Clinical Trial Leads to Remission for Man With Adverse-Risk Leukemia
CBS News reports a Florida man with adverse-risk AML entered remission after a 28-day trial regimen, with 25 of 36 trial patients seeing their cancer shrink or disappear.
The story in focus
CBS News reports that Ryan Goepel, a Florida father of three, entered remission from adverse-risk acute myeloid leukemia after enrolling in a clinical trial at Sylvester Comprehensive Cancer Center in Miami. Trial data published in Haematologica showed 25 of 36 patients saw their cancer shrink or disappear.
- CBS News reports doctors advised hospice care before Goepel sought a second opinion at Sylvester Comprehensive Cancer Center.
- His AML was adverse-risk, meaning standard chemotherapy likely would not have treated his disease.
- Trial data in Haematologica showed 25 of 36 patients responded during a 28-day course, with 19 entering complete remission.
- Only about 44% of adverse-risk AML patients see their cancer shrink or disappear with standard treatment, the trial data indicated.
- Goepel later received a bone marrow transplant from a sister and has been cancer-free for two years.
From hospice discussion to a trial opening
CBS News reports that Ryan Goepel, a Florida father of three, first attributed knee pain during a September 2024 trip to a gout flare. Blood tests at his doctor's office prompted an urgent hospital referral, where he learned he had leukemia and that his organs were nearing shutdown. Doctors told his girlfriend to consider hospice care.
After seeking a second opinion, Goepel was transferred to Sylvester Comprehensive Cancer Center in Miami, where a biopsy confirmed acute myeloid leukemia. Dr. Justin Watts, a hematologist-oncologist and chief of the center's leukemia section, said Goepel's disease was adverse-risk, meaning the chemotherapy most often given to such patients likely would not treat it.
Trial results
One opening remained in a trial Watts was overseeing. It studied whether two medications typically given to older AML patients would work for younger patients; Goepel's cancer had a genetic makeup similar to that of a 70- or 80-year-old, Watts told CBS News.
Treatment began a day after enrollment: a daily pill for 28 days and a daily infusion during the first week. CBS News reports Goepel was in remission just over two weeks later. Trial data published in Haematologica in September showed 25 of 36 patients saw their cancer shrink or disappear during the 28-day course, with 19 entering complete remission. Trial patients spent less time in hospital and needed fewer blood transfusions than those on standard treatment, according to the data.
Transplant and follow-up
Goepel still needed a bone marrow transplant after the trial regimen. Over half of trial patients had transplants, according to the data. Both of his sisters were matches, and he underwent the procedure in November 2024, followed by three weeks in hospital and 100 days of isolation at home. CBS News reports he has been cancer-free for two years. Watts said further research on adverse-risk AML treatment is underway.
What did the trial data show?
Published in Haematologica in September, the data showed 25 of 36 patients saw their cancer shrink or disappear during a 28-day course, with 19 entering complete remission.
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