By Deborah Borfitz  

February 3, 2026 | ORLANDO—For patients with a rare disease participating in clinical trials, hope can be a “doubled-edged sword,” potentially saving their lives but also burdening them with enormous, if unintended, hidden costs. Hope is also their “trust in us” as they desperately search for restorative treatments like chimeric antigen receptor (CAR) T-cell therapy, Jane Myles told a packed house during the opening day plenary keynote address at this week’s SCOPE event in Orlando.  

Myles, program director for the Decentralized Trials & Research Alliance and board member with The Myositis Association, was moderating a fireside chat with a pair of CAR T patients about the realities of their treatment journey and trial experiences. The stories were shared by Brad Watts, a life science and technology consultant and patient advocate with the Emily Whitehouse Foundation, and Lindsay Guentzel, a multimedia journalist and professional patient. 

Being in a clinical trial is hard even if everyone—sponsors, sites, regulators, and patients themselves—mean well, says Myles. “It’s our job not to make trials a super-human effort for people to be part of.” 

It has been more than two decades since Myles was diagnosed with dermatomyositis and, at the time, patients like her were devising “trial and error” treatment strategies with their doctors. Science has since then progressed to the point where many rare disease patients have trial options, possibly multiple ones, and, if they are lucky, have the chance to consider participating close to the time of their diagnosis, she says.   

But navigating the clinical trial system is an entirely new experience for most patients, says Myles. CAR T-cell immunotherapy being a complicated, high-stakes treatment just amplifies all the issues. 

The treatment journey, for Watts, was a nightmarish ordeal. In 2017, at the age of 29, he was diagnosed with diffuse large B-cell follicular lymphoma. It’s a particularly aggressive form of non-Hodgkin lymphoma that more typically strikes people around 75 years of age, he shares. Watts underwent the standard of care for four years before finally getting CAR T, the treatment he was hoping for. His disease is in remission. All told, he spent more than 250 days in the hospital receiving chemotherapy, radiation therapy, immunotherapy, an autologous stem cell transplant, and excision of a malignant node.   

Minnesota native Guentzel likewise credits CAR T with reversing a downward spiral that began suddenly in 2023 when she was diagnosed with dermatomyositis associated with a rare and incurable autoimmune disease (anti-synthetase syndrome), along with Sjogren’s disease.  “It’s how I know Jane,” she says of Myles. “I call it the worst club with the best members.” 

Over the past three years, she has been to more than 550 doctor’s appointments and spent 40 days in the hospital. Guentzel is on month 10 of getting CAR T via a clinical trial. 

Finding CAR T  

Before Guentzel got sick, she recalls that life couldn’t have been better. “I have an amazing partner, we have a beautiful home, great friends and family” and, workwise, everything she had been working for was finally within reach. “I honestly thought everything was ahead of me, and then one day it wasn’t.” 

Interestingly, the treatment landscape for myosis in 2023 looked about the same as it did for Myles years earlier, says Guentzel. “There were a lot of optimistic conversations happening ... [and] a lot of drugs they wanted to throw at me, but there was not a lot [enabling me to] get my old life back.” 

On the outside chance that could happen, Guentzel did everything in her power to improve the odds. “Very early on in my journey I found out about CAR T-cell transplants that were happening in Europe for autoimmune disease patients ... [and] very early results were incredible.”  

The wait wasn’t as long as her doctor had kindly prepared her for. At a 2024 conference put on in Baltimore by The Myositis Association, she met a representative from a pharmaceutical company recruiting for a CAR T-cell therapy trial enrolling patients with an autoimmune disease. Back home in Minneapolis, she made it her full-time job to secure her spot in the study, “because I knew how good it could be.” 

When CAR T entered Watts’ world, what he hoped for changed throughout his treatment path. “I was incredibly fortunate [because] I lived 30 minutes away from the University of Pennsylvania where cell gene therapy was born, and I knew about CAR T very early on,” he says. 

Watts remembers well the hope that arose in him in 2017 upon learning that the Food and Drug Administration had approved Kymriah (Novartis). He had just finished his first standard-of-care therapy, giving him his first and to this day only complete therapeutic response. “I didn’t know it at the time, but [Kymriah] was the therapy that I would be having four years later.” 

He was both “incredibly excited” and “genuinely curious” about the prospect of gaining access to Kymriah in the future. “I love the promise behind it,” he says, he likens to a vaccination against his specific type of cancer. 

Unexpected Hurdles 

As Watts got started on his treatment journey, hope became part catalyst and part burden, he shares. While dealing with his caregivers, he was simultaneously juggling work, paying bills, and dating his girlfriend who turned into his wife. “I wanted to get this over and done with and move on with my life.” 

