Researching a new treatment for twin

touching
lives
The Action Medical Research magazine
Autumn/winter 2014
Researching
a
new treatment for
twin-twin transfusion
syndrome to save
babies’ lives
Read Isabella’s story
Plus
A new surgical technique
that could save children’s
sight
Could you lend a hand?
It’s a high five for
volunteering
Dear supporter
Welcome to our autumn/winter
issue of
Touching Lives – where you can rea
d about
our work helping sick babies and
children
and the fundraising and suppor t
that
makes vital research possible.
Isabella, who lights up our front cov
er,
has a fighting spirit, a warm heart
– and a baby brother to play
with. But tragically, parents Laura
and Ryan lost her identical twin
sister Grace at birth through twin-tw
in transfusion syndrome. Read
about a new treatment being dev
eloped that Action is funding, and
the inspirational braver y of this you
ng family, on pages 8-10.
On page 4, you can find out how
research that Action funded back
in the1970s means that children
like Emma are now being saved
from lifelong learning disability.
New research includes work to help
children like Tom, six, who
has Duchenne muscular dystrophy
. A new medicine being tested
by researchers at Newcastle Univer
sity could slow down the
progression of this devastating, fata
l disease. On page 11 Tom’s
mum Rebecca explains what hop
e means to them.
Our celebrity suppor ters have bee
n busy again: TV chef Lesley
Waters fronted this year’s Action
Cream Teas in a box fundraiser ;
our cycling events were sprinkled
with stardust as big names from
the world of comedy, TV and spo
rt lent us their suppor t; and this
winter our friendly, furr y mascot
Paddington™ stars in his ver y
own movie!
Of course, you don’t need to be
famous (or furr y) to get involved
and make a difference. Why not lend
a hand as a community
volunteer? See page 15 to find out
more.
It won’t be long until the leaves star
t to fall, the air cools and
thoughts turn to the festive season
. Ordering your Christmas cards
from Action is a great way to sup
por t our work, so do check out
the wonderful, contemporary des
igns on our website. Every single
penny raised comes to Action, help
ing us to help sick babies and
children, and that’s something to
celebrate at any time of year.
Touching Lives is Action
Medical Research’s magazine
for supporters.
Interim Editor: Kate Lee
Front cover photo: Ben Rector
Print: NB Group, UK
2
Please send all
communications to:
The Editor, Touching Lives,
Action Medical Research,
Vincent House, Horsham,
West Sussex RH12 2DP
T 01403 210406
E [email protected]
Waction.org.uk
facebook.com/actionmedres
twitter.com/actionmedres
Touching Lives is also available to
download at action.org.uk
touchinglives Autumn/winter 2014 4
8
11
news We celebrate
3 Fundraising
celebrities in the saddle, sweet treats
in a box, and Paddington in his
own movie!
Our impact How newborn
4 screening
is saving children from
lifelong learning disability
Your money in Action
6 Read
about progress made in
research funded with your help
a legacy Big or small, every
7 Leave
legacy makes a difference
feature Research into
8 Cover
twin-twin transfusion syndrome
offers hope for the future
research Helping children
11 Nwithewcancer,
a new approach to
tackling asthma, surgery that saves
children’s sight, and a vital new
medicine for boys with Duchenne
muscular dystrophy
undraising news A Downing
14 FStreet
reception, the amazing work of
our corporate Action Partners, and a
feature on community volunteering
Kate Lee, Interim Editor
Action Medical Research is a
UK-wide charity saving and
changing children’s lives through
medical research.
Contents
We are keen to share, but you
must ask first! If you see an
article or a picture you would
like to copy or reproduce for
use elsewhere, please contact
the editor.
events Running, cycling
16 Corhallenge
walking … sign up for something
special in 2015
© Action Medical Research
2014
Action Medical Research
is a registered charity:
England and Wales no. 208701;
Scotland no. SC039284
action.org.uk
Fundraising news
Budding film star Paddington Bear™
The film follows the comic
misadventures of the young Peruvian
bear with a passion for all things
British, who travels to London in
search of a home. Finding himself
What have our other
celebrity supporters
been up to?
Davina McCall and
Fiona Bruce joined
Action guests for a
special reception
hosted by Samantha
Cameron at 10 Downing Street.
Davina McCall was on hand to
cheer riders at the DIVA Sussex
bike ride, having suffered an injury
that kept her off the bike this
time. She will be back hosting her
annual Dine with Davina ladies’
lunch in November.
Nicky Forster, former
professional footballer
turned Sky Sports
Football League pundit,
did the Castle Ride 100,
along with Location,
Location, Location’s
Phil Spencer.
action.org.uk Paddington has been at the heart
of fundraising for Action Medical
Research ever since Michael Bond
TV presenter
AJ Odudu
joined ladies
riding our DIVA
Chiltern event in June
and will be supporting us
again later this month in the
Great North Run.
