The SMA Trust Newsletter Issue 4

Issue 4 November 2013
MOMENTUM
NEWS
Funding Research into Spinal Muscular Atrophy
Celebrating 10 years!
It is 10 years since The SMA Trust was founded
Since then we have invested £2.4 million in people,
projects and places – all united by the common
aim of helping to find a cure and treatments
for Spinal Muscular Atrophy.
We’re marking this special occasion in two ways:
PARTY AT SPENCER
HOUSE!
SMA is currently
incurable and
often fatal
10th Anniversary Party
Research Impact Report
We are celebrating in style with
a wonderful drinks reception
at Spencer House, St. James’s
Place, London on 30th October.
This historic building, with its
stunning interiors, makes the
perfect setting for our party,
which will definitely be an
occasion to remember.
This new report (pictured
right) summarises research
funded by The SMA Trust
during the past decade, puts
it in the context of overall
SMA research, and looks
ahead to future challenges
and priorities.
Midsummer MADness with the
Cotswold Way MAD Challenge
Thanks to our amazing
walkers, this year’s event
raised £32,000, beating last
year’s total of £21,000!
On Midsummer’s Day, our intrepid walkers
met in Painswick to start their 50 mile walk
through the night along The Cotswold Way.
Caroline Warren, Peter Baughen and
Mark Dearlove (Trustee) had come back
for a second time (having vowed never
to do it again!) and did a fantastic job of
boosting morale. Mark, Chris South and
Kevin Talbot (another Trustee) forged
ahead, blazing the trail for everyone else.
Amazingly, Mark and Chris had already
walked the first half of The Cotswold Way
over the 3 days before joining us.
Other second-timers were Alan Lane
and Alistair Wilson, who were joined
by Mick Hunt – all chatting and laughing
throughout the night.
MAD
Challenge
Team News
Page 2
New walkers included a group from Tysoe
(led by Robert Philips). Rose Morris said:
“The miles flew by whilst chatting, striding
out and admiring the views.”
There were welcome food and drink stops
along the way, especially breakfast at
3am at Winchcombe Scout HQ, where
the Group (led by Jim Maher) barbecued
sausage and bacon for everyone. It was
hard to tell what was appreciated most –
the food, or the foot baths and massages
provided by Jessica Powell.
More walkers joined here for the 20 mile
walk, including a group of Year 10 Duke
of Edinburgh students from Chipping
Campden School, along with Dan
Rushworth, their Master.
Sam and Otis, our canine crusaders,
joined owners Caroline and Alan for the
next 20 miles, only stopping for a quick
chat with Chester the chocolate lab at
Broadway Tower, who was resplendent
in his SMA Trust t-shirt.
AIMING HIGH
AT BROADWAY
TOWER
Page 5
THE M AD
CH ALLE NG
ERS
We welcomed everyone back to the
Town Hall in Chipping Campden, where
local band Indie Toast provided some great
tunes and Gill, Daphne and Ebrington WI
provided a sumptuous tea.
Thanks to everyone who took
part or helped in any way with
this event. It was quite a night!
SIR JAMES
GOWANS
Interview
Page 6
FUNDRAISING
UPDATE
MAD Challenge
Team News
Katie Yeo held a fundraiser at her dance
studio in December raising over £300.
NSK staff went MAD in May. Phill
Dawson, Keith Slater, Alan Croft,
Lesley Gibson & Heather Moor, along
with co-workers, cycled Coast to Coast,
Whitehaven to Sunderland.
Team Joseph
Derrick Nundy, held a fabulous music
concert at the end of October last year
in memory of his nephew Joseph Wilson.
Derrick raised well over £1,000.
Team Tiegan
Vicki Jones raised over £1,500 with
The Windermere British Gas Swim.
Hannah Mckeown and her brother Andy
Mckeown continue to raise awareness
and funds for The SMA Trust.
Team Octavia
St. Mary’s School, Shaftesbury, continue
to fundraise for us.
Stagecoach of Shaftesbury made
a collection at one of the shows and
raised £295.
