lay Summary
We know that children who develop a high blood sugar whilst in ICU are more likely to die. We
are about to embark on a large trial of 1500 critically ill children to find out whether aggressively
controlling blood sugar with insulin will reduce mortality, as has been suggested in adults. We
propose to use this unique opportunity to improve our understanding of factors associated with
the development of high blood sugar and the response to insulin therapy. Specifically we aim to
investigate, for the first time, the importance of increased body weight and whether
hyperglycaemia is related to a lack of insulin or a resistance to the action of insulin .
Project Title: Bone metabolism, bone mineral density and bone strength in childhood IBD The impact of the biological agent infliximab versus the immune-modulator Azathioprine.
Lay Summary: IBD is a serious and incurable disorder of the digestive system and occurs in
children. It can cause severe symptoms including stomach pain, diarrhoea, weight loss and
lethargy. It can also lead to thinning of the bones (osteoporosis) resulting in fractures. There
are two treatments in common use – azathioprine and infliximab. Both can be very effective in
settling active IBD so that symptoms go away. We want to know if they prevent osteoporosis.
We plan to study children taking these drugs over a one year period. We can study bone
chemical processes using blood and urine tests. We intend to use two types of x-ray scanner
(DXA & pQCT) to study the density of bone in the spine and the arm. pQCT is a very new
technique and may be especially useful.
If these techniques prove effective we will also be able to use them in future clinical studies.
Amount
Requested
*Type of
Award
Ref No.
£45,499.00
RP
BCHRF207
BCHRF Awards – 2009
Applicant(s)
Project Title & Lay Summary
Amount
Requested
*Type of
Award
Ref No.
1. Dr C Patel, Prof E Maher, Dr H
Cox
Project Title: High Resolution Microarray Analysis in Congenital Heart Disease (CHD).
Lay Summary: We wish to identify genes that cause inherited congenital heart disease. This
will enhance the diagnosis and management of children with congenital heart disease. This will
also enable clinicians to counsel families appropriately on the risks to other relatives.
Identification of novel conditions associated with congenital heart disease will enable the
development of new tests to facilitate early diagnosis, provide information into the cause of the
condition, and help guide the development of novel treatment strategies. We are requesting
funding for a “Springboard Fellowship” for Dr Chirag Patel so that he can gain research training
prior to applying for a national research fellowship.
Project Title: Comparison of adrenal recovery after short- and long-term glucocorticoid
therapy.
Lay Summary: Many diseases in children require treatment with steroids (glucocorticoids,
GC). However, GC treatment suppresses the function of the organs that naturally produce GC,
the adrenal glands. Once GC treatment ends, the adrenals need to recover from long-term
suppression. This study investigates how long this period of adrenal recovery lasts. This is
important as the adrenals are urgently needed to fight off infections and react to stress.
£65,090.00
SPRF
BCHRF215
£29,000.00
RP
BCHRF226
2. Drs W Högler, AL McDermott,
M Velangi, N Krone, Prof W
Arlt
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Version 2, October 2013
BIRMINGHAM CHILDREN'S HOSPITAL RESEARCH FOUNDATION
Current Projects (as at October 2013)
Applicant(s)
3. Drs A Peet, P Gissen, C
Hendriksz, A Olson, Prof Z
Kourtzi
Project Title & Lay Summary
Amount
Requested
*Type of
Award
Ref No.
£62,224.00
RP
BCHRF230
Project Title & Lay Summary
Amount
Requested
Ref No.
Project Title: Whole body vibration as an osteogenic treatment for mild osteogenesis
imperfecta.
Lay Summary: Children with osteogenesis imperfecta (OI, brittle bone disease) have weak
bones, weak muscles, fractures and immobility. Milder forms of OI (type 1,4) may not require
drug therapy but would benefit from bone-forming treatment. Whole body vibration with sidealternating platforms uses the body’s neuromotor reflex system to train muscles. Recent
studies in children with cerebral palsy but also OI suggest that this muscle strengthening
therapy also improves bone strength. No study in OI children has compared vibration training
effects to a control group.
Our randomized controlled study assesses the effect of 6 months whole body vibration training
(2x10min/day) on muscle strength, mobility, bone structure and density. 20 children older than
5 years with mild OI will be randomized into a vibration group and a control group. Patients will
be equipped with vibration platforms for home use.