Long-term supporter Dr Dawn
Harper was the voice of our
charity appeal which aired on
Radio 4 in the summer.
Comedienne Vikki Stone got on
her bike for the Warwickshire 100
ride, tackling the toughest
100-mile route.
Matteo Carrara, Italian
pro-cyclist and winner of the 2010
Tour de Luxembourg, took part in
the Suffolk Sunrise 100.
TV chef Lesley
Waters fronted our
Action Cream Teas
event in June.
n and Co Ltd/Stu
diocanal S.A. 201
4
met the charity’s
founder, Duncan
Guthrie, in 1976.
Paddington Bear
always likes to help
wherever he can and
so, after chatting over
the matter with
Mr Bond, he agreed
to support Action as
the charity’s official
mascot. Paddington
Bear’s friendly face
has helped us attract
thousands of new
supporters who together have
raised millions of pounds to help
babies and children.
r™ © Paddingto
He takes to the big screen on
28 November and is supported by
a stellar cast of celebrities including
Hugh Bonneville, Nicole Kidman, Sally
Hawkins, Julie Walters, Peter Capaldi,
Jim Broadbent and Ben Whishaw as
the voice of Paddington.
lost and alone at
Paddington Station,
he begins to realise
that city life is not all
he had imagined –
until he meets the
kindly Brown family,
who read the label
around his neck
(‘Please look after
this bear. Thank
you.’) and offer
him a temporary
haven. It looks as
though his luck has changed until this
rarest of bears catches the eye
of a museum taxidermist …
Paddington Bea
Our much-loved
mascot Paddington
Bear is always busy
on and this year even
t
g
in
d
d
a
P
more so as he stars
Movie
in the first ever live
action film inspired by Michael
Bond’s best-selling books.
Look out for Paddington, coming to
a cinema near you!
Tweet, tweet. . .
Davina McCall @ThisisDavina
First pit stop of the
@actionmedres ride... Notice
which snack my ladies are going
for!!! Caaaaaaaaaaaaaarrrrbbbb
Thank you for helping
@actionmedres
Peter James @peterjamesuk
Wonderful children’s charity I
support, @actionmedres need
volunteers. Can you lend a hand?
http://bit.ly/1frJnXW
Phil Spencer @pipofftheTV
enjoyed his Action Cream Tea
with his family – Did you enjoy
it? Order yours now!
Fab kids charity and cycling
events @actionmedres
Can you lend them a hand?
#VolunteersWeek http://bit.
ly/1frJnXW
Follow us at @actionmedres
and @amr_events
touchinglives Autumn/winter 2014
3
How screening saves children
from lifelong learning disability
A research project funded by Action Medical Research in the 1970s led to routine
screening in the UK of newborn babies for the rare metabolic condition congenital
hypothyroidism (CHT). Now, early diagnosis and treatment spares children like Emma
from lifelong physical, developmental and learning disabilities.
For adults, the hormone thyroxine
is important in maintaining energy
levels to allow everyday activity. But
in children, and especially in babies,
it has another vital function: it is
essential for the normal growth and
development of the brain and body.
At around six weeks after
conception, a baby’s thyroid gland
moves from the back of the
tongue down the neck to its usual
position just below the Adam’s
apple. In babies with congenital
hypothydroidism (CHT) this gland
may not be fully developed, it may
not have developed at all, or it may
not have moved to the correct
position. Problems with the
production of the hormone
thyroxine, or very low levels
of thyroid stimulating
hormone, can also cause CHT.
If left untreated, the condition
can cause lifelong physical,
developmental and learning
disabilities, including severe
learning disability and difficulty
with walking, talking and
feeding. For some children,
this means they may
require lifelong care.
Yet the condition can easily be
treated with a synthetic form of
thyroxine in liquid form, suitable for
tiny babies, or in tablets.
In 1978, Action funded a one-year
pilot study led by the late Professor
Barbara Clayton at Great Ormond
Street Hospital, to screen for CHT
in babies in the north east and
north west Thames regions.
Professor Clayton was already
responsible for ‘heel prick’
testing (now known as ‘newborn
screening’) for the condition
phenylketonuria (PKU) in this
region, and proposed that CHT
screening could be linked to this.
During the pilot, more than 87,000
babies were screened and 26 were
diagnosed with CHT – around twice
as many as estimated at the start of
the study. Researchers found that
more than 92 per cent of babies
diagnosed with CHT through the
early heel prick test were identified
before their symptoms became
apparent.
The research, which was extended
following funding from the Medical
Research Council, showed that early
diagnosis and treatment helps save
the vast majority of babies with
CHT from lifelong learning disability.
In the early 1980s routine CHT
screening of all newborn babies was
introduced throughout the UK.
