“Miracle Pill for Cystic Fibrosis”
Frank Accurso, MD
Dr. Frank J. Accurso, Professor of Pediatrics at University of Colorado Denver, School of
Medicine and Children’s Hospital Colorado, talks about a new medication for patients with cystic
fibrosis.
Could you please tell us about this new medication, referred to as a “magic pill,” for
cystic fibrosis patients?
Dr. Accurso: In the past, we have only been able to treat the complications of cystic fibrosis.
Now, this new pill, Kalydeco, allows us to treat the very basic problem, the defect in cystic
fibrosis. This defect stems from one single protein that doesn’t work right in many organs of the
body. This protein is called, the CFTR, and when it doesn’t work right, there are many
complications and that’s what we have had to treat it in the past. Kalydeco actually makes the
protein work better; so it gets at the basic problem with the CFTR.
How big of a breakthrough do you think this is?
Dr. Accurso: We all consider it a great breakthrough. It’s been terrific for the kids who this
benefits. Remember, it’s only about 5% of the population now that actually benefits; they have
to have a specific type of cystic fibrosis.
The medication is effective in just 5% of the people with cystic fibrosis, is that correct?
Dr. Accurso: That’s correct. We are very hopeful that there will be similar treatments for a
much larger group of patients with cystic fibrosis; actually very soon within a few years.
What is cystic fibrosis?
Dr. Accurso: Cystic fibrosis is a condition that affects a number of organs in the body. The
most important organ that can be affected is the lung. Inside the lung, there is thick, sticky
mucus that leads to infection, and the body’s response to infection can destroy the lung over
time.
What is the expected lifespan for somebody with cystic fibrosis?
Dr. Accurso: The average life expectancy is around 37 to 40 years. It’s been getting better for
the last 30 years, but it’s still not improving fast enough.
Will the magic pill have an impact?
Dr. Accurso: We don’t know for sure if Kalydeco will change life expectancy, but we all think it
will. It has not been out long enough for us to get the data to show that changes life expectancy.
So far it is only taking care of symptoms, is that correct?
Dr. Accurso: At this point, it is taking care of symptoms, but it’s also improving lung function
and that’s a very, very good sign. This improvement in lung function has remained steady for a
couple of years, so that is another very good sign because most people with cystic fibrosis lose
some lung function every year.
Can you talk a little bit about the way in which this new drug was developed?
Dr. Accurso: The drug was developed in acollaboration between the Cystic Fibrosis
Foundation and Vertex Pharmaceuticals. I should mention that in order to do this, there was a
great deal of preparatory research done in academic laboratories supported by the National
Institutes of Health and the Cystic Fibrosis Foundation. So a toolkit had to be developed in
order to allow the development of this drug. So there are many, many scientists at universities
and institutes all across the country who contributed to the development of this toolkit.
Why wasn’t it done sooner?
Dr. Accurso: The gene was really only identified in 1989 and it took about 10 years to develop
all of the materials we needed so that this drug could be developed. So in 1999, they started in
earnest looking for a drug that would treat cystic fibrosis, but it took quite a while.
Why don’t the pharmaceutical companies want to represent this drug?
Dr. Accurso: One problem with development of a drug in cystic fibrosis is that it represents an
orphan disease, meaning that there are only 35,000 people in the country who have cystic
fibrosis and drug companies might find it more favorable to treat diseases where there are
millions of patients.
Why is it called an orphan disease?
Dr. Accurso: It means that the disease does not attract attention because a small number of
people are involved.
Was this drug developed with a nonprofit organization?
Dr. Accurso: Yes, it was really the first example venture philanthropy. This is where the Cystic
Fibrosis Foundation, made grants to bring industry into finding a new treatment for cystic
fibrosis.
Can you tell us a little bit about our patient?
Dr. Accurso: Yes, I know Caleb. He is a very active young person, 8 years old, very articulate,
and he has a great family. Caleb was admitted to the hospital a couple of times in the first year
of life; he had some of the other symptoms of cystic fibrosis including abdominal pain, and
malabsorption of nutrients. Now, with Kalydeco, he seems to be doing much better.
He has a regimen of therapy that is kind of complicated right?
Dr. Accurso: Yes he does. He has to do airway secretion clearance a couple of times a day
which means either pounding on the chest to help break loose the mucus, or putting a vest on to
shake the mucus loose. He has to do inhalation treatments every day, probably lasting close to
45 minutes or an hour; sometimes twice a day. He also has to take vitamins because people
with cystic fibrosis have trouble with some vitamins. He has to take a pancreatic enzyme
replacement every time he eats which is in the form of several capsules. That’s because the
pancreas doesn’t work right in cystic fibrosis.
Does he have to take a lot of medication?
Dr. Accurso: Yes, Caleb has to take a lot.
Does that also affect the tips of fingers and toes?
Dr. Accurso: Yes, it does. For reasons that we don’t understand, the capillaries are the little
tiny blood vessels and they begin to proliferate; they grow more than they should in the tips of
the fingers and the toes.
What are the ramifications of that?
Dr. Accurso: Fortunately, it’s only cosmetic. There is no problem with use of the fingers.
This information is intended for additional research purposes only. It is not to be used as
a prescription or advice from Ivanhoe Broadcast News, Inc. or any medical professional
interviewed. Ivanhoe Broadcast News, Inc. assumes no responsibility for the depth or
accuracy of physician statements. Procedures or medicines apply to different people
and medical factors; always consult your physician on medical matters.
If you would like more information, please contact:
Elizabeth Whitehead
Media Relations Specialist
Children’s Hospital Colorado CF Center
(720) 777-2522
cysticfibrosis@childrenscolorado.org
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