Despite being incredibly well looked after by his doctors at Penn, Watts wanted to get CAR T-cell therapy as soon as possible. “There were times when I was literally ... hoping to fail [other] treatments, so that I could gain access to clinical trials for CAR T.”  

In Guentzel’s journey into clinical trials, her discovery was that “despite the fact that patients are an integral part of [such studies] ... the process to get into one is not very patient friendly.” It took her a month “just to identify which sites were going to work with patients with myositis,” she says. Then it became an issue of getting someone at a recruiting site to return her calls. Never mind that all the sites she had to choose from were out of state.  

She has effectively put her life on hold since September of 2024, says Guentzel, adding that simply scheduling her initial screening trip took six months. “There were delays after delays” and, as desperation set in, all she could think was “if I don’t make myself available 110% when they call me are they going to give my spot away?”  

As she tells it, “If you had told me to go up to the roof and jump off, I would have done it—anything to get myself into that trial.” She read only the “CliffNotes version” of all the consent documents, motivated by hope. 

“Being a patient in a clinical trial is a lot of work,” says Guentzel. “You don’t know what you don’t know, you don’t know what to ask, and the unfortunate reality for me is I did not have a reliable point of contact at my clinical trial site and that has made things really difficult ... to this day.” 

From a clinical standpoint, she has had “the best response to this treatment,” she clarifies. “My symptoms are in full remission and have been for months ... [whereas] just a year ago, I wouldn’t have been able to travel to Orlando by myself.” 

Guentzel, who at one point couldn’t independently get out of bed, is today running for five minutes on the treadmill. “So, it has been worth it,” she says, “but I feel like I am learning all the things not to do.”  

Addressable Realities  

For Watts, decisions were being made under the pressure of time, and he longed to move past this stressful period of his life. He recalls naively thinking the entire ordeal would be over after his first six rounds of chemotherapy; he hadn’t considered what the treatment experience would be like, or life thereafter.  

“The aggressive nature of the cancer made it difficult to take a step back and think about what other clinical trials are out there” beyond those he was exploring at a few select institutions, says Watts. When genetic testing was eventually brought up, he didn’t recognize its significance.  

That changed in 2020 following Watts’ autologous stem cell transplant, which he was told would give him the best chance of getting 10-plus years of cancer-free remission. The procedure immediately failed, and genetic testing later revealed that it would never have worked because of a BRCA2 mutation he carries. His standard-of-care treatment would have gone differently had his doctors known. 

This was a telling example of how patients don’t necessarily understand the consequences of their decisions, Myles interjects. “It happens a lot.” 

One of the areas where Watts now advocates the most is better education of caregivers and physicians in whom patients put their trust. He also supports expanded access beyond the institutions where CAR T trials are currently conducted. “I’m very passionate about CAR T ... because it’s hard not to be when it saved your life.”  

All the chemotherapy and other standard, first-line therapies he underwent were “God-awful,” says Watts, calling the treatments comparatively “archaic and barbaric.” Could he go back and begin treatment anew, he would opt for CAR T “10 times over.”  

Watts says this with the knowledge that his cancer ordeal is not yet over. When he went on a drug holiday for his wedding, he was told “it’s not a matter of if [but] ... when it comes back,” he points out. He continues to stay up to date on clinical trials and new drugs and therapies coming out because his life could literally depend on it.  

As for Guentzel, she realizes now that she had “turned a blind eye” to what she endured in the clinical research process. “I should have shopped around. I love my clinical trial care team ... but I didn’t realize different sites were going to cover different things” and in some cases use a concierge to book her travel. 

Inexplicably, she was expected to pay for everything out of pocket and wait for reimbursement. “It feels like in 2026 when I can send any of you in this room money in a matter of moments ... patients shouldn’t be paying out of pocket for anything,” Guentzel reasons. “I shouldn’t have to scan receipts at the end of the day for travel, and to benefit the greater good. 

“And yes, I am benefiting,” she continues, “I’m a walking advertisement for how important [CAR T] treatment is for the autoimmune community, and my messages on social media are a sign of that.” She and Watts often hear from patients whose doctors have misled them into thinking CAR T-cell therapy is “more intense” than other medications they’re already taking every day. 

Guentzel’s best friend, who has been at her side throughout her treatment journey and heard about all the logistical issues, was as surprised as anyone that she wasn’t taken care of better, believing as many do that participants who willingly give their body to science get catered to in return. While grateful for getting a treatment that is working well for her, Guentzel unapologetically asks for what every patient needs—help in “dealing with all the other stuff on the back end.”