The team at Evelina
London Children’s
Hospital monitor
Emma on a six
month basis
4
touchinglives Autumn/winter 2014 action.org.uk
For Nicky and Simon, whose
daughter Emma was diagnosed
with CHT as a newborn baby, the
research funded by Action has
made an enormous difference.
“We are so grateful for the research
which has been done to reach this
point, where this rare condition can
be picked up at an early enough
stage to prevent as much as possible
any negative impact on new lives,”
says Nicky.
Emma’s arrival was problem-free,
as Nicky explains: “By the time
Emma was born I was already 40
and so we were delighted when
she arrived with 10 fingers, 10 toes,
and everything appearing to be
in the right place. There were no
complications with the pregnancy or
birth and no signs that Emma had
congenital hypothyroidism.”
But when Emma was just nine
days old, Nicky received a call
from Lewisham Hospital Paediatric
Team asking to see her daughter
as a matter of urgency. “I cannot
remember much about the actual
call itself, other than that a doctor
was telling me that my newborn
daughter, most precious and tiny, was
fundamentally unwell in some way,”
says Nicky.
Photos: Ben Rector
CHT screening had identified a
problem which indicated that Emma’s
body was attempting to ‘kick start’
the function of her thyroid gland.
“We are so lucky that
CHT screening exists
because it enabled
Emma’s condition to
be treated as early as
possible”
A scan showed that Emma’s thyroid
gland was not in the usual position.
Doctors prescribed thyroxine, which
Emma now takes daily as part of
the family’s breakfast routine.
“Had the heel prick test not been
done, then I am unsure how long
it would have been before the
condition was picked up on, by which
point Emma’s mental and physical
development could well have been
compromised,” Nicky reflects.
“We don’t know if there will be
any further challenges ahead for
Emma due to her condition, but
her current physical growth and
mental development appears
to be unimpaired. She is such a
cheeky monkey with a smile that
would melt the coldest heart.
She blows our socks off every
day and we could not be more
proud of her and her brother!”
Around
1,000
babies in the UK have
been saved from
lifelong learning
disability since
the 1980s
Two-year-old Emma is very active and
loves to do the same things as big
brother Oliver
action.org.uk touchinglives Autumn/winter 2014
5
Your money in Action
Brain tumour research to help
children like Jack
If you’re a regular reader of Touching Lives you may
remember Jack, who featured in our spring 2011 issue.
Jack was diagnosed with a brain tumour when he was just
14 months old.
Following a gruelling year of
treatment, involving brain surgery,
chemotherapy and radiotherapy,
Jack thankfully recovered. He is
now nine years old and has been
clear of cancer for almost seven
years. However, having radiotherapy
at such a very young age
has affected his working
memory and he needs
extra support at school.
Ben Rector
His mum Lisa says: “He
is doing amazingly well
and only has an annual
check up now. He
is a very happy
boy and works
very hard. He
likes to play on
his scooter and
loves Lego!
“We met so many lovely families
who weren’t as lucky as us,” she
adds. “That’s why it’s really important
that more is known about this form
of cancer.”
Action funding awarded in 2010
supported Research Training
Fellow Dr Chris Howell in his
bid to improve understanding
and treatment of the type of
brain tumour Jack had, called
medulloblastoma. This is the
most common cause of brain
cancer in children and around
one in 10 of the children
who die from cancer in
the UK have this type
of tumour.
Jack as he appeared in
Touching Lives 2011
Dr Howell has been studying what
changes in genetic factors cause
medulloblastoma in different children
under three. This has revealed
a link between certain types of
genetic change and how well a
child responds to treatment; some
children are more likely to recover
more quickly than others.
This knowledge will help doctors
to better plan and design treatment
for individual children, as well as to
develop new treatments. It means
that some children could be spared
the damaging effects of radiotherapy,
while those with the poorest
chances of survival could receive
more aggressive treatment, newly
developed therapies or focus given
to providing special care to maximise
their quality of life.
This project was supported
by generous donations from
The Freemasons’ Grand Charity
and The Catherine Cookson
Charitable Trust.
Preventing premature labour
Research funded by Action has given
new insights into why some women
go into labour too soon and shown
that it could be possible to develop
a simple test to identify those at
highest risk.
Over 60,000 babies are born
prematurely every year in the UK
and tragically around 1,300 of them
die. Very premature babies who do
survive can spend many weeks in
hospital and are at risk of developing
lifelong disabilities.
Professor Nigel Klein and his team
at the Institute of Child Health and
the Institute for Women’s Health,
University College London, were
6
touchinglives Autumn/winter 2014 awarded £119,332 in 2010. Their
aim was to investigate links between
early labour and changes in the
mother’s immune system. They
believed this made some women
more vulnerable to infection, allowing
bacteria to grow and spread within
the womb, creating an inflammatory
response that could trigger labour.
