Local funding. Global solution.
www.cure4cf.org
What living
with CF means.
Cystic fibrosis is the most common life-shortening
inherited disease in the developed world, with
one in every 2,500 children being affected.
Even with the best medical care, about half of
those with cystic fibrosis die by their late 30s.
Cystic fibrosis results in progressive loss of lung
health due to repeated airway infection and
resulting damage, however it also affects the gut,
liver, pancreas and reproductive tissues. The lung
infections in cystic fibrosis patients are extremely
difficult to treat, even with the strongest antibiotics
and most innovative therapies available.
Current treatments for cystic fibrosis are very time
consuming, can have debilitating side effects,
and can only slow the effects of the disease and
the destruction of the lung.
WHAT IT IS LIKE TO LIVE WITH CF?
“Try breathing for a while through a short length
of garden hose. Soon, you will feel an increasing
urgency to breathe more than you are able to
– you will probably make increasing efforts to
move more air in and out of the hose, but your
lungs cannot do that, no matter how much effort
is put into it. Now imagine this feeling of “not
enough breath“, starting off imperceptibly in
childhood, and steadily increasing, never halting
or reversing...”
Dr. David Parsons, Team Leader,
Adelaide CF Gene Therapy Research Group
For children like Mae, having cystic fibrosis can mean:
Cure4CF is working with leading
researchers in Adelaide to find a
global cure for Cystic Fibrosis.
(Front cover) Ten year old Mae (right) with her older sister Olivia. Mae has cystic fibrosis airway disease.
(Inside cover) Mae must use a nebuliser every single day in order to inhale the numerous medications she requires.
• Taking up to 40 tablets per day to help digest food
• Up to two hours of intensive physiotherapy every day to help expel mucus from the airways
• Taking nebulised drugs (i.e. in an inhaled mist) every day
• Regular but unpredictable week(s) long hospital admissions each year
• A shortened life-span (37 years on average)
• Never knowing what it feels like to live a ‘normal’ life because of the impact of numerous
hospital visits each year.
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World leading
research.
The research being undertaken is both innovative
and cutting edge, having already delivered a
number of world first outcomes. The team is
developing a gene transfer approach to treat or
cure cystic fibrosis airway disease. Importantly,
this method is targeted to include correction of
the airway stem cells. This means that every new
airway cell that is produced over a lifetime by
the corrected stem cells should already have the
corrected cystic fibrosis gene operating within it.
The treatment aims to use a heavily modified
virus to take the correcting cystic fibrosis gene into
the defective airway cells to rejuvenate proper
cystic fibrosis gene function. These corrected cells
should then take over the job of producing the
normal airway physiology, and so prevent, halt or
reduce the lung disease process.
Cure4CF, along with expert research institutes
such as the USA Cystic Fibrosis Foundation and
the UK CF Trust, believe that gene therapy has
the potential of curing cystic fibrosis because it
addresses the cause of the disease (the faulty
cystic fibrosis gene) and not merely the symptoms.
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The team has shown their gene transfer approach
works in animal models and are now scaling up
their work and modifying techniques to make them
suitable for use in future human clinical trials.
The technique being developed by the Adelaide
CF Gene Therapy Research Group has the
potential to cure, or at minimum provide a longlasting and effective treatment for cystic fibrosis
airway disease, transforming the lives of the 3,000
people living with cystic fibrosis in Australia and
more than 70,000 worldwide.
The cutting edge processes being developed may
also have applicability beyond the treatment and
cure of cystic fibrosis, with the potential to benefit
researchers working on other genetic disorders.
The most common life-threatening
genetic disease affecting the
developed world.
PhD student Nigel Farrow has been a member of the Adelaide CF Gene Therapy Research Group for more
than four years. Nigel’s daughter, Ella, has cystic fibrosis airway disease.
Achievements
to date.
Cure4CF Foundation initiated and was at the
forefront of fundraising efforts to establish the
Allan Scott CF Research Laboratory at a cost
of more than $1.5M. Located at the Women’s
and Children’s Hospital in Adelaide, the Allan
Scott CF Research Laboratory was opened
in 2012 and is a dedicated research facility
designed for the explicit use of the Adelaide
CF Gene Therapy Research Group.
