Donna Culton is expanding dermatological clinical trials

NC TraCS' Clinical Trialist Training Program helps researchers develop, coordinate, and oversee clinical trials in their field of study.

Donna Culton, a dermatologist at the University of North Carolina School of Medicine, specializes in autoimmune disorders of the skin and mucus membrane such as pemphigus and pemphigoid, two groups of disorders that can cause painful or itchy blisters and sores. Pemphigus and pemphigoid are relatively rare—each affect only a handful of Americans out of every 100,000—but these conditions can make life incredibly uncomfortable for the people who suffer from them.

As a doctor, Culton helps patients from across North Carolina and nearby states find care. As a researcher, she also coordinates clinical trials that test potential new therapies for pemphigus and pemphigoid, as well as more common dermatological conditions like psoriasis and alopecia.

Culton first started working on clinical trials more than 10 years ago and has since been involved in more than two dozen different trials, studying the safety and effectiveness of topical creams, oral medications, and injectable or infusion-based therapies. And about four years ago, Culton decided to bolster her clinical trial bona fides by enrolling in the Clinical Trialist Training Program (CTTP) offered through the North Carolina Translational and Clinical Sciences (NC TraCS) Institute.

The CTTP gives researchers an intensive study in the process of overseeing clinical trials, from recruiting participants to collecting data. For Culton, completing the program helped her make the switch from being someone who works on clinical trials to being someone who has the skillset to plan, design, oversee, and empower the kinds of trials that drive her field of research forward.

"There's a seriousness and gravity to being principal investigator on a study—and I needed to know more, to really have a deep understanding of the entire process," Culton says. "The Clinical Trialist Training Program provided that for me."

Culton's work on clinical trials began through a partnership with Aida Lugo-Somolinos, the Director of the Dermatology Clinical Trials Unit at the UNC School of Medicine, who Culton says has been an "amazing mentor." The department had been approached to participate in a trial of a potential therapy for pemphigus, and the two researchers had the complementary skillsets needed to make that trial work. While Lugo-Somolinos wasn't a specialist in pemphigus, she had years of experience with clinical trials—and while Culton didn't have much experience with clinical trials, she knew a lot about pemphigus.

Often, clinical trials are organized by pharmaceutical companies or research organizations that want to test out a new drug or see if a drug already approved for one condition might be useful for another condition. In this case, the company or organization will write the trial protocol, lay out the testing parameters, and fund the work, which is then undertaken by investigators at a network of hospitals, clinics, and research institutions across the country or around the world.

For an early career researcher, being a local investigator for a clinical trial can be a daunting task. That's true even if that trial is sponsored, funded, and organized by a drug company or external research organization, as the local investigator is still responsible for things like patient safety and compliance with FDA regulations. And typically, you don't learn how to do any of this in medical school.

"There are a lot of moving parts," Culton says.

The Clinical Trialist Training Program at NC TraCS helps researchers begin to master these skills. The year-long program kicks off in July and enrolls early career scientists from across the UNC School of Medicine, who also receive 10% salary support for the year. Participants go through a "Clinical Trialist Training Bootcamp," covering everything from institutional review boards to budgeting needs to what to do in case a trial deviates from protocol. Many of these topics are also covered in once-a-month "deep dive" sessions, and participants have regular check-ins with Catherine Barnes, a research program manager on the Training & Career Development team at NC TraCS.

Some people come into the program with a specific trial in mind that they want to work with, while others come in with only ideas of future research plans. But over the course of the year, all participants are expected to find a trial and begin working on it with a clinical mentor, using the resources of the CTTP program to work through any challenges and gain a better understanding of the clinical trial process.

Barnes notes that while the local investigator of a clinical trial will usually end up delegating many of the study's day-to-day tasks, the buck stops with them when it comes to what happens during the trial at their hospital or clinic—and they need to know how every detail of this process works.

"Being a principal investigator is a great responsibility," Barnes says. "The Clinical Trialist Training Program is here to support and train researchers as they take on that responsibility."

Culton has since served as the principal investigator on a number of industry-sponsored clinical trials and is now the Associate Director of the UNC Dermatology department's Clinical Trials Unit, which oversees clinical trials relating to an array of dermatological conditions. After completing the CTTP, Culton was also able to take on a whole new kind of challenge: an investigator-initiated study of a potential medication for oral lichen planus, an inflammatory condition of the mouth.

For this project, Culton, not a drug company, wrote up the protocol, determined the endpoints for the study, and is overseeing every aspect of the trial, from concept to publishing the results. "An investigator-initiated study is when you as the investigator think: I have this great idea, I have this patient population, I think this drug will work," Culton says.

I definitely would not have had the confidence or knowledge base to be able to initiate an investigator-initiated study without being part of the Clinical Trialist Training Program.

"I definitely would not have had the confidence or knowledge base to be able to initiate an investigator-initiated study without being part of the Clinical Trialist Training Program," she adds.

Some of the studies Culton has worked on have even started to yield new hope for patients. In September, the drug company Sanofi (which is developing the drug with Regeneron) announced that dupilumab, an injected monoclonal antibody, demonstrated success in helping patients with moderate to severe bullous pemphigoid. Dupilumab is already used to treat conditions such as eczema, and in February, it was accepted for priority review at the FDA for possible approval in patients with bullous pemphigoid.

That study took place at sites around the world, including UNC, where Culton served as the local principal investigator. If it is granted approval, dupilumab would become the first drug specifically approved for bullous pemphigoid in the US—and Culton says it could be especially helpful to older patients, who have traditionally been harder to treat with existing therapies.

"It would be a game changer for the treatment of this disease," Culton says.

Through these kinds of clinical trials, UNC scientists help spur progress in all kinds of medical fields. It's not simple work—but through the CTTP program, dedicated researchers can ensure that they're prepared and engaged to take on clinical trials, develop their own career, and bring cutting-edge medical science to the people of North Carolina.

"Clinical trials are complicated—and working with sponsors and industry and with UNC to make sure everything is done properly is key," Culton says. "The CTTP program is really meant for people who want to take this work to the next level, have it be a part of their career, and do it really well."