Project Title: Molecular studies of hepatitis C virus mother-child transmission.
Lay Summary: Hepatitis C virus (HCV) is the main cause for liver transplantation in adults.
HCV is transmitted via infected blood and most children with HCV are born to infected mothers.
They have a lifelong risk of developing serious liver disease and liver cancer. We do not
understand why some mothers infect their children during pregnancy and why children have a
milder illness despite being infected with the same viral strain. We plan to study the genetic
£68,044.94
*Type of
Award
RP
BCHRF252
£69,633.00
PhD
BCHRF253
This study compares adrenal recovery after short-term (5 weeks) GC use in children treated
for acute leukaemia and long-term (>2 months) GC use in children treated for airway
disorders, in particular haemangiomas (benign condition affecting blood vessels). Blood tests
and short adrenal stimulation tests will assess adrenal function at various time points after the
last GC dose. In addition, the study explores the comparison of two GC weaning strategies in
the long-term GC group.
Project Title: Non-invasive investigation of brain metabolism and function in children with
neurodegenerative diseases.
Lay Summary: Our research will measure activity in the brains of children with
neurodegenerative diseases so we can detect early signs of the disease and understand
changes that occur as the disease progresses. At the moment, most of these devastating
diseases cannot be cured. Understanding the early signs is critical for diagnosis and for
measuring the success of promising new treatments. Treatments that replace genes, for
example, are now starting to become a real possibility. We will measure brain chemistry and
brain connections. We will also measure brain activity during tasks that exercise vulnerable
areas. We want learn to detect reliable changes in chemistry or activity that signal problems
before the changes in brain structure become clear and advanced.
BCHRF Awards 2010
Applicant(s)
1. Dr Wolfgang Högler, Dr Nick
Shaw, Dr Nicola Crabtree, Ms
Lindsay Rae, Dr Paul Davis
2. Professor Jane McKeating,
Professor Deirdre Kelly
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Version 2, October 2013
BIRMINGHAM CHILDREN'S HOSPITAL RESEARCH FOUNDATION
Current Projects (as at October 2013)
3. Dr Welch, Dr Shaw, Dr Hackett,
Dr Lammas, Dr Akula
4. Dr Paul Gissen, Dr James
Davison, Dr Phillip Newsome,
Professor Jon Frampton
5. Andrew Peet, Pam Kearns,
Lesley MacPherson, Nigel
Davies, Theo Arvanitis
structure of the virus in mothers and their children using molecular and biological methods to
discriminate between transmitting and non-transmitting virus strains. These studies will help us
understand the molecular events that control vertical HCV transmission and will allow us to
direct future treatment.
Project Title: Vitamin D and innate immunity in children screened for tuberculosis.
Lay Summary: Vitamin D is made by the action of sunlight on skin, with a small amount being
obtained from diet. Lack of vitamin D is known to cause the skeletal disease rickets, but it also
has many other actions, including an effect on the immune system. Both vitamin D deficiency
and tuberculosis are more common in non-white populations in the UK. This may be because
of sociological association or a direct biological effect. We plan to measure firstly whether
vitamin D levels differ between groups of children with different outcomes after household
exposure to TB. In those children with active TB disease, we will investigate whether
supplementation with vitamin D has a measurable effect on activation of their immune system.
Project Title: Generation of Induced Pluripotent Stem Cells from Fibroblastic Cells obtained
from Patients with Inherited Disorders of Protein Metabolism.
Lay Summary: In this project we aim to study severe genetic disorders in which patients
cannot normally degrade proteins in the liver. Liver transplantation can prevent brain damage
in these disorders but is a high-risk procedure. Recent developments allow patient skin cells to
be converted in the laboratory into normal liver cells. The converted cells can be used for
designing treatments in PA and OTC. Potential drugs can be tested on the patient-derived
cells for their efficacy. Novel technologies allow correction of the genetic defect in the patient
cells, which can then be given back to the patient to provide a life-long cure. As the cells come
originally from the same patient, they will not require immunity lowering drugs to combat
rejection.
Project Title: 31P Magnetic Resonance Spectroscopy as an early biomarker of tumour
response in childhood tumours.