Scott CF Research Laboratory with several pieces
of major scientific research equipment.
Cure4CF Foundation has taken on the challenge
of ensuring that the researchers have sufficient
funding to effectively utilise this exciting new stateof-the-art Allan Scott CF Research Laboratory and
advance their world leading research.
The Foundation has been actively involved in
campaigns to raise awareness of cystic fibrosis,
and has been successful in furnishing the Allan
1998
First demonstration of an
effective pre-treatment to
enhance airway gene transfer.
2001
Airway surface
changes detected
in CF mice.
2002
First successful
corrective gene transfer
in CF mouse airways.
2005
Gene delivery
optimised for
maximum
effectiveness.
2008
First demonstration of
long-term (>12 month)
corrective airway gene
transfer in CF mouse
airways.
First synchrotron
X-ray imaging of
mouse airways to
improve measurement
of gene transfer success.
2009
First X-ray
detection and
measurement
of particle
movement in
live mouse
airways
2010
Successful
translation of
airway gene transfer
techniques into a
large lung (sheep).
Optimisation of
pre-treatment
method for airway
gene transfer.
2012
Allan Scott
CF Research
Laboratory
opened.
2013
New method for
X-ray detection
of airway surface
changes after
treatment.
Where to
from here?
“Having CF and knowing what it’s like, I wouldn’t wish it on
anyone. When I am sick, some of my friends and people that are
closest to me start to worry. They are worried about how sick I will
actually get and how much longer I have left.”
Andrew, cystic fibrosis patient (1991-2009)
Babies are born with cystic fibrosis every day across the globe, and
their greatest hope lies with medical research. The greatest barrier
to finding a cure for cystic fibrosis is not inspiration, ideas, research
pathways or smart researchers; the greatest barrier is funding.
By supporting Cure4CF you will enable the Adelaide CF Gene
Therapy Research Group to continue their ground-breaking
research, without delay or limitation. This means that they can work
to complete preclinical trials of their cystic fibrosis airway gene
transfer approach. Once preclinical trials have been successfully
completed it is then possible to begin the long awaited move to
human clinical trials.
Cure4CF believes that a future without cystic fibrosis airway disease
is possible, and the work being undertaken by the research team is
already showing great promise. Your support will help ensure that
the research team can continue their vital work and take it to the
next level. For people living with cystic fibrosis, and their families,
progress towards a cure for cystic fibrosis airway disease simply
cannot come soon enough.
CF kids often feel a strong sense of social isolation, as deteriorating lung health can make
it difficult to participate in sport and other physical activities.
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How you
can help.
There are hundreds of ways that you can make
a valuable contribution to the work of the
Cure4CF Foundation.
The most direct and quickest is to make a
donation via www.cure4cf.org
You could also purchase a case of superb value
Climbing Rose wine (details at www.cure4cf.org)
Join many Cure4CF supporters and put together
a team for events such as the annual City-Bay Fun
Run or Tour Down Under Bike Challenge.
Be a part of what may be one of the
most exciting health breakthroughs
of the 21st century.
In fact, you could organise any fundraising activity
that you could think of and utilise the convenience
of Everyday Hero www.everydayhero.com.au to
set up a fundraising page to promote your activity
and process the donations your receive.
Involve your workplace or school and nominate
Cure4CF as a charity to support through your
fundraising program.
Finally, make sure that you “like” the Cure4CF
Foundation Facebook page to stay in touch with
all the developments in pursuit of a cure and
share your support - locally and globally.
For more information on cystic fibrosis airway disease and the work of the Cure4CF Foundation
please visit www.cure4cf.org For further details on making a donation, corporate sponsorship or
hosting a fundraising event please contact:
Rosie Lillas, Executive Director
0411 046 915 | [email protected]
David Coluccio, Chairman
0408 088 829 | [email protected]
All donations to Cure4CF Foundation are fully tax deductible.
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Cure4CF Foundation Limited
PO Box 181
Unley BC 5061
South Australia
Telephone: 1300 131 480
[email protected]
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Local funding. Global solution.
www.cure4cf.org