Team Rowan
HOLLIE , TOBY & PA
ULA
Team Hollie
The Team Hollie Ball was held on May
3rd 2013 at the David Lloyd Health Club,
Dudley. It was a fantastic success for
Paula and Toby Flatman, parents of
the bubbly, ever-smiling Hollie who has
SMA. Guests enjoyed strawberries and
marshmallows to dip in the chocolate
fountain and participated in a brilliant
auction and raffle whilst revelling in
entertainment from a Take That tribute
band. The dynamic duo are organising
another ball next year on 10th May 2014.
Team Jaiden
A very warm
welcome to Team
Jaiden – Pauline
and Shannon
Wright. Pauline has
been heading up
the team along with
daughter Shannon
raising awareness
Pauline and Shannon
and funds since
September last year. Their gorgeous boy,
Jaiden tragically lost his fight with SMA
last September. Mother, Shannon has
set up her own dance studio in Peterlee,
in memory of her beloved son, Jaiden’s
Dancing Stars (JD*s). “This is the perfect
way to keep my son’s memory alive and
continue fundraising for The SMA Trust
to help them find a cure. My ultimate goal
is to make him proud” said Shannon.
Pauline has held fundraisers, along
with her employers NSK Bearings.
Lynne Flanagan collected at her dance
shows and donated a brilliant £1,050.
Gemma Fowler ran the Great North Run
in September last year raising £250.
2
COAST TO CO
AST CYCLE RI
DE
Team Benny
Alex Smith and friends, along with Benny
Knight as Cox, took part in the Great River
Race along the Thames, from London
Docklands to Ham raising a whopping
£3,500 in the process.
Team Elliot
Steven Rotherham made a huge effort
with his Iron Man Challenge on September
16th 2012 raising almost £1,000 for Team
Elliot and The SMA Trust.
Petra Rezacova ran the Great North Run
last year raising £1,000.
Elliot’s parents, Simon & Louise Granger
did a series of bag packs in their local
supermarkets and then took part in the
‘Way of the Roses’ Cycle challenge.
Cycling 170 miles from Morecombe Bay to
Bridlington over 3 days and raised £5,000.
A very heartfelt
welcome to another
new Team, headed
up by Sarah &
Matt Poole. Team
Rowan has been
very active indeed
with their fundraising
activities since
Rowan Poole
December 2012.
Sarah & Matt wanted to raise £10,000
for research into Spinal Muscular Atrophy,
they have totally SMAshed that target,
even doubled it!
Sarah herself has been making keyrings for
The SMA Trust. Trish Taylor, Matt’s mum
has made adorable Sock Monkeys, a real
cottage industry going on in Denby Dale.
Super girl Naomi Clayton, started the ball
rolling with her 5, yes that’s 5, Half Marathons.
Naomi also joined the Onesie walk.
TON
NAOMI CLAY
WA Y OF THE RO SE S
CYC LE CH AL LE NG E
www.smatrust.org
Sarah herself, swam the 3.8 kilometres
Epic Lake swim in June.
Sarah’s parents, Christine & Steven
Pearson organised and hosted a series
of ‘Come Dine with me’ evenings.
FUNDRAISING
TEAM RO WA N THR
EE PE AK S CH AL LE
NG E
The Awesome Andrew Pearson took part
in the Yorkshire Three Peaks Challenge with
his friends. Andrew was slightly different
though, he was wearing a surfboard
on his back! Andy wrote a poignant piece
afterwards about the walk and his feelings.
There wasn’t a dry eye in this office and it
was our biggest ‘hit’ on Facebook to date.
Emma Ashton, Julie Elliott and Lizzie Davis
have been doing a brilliant job of fundraising.
FMG, the company Matt Poole works for
has chosen The SMA Trust as one of their
charities to support this year, starting with
a Rounders match in August. They are also
involved with the 20-20 cricket in September,
organised by Amy Bissett and friends.
UPDATE
Team David
Team Lily-Bea
The second Platinum Ball was another
glittering occasion hosted by BBC
Breakfast’s Bill Turnbull on 5th October.
The fantastic £52,000 raised from this event
will go to our Edinburgh University project.
We warmly welcome
Team Lily-Bea,
fundraising for
The SMA Trust
since September
2012. Hannah
and David Razzell
lost their adorable
daughter Lily-Bea
Lily-Bea Razzell
to SMA last year.
Sarah Razzell and friends took part in the
Thames Path Challenge over night walk
in London this year.
