Jason Klok

Professor Presnell
UWRT 1103
10/26/14
Cystic Fibrosis: Will It Always Be a Death Sentence?
My sister was born one year and eight months following my own birth. Of course I
do not remember this event, but I have been told I was present. Ive even been told I started
to cry when she was quickly whisked away from her family only minutes after her entrance
into this world. As she was tested and prodded by a team of professionals my mom held me
tight as she cried and my dad fought with the hospital staff to know what was wrong. It
scares me that such an important part of my life happened without my being aware. The
world handed me a little sister, and gave her a death sentence on the same day and I didnt
even know it.
The Impact of the Disease
My first memories are of the florescent lights, and sterilized air of the hospital room
my sister lived in for the majority of her young life. The fact that I was surrounded by
grown men and women wearing blue gowns and the fact that most of the children I played
with in the common room of our floor were bald and frequently disappeared from my life
were not strange to me. This was simply the world I lived in.
It was not until about second grade of elementary school, that I began to realize that
my life was strange to others. I often went home to my grandparents while my dad was on

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business and my mom was at the hospital with my sister and only as I started to visit my
friends houses did I realized that this wasnt normal. I began to miss my parents and my
little sister, realizing that a life without them was unfair.
My dad quit his job, which required his frequent traveling and absence from the
house, and luckily my sister was released from the hospital with many treatments and
private nurses that could care for her in the comfort of her own home. This of course came
as a great relief to me. I continued my life as a very bubbly young boy and lived my very
normal life given to me by my caring parents who tried every day to remove me from the
stress of the fight to keep my little sister healthy. However, some time during my middle
school education, I was made aware that my little sister had a very low life expectancy, and
this began to eat away at my heart. She could actually die at any time and this stuck to the
back of my thoughts every day. She was my best friend, I had never lived in a world without
her and the thought scared me.
As the years went on, treatments improved exponentially and my sisters life span
increased more and more, bringing me more and more hope and alleviating the pain from
the back of my thoughts. One day during my Junior year of high school, My sisters doctor
called the house to inform us of an experimental cure that could possibly allow my sister to
live a normal life. The cure was said to be compatible with the strain of the disease present
in my sister and we were told to think about whether or not we were willing to try this
cure. The very same night however, we were called again by the doctor to tell us that my
sister also had part of another strain of the disease which would likely cause complications
in the treatment. Just like that, the hope of a normal life was given, and taken from us in
only a matter of hours.

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Ever since this night I have been left with a small amount of hope that a cure for my
sister will be made and, in a split second, I will be able to have my grandchildren meet their
aunt. I will be able to introduce the future love of my life to the love of my life that has
always been there, and now, always will.
What is Cystic Fibrosis?
The origins of Cystic Fibrosis come from the Dutch peoples of northern Europe
(Rosaler, Maxine). Still mostly only being seen present in White races, it is commonly
thought of as a solely Caucasian ailment. The major symptoms of CF include a thickening of
Mucus within the lungs of the child and an inability for the stomach to produce digesting
enzymes (Rosaler, Maxine). This results in the inability for a newborn with CF to be able to
breath or digest food, resulting in a quick death for the baby up until about 30 or 40 years
ago.
The disease also causes liver and pancreas deficiencies in the production of import
agents in the body such as white blood cells and insulin, resulting in easy contraction of
diseases and the common development of diabetes by those who have CF (Rosaler,
Maxine).
How is CF Treated Now?
Treatments for CF have increased at an amazing pace in the last ten years. My
sisters expected life span has doubled since the day she was born and she is now able to
live a pretty normal life. Drugs such as Pulmizine can be inhaled directly into the lungs to
thin mucus and fight infection. Oral enzymes can be ingested before each meal to allow for
more efficient digestion. Physical therapy machines that have been created allow for a

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much more efficient way of loosening the mucus within the lungs. All of these treatments,
as well as increased treatments for diseases that may branch off of CF such as Diabetes,
have allowed my sister to hide her disease from all but her closest friends and enjoy an
almost average day to day life.
How are Treatments progressing?
The hope I spoke of earlier towards a future including my little sister, sparked my
curiosity and resulted in my digging towards possible cures and more effective treatments.
In my research I found many, very promising cure and treatment opportunities. Personally,
the possible cure that brings me the most hope, the same possible cure offered to us that
night, is genetic therapy.
In genetic therapy, virus cells are injected with healthy genes to replace the faulty
CF genes in the patients body. The virus is then injected into the body and the virus cells
replace the faulty genes with the healthy ones. Great progress has been made in the
research towards a practical use of this method. Keith Wailoo and Stephen Pemberton
write Twenty years from now, predicted gene therapy pioneer W. French Anderson in
1996, gene therapy will have revolutionized the practice of medicine. Virtually every
disease will have gene therapy as one of its treatments. In the last ten years we have gone
from only being able to cure CF in a test tube, to being able to cure every cell in a single
body part of a patient (Drexler, Michael). This being so, the cure is still not ready to be
released. The virus cells used to implant the gene can damage the body if used in the
amount needed to cure a significant portion of the body, such as the lungs (Wailoo, Keith
and Pemberton, Stephen). These cells are also attacked by the bodys natural defenses and

