A Florida man diagnosed with advanced acute myeloid leukemia (AML) entered remission following a clinical trial that tested non-traditional treatments for younger patients. Ryan Goepel, then 49, sought medical attention in September 2024 for persistent knee pain he initially attributed to gout. Blood tests and a subsequent biopsy revealed his organs were failing due to adverse-risk AML, a form of blood cancer with a genetic makeup typically found in patients in their 70s or 80s.
Doctors at Sylvester Comprehensive Cancer Center in Miami informed Goepel that standard chemotherapy would likely be ineffective and could potentially be fatal within weeks due to his weakened state. Dr. Justin Watts, chief of the leukemia section at the center, instead enrolled Goepel in a clinical trial investigating two medications usually reserved for older patients. The trial examined whether these drugs could effectively treat younger patients whose cancer possessed specific genetic characteristics.
Goepel began a 28-day regimen involving a daily pill and one week of daily infusions. According to data published in the journal Haematologica in September, 25 out of 36 patients in the trial saw their cancer shrink or disappear, with 19 achieving complete remission. Goepel reached remission within two weeks of starting treatment. Following the trial, he received a bone marrow transplant from one of his sisters in November 2024 to rebuild his immune system.
For the individuals affected, such as parents like Goepel, this shift in treatment protocol may mean the difference between entering hospice care and achieving long-term remission. Dr. Mihir A. Shukla of NYU Langone’s Perlmutter Cancer Center noted that the results are prompting questions about whether decades-old standard approaches to AML should be changed. The data indicates that for specific genetic profiles, a less toxic regimen of pills and infusions may be more effective than the aggressive chemotherapy traditionally administered to younger patients.
The success of the trial sets a precedent for personalized genetic matching in leukemia treatment rather than relying solely on patient age. Goepel, who has remained cancer-free for two years, married in November 2025. Further research into treatment options for adverse-risk AML is currently underway. Goepel continues to meet regularly with his medical team for monitoring, while other institutions, including Boston Medical Center, continue separate research into gout through the TRUST trial, which aims to enroll 650 adult patients to compare symptom-based versus uric-acid-level-based treatments.