Ryan Goepel had gout, and admits he didn’t always manage it as well as he should have. So in September 2024, when his knee started hurting on a trip, the Florida father of three assumed it was a flare-up. The ache stayed, so he saw his doctor, who gave him some medication and drew blood. Two days later she called him back. “She said, ‘Stop whatever you’re doing, and go straight to the hospital,’” he recalled. “She says, ‘Just go. You have no immune system. I don’t know what’s wrong. Just go.’”
He went to a nearby sports medicine hospital, had more blood drawn, and got worried when an oncologist walked in. “I’m like, ‘Why are you here? I have a sore knee,’” he said. “And she’s like, ‘Well, actually, you have leukemia.’” He was 49. His organs were close to failing. The doctors took his girlfriend aside and told her to think about hospice. “They just basically told her: ‘You should make arrangements. He’s not leaving alive.’”
He did leave alive, and is still alive, which is why Kerry Breen told his story for CBS News. His girlfriend pushed for a second opinion, and he ended up at Sylvester Comprehensive Cancer Center in Miami. A biopsy there confirmed acute myeloid leukemia, or AML, a blood cancer the Cleveland Clinic puts at about 30% five-year survival. His version was worse than that: the genetics made it adverse-risk, said Dr. Justin Watts, the hematologist-oncologist who heads Sylvester’s leukemia section. In practice that meant the intensive chemotherapy usually given to someone his age probably wouldn’t work. It could wreck his body without controlling the cancer and kill him within weeks. And if it did work at first, he would still face a higher risk of relapse.
Here is the part I find interesting. AML treatment sorts patients largely by age and fitness. Younger, sturdier patients get intensive chemotherapy. Older or frailer patients, who couldn’t survive that, get a gentler combination of venetoclax, a daily pill, and azacitidine. According to the paper reporting the trial, that combination is the standard of care for newly diagnosed patients who aren’t candidates for intensive chemo. Age stands in for something else, though. The underlying question is what the cancer will respond to, and age is just a rough guess at the answer. The authors argue that the factors predicting success with venetoclax-azacitidine are different from those predicting success with chemo, so a patient who is fit enough for chemo might still do better on the gentler drugs.
Watts was overseeing a trial built to test exactly that, and it had one slot left. Goepel was 49, but his disease had the genetic profile of a 70- or 80-year-old’s, so he looked like a good fit. He said joining was a no-brainer. His girlfriend had given him a journal, and on the first day he wrote just one line: “I’m not done yet.”
He started the next day: the pill every day for 28 days, plus an infusion every day for the first week. The paper gives the doses as venetoclax escalated to 600 mg a day and azacitidine at 75 mg/m2 for seven days, in 28-day cycles. A little over two weeks later he was in remission, which he called “bonkers.” He went into remission in the same month his knee first started hurting.
The numbers
The results came out in Haematologica in September. The trial enrolled 36 patients aged 60 or younger with non-favorable-risk AML. Of those, 25 responded, or 69%, and 19 had complete remissions, or 53%. Among the responders, 68% reached MRD-negativity, meaning no measurable residual disease. That leaves 11 who didn’t respond, and the paper says most of them were then successfully treated with intensive chemo. So the gentler drugs going first didn’t use up the backup option.
The comparison group was patients matched from the past who had received intensive chemotherapy. Their response rate was 44%, the figure CBS gives for standard treatment. Trial patients reached transplant more often, 53% against 28%. Median progression-free survival hasn’t been reached for the trial group yet, against 60.8 months for the controls. The difference in hospital use is striking: in the first 30 days, trial patients spent a median of 9 days in hospital against 30, and 43% had infectious complications against 94%. Twenty-one fewer hospital days a patient adds up to real money for whoever is paying, and it is also three weeks of someone’s life spent at home rather than in a ward.
There are caveats, and the paper states them. Overall survival wasn’t significantly different, not reached against 5.0 years, P=0.3206. The study was small and not randomized, and the matching of controls was done after the fact without being planned in advance. Patients with monocytic disease responded only half the time, 4 of 8, and were later excluded from enrollment. An interim analysis presented in 2024 covered 28 patients and reported a 61% response rate. The authors’ conclusion is that the combination “appears at least as effective as IC.” In journal language, that means it’s promising, it didn’t hurt anyone, and the next step is a proper randomized trial.
Dr. Mihir A. Shukla, a hematologist at NYU Langone’s Perlmutter Cancer Center who wasn’t involved in the study, said that this trial and another Phase 2 study of a similar regimen in patients who can’t have standard chemo are “generating questions about whether we can change our standard, decades-long approach to treating these patients.” He also said the findings “suggest that there may be certain populations that could benefit.” Other researchers are asking the same question. A randomized phase 2 trial called PARADIGM reported that azacitidine-venetoclax beat induction chemo in fit patients, as Medscape covered it. Watts says further research on adverse-risk patients is under way.
After
Remission wasn’t the end of treatment. Goepel still needed a bone marrow transplant, which Watts said can rebuild the immune system and cure leukemia. More than half the trial patients had one. Both of Goepel’s sisters turned out to be perfect matches, and he had the transplant in November 2024. “Literally: Right place, right time, right people,” he said. “And it really worked.” He then spent three weeks in hospital and another 100 days isolated at home, and caught rotavirus and pneumonia along the way. Both were treated, and tests show the transplant is working. He has been cancer-free for two years.
“I don’t know for certain, but he probably would not be alive today if we would have gone the other path,” Watts said, meaning standard treatment.
Goepel and his girlfriend married in November 2025, which CBS describes as the one-year anniversary of his remission. He has started traveling again. He is also trying to stop treating every symptom as a relapse. “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.”
The drugs that saved him were already on pharmacy shelves, approved and in routine use. He needed two other things: a doctor willing to ask whether a 49-year-old should get them, and one empty slot in the study asking that question.