Lay Summary: How quickly tumours respond to treatment is a very good indicator of long term
outcome. Improving methods for determining tumour response earlier in treatment is an
important research goal allowing effective treatments to be continued and ineffective ones to
be stopped in a timely manner. In this project we will assess a new method 31P Magnetic
Resonance Spectroscopy (31P MRS) for determining treatment response in children with
cancer. The method is closely related to the MRI scans which are a routine part of the
children’s care ensuring that the method could be incorporated into current practice quickly and
easily. 31P MRS shows particular promise for assessing new drugs which are being developed
for combating cancer.
£9,004.00
SP
BCHRF255
£67,228.00
RP
BCHRF258
£69,737.00
RP
BCHRF261
Project Title & Lay Summary
Amount
Requested
*Type of
Award
Ref No.
Project Title: Generation of hepatocytes from fibroblastic cells obtained from patients with
progressive familial intrahepatic cholestasis (pfic).
Lay Summary: Progressive familial intrahepatic cholestasis (PFIC) is a devastating childhood
£10,000.00
SP
BCHRF264
BCHRF Awards 2011
Applicant(s)
1. Dr N V Morgan, Prof D Kelly,
Prof J Frampton
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Version 2, October 2013
BIRMINGHAM CHILDREN'S HOSPITAL RESEARCH FOUNDATION
Current Projects (as at October 2013)
Applicant(s)
2. Dr C McConville, Dr G Kapatai,
Dr MA Brundler, Dr H
Jenkinson, Mr M Parulekar
3. Dr François-Xavier Li, Dr Clive
Ryder, Dr Penny Davis, Dr
Kathy Bailey, Ms Janis Scott,
Ms Rosanne Wilshire, Nigel
Jones, Ms Kate Cotter and
Professor Tauny Southwood
4. Prof. Stefano Seri , Dr. N. J.
Thai, Prof. P. L. Furlong, Dr.
Lesley McPherson, Mr. A. R.
Walsh
Project Title & Lay Summary
disorder. There is no specific medical treatment that can prevent progression of liver disease.
In this project we aim to generate liver cells from patients with PFIC and then use these cells to
help develop new treatments.
Recent scientific developments have allowed patient skin cells to be converted in the laboratory
into liver cells very similar to those that come from the patients. The patient-derived liver cells
can be cultured in the laboratory in large quantities and used to study the molecular changes
that occur with the disease progression.
The knowledge of these molecular changes will help to design specific treatments for PFIC,
which will then be tested in patient-derived liver cells.
Project Title: Clinical Validation of a Molecular Classification of Retinoblastoma.
Lay Summary: Continuing efforts to improve the effectiveness of cancer treatment emphasize
the importance of recognizing that not all cancers of a particular type e.g. retinoblastoma, are
exactly the same. Some retinoblastomas may benefit from one type of treatment while different
treatments may be better for others. In most other types of cancer, assessment of a small
sample or biopsy of the tumour taken at the time of diagnosis may be used to decide on the
best treatment. This is not possible in retinoblastoma, because the cancer is difficult to get at
and trying to remove a piece might increase the risk of the retinoblastoma spreading outside
the eye.
The purpose of our research is to develop alternative non-invasive methods for assessing
retinoblastomas at diagnosis. We will investigate a new technique called MRS. MRS is similar
to an MRI scan, but gives much more detailed information about the retinoblastoma. This will
allow us to understand more about what makes the retinoblastoma grow, and what type of
treatment it is most likely to respond to.
Project Title: Assessing the influence of custom moulded foot orthotics on the gait
characteristics of hypermobile children and young people.
Lay Summary: Children with flexible joints and flat feet may develop musculoskeletal pain.
Treatment with shoe orthotics reduces pain in some children. We want to understand if
wearing orthotics changes walking patterns, and if those changes are associated with
improvement in pain. In this small pilot project, we will ask 40 children and young people (20
controls and 20 with flat feet and musculoskeletal pain) to have their walking patterns analysed
before and during the use of shoe orthotics. We will also ask them to fill out a simple pain
questionnaire to see if their pain is reduced when they use orthotics. We will use the
information from this project to apply for a larger, externally funded research grant.
Project Title: Identification of seizure-related secondary neural damage with MRI morphometry
in children with symptomatic focal epilepsies.
Lay Summary: In many children with epilepsy, attacks start in one part of the brain; we call
these “focal”. We have learned from research in animals that with repeated attacks other parts
of the brain might “learn” to fire independently from the original area. Today we can have very
detailed pictures of the brain using a test called MRI and measure accurately with
computerised methods the composition of specific brain areas. By comparing the MRI pictures
of children with epilepsy with those of non-epileptic children, we will start to know how the brain
Amount
Requested
*Type of
Award
Ref No.
