Clinical trial saves man initially given weeks to live: "Not done yet"

Ryan Goepel knew he didn't always treat his gout as well as he should. The inflammatory condition can be triggered by diet and lifestyle factors, and flare-ups can cause pain and discomfort.

When the Florida father of three's knee started hurting during a trip in September 2024, Goepel thought it was just one of those flares. But when the ache didn't go away, he went to his doctor. She gave him some medication, and took blood to run tests. Two days later, she called with alarming results.

"She said, 'Stop whatever you're doing, and go straight to the hospital.' And I'm like, 'What do you mean?'" Goepel recalled. "She says, 'Just go. You have no immune system. I don't know what's wrong. Just go.'"

Goepel rushed to a nearby sports medicine hospital, where he underwent another round of blood work. He became alarmed when an oncologist came to discuss the results.

"I'm like, 'Why are you here? I have a sore knee,'" Goepel said. "And she's like, 'Well, actually, you have leukemia.'"

His condition was critical, and his organs were on the verge of shutting down. The doctors told Goepel's girlfriend that they should consider hospice care for the then-49-year-old.

"It was so bad, they didn't tell the patient, they told the family," Goepel said. "They just basically told her: 'You should make arrangements. He's not leaving alive.'" 

"Not done yet"

Goepel's girlfriend refused to give up hope. She and Goepel sought a second opinion. Eventually, Goepel was transferred to Sylvester Comprehensive Cancer Center in Miami. There, a biopsy confirmed he had acute myeloid leukemia, or AML, a kind of blood cancer that the Cleveland Clinic says has a five-year survival rate of about 30%.

Complicating matters was the genetic makeup of Goepel's disease. His AML was adverse-risk, said Dr. Justin Watts, a hematologist-oncologist who treated Goepel, and the cancer center's chief of its leukemia section. That meant the chemotherapy most often given to patients like Goepel likely wouldn't treat his disease. It could weaken his body without managing the cancer, potentially killing him in a matter of weeks. Even if the chemotherapy initially worked, Goepel would have been at a higher risk of relapse and future disease.

There was one bright spot in the sea of bad news: There was one opening left in a clinical trial that Watts was overseeing. The trial was studying if two medications typically given to older AML patients would be effective for younger patients. Because Goepel's AML had a genetic makeup similar to that of a 70- or 80-year-old, he seemed like a good fit, Watts said. 

Goepel said that signing up for the trial was a no-brainer.

"My girlfriend gave me a journal that first day, like, 'Maybe it'd be good for you to write,' and I didn't know what to write, and I just wrote, 'I'm not done yet,'" Goepel said. "I kind of just went to the doctors and said, 'OK, what do I have to do? I have to stand, jump, hop on one foot, take this pill, take the whatever. Just, what do I have to do?'"

A clinical trial with "bonkers" results

One day after joining the trial, Goepel began his treatment regimen. He received two medications: a pill taken daily for 28 days and an infusion given daily during the first week of that period. His cancer quickly started responding. Just over two weeks later, he was in remission, a development that Goepel called "bonkers."

He wasn't the only member of the trial to see such results. According to the data, published in the academic journal Haematologica in September, 25 of 36 patients saw their cancer shrink or disappear during a 28-day course of treatment, with 19 of them entering a complete remission. Goepel was one of those patients, entering remission the same month he felt the soreness that made him go see his doctor.

Only about 44% of patients with adverse-risk AML see their cancer shrink or disappear with standard treatment. Patients enrolled in the trial also spent less time in the hospital, needed fewer blood transfusions and experienced fewer infectious complications compared to those who underwent standard treatment, according to the trial data.

The results of the trial, along with data from another Phase 2 study looking at a similar treatment regimen for AML patients who are ineligible for standard chemotherapy, are "generating questions about whether we can change our standard, decades-long approach to treating these patients," said Dr. Mihir A. Shukla, a hematologist at NYU Langone's Perlmutter Cancer Center who was not involved in Watts' study or Goepel's care.

The findings "are interesting and suggest that there may be certain populations that could benefit" from such treatment, Shukla said.

Life after leukemia

After completing the trial regimen, Goepel still needed a bone marrow transplant. Such procedures can essentially rebuild the immune system and be curative for someone with leukemia, Watts said. Over half of the trial patients had transplants, according to the data. Both of Goepel's sisters were perfect matches. He underwent the transplant in November 2024.

"All these things came together, and it happened so fast, and then it was just kind of a blur of luck," Goepel said. "Literally: Right place, right time, right people. And it really worked."

After the transplant, Goepel spent three weeks in the hospital as his immune system began to rebuild. Then he spent another 100 days isolated at home. He had a few post-transplant complications, including a bout of rotavirus and a case of pneumonia. But both illnesses were treated, and tests show the transplant is working correctly.

Meanwhile, Goepel has been cancer-free for two years. It's a result that seemed unlikely when he was first diagnosed, Watts said.

"I don't know for certain, but he probably would not be alive today if we would have gone the other path," Watts said, referring to the standard treatment.  

Further research studying treatment options for adverse-risk AML patients is underway, Watts said. Goepel will also regularly meet with his care team to ensure the transplant is continuing to succeed and that his cancer is staying in remission.

Now, Goepel is finding a new rhythm. He and his girlfriend wed in November 2025, on the one-year anniversary of his remission. He's starting to travel again. He's also processing the past two years and determining what life beyond cancer looks like.

"It's not so much normal, because you kind of lose sense of what normal is, but just kind of not living in fear that it's coming back," Goepel said. "Because if I've got a sore throat, (I think) 'Oh s***, is it cancer?' You jump to it. But no, it's just a sore throat. Relax, and just kind of enjoy life again."