Their study, involving women
attending a preterm birth clinic,
found that failure to detect certain
infection-fighting blood cells was
indeed a feature in a majority of
pregnancies that ended too soon,
especially where babies were born
before 34 weeks.
The absence of these cells could be
detected as early as 12 weeks into
a pregnancy, creating a window of
opportunity for treatments that may
prevent premature birth. The team
now aims to develop a simple test to
identify women most at risk.
This project was supported by a
generous donation from The Henry
Smith Charity.
action.org.uk
Big or small, your legacy gift
can make a real difference
Despite what many people
think, you don’t have to be
hugely wealthy to leave a
legacy gift and nor do such
gifts need to be large to
make a difference.
We are always extremely grateful to
all those supporters who remember
Action with a gift in their will, helping
us fund more medical breakthroughs
and leaving a brighter future for
children.
While big legacy donations are,
of course, always very much
appreciated, over half of our legacy
gifts last year were for £5,000 or less,
but collectively our legacy income
amounted to more than £1 million
– making a significant difference to
the research projects we could fund.
It costs around £265 to fund a day’s
research. Gifts, whatever their size,
enable us to fund vital research to
help save children from a lifetime
of suffering. We currently can only
afford to fund around half of the
children’s research that we want
to simply because of lack of funds;
research which could well deliver
new treatments or cures for sick
babies and children.
Many people may have limited
opportunity to make significant gifts
to charities during their lifetime, even
if they would like to do so. In such
cases a gift in your will can be an
ideal way to support Action Medical
Research. It costs nothing now but
could have a huge impact on future
generations.
Action’s Chair, Phil Hodkinson,
recognises the vital importance of
legacy funding and together with
his wife Julia, both have pledged
to support us in their wills. “To be
able to play a part, however small,
in potentially unlocking a medical
breakthrough in the future is a
great feeling today,”
he says.
We have produced a new booklet
on legacy giving, explaining how
legacies can help our work and
providing more information on
how to leave a gift in your will.
You can request a copy online at
action.org.uk/legacy, or
contact Jane Tarrant by email at
[email protected] or on
01403 327414.
Recent legacies left to the charity
have included a share in the estate
of Hilda Dodsworth of South
Shields, and gifts from Clifford Seller
of Slough, Patricia Bailey of Stockport
and Betty Cowieson of Fife. We are
extremely grateful to them all for
their generosity in remembering
Action Medical Research
and their names will be
recorded in our Book
of Remembrance at
Vincent House.
touchinglives Autumn/winter 2014
7
“Grace is held
in our hearts if
not in our arms”
8
touchinglives Autumn/winter 2014 Better
treatment
for tiny
Laura and Ryan were thrilled
when they discovered
they were expecting
identical twin girls. Laura
was monitored closely
throughout the pregnancy
and recovered well each time
complications arose. But the
serious condition twin-twin
transfusion syndrome (TTTS)
developed unexpectedly at
a late stage and, tragically,
their daughter Grace did not
survive her birth. Celebrating
baby Isabella’s arrival whilst
mourning the loss of her
twin was, as Laura explains,
agonising.
twins
In TTTS, which estimates suggest
affects around 10 per cent of
identical twins in the womb, blood
moves from one baby to the other
via connections in their shared
placenta. One twin can become
dangerously short of fluid and the
other overloaded. The condition puts
babies at risk of premature birth,
death or disability.
Ben Rector
One of the first signs of this
syndrome is when the babies grow
at different rates. But throughout
Laura’s pregnancy, her twins were
always identical in estimated weights.
Every two weeks Laura watched
her girls growing and getting
stronger. “I felt like the luckiest
girl in the world,” she recalls.
The 20 week scan revealed that
Isabella had a rare abnormality,
congenital cystic adenomatoid
malformation (CCAM), which meant
touchinglives Autumn/winter 2014
9
Surviving twin Isabella,
who underwent open lung
surgery aged six months, is
now a happy two-year-old
the lobe of her developing lung
contained cysts. Most often, the cysts
do not grow rapidly or at all so
cause no problems. Once this was
explained to Laura and Ryan their
minds were put at ease.
A caesarean section was booked for
36 weeks. The delivery day arrived
as scheduled, but tragically their
twin daughter Grace did not survive
the birth. TTTS had developed at
this late stage. Grace had become
critically short of fluid and died.
“We didn’t know how to feel happy
for our surviving baby girl, Isabella.
We felt empty, lost and angry,” Laura
says. “We spent time with Grace
after she was stillborn, kissing her,
taking photographs and making the
only memories we would ever have.”
Isabella’s birth was not without
complications. She was resuscitated
and transferred to a special
care unit where she stayed for
three weeks. “To not be able to
take her home with us and try
to fit in with visiting hours, all
whilst writing funeral invitations
from her cot, was sickening.”