Childrens Salon, Pritti Ansome, in Saffron
Waldon has been selling Lily-Bea hair clips.
Hannah also made special Jam to sell at
coffee mornings.
As usual for Team David the year has
been action packed. Cheadle based
Stuart Ingleby, a Specsavers store
director, managed to cycle to all 11
Specsavers stores in the Manchester
area in just one day on Saturday 29th
September, raising funds and awareness.
This was just a warm up for Stuart before
his mammoth N4N cycle challenge, the
UJIA North4North Israel Cycle challenge.
On the 5th November 2012 Stuart, along
with Jonathan Dee, Ilan Lieberman,
Laurence Sugarman, Jonathan Tuck,
Simon Israeli-Korn and about 50 other
crazy riders cycled the length of Israel.
Up and down hills and into deserts it took
6 days of approximately 80 miles a day –
gruelling work and they raised a staggering
£22,000. Team David, we salute you!
LILY-BEA JAM
Team Millie
Mayfair Capital, for Team Millie, got
a group together and completed the
Three Peaks Challenge. Selina Brock,
William Parker, James Feilden, Simon
Martindale, Edward Pumphrey, James
Lloyd, Robert Palmer, James Thornton,
Emily Osmond and Guy Brogden all took
part in the challenge, walking a distance
of 40Kms. They raised a fantastic £17,000
which was split between The SMA Trust
and another charity.
William Hewish completed a 100 mile
cycle challenge in May.
Alex Murray – see article on page 4.
Team Doingit4Alfie
Chelsea Leach, along with Leanne
Pearce are raising funds for their skydive
which they hope to arrange this year.
Team Travis
STUART INGL
EBY AND TE
AM
Lucy Keseru, Mum to Travis, along with
her husband Shane, arranged a walk
up Pen-y-Fan in May. They also held
a fundraising event in June.
Sadly, little Travis died in October.
Our thoughts are with his family.
A Comedy Night on
behalf of The SMA Trust
We found the perfect way to chase away the
post Christmas blues by holding a Comedy
Evening at the Chelsea Theatre in January
this year. Lead Pencil (pictured right) are an
up and coming Comedy Sketch Group, who
performed to rave reviews at the Edinburgh
Fringe in August 2012. Maddie Rice (Team
Millie) said “It was great to use comedy
to raise awareness of SMA. It meant that
everyone had a really good night, but went
home with something to think about”.
LK
PE N-Y-FA N WA
TEAM TR AVIS
1 in every 6,000
births is affected
by SMA
www.smatrust.org
3
FUNDRAISING
UPDATE
The Onesie Walk
On a fresh, early summer morning at 9.00am, on May 26th
2013, on what would have been Rowan Poole’s 2ND Birthday a
group of 52 MAD people dressed up in Onesies met up at Greyfell
Academy, Shepley, Yorkshire and set off on a 17 mile walk.
Friends and family of Sarah and Matt Poole
(Team Rowan) came from far and wide to
join them in this poignant walk to celebrate
Rowan’s birthday. They walked through
Holmfirth and joined the Pennine trail over
Saddleworth Moor to finish at Dovestones
Reservoir where Matt & Sarah have planted
a Rowan Tree for their beloved little girl.
The back-up car support team were
Sarah’s parents, Christine & Steven
Pearson, who followed the walkers
providing water, huge smiles and cheers
and the best flapjacks ever!
Other walkers up on the Moor were
fascinated to know what was happening
and the collection buckets filled up nicely.
Julie Elliott, who had the original idea for
a Onesie walk helped organise the event.
At the start everyone was given a label for
their back pack with a gorgeous photo
of Rowan attached.
E. Stott & Sons, local coach company,
took the weary but exhilarated walkers
back to The White Hart Pub in Denby
Dale for much needed beers, a raffle,
selling of SMA Trust merchandise
and a fantastic collection.
The walk raised over £8,000.
4
Debbie Slatter, completed the Wiltshire
100 mile bike ride for The SMA Trust.
Thank you Debs for raising £300.
We were chosen as one of the recipients for
Kumho Tyres, cash for causes last year.
Thank you so much Kumho for the donation.
Ebrington W.I had a collection at
their October meeting, thank you to June
(president) and all of the wonderful ladies
at Ebrington W.I.