£50,934.00
RP
BCHRF269
£9,968.00
SP
BCHRF272
£40,770.00
ER
BCHRF280
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Version 2, October 2013
BIRMINGHAM CHILDREN'S HOSPITAL RESEARCH FOUNDATION
Current Projects (as at October 2013)
Applicant(s)
5. Aaron Ranasinghe, Shiao-Yng
Chan, Robert Bonser, Timothy
Jones, Christopher McCabe,
Jayne Franklyn
6. Andrew Peet, Martin Wilson,
Nigel Davies, Theo Arvanitis
7. Dr Nigel P Davies, Dr Andrew C
Peet, Dr Theodoros Arvanitis
Project Title & Lay Summary
reacts to repeated attacks, what events in the history of the patient play a facilitating role, and
in the future how to limit the effect of these attacks.
Project Title: Tissue specific hypothyroidism and the heart.
Lay Summary: Children born with complex heart conditions may have reduced circulating
levels of oxygen. Surgical repair of these conditions is complex and after such surgery children
will be looked after on an intensive care unit with drugs and machines helping both their heart
and breathing functions. One potential reason for the heart needing support after surgery could
be a reduction in levels of crucial hormones (thyroid hormone) that influence the function of the
heart. We aim to study the hormonal deficiencies that occur within the heart of babies
undergoing cardiac surgery with the aim of understanding why some have poorly functioning
hearts and how it may be possible to best direct treatments to make the heart function better
after such operations.
Project Title: Novel metabolite biomarkers of prognosis in childhood tumours.
Lay Summary: Brain tumours are an important cause of death in childhood and new strategies
are required to improve their treatment. MRI scans are used extensively for diagnosis and
monitoring response to treatment. Recent advances have allowed these scans to measure the
chemical composition of tumours and we have established that four chemicals are linked to
tumour aggressiveness. Measuring these chemicals is very challenging but a new research
scanner gives us the opportunity to measure them accurately enough to be used clinically in
tailoring treatment to the individual. In this project we will optimise the measurement of these
chemicals in children with brain tumours and investigate why they are important by performing
experiments on tumour cells grown in the laboratory.
Project Title: Probing the tissue microenvironment of tumours by Magnetic Resonance
Imaging.
Lay Summary: Cancer kills more children than any other disease with diminishing
improvements in survival rates implying that new strategies are required to improve their
treatment. MRI scans are routinely used for diagnosis and monitoring response to treatment,
but the results are often inconclusive. Research has shown that tumour aggressiveness and
resistance to treatment may be linked to certain factors of the tumour microenvironment. In this
project, a unique analysis of advanced MRI data will be used to make several independent and
complementary measurements sensitive to the tumour microenvironment. We will investigate
how these factors are related to each other and their importance in relation to the
aggressiveness and response to treatment by applying these measurements to new and
existing data from childhood brain tumours.
Amount
Requested
*Type of
Award
Ref No.
£69,807.00
RP
BCHRF281
£68,276.00
PhD
BCHRF287
£65,082.00
RP
BCHRF288
*Type of
Award
Ref No.
RP
BCHRF297
BCHRF Awards 2012
Applicant(s)
1. Dr. Wenbin Wei, Dr. Pamela
Kearns, Prof. Paul Murray, Dr.
Project Title & Lay Summary
Project Title: Discovery and prioritization of frequently mutated genes in tumour cells of
paediatric Hodgkin’s lymphoma through whole genome amplification and whole exome
Amount
Requested
£69,539.00
7 of 12
Version 2, October 2013
BIRMINGHAM CHILDREN'S HOSPITAL RESEARCH FOUNDATION
Current Projects (as at October 2013)
Applicant(s)
Fathima Zumla Cader
2. Profs. Mark D. Kilby & Paul
Moss, Drs David Lissauer &
Philip Cox
3. Dr A K Ewer, Prof N Hall, Prof C
Probert,
4. Sharon Evans, Anita MacDonald
Project Title & Lay Summary
sequencing.
