Voices of Vertex

THE CF TRAILBLAZERS

Our researchers have spent the last 20 years in the relentless pursuit of medicines that strike at the core of cystic fibrosis. We call them the CF Trailblazers. These are their stories.

THE VISIONARY

PAUL NEGULESCU
Years working on CF: 20
SVP, San Diego Research & San Diego Site Head
Paul_N

AH-HA! Moment

I remember when we were first getting started with our CF research, and we were considering various options. During this time, I met a man living with CF who told me: "whatever you do, don't make another inhaled therapy." That moment helped me see that we needed to focus on a pill that would treat the underlying cause of this disease.

I learned about CFTR from scientists, but I learned about CF from patients and their families.

THE MEMENTOS

"Every person with CF who I've met and every momento I've received along the way is a reminder that we aren't done. Over the years, I've had the incredible opportunity to meet so many people living with CF. I have a bookmark given to me by a 6-year-old child with CF, who is now 18. In one of the most emotional experiences of my life, a man living with CF in Germany gave me a ring he's worn his entire life. I experience so much joy when I meet someone who is taking one our treatments, but then I'll meet someone who isn't eligible and I'm hit with the reality of how much more work we have to do."
 

- Fred Van Goor, Principal Research Fellow (16 years working on CF)

THE INNOVATOR

FRED VAN GOOR
Years working on CF: 16
Principal Research Fellow
Fred_VG

AH-HA! Moment

When I first saw the proof-of-concept data for our first CF medicine, it was the day when all the science came together. It confirmed that we were on the right path. But I also recognized we had more work to do to bring a medicine to all people with CF.

The field of CF research has completely shifted throughout my career to a focus on CFTR science and our first CF medicine played a huge role in that. I don't take for granted how incredibly lucky I am to be a part of this for so long.

USSING CHAMBER

Ussing

"The Ussing chamber allows us to test the activity of our compounds in human bronchial epithelial cells. It's like creating little lungs in a dish. The first time we received these kinds of cells from a patient with CF, we couldn't wait to test to see if there was activity when we added one of our investigational medicines. I had the experiment scheduled for a Thursday. But when I came in that day, the lab was a mess. It turns out that Fred Van Goor was so anxious to run the test that he had come in at 1 a.m. and ran the experiment! The rest is history as it was the first time we saw activity – the cilia beating and a decrease in mucus. For me, that's when I knew we were doing something special. I knew I had more than just a job and a real shot at helping people living with this disease"
 

- Bill Burton, Research Scientist II, Biology