For 20 years, Theresa Brennan knew something was wrong. Her energy was sapped, she slept a lot and she suffered from mysterious aches. Doctors thought the pain in her joints might be rheumatoid arthritis or fibromyalgia. But on Christmas Eve 2012, she received a surprising diagnosis: She had hepatitis C.
“I had them run the test again because I was pretty sure it had to be a mistake,” says Brennan, who appears to have contracted the virus after receiving blood transfusions as a baby.
Dissatisfied with existing treatments, Brennan volunteered as a patient for an experimental treatment developed by Duke University. For Brennan, the expert would become the patient; unlike the thousands of other patients who volunteer for experimental drug trials, Brennan has spent her career in medical research.
“I was looking for a cure, and standard treatments at the time weren’t going to get me there,” Brennan says. She’s the senior director of customer solutions at Quintiles Transnational, an outsourcing firm that helps pharmaceutical companies in drug development.
“I’m a mom of twins, a wife, an employee … I just couldn’t be down for the count,” she says.
The advancement of drug therapies into potential medical breakthroughs requires clinical trials on patients before receiving government approval. From start to finish, most drugs take more than a decade to develop through rigorous clinical trials to confirm the safety and efficacy of the drug. For patients like Brennan, volunteering for clinical trials carries hope that outweighs any potential risks.
How it Works
Even though the virus was spreading and she didn’t feel good, Brennan decided to look into clinical trials, including at Duke. At the time, there were no hepatitis C trials available, so she settled for the waiting list. Almost a year later, in October 2013, she got a call: A trial was available, and she was a potential candidate.
Having so much experience on the business end of clinical trials, Brennan had some ideas about what to expect. Before entering any clinical trial, subjects must read and sign a lengthy document called an informed consent form. It exists as one of many protections for clinical trial subjects, and contains a schedule for the study, potential risks and benefits, and numbers to call for help.
Then a screening process occurs to ensure patients meet all criteria to enter the treatment phase. The criteria are universal and set by doctors overseeing the study. They include things such as appropriate medical history, qualifying laboratory values and lack of interfering risk factors. Once a person has met all these criteria, the physician in charge, or principal investigator, enrolls him or her in the trial and starts treatment.
The local research staff, consisting of doctors, nurses and other coordinators, must verify the criteria to ensure patients are healthy enough to participate in research. The screening process and entry criteria also help ensure the quality and integrity of the overall research by only allowing participants who meet certain metrics.
Before she could finish the screening process, which typically takes one to four weeks, Brennan found out the trial was full. She didn’t finish screening in time to be enrolled before the trial met its capacity.
“I couldn’t believe how devastated I was,” she recalls. “Running trials, closing recruitment is a good thing — it brings the drug closer to market. I never thought about the patients who didn’t make it.”
She resolved to wait. Two weeks later, she got a call from Duke. Someone had dropped out of the trial, and she was invited back to finish screening. Brennan jumped on the opportunity and enrolled in the trial.
Treatment
Archie Franklin was diagnosed with psoriasis in the mid 1980s. A couple years later, he saw an ad in the newspaper for a trial for psoriasis patients. There were few treatment options available for his autoimmune disorder at the time, so he entered his first clinical trial riding on hope.
Thirty years later, some initial details are hard to recall. “It’s been so long since I’ve been involved, I can’t remember the year I started anymore,” Franklin says.
Since then, he’s participated in at least seven more psoriasis trials, having been screened for the latest this month.
Being a research subject in multiple trials doesn’t come without its challenges, however. Although Brennan wasn’t in a trial that used a placebo, Franklin has been in many. Whenever a placebo is involved in a trial, patients run the risk of receiving no drug at all; after completing screening, their treatment is randomly selected.
Plus, research subjects must be exemplary patients. Typically, they need to be OK with needles and frequent blood draws, as well as more-frequent-than-average doctor visits. In order for the data they provide to have any integrity, visits must fall along certain timelines. Patients usually have a narrow window of three to seven days to make it to each appointment. Those who don’t show up, don’t take the drug properly or are late to enough visit windows are taken out of the trial early at the physician’s discretion.
Even so, the physician’s job is primarily to ensure patient safety rather than integrity of the trial. In fact, the No. 1 priority of all parties responsible for conducting any clinical trial is subject safety. Patients are also often discontinued from trials when they have alarming lab values, bad reactions to the drug, severe injuries or illness, or just don’t feel like being in the trial anymore. Above all else, research must be safe and voluntary.
Following Up
When a patient exits a trial or stops study medication, he or she still has study visits to attend. Lab draws and physical exams after investigational treatment help ensure patients’ safety once a trial is closed.
Franklin, a pharmaceutical representative, says he’s enjoyed his run — but promised his wife this will be his last clinical trial. That’s not because the trial treatment is problematic, but because he must completely wash out of one experimental treatment before starting another. As a result, Franklin sometimes spends weeks or months without treatment for his psoriasis.
“When you’re not in a trial and you’re not in treatment, it’s just hard,” Franklin says of the plight of a longtime research subject.
Psoriasis is a disease that tends to flare up and remit, and when he’s not on treatment for it, flare-ups are more likely to occur.
“It affects your whole life,” he says of his condition during flare-ups. “You’re itchy, [and] your skin is breaking and bleeding. It gets everywhere, and you’re irritable.”
But the treatment he receives in trials is often more effective than anything on the market for treating those flare-ups. Emerging research on psoriasis includes a number of monoclonal antibody treatments, which are in the super-expensive class of medications known as biologics. Franklin also receives his treatment for free as part of his clinical trials.
Some people enter clinical trials because they have no other means of affording costly biologic treatments. “I could probably afford them if I needed to, but that’s not why I do it; for me it’s not financial,” Franklin says. “This helps people. By doing this, I can help people in the future in my own small way, and I just like to be a part of that.”
Hope
Brennan knows what he’s talking about — she’s made her career in research because she likes helping people. But by the time her hepatitis treatment started, she had to set all that aside.
“It was time to just be a patient,” Brennan says.
It wasn’t long before she got good news. During screening, her viral load, or severity measure of her virus, was more than 10 million. After only a week taking the trial drug, it was down to 47. She arrived in the office for her study appointment to the elated high-fives of office staff and nurses.
Brennan cried tears of joy when they told her the number, then immediately called her husband with the lab results. Throughout her entire ordeal, he’d been stoic and strong for her and their daughters, but the news got him, too, Brennan says. “He dropped the phone. I could hear him — I can still hear him — crying in his office.”
A week or two later, her energy had been restored, and Brennan began feeling better. By the end of study treatment, her labs showed no evidence of the hepatitis C virus in her system. Brennan had found the cure she was looking for.
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What It’s Like to Volunteer for a Clinical Research Trial originally appeared on usnews.com