Lay Summary: Hodgkin's lymphoma is a type of blood cancer that affects children and
young people. Many kinds of blood cancer are driven by genetic changes or mutations
that occur in the cancer cells. Identifying these mutations has already allowed the
development of new therapies for some forms of blood cancer. Unfortunately to date our
knowledge of the nature of the genetic alterations that characterize Hodgkin’s lymphoma
has been limited to a handful of studies of single genes in adults with this disease. This
research will use new highly sensitive technologies to discover the genome-wide genetic
changes responsible for driving Hodgkin’s lymphoma in children. This knowledge will
provide the scientific basis for the development of better and less toxic treatments for
children with this disease.
Project Title: Villitis of unknown aetiology: A potential process of fetal allograft rejection.
Lay Summary: Babies not growing properly in the womb are born too small and too soon.
This can have long term health consequences for that child, which even last into
adulthood.
A relatively common problem called “Villitis of Unknown Aetiology” is associated with poor
fetal growth and complications. In this condition white blood cells invade the placenta and
cause damage but we don’t understand why. We propose that these cells are recognising
the baby as foreign and are trying to ‘reject’ the baby.
This project aims to identify in detail the processes occurring in this disease and
understand exactly what the role of these white cells are and what attracts them to the
placenta. We hope this knowledge will help explain why this problem occurs and suggest
ways it can be prevented or treated.
Project Title: Faecal microbiota in preterm infants developing Necrotising Enterocolitis
(NEC).
Lay Summary: NEC damages the bowel of premature babies. NEC is a serious condition;
a lot of babies die from it and may need emergency surgery. Babies who survive after
serious NEC often have long-term health problems. The main difficulty with NEC is that we
are not sure what causes it and therefore we don’t have an effective treatment. Bacteria
are thought to be involved but they are difficult to identify and we don’t know which ones
are harmful. We are proposing to use new, state of the art techniques to see if we can
identify differences in the bacteria in the faeces of babies who get NEC compared to those
who don’t. If we can identify differences then we may be able to target treatment to reduce
the risk of them developing NEC.
Project Title: Taste preferences of young children with phenylketonuria (PKU).
Lay Summary: In this observational 2-part study the flavour preference of 25 children,
aged 4-8y with phenylketonuria (PKU) on a restricted low protein diet will be compared
with 25 age/gender-matched children on normal diets. Part 1: accompanied by a Play
Therapist subjects will taste 10 familiar puree foods in random order rating each as
“yummy”, “just okay” or “yucky” using pictorial representations of faces and then rank the
foods in order of preference. For validity, children will be asked to feed the “yummy”
Amount
Requested
*Type of
Award
Ref No.
£70,214.00
PhD
BCHRF298
£69,150.00
RP
BCHRF304
£10,000.00
SP
BCHRF308
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Version 2, October 2013
BIRMINGHAM CHILDREN'S HOSPITAL RESEARCH FOUNDATION
Current Projects (as at October 2013)
Applicant(s)
5. Dr Girish Gupte, Prof David
Adams, Sister Lindsay Hogg, Mrs
Lisa Morris.
6. Springboard Fellow: Dr Archana
Singh
Applicants: Dr Andrew Peet, Dr
Zumla F Cader, Dr Jessica
Edwards, Prof Paul Murray.
7. Drs Andrew Peet, Jan Novak,
Theodoros Arvanitis, Nigel
Davies, David Hobin, Lesley
MacPherson.
8. Drs Reinout J Mildner, Hari
Krishnan, Jim Gray.
Project Title & Lay Summary
purees to a “friendly” doll and the “yucky” purees to an “unfriendly” doll. Part 2: this
procedure will be repeated 2-weeks later. A food frequency questionnaire will be
completed by caregivers to indicate how often children eat certain foods.
Project Title: Immunological markers in liver and small bowel transplantation.
Lay Summary: Long term survival of liver and intestinal transplant grafts are adversely
affected by rejection and opportunistic infection. Differentiating between rejection and
infection (usually by invasive procedures done in hospital ) is important as treatment is
exactly opposite and if not treated at an early stage can lead to graft loss & death . In
other inflammatory conditions involving the intestine and liver, key players of the body’s
immune system are activated and we propose that the same markers can be identified in
blood/stomal fluid after transplantation. These tests may enable us to detect
rejection/infection at an early stage on blood /stomal fluid tests avoiding hospital
admission for invasive procedures, disruption of family life and improve long-term survival.
Project Title: Therapeutic targeting of autotaxin in poor prognosis paediatric high grade
gliomas.