Ian & Suzanne Hewertson from
Blackpool have launched their fundraising
with a balloon race from their Golf Club,
Poulton le Fylde.
Alex Hickman spent a day in a
wheelchair to raise funds and awareness.
Toby Hull, ran the London marathon
for his Grandfather, who suffers with SMA.
Toby raised a whopping £1,600.
John Lewis at home, Chester, held
a couple of fundraisers for The SMA Trust
raising almost £400. These were in memory
of Theo Parker of Cheadle.
THE ONSIE
WALKERS
Mark Lambeth from Crawley, ran the
Paris Half Marathon in March for his friend’s
son George who has SMA.
“At the start everyone
was given a label for
their back pack with
a gorgeous photo
of Rowan attached.”
UK Perimeter
Cycle Challenge
Alex Murray (Team Millie) along with
his friend Sam Kania took part in the
UK Perimeter Cycle challenge starting
on 16th July. They cycled 2,500 miles,
averaging over 100 miles a day over 24
days. They left Mathry fully prepared
from their training and ready to go, with
back up vehicle at the ready. They met
up with the Cardiff Morris Dancers at
the Gwaelod-y-Garth Inn and stayed
with family and friends along the way.
By day 12 the ride was taking its’ toll on
their bodies and they were feeling the
early onset of saddle sore. By day 14
the weather, thankfully, had started to
improve and the Just Giving donations
were following the temperature rise in
an upward direction!
Day 16, they had made it to their final
destination, John O’Groats. At the end
of their amazing challenge a wonderful
homecoming party had been arranged
to celebrate this immense achievement.
Here in Alex’s words; “6 months ago,
I was lying in a hospital bed having my
Other fundraisers
THE BRITISH 10K
LONDON RUN
The British 10K
London Run
A
AN D SA M KA NI
ALEX MURR AY
ankle reconstructed with a lot of metal.
Yesterday, I finished a 2,500 mile cycle
challenge with Sam in 24 days. 3 months ago
I was learning to walk again. If somebody
had said to me in February that in 6 months
time I would have cycled 2,500 miles
around the perimeter of the UK, I would
have said not a chance. But through
some incredible support from friends
and family, through encouraging words
and incredible donations, we found
all the motivation we needed. It’s hard
to know how we go bigger and better
for our next fundraising challenge!”
www.smatrust.org
On a very hot, sticky morning in June
The SMA Trust cheered on our brave
runners. Team Millie gave us Maddie,
Marcus and Mimi Rice. Laurel Henning
flew over from Brussels, Jack & Alan
Lane joined us once again along with
Stephen Wickenden. Flora and
Hugo van Vredenburch met up with
The SMA Trust’s Tessa Rice at Trafalgar
Square and Lucy & Robert Wright
joined the group running on this incredibly
sweltering day. Well done to you all!
The child of two
carriers has a one
in four chance of
developing SMA
ACTIVITIES
UPDATE
Thank you for an
incredible first year...
Joanna Mitchell, Executive Director, The SMA Trust
I can’t believe I have
been with The SMA
Trust for a year. It has
been a busy time and
a wonderful one and
I’ve learnt so much;
•I’ve learnt a great deal about SMA and
SMA research, BUT that my knowledge
is a tiny fraction, compared with that
of the incredibly bright people who are
working with this disease.
•I’ve learnt that what we are doing (giving a
UK focus to SMA research) is really important
BUT we are only a small part of the global
effort that is currently going into SMA.
•I’ve learnt that our MAD Challenge
teams and other donors are phenomenal
and work tirelessly to raise funds and
awareness around the country, BUT we
need more of them if we are to grow and
be able to fund future research projects.
As I write this, we are busy finalising
arrangements for our 10th Anniversary
party at Spencer House in London.
We are so lucky to have the opportunity
of this stunning venue and are very grateful
to Spencer House and The Rothschild
Foundation for making it possible. We
will be using the event to launch our 10th
Anniversary Research Impact Report,
which looks back over the first 10 years
of the Trust – what we have funded, and
how it has contributed to overall progress
in SMA research.
In his foreword to the report, Trustee
Professor Kevin Talbot summarises:
“It is now possible to see how, in children
affected by SMA, we might prevent the
degeneration of the motor nerves that
are crucial for movement. SMA may
indeed become one of the first inherited
neurological diseases to be treated
successfully with therapies that directly
target the genetic error.”