“In the very early days I found it
difficult to bond with our surviving
twin, Isabella. I had spent an entire
pregnancy preparing for the safe
arrival of our two beautiful girls and
that was taken away from us over
night,” she continues.
Two years on, Laura admits: “We still
have difficult days but they are less
frequent. We live our life including
10
touchinglives Autumn/winter 2014 Grace, we talk about her frequently
and have mementos around the
house which make us smile.”
Isabella has a suspected bicuspid
aortic valve in her heart which
still requires regular hospital check
ups, but has already impressed
her parents with her fighting spirit.
After open lung surgery at just six
months old to remove the cysts she
developed due to CCAM, Isabella
came home just two days later.
Having each other has been a
constant source of support for Laura
and Ryan, who are getting married
at the end of this year. And having
a baby son, Blake, was “our rainbow
after the storm,” says Laura.
Setting up their own non-profit
organisation ‘Our Saving Grace’ has
also helped the family. The group
organises teams of befrienders to
help families who have lost one or
both of their twins to TTTS.
In August this year Laura and Ryan
cycled in memory of Grace from
Bristol to London on the Action 100
ride for Action Medical Research.
“We did the ride because we know
we can never change what has
happened to us but we can, and
we will, change it for other families
like us,” Laura explains.
Grace will be mentioned in the
marriage service, in the speeches
and throughout Laura and Ryan’s
wedding day. “She is held by
everyone in their hearts if not in
their arms,” says Laura.
Dr Christoph Lees of Queen
Charlotte’s and Chelsea Hospital,
Imperial College, London, is
in the laboratory stages of
developing a new treatment for
babies with twin-twin transfusion
syndrome (TTTS) using high
intensity focused ultrasound.
The aim is to stop blood moving
between identical twins in
the womb by blocking blood
vessels in their shared placenta.
Currently, women whose babies
develop TTTS may be offered
laser therapy which, although
it boosts babies’ chances of
survival, involves potentially
risky surgery in pregnancy
and possible complications
including miscarriage, premature
birth and infection.
“We believe this new treatment
could be safer than laser therapy,”
Dr Lees says. “It could mean
babies could be treated earlier
in pregnancy, hopefully improving
their chances of survival and
escaping disability.”
Laura fully supports Action’s
research into this new treatment.
“We think the research funded
by Action Medical Research into
TTTS is brilliant. Any treatment
that may effectively be able to
offer the same, if not higher,
survival rates for babies without
the added complications of
surgery would be a breakthrough
with worldwide benefits. We will
continue to support causes such
as this in the hope that one day
the risks for an identical twin
pregnancy are minimal when
compared with current statistics.”
action.org.uk
New research
A new medicine to help children
with Duchenne muscular dystrophy
While there is still no cure for this fatal degenerative disease, a new medicine could slow its
progression. For six-year-old Tom and his family, that means vital hope for the future.
Tom was diagnosed with Duchenne when
he was four, but his parents were concerned
about him from his earliest days. “The
health visitor stressed the importance of
‘tummy time’ to help Tom lift his head, but
he couldn’t do it. He just screamed,” mum
Rebecca remembers. “He was slow to reach
milestones like crawling and walking, too.”
When Tom started school, Rebecca
insisted on a referral to a paediatrician who
explained that several conditions might
be causing Tom’s problems, including mild
cerebral palsy and dyspraxia. He asked to
do a blood test to rule out a couple of
rare conditions, but phoned at 9.00am the
next day with devastating news – Tom had
muscular dystrophy. Children affected by this
rare disease suffer a relentless deterioration
of their physical abilities. They eventually
become paralysed, depending
on others for round-the-clock
care, and the disease is fatal.
Ben Rector
Tom fell over a lot and walked with an
awkward gait. A specialist diagnosed femoral
anteversion – a problem with the way
Tom’s thigh bones were aligned. Rebecca
and husband Gary were told this would
right itself in time, and not to worry.
As a parent, it is difficult to know how
honest to be when Tom asks questions,
Rebecca finds: “If my son asks me when he
will be able to hop, how do I answer that?”
New types of gene therapy may be
able to treat some but, at
present, not all boys with
Around
Duchenne. So Rebecca
is encouraged by the
work led by Professor
boys are diagnosed Volker Straub at
Newcastle University.
