skipDuchenne to raise £5m
over the next five years, and to lobby Government for matched funding to
support gene therapies for all those living with Duchenne
Action Duchenne,
the UK’s leading charity dedicated to finding treatments and a cure for
Duchenne Muscular Dystrophy has today launched its skipDuchenne
research fund. The fund will raise £5m over the next five years and
will be seeking Government matched funding (from the fund announced by
David Cameron in April) to support clinical trials of gene therapies for
Duchenne patients in the UK. Action Duchenne will be working closely
with the International Duchenne Exon Skipping Consortium (iDESC) to
facilitate development of systemic anti-sense therapeutics for
neuromuscular disease, with the goal of providing access to treatments
to as many Duchenne patients as quickly as possible.
skipDuchenne is already supporting cutting edge research and earlier
this year provided a grant of £160k to Royal Holloway University College
to develop full length dystrophin, effectively a cure. A grant of £23k
was secured by skipDuchenne from Jeans for Genes to further support the
project.
The first exon skipping drug is expected to be on the market in 2014,
but due to the way that genetic medicines are currently approved, it
will only benefit 14% of Duchenne patients. skipDuchenne is currently
building a fund of £180k to sequence rarer exons, which will help to
ensure that treatments that will benefit between 60% and 80% of boys
living with Duchenne are available by 2014.
Martin Bashir, broadcaster, patron of Action Duchenne and whose brother
Tommy had Duchenne commented, “My own brother lived until he was 29, but
even now this is very much the exception. We are on the verge of
perhaps the most significant breakthrough in the treatment of Duchenne.
This is a pivotal and critical moment in the fight against this disease
and the funds needed are a drop in the ocean when compared to the
social and human cost of caring for these young men. We urgently need
the Government to provide the matched funding required to bring these
treatments to market as soon as possible.” Martin Bashir, who will be
covering the Olympics for the broadcaster NBC, is advocating on both
sides of the Atlantic for those with Duchenne.
In April 2012 the Prime Minister, David Cameron launched a fund of £180
million to provide grant funding for innovative small and medium sized
enterprises and academics to develop solutions to healthcare challenges.
Mr Cameron said:
"The UK boasts a world-leading life sciences sector which is changing at
an incredible pace. And I'm absolutely committed to helping it widen
its significant foothold in the global market. Benefiting both patients
and the British economy, this £180 million programme will support both
SMEs and academics and help to ensure that they can turn their promising
ideas into innovative technologies."
Action Duchenne’s Boys to Men Campaign which was the focus of recent
lobbies to Westminster, Holyrood and Stormont (the lobby to the Welsh
Assembly will take place on 17 July) is calling on the government to
provide matched funding for skipDuchenne from this fund.
Nick Catlin, Head of Research at Action Duchenne commented, “Since we
launched Action Duchenne in 2001 so much has happened in the race to
find a cure for Duchenne. In those days they said there was no hope,
now we are on the edge of a breakthrough and a potential cure for this
devastating condition. Now that we are so close to viable treatments
and a cure, skipDuchenne aims to raise vital funds to ensure that these
medicines are developed quickly and made available so that they benefit
the current generation of children living with this cruel condition. We
are asking our members, supporters, funding partners and the Government
to support us in this goal.”
Tyran Hawthorn, aged 25, who is living with Duchenne, recently featured
in an ITV Meridian news piece, “Out of the sixteen boys at Treloars
College who were there at the same time as me only one other friend is
still alive. Unfortunately, he is in intensive care where he has been
since January. We keep hearing how young men with Duchenne should live
into their 30s and 40s, but at the moment that simply isn’t happening.
We need these gene therapies urgently.”
Professor Francesco Muntoni, Chair of Paediatric Neurology, ICH -
Dubowitz Neuromuscular Centre, Dept of Neurosciences & Mental
Health, Faculty of Population Health Sciences and a founding member of
iDESC said, “The landscape of potential treatments for Duchenne Muscular
Dystrophy has improved out of all recognition in the last ten years.
We believe we are now getting closer to a viable treatment and iDESC has
been set up to ensure a high level of focus to delivering these
treatments as soon as possible. We are delighted that skipDuchenne is
supporting iDESC with much needed funds to ensure that we deliver for
these children.”
Matthew Wood, Professor of Neuroscience, Department of Physiology,
Anatomy and Genetics, University of Oxford said, “Exon skipping is the
most promising of several new potential treatments for Duchenne
Muscular Dystrophy to emerge in very recent years. It is crucial now
to capitalise on these scientific advances and to ensure that the
clinical potential of exon skipping is maximised and that its benefits
reach as many Duchenne boys as possible as quickly as possible.
skipDuchenne is poised to play a very important role in achieving these
goals”
Dr Keith Foster at the School of Biological Sciences at Royal Holloway,
who is looking at a system to produce full length dystrophin, said,
“Gene therapy research is progressing at a faster pace than ever before.
It is crucial to raise the funds so that we are able to finish the
project and to change the regulatory climate so that the treatments are
made available to Duchenne patients as quickly as possible. The
skipDuchenne research fund will help support our efforts and ensure that
we keep awareness levels high.”
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