The SMA Trust aiming high
at Broadway Tower with the kids
from Chipping Campden School.
However, he goes on to talk about
future priorities: “We need better ways of
measuring change in SMA patients so that
we can be sure that new drugs can be
properly tested. Replacing the missing gene
should prevent loss of nerves, but other
therapies will be needed to help preserve
and strengthen the connections between
muscles and nerves...A better understanding
of how the SMN gene, which is missing in
SMA children, actually functions normally
in the development and maintenance of the
nervous system will also help the discovery
of new medicines to treat SMA.”
We need to ensure that we have the funds
available to quickly support world-class
projects and scientists, here in the UK and
abroad. We are entering an era of real hope
for people living with SMA. The SMA Trust
is determined to continue helping to make
that hope a reality.
Thank you for everything you do to
make it possible for us to do that.
Leaving a gift
in your will TO
THE SMA TRUST
Leaving a gift in your will will help to
ensure that work to fund research and
raise awareness of this inherited disease,
which is the leading genetic killer of
children under 2 years old, continues.
By leaving a gift, no matter the size, you
will continue to make a positive impact
and motivate change beyond your
lifetime. If this is something you choose
to do, it is important that our name,
address and charity number is featured
in your will:
The SMA Trust, Compton Scorpion,
Shipston on Stour CV36 4PJ
Registered Charity Number: 1097765
A group of 50 enthusiastic students from
Year 10, Chipping Campden School,
braved the snow and wind and gathered
at Broadway Tower to form The SMA
Trust chromosome for use in our publicity
campaign to launch and promote the
Cotswold Way 50 mile walk.
Having donned T-shirts in 2 different
colourways which comprise The SMA
Trust logo, the excited students alighted
from the coach at Broadway Tower to be
faced with icy winds and snow.
Photography student Annie Ounstead
drove up from Bath to help us and was
very concerned with the atrocious weather
conditions as she had to perch at the very
top of the tower to take the shot!
We were thrilled with the end result and the
youngsters were fantastic, not complaining
about the cold, with lots of squeals and
howls of laughter whilst holding each other
up in the driving snow.
If you have any questions about leaving
a gift to The SMA Trust, please contact
our Head of Fundraising on 01608 663415.
1 in every 40 people
is a carrier of the
defective gene that
causes SMA
www.smatrust.org
5
QUICK
INTERVIEW
Sir James Gowans
CBE FRS
Sir James Gowans, one of our longeststanding trustees, has had a long
and notable career in pathology and
immunology. During the 1950s he did
pioneering work on the life cycle of the
lymphocyte, establishing that the small
lymphocyte continuously recirculated
from the blood to the lymph and back
again, and that this cell was responsible
for initiating immunological responses.
His career included spells as Henry Dale
Research Professor of the Royal Society
School of Pathology, University of Oxford
(1962-1977); as Director of the Medical
Research Council’s Cellular Immunology
Research Unit (1963-1977); as Secretary
of the Medical Research Council (19771987), and as Secretary General of the
Human Frontier Science Programme
Strasbourg (1989-1993).
Sir James has received many awards and
honours for his work, including a knighthood,
and continues to use his medical knowledge
as a Trustee and Scientific Adviser to
The SMA Trust.
Q: When did Andrew Knight approach
you to join The SMA Trust?
Sir James: Andrew came to Oxford and
spoke to Professor Dame Kay Davies (British
Human Geneticist & Director of the MRC
Functional Genetics Unit), who recommended
me to him. Andrew was interested in my
previous work and connections with the
Motor Neurone Disease Association.
Q: What did you know of SMA
at that time?
Sir James: I knew of it as ‘floppy baby
disease’ and not much else. However, I did
know it was caused by a single gene defect
which made it particularly interesting.
“I knew of SMA as
‘floppy baby disease’
and not much else.”
Q: How did you feel about being
approached to become a trustee?
Sir James: Oh I didn’t have any objections
at all. I was very interested in finding out
more about SMA. I was, in fact, delighted
to have been asked.
Q: You recommended Professor Angela
Vincent as a Trustee didn’t you?