“It was such a shock,” Rebecca
with Duchenne
The focus of the
says. “It seemed so improbable that
each year in
project, funded by
my gorgeous son would eventually
the
UK
Action
Medical Research,
become locked in a body he could
is to develop a new
not move and die. Even now, two years
medicine that could increase
on, you feel your heart crack and smash.”
muscle strength and slow down disease
Sadly, further tests revealed that Tom has
progression, by reducing calcium overload
the severe form of the disease known as
in the muscles, and thus improve quality
Duchenne muscular dystrophy. By 10 to
of life. For Tom, this could mean he walks
14 years of age, boys with Duchenne lose
for longer and keeps upper-body skills
the ability to walk and require a wheelchair
such as feeding himself for longer, too.
full time. Advised by Great Ormond
“These potential treatments mean
Street Hospital to be proactive, Rebecca
everything to us,” says Rebecca. “Too many
and Gary bought a dilapidated bungalow
to adapt for Tom and plan to build a
parents have lost their children to this
hydrotherapy pool. “The warmer water is
horrendous condition. It is the thought of
perfect for doing Tom’s stretches and good
those mothers and fathers that makes me
for pain management,” Rebecca notes.
appreciate every second with my son.”
action.org.uk 100
“These
potential
treatments
mean
everything.
Hope keeps
us going”
This project is
supported by
generous donations
from The Henry
Smith Charity
and The Catherine
Cookson Charitable
Trust.
touchinglives Autumn/winter 2014
11
New research
Helping children with cancer
Germ cell tumours develop from cells that
produce eggs or sperm so mostly occur
in the ovaries or testes, but they can also
develop in other parts of the body including
the brain.
Although survival rates are high, sadly some
children still die and current treatments can
have damaging long-term side effects for
those who survive.
Science Photo Library
Action recently awarded a
prestigious grant to a talented
young doctor who plans to
dedicate her career to helping
young cancer patients. Dr Shivani
Bailey received a Research Training
Fellowship of almost £200,000
for a three-year study that aims
to develop a new treatment for
children with germ cell cancers.
amounts in germ cell cancers. Dr Bailey is
investigating whether substances that block
the action of these molecules could be
developed into safer, more effective drug
treatments. If her results are positive, the
next step would be a trial in patients.
Dr Bailey, who is based at Cambridge
“Our ultimate aim is to develop a better
University, says: “Children are currently
treatment for children and young
treated with a combination of
people with germ cell cancers
surgery, chemotherapy and
– one that improves survival
radiotherapy. Parents worry
rates and causes fewer
not only about whether their
long-term side effects, so
child will survive, but also
that patients can live long
are diagnosed with
about the toxicity of these
and healthy lives after
germ
cell
cancer
therapies and how this might
treatment,” she says.
every year in
affect the rest of their
Germ cell cancers can affect
child’s life.
the UK
people of all ages, with testicular
“Children who survive can go on
germ cell cancer being one of the
to develop long-term complications.
commonest causes of cancer in young
For example, chemotherapy is associated
men. Better treatments would be
with hearing loss, and kidney, heart and
welcomed by all.
lung problems.”
Action’s Research Training Fellowship
Dr Bailey hopes to develop a new, non-toxic
scheme has been running for more than
treatment for these young cancer patients.
40 years, supporting promising doctors and
She says: “I strongly believe that clinicians,
researchers early in their careers. In this time
like me, who care for children with cancer
the charity has funded 170 fellowships, worth
are perfectly placed to lead research into
more than £12 million, and many of these
new treatments, with the aim of changing
recipients have gone on to become leaders
children’s lives for the better.”
in their field.
Cells from a germ cell
tumour
45
Up
to
children
With Action funding, Dr Bailey is focusing
on molecules called microRNAs – these
are small pieces of genetic code that help
control protein production in cells. Certain
microRNAs are present in excessive
12
touchinglives Autumn/winter 2014 Dr Bailey says: “I am honoured that Action
Medical Research, a charity that has played
a key role in supporting research into
childhood diseases for many years, has
placed its faith in me and my project.”
Dr Shivani Bailey
“Our
ultimate
aim is to
develop
a better
treatment
for children
and young
people”
action.org.uk
New research
Tackling asthma: a new approach
Around
1.1m
new approach is known as ‘personalised
therapy’ and could help people with
asthma to better control their symptoms.
Every 18 minutes a child is
admitted to hospital in the UK
because of asthma. It is the
most common long-term
medical condition, affecting
one in every 11 children.
When asthma is well managed children can
lead a full and active life but researchers have
found that a commonly prescribed medicine,
called salmeterol, may offer little benefit to
some of those taking it. They have discovered
that a person’s genetic make-up can affect
their response to the drug.
With funding from Action researchers will
investigate if using a simple genetic test and
prescribing medicine accordingly leads to
improved quality of life for sufferers. This
children in the
UK have
asthma
Children with poorly controlled asthma
can struggle to participate in playground
games and sports. They may also miss
school and fall behind in their work, leading
to problems with low self-esteem.
