Four Questions and Nine Things To Consider Before Committing to a Gene Therapy Trial

Bobby Wiseman Offers Tips from Personal Experience

By Ranae Baker

At 50, Bobby is an active member of The Coalition for Hemophilia B and an avid Star Trek fan. As an African American gay man with severe hemophilia B, Bobby has found his voice advocating for more than a couple of underrepresented groups.

He is an avid researcher who tenaciously works to illuminate important issues with which he is intimately familiar. Gene therapy is one of those issues, and he wants to help you have a well-informed experience when deciding whether a gene therapy trial is right for you.

Bobby has been participating in a phase 2 clinical gene therapy trial since September 2018. Deciding to participate was a big decision that he took seriously. He dove deeply into as much research on the subject as he could. Indeed, his research began about ten years ago when he first started hearing “rumblings” about gene therapy at national and international bleeding disorder meetings.

He opened a dialogue with his doctor about it, and they began sharing new information they learned with each other. Gene therapy was such a new field that Bobby felt the need to reach a comfort level with what he was learning before volunteering his time and body to the scientific study of it.

Bobby also felt it was important to have serious conversations with family and close friends about the possibility. “It was so new,” he explains, “that I wanted to tell them, ‘Hey, I may need some support along the way.’” Bobby’s spirituality has gotten him through a lifetime of challenges, and prayer was a big piece of his process of navigating through his fears.

On the other hand, his desire to give back to the hemophilia B community and to see what gene therapy could do for hemophilia helped him make his decision. A conversation he had with his mother clarified that he would be helping other people by participating in the trial.

He was also reminded that very few people of color participated in many of these trials, and he wanted to help add some of those data points to the research. After a few weeks of weighing the pros and cons, he decided he would like to be part of the trial. He thought he was as prepared as he could be to make this decision, but he would soon find himself in unforeseen situations.

He doesn’t regret participating in this trial, but there are questions he wishes he had known to ask in advance. He shares his hard-won wisdom with us so that you may make a well-informed decision should you consider participating in a gene therapy trial.

Things to consider

1. Time commitment.
The first point Bobby wants to stress is that being in a gene therapy trial is a big commitment! “You can’t schedule these visits around your calendar and location. The samples need to be drawn at the study site, because there are certain protocols around the storage of the tubes,” Bobby emphasizes.

In his case, the trial is a five-year trial. Although the treatment (the vector) may only be administered one time, his commitment to be a monitored study subject will continue until the five-year mark, and maybe even beyond.

2. Consider the travel time involved.
When Bobby started the trial, he lived in Chico, California, and traveled an hour and a half to UC Davis. The devastating “Campfire” in 2018 prompted Bobby to move his family to Phoenix, AZ. After the move, he had to fly back and forth from Phoenix to San Francisco for the study. Bobby is now able to have his gene therapy visits in Phoenix.

3. The study visits may be more intimate than you expect.
Bobby was taken by surprise when, in the beginning, phase of the trial, he was asked to give a semen sample on-site. “Yes, you may feel comfortable signing up for the study,” Bobby understands, “but if you’re a male in a study with traditionally female research coordinators, that may be an issue.”

4. The frequency of visits may surprise you.
At the onset of the trial, Bobby had to be seen by the trial team weekly. That transitioned to once every other week, then every three weeks, once a month, once every other month, every four months, and then every six months. Bobby is now being seen every six months.

When does it end? “That’s the unknown,” Bobby cautions. He remembers back to a hep C trial he did. “It was supposed to be a five-year study, but they kept extending the deadline.”

5. There is homework!
“I got in trouble,” he admits with lingering aggravation, “because I would not fill out the diary. You had to do it daily! And by a specific time! You must report every little condition and medication, even if it’s over the counter.”

Bobby worked full-time as a project coordinator for people with IDD and co-parented four foster kids with his husband. The daily journaling was just too much for him, and he let them know. “It got to the point that they would email the research coordinator to ask me to do it.”