Sir James: Yes I did. I was concerned that
it was getting more difficult for me to get to
meetings for health reasons so I suggested
Angela. Kevin Talbot (who became a Trustee
in December 2011) also knew her and was
enthusiastic about her joining the Trustees.
How can you help?
Q: Now that you have professionally retired,
what is your main role as a Trustee?
Sir James: Andrew suggested I might just
make myself available for extra scientific
advice when necessary.
Q: You recently gave Vanessa C-B
advice on writing the impact report,
what was that?
Sir James: I think I said when writing a
report or document, try not to make it too
long as you want busy people to read it.
Always include appendices for technical detail,
as this allows you to keep the flow going in
the main body of the report. Busy people do
not have time to read massive reports. The
key thing in scientific writing is clarity. Be clear
and precise. The summary is also important:
the ‘take home message’ is key.
Q: What are your hobbies?
Sir James: Now it’s funny you should ask
me that. Many years ago I gave a talk at
Abingdon Girls School which I really enjoyed.
The Biology Mistress said she wanted to
introduce me and could I tell her what my
hobbies were. I tried to think of one and
came up with fretwork. I had no idea what
fretwork was but thought I ought to say
that I had a hobby!
Q: What is your favourite book?
Sir James: I don’t have a favourite book!
I have lots of favourite books, among them
are Anton Chekhov’s, Short Stories.
Q: What do you enjoy most about
being retired?
Sir James: One of the nice things is that
there are no deadlines, apart from this
newsletter of course.
SMA is the leading
genetic killer of
infants and toddlers
Christmas
Cards 2013
➜ You can make a regular gift to The SMA Trust, see back page for details.
➜ Donate directly to The Trust www.smatrust.org/fundraising or through
our JustGiving page www.justgiving.com/smatrust/Donate.
➜ Do your own MAD Challenge – the MADder the better. It can be anything from running,
walking, tea parties (MAD Hatters Tea Party!), cake sales to car washing. Come up with
your own ideas. For more inspiration or help contact us on 01608 663415.
We are just putting together an exciting Calendar of Events for next year, keep an eye
on our Facebook page facebook.com/smatrust and website www.smatrust.org.
GET IN TOUCH
Fundraising: Mandy Lane Email: [email protected]
SMA Research: Vanessa Christie-Brown Email: [email protected]
The SMA Trust Compton Scorpion, Shipston-on-Stour, Warwickshire CV36 4JP
Follow us on Twitter twitter.com/smatrust
Like us on Facebook facebook.com/smatrust
For further information please visit www.smatrust.org
Registered Charity Number: 1097765
6
www.smatrust.org
We have four single image Christmas
cards this year, Deer in the Snow,
Team Spirit, Christmas Delivery and
We Three Kings. £4 per pack of 10 cards.
If you would like to purchase any cards
please contact: Mandy Lane on 01608
663415, email [email protected]
or buy online at www.smatrust.org/shop
The charity has donated £2.4M to SMA research since its inception
RESEARCH
ACTIVITIES
UK Initiatives
Since January 2013, the SMA Trust has contributed £174,479
on new international and UK research initiatives.
We are pleased to report that the
staff recruitment process for our SMA
REACH UK Clinical Trials project, in
partnership with Great Ormond Street
Hospital, has been completed and the
members of the External Advisory Board
have been selected.
Through this initiative, The SMA Trust
will seek to:
•Establish patient registries
•Standardise outcome measures
•Prepare key neuromuscular centres
for inclusion in clinical trials
To mark the 10 year
anniversary of The SMA
Trust, Great Ormond Street
Hospital Charity has made
a short film about the
project and how it will help
children with SMA. Follow the
link at www.smatrust.org/projects
to view the film online.
In partnership with the Motor Neuron
Disease Association, The SMA Trust
continues to support the Chair of Motor
Neurone Biology, Professor Kevin Talbot,
at Oxford University.
New Awards
Meanwhile, The SMA Trust continues
to support eminent UK scientists and
we are delighted to announce the
£89,710 funding of Professor Thomas
Gillingwater’s new project entitled
Developing novel small molecule-based
therapies for SMA. Professor Gillingwater
and his team are looking at the molecular
pathways that carry messages from the
spinal cord to the muscles, disruption
of which contribute significantly to SMA.
The team will also be looking at the
potential therapeutic effects of a drug
known to target these pathways.