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so small amounts would do very little to help the patient. Overcoming this is the next
crucial step in developing this cure.
The most promising progress in further treatments comes from cocktail drug
research. At the most basic level of the Cystic Fibrosis disease, it is observed that the cells
of a patients body do not sufficiently transfer electrolytes. This results in the thickening of
mucus and many other symptoms experienced by a patient. Drugs being produced by
research teams, attempt to fix the symptoms of CF by attacking this most basic cause of the
symptoms. One drug allows the cell membranes to become much more fluid and
permeable, as is found in the cell membranes of healthy human beings (Rosaler, Maxine).
Another drug excites the parts of the cell, which transfer electrolytes to and from other
cells, to the point found in healthy cells (Rosaler, Maxine).
Both of these research subjects stands on the brink of application in patients lives,
however, the time needed to make these applications possible, is unknown. Unfortunately,
there is no clear answer to my question of will my sister be able to live a normal life? This
being said, there are ways for me to fight for this possibility.
Funding of CF Research
The vast majority of funding towards cystic fibrosis treatments and possible cures
comes from the Cystic Fibrosis Foundation. Fund raising events such as Pipeline Towards a
Cure and Sixty Five Roses, raise millions of dollars a year towards this research (PRWeb).
Auctions, races, and dining events are held annually, and more and more fund raising
opportunities are being created every year. If I want to help fight for a cure for CF, I should
support one of these events.

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The Sixty Five Roses fund raising initiative stands as the most compelling and
relatable event. The origins of this event come from a young boy who was stricken with the
illness, and his mom (Bjorklund, Ruth). As the mother spoke with others over the phone
that wished to put together a fund raising event, the little boy told his mom that he knew
what she was talking over the phone about (Bjorklund, Ruth). When the mom asked what
he thought she was talking about he replied with sixty five roses (Bjorklund, Ruth). This
young boys confusion between the pronunciation of Cystic Fibrosis and Sixty Five Roses is
how the event got its name and received the sympathy it has needed to create such a huge
impact towards the Cystic Fibrosis Foundations efforts to support treatment research.
Is There Still Hope?
After my research of Cystic Fibrosis, My hope of having my sister live a long healthy
life has increased. I now know how extremely close we are to these amazing cures and
treatments that could save my little sisters life. Realistically, These cures could be fully
completed tonight as I write this paper, and able to be used on my sister within a month.
This being so, I also know that we may never be able to complete these cures and my sister
may have to struggle to reach her 50s. I am left with the determination to fight for the
organizations that are trying their best to allow my sister to live a full life. Because of these
organizations, I have my sister healthy and happy right now and I will be forever grateful
for that, but the fight is not over until my little sister sits down for a thanksgiving meal with
my grandchildren, and is able to have a meaningful talk with them about what it really
means to overcome hardship.

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Works Cited
Drexler, Michael. A Cure for Cystic Fibrosis? Rainbow Cheers New Gene Therapy The
Plain Dealer. [Cleveland, OH]. October 16, 1993: Newsbank Web. 16

November

2014
PRWeb (USA). Fifth Annual Pipeline to a Cure Gala Successfully Raised $465,000 for
the Cystic Fibrosis Foundation PRWebDateline. [Huntington Beach, CA]. Jul 17,
2012: Newsbank Web. 16 November , 2014
Rosaler, Maxine. Cystic Fibrosis The Rosen Publishing Group, 2007 Googlebooks,

web.

16 November, 2014
Gibbons, John H. United States. Congress, Office of Technology Assessement, 1992
Cysitic Fibrosis and DNA test: implications of carrier screening Googlebooks
Web. 16 November 2014
Wailoo, Keith and Pemberton, Stephen. The Troubled Dream of Genetic Medicine:
Ethnicity and innovation in Tay-Sachs, Cystic Fibrosis, and Sickle Cell Disease JHU
Press. 1 April, 2008. Googlebooks Web. 16 November 2014
Bjorklund, Ruth. Cystic Fibrosis Marshall Cavendish, 1 September, 2008
Web. 16 November 2014

Googlebooks.