AML Master Trial Offers Patients Renewed Hope

In 2018, Susan Powers was preparing for hip replacement surgery when her blood tests revealed a more pressing issue: she had acute myeloid leukemia (AML), an aggressive form of cancer. The diagnosis was devastating, with doctors giving her a six-month life expectancy. However, a groundbreaking master clinical trial offered her hope.

Master trials, unlike traditional trials that focus on a single intervention, test multiple drugs and patient groups simultaneously. This approach has been gaining popularity due to its success in accelerating cancer drug development. The Beat AML trial, initiated by the Leukemia & Lymphoma Society (LLS) in 2016, has since enrolled over 1,300 patients and led to the FDA approval of more than 10 AML treatments.

AML is a blood and bone marrow cancer that is the most prevalent type of leukemia in adults. It has a five-year survival rate of 31.7%, but this drops significantly for those diagnosed at 60 or older. The poor prognosis in older individuals is attributed to weaker immune systems, lower medication tolerance, and the nature of the cancer itself.

For Powers, participating in the Beat AML trial was a clear choice. The trial's design, known as an umbrella or basket trial, has been increasingly adopted in recent years due to the rise in targeted treatments and genomic sequencing. Patients are assigned to treatment arms based on genetic or biological markers, which can expedite drug development by allowing for the removal of ineffective drugs and the addition of new therapies.

The FDA has also recommended the use of a shared standard-of-care control arm in these trials, reducing the number of patients needed to show significant effects. Early master trials have shown their effectiveness, with Novartis' Signature Program demonstrating a faster setup time and reduced exposure to toxic drugs compared to traditional trials.

In the case of Powers, after undergoing genomic sequencing to match her to a treatment arm, she initially faced complications from a combination treatment. However, when she was switched to a targeted therapy consisting of a daily pill, her health improved dramatically. Now, nearly seven years post-diagnosis, Powers is cancer-free and enjoys a full life with her family, including 10 grandchildren and two great-grandchildren.

The success of the Beat AML trial and others like it highlight the potential of master trials to revolutionize cancer treatment and drug development, offering patients like Powers a chance at survival and a better quality of life.