Lay Summary: High grade gliomas are devastating in childhood, being a leading cause of
deaths due to cancer. One of the key problems is that these tumours invade surrounding
normal brain tissue making them difficult to treat effectively. For this reason, inhibiting
glioma cell invasion has the potential to improve outcomes for these children. We have
recently identified a new pathway which can promote glioma cell invasion. In this study we
will test a range of therapeutic agents for their ability to disrupt this pathway. We will also
explore if the measurement of certain by-products of this pathway using a non-invasive
imaging method, can be used to assess how well these inhibitors work in our experimental
models prior to application in patients.
Project Title: Imaging tumour perfusion using magnetic resonance without injecting
contrast agent.
Lay Summary: One of the ways children with cancer are treated is by using drugs which
disrupt blood vessels in the tumour thereby starving it of oxygen and nutrients. The effect
of these drugs on the blood vessels can be measured by injecting a dye into one of the
patient’s veins and scanning the tumour using MRI to see its uptake. However, this is very
difficult to do, particularly in young children. The aim of this project is to measure blood
flow in tumours using MRI without injecting a dye. We will then see if this method, called
arterial spin labeling, can detect changes in blood flow in the tumour when the children are
treated. Doing this will help us to see whether the treatments we give are working.
Can a procalcitonin guided algorithm reduce antibiotic exposure in children admitted to
Paediatric Intensive Care Unit?
Lay Summary: When children are treated with antibiotics for severe infections the
duration of treatment is usually determined empirically, which may cause antibiotic
overuse. This increases the risk of bacteria becoming resistant, risk of complications and
overall costs. During severe bacterial infections the levels of procalcitonin (PCT, a
naturally occurring hormone) rise sharply. Measuring PCT levels to guide antibiotic
Amount
Requested
*Type of
Award
Ref No.
£10,000.00
SP
BCHRF311
£68,019.00
SPRF
BCHRF314
£64,163.00
RP
BCHRF316
£9,600.00
SP
BCHRF323
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BIRMINGHAM CHILDREN'S HOSPITAL RESEARCH FOUNDATION
Current Projects (as at October 2013)
Applicant(s)
9. Scholefield B, Duncan H, Morris
K, Wassemer E, Gupta R, Notghi
L, Bill P, Matam R.
Project Title & Lay Summary
treatment has been shown to reduce antibiotic exposure in adults. We want to study 50
children undergoing treatment for severe bacterial infections in PICU. By measuring the
PCT level daily and looking back, we will assess how many days of antibiotic treatment
could have been avoided had the clinical team known the PCT level and what the
potential cost saving would be.
Project Title: Optimising the detection of seizures on the paediatric intensive care unit
(PICU) using cerebral function monitoring (CFAM) (The CFAM-PIC Study).
Lay Summary: Critically ill children in intensive care are at risk of developing seizures. If
seizures occur and are not quickly treated, brain injury and eventually death can occur.
Identification of these seizures is often difficult due to administration of medication which
stops muscle movement and therefore masks the seizure. We plan to evaluate the use of
a continuous brainwave monitoring system (CFAM) which can identify seizures in
intensive care patients. We will be assessing if CFAM allows bed-side clinical staff to
quickly and accurately identify seizures and in addition whether automatic computer
software can be helpful. We hope that by understanding any problems with these two
methods, we can improve identification rates, potentially reducing brain injury and death in
children.
Amount
Requested
*Type of
Award
Ref No.
£37,621.34
ER
BCHRF329
Amount
Requested
£45,726.21
Type of
Award
PhD
BCHRF349
£68,287.00
RP
BCHRF330
BCHRF Awards 2013
Applicant(s)
1. Prof S Seri, Dr G Woodhall
2. Dr F Mussai, Dr C DeSanto
Project Title & Lay Summary
Project Title: A translational study of neural mechanisms of drug resistance in paediatric
epilepsy.
Lay Summary: Advances in understanding why certain people with epilepsy don’t
respond to medications have relied on creating small lesions in animals’ brains to study
the mechanisms of generation of the attack and how specific chemicals modify these.
These models cannot capture what happens in the human brain and what brain changes
underlie drug-resistant epilepsy in children. We propose to study the electrical activity of
the brain in children before surgery and from brain tissue taken during surgery to record
the activity from individual brain cells and networks of cells to understand what leads to
seizures and how these respond to new and old anti-seizure drugs. This will enable us to
elucidate the mechanisms of seizure generation in paediatric epilepsy.