Lead researcher Professor Somnath
Mukhopadhyay, from Brighton’s Royal
Alexandra Children’s Hospital, says: “Our
ultimate aim is to make the most of the
treatments we’ve already got for asthma, so
children get the medicines that suit them
best – and they can enjoy the best possible
quality of life.”
Professor Somnath
Mukhopadhyay
This project is supported by a generous
donation from the Ward Family Charitable
Trust.
Revolutionary new surgery for cataracts
Although more commonly associated with
older people, around 200 babies in the
UK are born each year with cataracts and
children can also develop them at a young
age. The condition affects the eye’s lens,
causing blurred or misty vision. Sadly, some
may lose their sight entirely.
Based at the University of
Aberdeen, an expert team
is studying a new approach
that they believe could
revolutionise the treatment
of cataracts, especially
in children.
Brendan McIlhargey / istock
Prompt surgery can help restore a child’s
vision but currently their sight will often
remain less than perfect. Children are
also more susceptible to a post-surgery
complication, like a secondary cataract,
that can further impair their sight and
require yet more intervention.
removed lens. The problem is that artificial
lenses cannot focus as well as natural ones,
meaning children can still face a life
of restricted vision.
Estimates
suggest up to
With Action funding, the
research team are developing
a new technique which they
children worldwide believe could encourage
the eye’s lens to grow back
have lost their
naturally after surgery
sight to
During current surgery the
– something that was previously
cataracts
cataracts are removed and the
thought impossible. This could
lens is replaced with an artificial
greatly improve the sight of affected
one, or the child will wear contact
children and could eventually benefit older
lenses or glasses to compensate for the
people with cataracts too.
action.org.uk 210,000
touchinglives Autumn/winter 2014
13
Fundraising news
Downing Street reception
Action guests were welcomed
behind the door of number
10 at a Downing Street reception
hosted by the Prime Minister’s
wife Samantha Cameron.
Action Celebrity Supporters
attended this special event, including
Davina McCall and Fiona Bruce,
who spoke about the importance
of our research. A group of doctors
and professors also shared their
vital charity-funded work, focusing
on premature birth, seizures in
babies, Down syndrome and the
rare genetic condition X-linked
lymphoproliferative disease.
The event was a wonderful
opportunity for the charity to
highlight its successes, as well as the
ongoing need for further funding.
Action gave certificates of
acknowledgement signed by
Fiona Bruce to Allen Ford, Quai
Administrative Services and One
Stop, all companies who have raised
considerable funds for the charity.
Thank you, Action Partners!
Arun Estates, the estate agency
business with 110 branches across
the south east, has supported Action
for the past four years, raising over
£70,000. The partnership started
when a small group, led by Managing
Director David Lench, entered
the Three Peaks Bike and Hike in
2010. Since then they have tackled
Kilimanjaro and Race the Sun, and
David is taking
on the personal
challenge of
cycling from
Land’s End to
John O’Groats
in September.
whole Group for their enthusiasm,
dedication and creativity which will
enable us to help more sick babies
and children.
In 2013 staff committed to
fundraising for Action and charities
local to their offices through runs,
rides and triathlons, cake sales, dress
down days, cabaret evenings and
family days. 73 staff teams entered
the 2014 Blenheim Triathlon and
Arun Estates also supported
our Action Cream Teas in a box
event. We would like to thank the
One Stop, the retail convenience
business with some 700 stores
across England and Wales, are
stalwart supporters of Action. The
company sponsored the drinks at
our Champions of CycleSport dinner,
and from 16 March to 27 April
placed Paddington-themed collection
boxes on their countertops, raising
almost £30,000.
14
touchinglives Autumn/winter 2014 Smeg UK have adopted Action
as their charity of the year. Nine
staff members are taking part in the
Action London to Paris bike ride in
September. Managing Director Mike
Giddings was inspired to choose
Action because of our work helping
sick babies and children and our
expertise in organising cycling events.
We wish the intrepid cycling crew
bonne chance! Mike is running the
Great North Run and the Chicago
Marathon for Action in 2015 too.
Lucy Hynes and Mike Phillips
with Paddington BearTM
Allen Ford and Allen Kia,
the Midlands-based car dealership,
hosted two ‘red carpet’ VIP sales
weekends in February and
generously donated £50 to Action
from each sale, raising an amazing
£12,900. Allen Ford entered four
teams in the 40-mile Rutland Round
PLOD event, raising over £5,000.
Tomas Phillips, son of Managing
Director Mike Phillips, also ran the
London Marathon for Action, raising
£1,250. We want to say a big thank
you to all Allen Ford staff for their
fantastic support.
action.org.uk
Fundraising news
High five for volunteering
Community volunteers play a vital role in promoting awareness and
raising funds for Action. We caught up with Volunteer Development
Manager Jane Charlton to find out more about how community
volunteering works, and what to do if you’d like to get involved.
Q What does your role
involve, Jane?
