In an interview with Pharmacy Times®, Chip Hailey, a 74-year-old retired pharmacist from Lebanon, Tennessee, candidly discussed his lifelong struggle with hemophilia B, detailing the challenges of growing up without effective treatments and how participating in a gene therapy clinical trial of etranacogene dezaparvovec (Hemgenix; uniQure, CSL Behring) dramatically transformed his quality of life, providing him unprecedented freedom from constant medical management.
Pharmacy Times: What was it like growing up with hemophilia B in an era without treatment options? How did that shape your view of health and independence?
Chip Hailey: It was very frightening as a child, not knowing, not having the maturity to understand your situation, especially a complicated health care condition like this. I was scared to death. My parents were great. My family was great. Everybody was looking out for me. "Don't do that. Watch what you're doing." So many restrictions; it was a nightmare. I'll give you an example. There was nothing to take a day. I was a child in the '50s, and there was essentially no treatment. Sometimes I'd wake up in the middle of the night, and my knee would be swelling, and there was absolutely nothing anybody could do. I didn't even want to keep my parents up; I just laid there and suffered with the pain and the immobility of it the next day and just essentially wore myself out. That happened dozens and dozens of times.
Regarding my independence, certainly all during my adult life, I've had to make decisions about jobs based on health care: whether I could get health care coverage and proximity to the hemophilia treatment centers. I actually worked a job at one time and considered moving out west to a contract and start work there, but I looked, and there wasn't a close hemophilia treatment center. I just said, "Nope, I can't do it." I've kind of been trained to middle Tennessee, which is fine; this is where I'm from. But again, it's always my proximity to Nashville. I actually live in Wilson County, which is just a little bit east of Nashville, and I built a house closer to Nashville so I could actually be there quicker.
Key Takeaways
1. Growing up with hemophilia B in the 1950s meant living with severe restrictions and no effective treatment options, causing significant physical and emotional challenges.
2. As a pharmacist, Hailey maintained a proactive approach to understanding hemophilia research, actively seeking clinical trial opportunities for potential treatments.
3. Gene therapy has eliminated his need for regular factor IX injections, giving him newfound independence and reducing the anxiety associated with managing his condition.
Pharmacy Times: As a pharmacist, how did your professional background influence your understanding of the evolving hemophilia treatments?
Hailey: It was kind of dumb luck that I became a pharmacist. A friend of mine was going to pharmacy school, and he said, "Hey, Chip, come on, why don't you go with me?" And I said, "Fine." That's how I decided to be a pharmacist. But it actually turned out to be a great choice. The education I got at the University of Tennessee College of Pharmacy in Memphis was great. It was a great foundation for my knowledge. One thing that pharmacy school taught me was that if you didn't know the answer to something, it wasn't a crime. It was a crime if you didn't know where to find the answer. Being a pharmacist and knowing the available literature and where to find it, and the doctors to ask, that was just great. I couldn't have asked for a better situation.
Pharmacy Times: What were you thinking about when you decided to enroll in the etranacogene dezaparvovec clinical trial? What ultimately convinced you to take that leap?
"How many times as a patient do you get the opportunity to make a choice that can change your outcomes for the rest of your life?"
Hailey: Well, this is a long journey. This didn't sneak up on me. At 10 years old, an aunt that lived in Charlotte, North Carolina, sent me a newspaper clipping. It's about 1960, and it was about Duke University doing basic research on hemophilia. I didn't keep that article for a year or two; I kept it for decades. At a young age, I knew somebody was out there barking on my behalf. I go off to pharmacy school in Memphis. We had a great library. I'd search all the periodicals for news on hemophilia--never found any. Twenty-five years ago, I got a letter from one of the early researchers asking me to be in one of their clinical trials—I mean, one of the very first. For many reasons, that didn't come about. Then 10 years ago, my hematologist called me up and asked me to be a participant in a gene therapy clinical trial, specifically for factor IX—that fell through. I actually found another gene therapy clinical trial, and I got to within 2 days of having that done. Due to circumstances, it didn't work out. But that didn't stop me there. I actually got online, found uniQure, and I told them I'd go anywhere east of the Mississippi on my own dime to be in a clinical trial. They said, "Well, just get in touch with your doctor at Vanderbilt; we have a spot for you." It was an opportunity that I couldn't pass up. The 5 years from the very first meeting that I had with my hematologist, all of those 5 years were filled with me doing research. So, it wasn't hard to make that decision.
Pharmacy Times: How has receiving gene therapy changed your daily life, both physically and emotionally, compared with managing your condition with weekly injections?
Hailey: It's freedom from the need. It's freedom from having to sit down with a calendar and figure out when your next infusion is going to be. I calculated a few days ago that gene therapy has saved me at least 280 times not having to infuse. I've been very lucky with this gene therapy, to the point that I don't use factor IX anymore, nor do I actually have any at home. I don't take any with me on vacation. Let me tell you a little story. About 11 years ago, I was fortunate to travel to Europe, and the morning I was going to get ready and go to Nashville, I got my factor IX out, mixed it up, and I stuck myself 10 times before I gave up. At that time, I was using a short half-life medication called BeneFix (Coagulation Factor IX [Recombinant]; Pfizer). I had to get that at least 3 times a week. I actually went to Nashville, got on a plane, and went to Geneva, Switzerland. Twenty-two hours later, I got to a B&B and got my medication out and mixed it up. I missed again. I missed infusions—missed a vein—11 straight times in 2 different countries across the Atlantic Ocean. I did hit it the 12th time. That anxiety that I had, I can still relive it today. I don't miss that one bit.
Pharmacy Times: What does freedom from constant treatment and bleeding concerns mean to you personally?
Hailey: One thing that I found out was that I was thinking about things that I didn't have to think about anymore. First of all, no infusions. Very few doctor visits; I did get followed up because I was in the clinical trial, and hopefully they'll follow me for the rest of my life. I don't have to worry about the storage of my factor IX. I don't have to worry about procurement of it. I don't have to worry about the supply chain.
"That anxiety that I had, I can still relive it today. I don't miss that one bit."
Pharmacy Times: Is there anything else that you would like to add?
Hailey: The Hemgenix that was used with me, you have to remember, is just a one-time deal. It was easy. I had some appointments running up to it. Of course, they were checking how my health was. The day of the infusion was just a day at the office. It took about an hour for infusion. Again, I was one of the lucky ones that had no [adverse] effects. One thing about this, getting to participate in this, getting to choose Hemgenix, it's like, how many times as a patient do you get the opportunity to make a choice that can change your outcomes for the rest of your life? I made that choice, and it was the best choice I've ever made in health care.