Professor
Tom Gillingwater
“The Sma Trust continues to
support eminent UK scientists.”
Research Impact
For the 10 year anniversary of the inception
of The SMA Trust, we have written a report
summarising the impact The SMA Trust has
had on SMA research over the past decade.
The report includes a piece on the foundation of
The SMA Trust by Chilla Knight, grand-daughter
of co-founder Andrew Knight, summaries of all
the grants funded and our vision for the future.
The report can be viewed at www.smatrust.org
www.smatrust.org
New Publications
funded BY
THE SMA Trust
2013 has seen the publication
of 7 papers resulting from
projects funded by the SMA
Trust directly or through
SMA Europe.
Publications are extremely important as
they demonstrate peer-reviewed advances
in medical research:
•SMA-EUROPE workshop report:
Opportunities and challenges in developing
clinical trials for Spinal Muscular Atrophy
in Europe. N. Kayadjanian et al. Orphanet
Journal of Rare Diseases 2013, 8:44.
•Six minute walk test in Type III Spinal
Muscular Atrophy: A 12 month longitudinal
study. E. Mazzone et al. Neuro-muscul
Disord (2013).
•Increasing SMN levels using the histone
deacetylase inhibitor SAHA ameliorates
defects in skeletal muscle microvasculature
in a mouse model of severe Spinal Muscular
Atrophy. Somers et al. Neuroscience Letters
2013, 544:100-4.
•Spinal Muscular Atrophy: going beyond the
motor neuron. Hamilton & Gillingwater. Trends
in Molecular Medicine 2013 19.1:40-50.
•Intracisternal Delivery Results in Efficient
Gene Transfer to the Central Nervous
System of a Feline Model of Motor
Neuron Disease. Hum Gene Therapy 2013
Jul;24(7):670-82.
•Rasch analysis of clinical outcome
measures in Spinal Muscular Atrophy.
Muscle Nerve 2013. doi: 10.1002/
mus.23937.
•Chonotrolectin affects cell survival
and neuronal outgrowth in in-vitro and
in-vivo models of Spinal Muscular Atrophy.
Sleigh et al. Human Molecular Genetics,
2013, doi: 10.1093.
Rare Disease Day
Joanna Mitchell and Vanessa
Christie-Brown took part in the 6th
Rare Disease Day at the Royal Holloway
in London on 28th February 2013.
Rare Disease Day is an international
initiative to raise awareness amongst
the general public and decision-makers
about rare diseases and their impact
on patients’ lives.
7
RESEARCH
ACTIVITIES
INTERNATIONAL RESEARCH
COLLABORATION
The SMA Trust continues to be A driving force behind
international research collaboration and
co-ordination through SMA Europe.
Call 5 (2012) Awards:
Columbia University, USA, Dr. Shingo
Kariya: Elucidate the molecular
mechanism underlying maturation
and remodelling defects of the
neuromuscular system in SMA.
The Board of SMA Europe held their annual
meeting in The Hague, The Netherlands,
on 9th March 2013, where research
projects were awarded grants following
recommendations from the Scientific
Advisory Board (SAB) who had reviewed
all Call 5 applications the previous day.
The SMA Europe Board also added
new initiatives to its remit: a new strategy
and the development of new strategic
partnerships with the pharmaceutical
industry and regulatory authorities in order
to facilitate the implementation of SMA
clinical trials in Europe.
Call for Projects
A total of 3 Operating Grants were selected
for funding in Europe and the USA.
The SMA Trust committed €100,264, or
25% of the total budget to these projects.
INSERM, France, Dr. Martine Barkats:
AAV9-SMN Gene Therapy for SMA.
THE BOAR D OF
SM A EU ROPE
Call 6 (2013) was launched on 30th July
on the SMA Europe website as well as
on national websites and in Treat-NMD
and Orphanet newsletters. The deadline
for applications was 4th October and the
best projects will be selected for awards
in March 2014.
Swiss Federal Institute of Technology,
Switzerland, Dr. Frédéric Allain:
In search of small molecules
targeting protein-RNA complex:
a novel approach against Spinal
Muscular Atrophy.
Contact
Vanessa Christie-Brown
The SMA Trust Research Co-ordinator
Email: [email protected]
Website: www.smatrust.org
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