The Clinical Significance of Clinicaltrials.gov


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“I think I found the trial that is going to save your life,” Stefanie Joho’s sister said after checking out the ClinicalTrials.gov website.

“And sure enough, it did. That is not an exaggeration. That is exactly what happened,” Ms. Joho, a health advocate and consultant based in Philadelphia, told The ASCO Post.

Ms. Joho was diagnosed with colon cancer 5 years ago, at age 22. “I had just graduated from New York University. It was, of course, very unexpected,” she said. She was treated with two surgeries, FOLFOX (leucovorin, fluorouracil [5-FU], and oxaliplatin), and FOLFIRI (leucovorin, 5-FU, and irinotecan), but the cancer was aggressive and “just grew exponentially to the point where it was totally inoperable, and the doctors told me that there was nothing left to do for me. This was the summer of 2014. I went home with no options and no hope at all.” 

Up until that point, she had been optimistic, she said, “but now I felt it was my time to go. I truly did,” she said. “I was barely 100 pounds. I was sleeping 18 hours a day. I wasn’t living a life at all. I was very, very sick.” 

She had heard from some physicians, however, that there was “interesting work being done in immunotherapy,” she said. So, her sister “scoured the Internet and went on ClinicalTrials.gov. She found this study that had just opened a couple of months prior.” It was a trial at Johns Hopkins evaluating pembrolizumab (Keytruda) for patients with unresectable or metastatic, microsatellite instability–high (MSI-H) or mismatch repair–deficient (dMMR) solid tumors that have progressed despite prior treatment. 

Reaching Out 

“In the next few days, I reached out to my network of doctors throughout the tristate area,” Ms. Joho said. “One of my physicians in Philadelphia confirmed that yes, this is a fantastic study” and facilitated the process for Ms. Joho to enroll. 

Ms. Joho had MSI-H colon cancer and qualified for the trial. “Within 3 months, my tumor had shrunk by 65%, and within about a year, there was no evidence of disease in my body,” she reported. She stopped receiving pembrolizumab in August 2016 and was pleased to report that her most recent scan was termed unremarkable. 

In May 2017, the U.S. Food and Drug Administration (FDA) granted accelerated approval to pembrolizumab “for adult and pediatric patients with unresectable or metastatic, MSI-H or dMMR solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options or with MSI-H or dMMR colorectal cancer that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan.” The approval was based on data from 149 patients, including Stefanie Joho.

On the day the FDA issued that approval, one of the physicians involved in the trial texted her, “We did it,” and another told her, “We couldn’t have done it without you.” Ms. Joho said that exemplified the collaborative spirit and partnership she experienced throughout her participation in the clinical trial and beyond. 

Reassuring to Family 

Ms. Joho is a member of a Lynch syndrome family. Other than her mother, no other members have been diagnosed with colorectal cancer, but “they are very much on top of their surveillance and also very optimistic now. Not that immunotherapy would necessarily work perfectly [for them], but to know that I responded so remarkably to a treatment I think is reassuring to my family as well,” she said.

“I try to be an advocate for clinical trials,” Ms. Joho stated, and clear up misconceptions, such as those she once held, about them. “I was one of those people who thought clinical trials were last-ditch efforts, and participants were guinea pigs,” she said. She now realizes the value of clinical trials for current and future patients. “Everyone is working toward the same goal.” ■


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