Project Title: The role of myeloid-derived suppressor cells (MDSCs) in Neuroblastoma.
Lay Summary: Neuroblastoma is the common extracranial solid cancer of childhood and
patients with metastases (spread) have an extremely poor survival. The immune system is
a powerful way in which the body can remove abnormal cells.
We have preliminary evidence that neuroblastoma tumours can switch off the immune
system, preventing them being attacked and killed. This occurs due to a group of cells,
called MDSCs, found in the blood and tumours of patients. This study will investigate how
MDSCs in neuroblastoma tumours switch off the immune response. In addition we will
Ref No.
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Version 2, October 2013
BIRMINGHAM CHILDREN'S HOSPITAL RESEARCH FOUNDATION
Current Projects (as at October 2013)
Applicant(s)
3. Dr C McConville, Prof T
Stankovic, Dr H Jenkinson, Mr M
Parulekar
4. Prof A Peet, Dr M Wilson, Dr T
Arvanitis, Dr B Coyle, Prof R
Grundy
5. Prof T Barrett, Dr C Hardy, Dr H
Cangul, Prof E Maher
KEY:
Project Title & Lay Summary
identify whether molecules released can predict which patients will respond to therapy.
The Mussai laboratory is leading the research, in collaboration with Great Ormond Street,
Children’s Hospital Oxford, and international partners.
Project Title: Development of new approaches to chemotherapy for retinoblastoma.
Lay Summary: Retinoblastoma is a cancer of the eye which occurs in very young
children. If diagnosed in good time, and treated promptly, usually by removing the eye
(enucleation), survival is excellent (≥ 95%). Untreated retinoblastoma which spreads
outside the eye is often fatal. There is increasing interest in looking for better ways to
treat retinoblastoma which would allow some preservation of sight, particularly for
children with retinoblastoma in both eyes (approximately 40% of all patients).
The purpose of our research is to investigate new approaches to chemotherapy for
retinoblastoma. By studying retinoblastoma cells in the laboratory, we will find out how
they respond to different types of chemotherapy drugs (including some very new types of
drugs) and how these might best be used to kill the cancer cells while at the same time
minimizing damage to the rest of the eye.
Project Title: Metabolite profiles as a means of identifying genetic subtypes of
medulloblastoma.
Lay Summary: Medulloblastoma is the most common brain cancer in children. We have
invented a scan called MRS which can diagnose these tumours without a biopsy.
However, recently it has been found that there are actually four different types of this
tumour and we wish to find out whether our new scans can tell the difference between
these four types. The MRS scans are based on looking at chemical makeup and so we will
first look at the chemical make-up of the tumours in the laboratory. When we have shown
that the chemical make-up is different between the four types of medulloblastoma we will
use this to adapt our MRS scans to diagnose tumours more accurately in the children.
Project Title: Development of molecular genetic testing service for children with
congenital hypothyroidism
Lay Summary: About 1 in 4,000 children are born without a working thyroid gland. The
thyroid is needed to make thyroxine, an essential hormone for health and wellbeing. We
don’t know why this happens, but believe there is a combination of genetic and
environmental factors. Over the last 15 years, genetic causes of this problem have been
identified; but so far, there is no genetic test available on the NHS; so it is difficult to
counsel families about future risks, or what the long term outcome will be for their child.
This project builds on our previous work in identifying genetic causes of congenital
hypothyroidism, to develop a genetic test on the NHS that we can offer to families in the
future.
Amount
Requested
Type of
Award
Ref No.
£51,994.00
RP
BCHRF344
£59,520.00
RP
BCHRF353
£10,000.00
SP
BCHRF347
* Type of Award:
SPRF = Springboard Paediatric Research Fellowship (Max. £70,000)
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Version 2, October 2013
BIRMINGHAM CHILDREN'S HOSPITAL RESEARCH FOUNDATION
Current Projects (as at October 2013)
SNAHCPRF = Springboard Nursing & Allied Health Care Professional Research Fellowship (Max. £70,000)
PhD = PhD Studentship (Max. £70,000)
RP = Research Project (Max. £70,000)
SP = Small Project Grant (Max. £10,000)
NP = Nursing Project (Max. £10,000)
ER = Epilepsy Research (Max. £50,000 or small project grants up to £10,000)
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