6. Misconceptions.
There are misconceptions about gene therapy due to varying degrees of education among the general public and even within the local chapters of the various health focused societies. “I think there is a strong belief in the bleeding disorders community that if you get gene therapy, you are cured, and you won’t have to treat anymore,” Bobby says. “That is not the case.”

When Bobby looks at his gene therapy trial experience, a key element fueling it is the very interpersonal relationship he has been maintaining with his doctor and the research coordinator.

7. You may have to educate your health providers.
For instance, Bobby is typically prone to sinus infections. At one point, during the trial, he had to go to the ER for acute sinusitis. The doctor looked at his chart and saw that Bobby had been diagnosed with severe hemophilia b, making him unsure of what to do. It was hard for the doctor to understand that they only needed to treat the sinusitis.

8. You may be confused.
Being part of a gene therapy trial can be amazing and confounding at the same time. “My definition of gene therapy used to be that I wouldn’t have to treat anymore,” Bobby admits.

“In the trial, my levels were going up from my less than 1% and steadily increasing. My whole concept of being a “severe hemophilia B” was changing weekly. My levels were going up so much that the doctor called me to ask me if I was treating in between study visits.”

As his levels increased, Bobby went about living his life. “Things happened,” Bobby divulges. “I was doing stuff around the house, and I got a bleed in my calf. My brain said, ‘Treat!’ But then I thought, ‘Wait a minute. I can’t do that. I’m on a trial, and I have to talk to the doctor about what to do.’”

Bobby knew if he treated it, he might invalidate the data of the trial. “So, I called the doc, and he said, ‘Well we want to make sure it’s a bleed.’ That was one part of the conversation. And then he also said, ‘We still want you to be proactive and treat it if it’s a bleed.’” So, Bobby felt “stuck in the middle.”

9. You may find yourself in unexpected emotional states.

Everything he’d been taught for so many years was shifting. Traveling was much easier. He found himself able take extended trips without worrying about factor conditions. But there is also a no man’s land aspect for people who have participated or who are currently participating in gene therapy trials. There aren’t established support systems for them, at this early stage, and yet, there are unanswered questions and insecurities.

What happens at the end of the trial? How do we proceed? What happens if our levels start to decline? Nobody can answer these questions. The product on trial may or may not come to market. How will that affect us?

Bobby reiterates that he doesn’t regret being in this study. He is happy to be a guinea pig for the community, and he is hopeful that these trials will yield improvements for his hemophilia B community. For those who decide to take this on, he offers this advice:

“Be patient with the process and with yourself. The nurses and social workers don’t yet have the tools and resources to offer their patients, which can lead to frustration.”

Questions to Ask

1. What is the actual time commitment? “

2. Will the study team be able to provide transportation to and from the study visits?

3. Who do I talk to besides the doctor, the nurse and research coordinators?

4. How will this affect my intimate relationship(s)? Bobby encourages people looking into gene therapy trials to invite your primary support person to the initial discussion sessions with the treatment center. Not only may they ask questions you may not have thought of, but it may be helpful to have their set of ears taking in the discussion about how the trial will impact your life.

Conclusion

“My fear for gene therapy,” he begins, “is that when it does become a readily available product that is successful for those in the bleeding disorders community, it may be cost-prohibitive.

I’m also concerned that not all the community will have access to the information and be able to have someone advocate for them because of the cost.”

Bobby’s philosophy:

“Just because it’s new does not make it right or wrong. Do your own research. If you don’t understand it, Google it, ask your doctor, or call up your nurse hotline.

Don’t limit yourself to the hemophilia B community. Reach out to other communities who are doing gene therapy and learn from their experience.

If you do decide to participate in a gene therapy trial, know that your pioneering efforts to go ‘where no man has gone before’ will be a tremendous help to the bleeding disorder community and would be worthy of Captain Kirk, Piccard, and even Janeway!