A I develop new opportunities to
maximise volunteering. There are
two main strands: strengthening our
volunteer groups, including setting up
new ones, and developing new roles
such as Speaker Ambassadors and
School Champions.
Q What does that mean in practice
for volunteers?
A Our long-established, richlyexperienced community fundraising
groups do an amazing job for us, and
I’m building on that as well as looking
for new opportunities in all parts of
the community to complement it. It’s
exciting because we are exploring
new groups in areas such as Glasgow,
Oxford, Belfast and Horsham.
Volunteer groups can comprise
young professionals who’ve met in
the office, mums who know each
other through a blog or the school
gate, or simply a group of neighbours
who’ve been inspired to support us.
Q What do volunteer groups
typically do for Action?
A Some groups organise an annual
fundraising extravaganza, such as
the amazing Dine with Davina
ladies’ lunch which takes place in
Southampton every autumn
or the vibrant Killer Heels
and Cocktails event
in Aberdeen. Others
arrange a variety of
events, such as pub
quizzes and golf days,
throughout the year.
The key theme that I
hear over and over
again is how dedicated volunteers
are to Action and how much fun
they have, no matter what the event!
Q Are any special skills needed?
A Community volunteers don’t
need any particular skills, just a love
of meeting people and working in a
team. Some people join an existing
group, others start a new one, and
some work outside of Action groups
but within virtual groups,
such as breakfast or lunch clubs.
‘By volunteering
with us you’ll be
helping to make a
big difference to the
lives of sick babies
and children’
Q You mentioned Speaker
Ambassadors. How do they
help Action?
A If you’re a strong communicator,
you like meeting new people, and
you have a little time to spare, then
you could be well placed to
be a Speaker Ambassador
Jane Charlton,
Volunteer Development Manager
for Action. Volunteers who help us
in this role talk about our work in
settings such as cricket clubs and
Rotary meetings.
Q And School Champions?
A When our School Champions
nurture links with schools, nurseries,
and colleges, we gain the opportunity
to inspire young people to fundraise
for Action, or nominate us as their
Charity of the Year. We then benefit
from events like summer fêtes and
non-uniform days.
Q And what’s next?
A I’d like to explore student
fundraising and get some of our
universities fundraising for us.
Q If someone wants to get
involved, what should they do?
A My number is 01403 210495
or you can email me at
[email protected]. Lots of
people like using our Twitter feed
@actionmedresvol and there’s lots
of information on our website at
action.org.uk/volunteering too.
I’m always really pleased to hear
from anyone who may be able to
lend a hand.
From left to right:
Yvette Mayo, Tina Middleton,
Julie Willford and
Dr Rosemary Anderson
action.org.uk 15
Challenge events 2015
Christmas cards designed
to delight
Cycling
Prudential Ride London-Surrey
100 August 2015
RIDE100 series
Dates all through the year
Tours
Action London to Paris
Cool and Classic routes
22-26 July 2015
Action London to Paris
Champion 24-hour challenge
24-26 July 2015
Action London to Paris
Brand new route!
9-13 September 2015
Every penny raised from
sales of our fantastic
range of contemporary
Christmas cards will come
straight to Action – and
they are on sale now!
There are twelve beautiful new
designs to choose from this year.
Why not take a look at action.org.uk/xmas and order yours ahead
of the festive rush? Cards cost £3.50 for a pack of five. Order forms
are also available from Chris Tanner; you can email him at:
[email protected] or call him on 01403 210406.
Last year, we raised more than £12,500 from Christmas card sales.
The project is the brainchild of longstanding Action supporter Lynn
Tait, who has owned a greetings card business for more than 20 years.
Using her skills, contacts and powers of persuasion, she has again secured
sponsorship for every aspect of producing the range, from original
artwork to the card used, envelopes, printing and even cellophane bags.
Thank you, Lynn!
And our thanks also go to all the Ladder Club artists who donated
their exclusive designs and to Sherwood Press, The Imaging Centre,
Enveco and G F Smith who have all donated their services free to
produce the cards.
Running
Adidas Silverstone Half
Marathon 15 March 2015
Virgin London Marathon
26 April 2015
BUPA 10,000 25 May 2015
BUPA Great North Run
September 2015
Team Events
PLOD – Mourne Mountains
June 2015
PLOD – Cotswold Way
June 2015
Race the Sun Brecon Beacons
20 June 2015
PLOD – Rutland Round
July 2015
Three Peaks Challenge
4-5 July 2015
PLOD – South Downs Way
July 2015
PLOD – Trossachs
August 2015
Race the Sun Lake District
29 August 2015
You can register for an event on
our website, give us a call or send
an email:
action.org.uk/get_involved
T 01403 210406
E [email protected]
action.org.uk touchinglives Autumn/winter 2014
16