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

Ryan Goepel, a Florida father of three, was initially given weeks to live after being diagnosed with leukemia. The 49-year-old had been experiencing knee pain that he initially thought was a flare-up of his gout. However, when the pain persisted, Goepel sought medical attention and was told he had acute myeloid leukemia (AML), a cancer with a five-year survival rate of about 30%.

Goepel's condition was critical, and his organs were on the verge of shutting down. His doctors considered hospice care for him, but his girlfriend refused to accept this prognosis. She and Goepel sought a second opinion, eventually leading to Goepel being transferred to Sylvester Comprehensive Cancer Center in Miami, where a biopsy confirmed his AML diagnosis.

The genetic makeup of Goepel's disease was adverse-risk, meaning standard chemotherapy likely wouldn't treat his disease. There was one clinical trial that seemed promising for Goepel's case, overseen by Dr. Justin Watts, the leukemia section chief at the cancer center. Goepel volunteered for the trial, signing up the same day he learned about it, stating, "I'm not done yet."

After joining the trial, Goepel began a treatment regimen involving two medications. Just over two weeks later, he was in remission. According to the trial data published in Haematologica, 25 out of 36 patients saw their cancer shrink or disappear during a 28-day course of treatment, with 19 entering complete remission. This is significantly higher than the 44% of patients with adverse-risk AML who see their cancer shrink or disappear with standard treatment.

Goepel's cancer-free status has lasted for two years now. However, he still needs a bone marrow transplant, which can rebuild his immune system and potentially be curative. In November 2024, Goepel underwent the transplant, with both of his sisters being perfect matches. He spent three weeks in the hospital as his immune system began to rebuild, followed by 100 days of isolation at home to prevent infection.

While further research is underway to study treatment options for adverse-risk AML patients, Goepel's case suggests that certain populations could benefit from this approach. His experience serves as a reminder of the importance of hope and the potential for unexpected positive outcomes in medical treatments.