MCW clinical research teams launched a phase 1 study, led by Guru Subramanian Guru Murthy, MD, MS, Assistant Professor of Hematology and Oncology, to evaluate the safety and best dose of iadademstat in combination with azacitidine for adults with myelodysplastic syndrome (MDS), a condition that affects the bone marrow’s ability to produce healthy blood cells. While the combination therapy has been effective in acute myeloid leukemia (AML), this investigator-initiated trial is the first to evaluate its potential in MDS.
“Iadademstat is a drug that blocks an enzyme called LSD1, which plays a role in blood cell development. By combining it with azacitidine—a widely used treatment for MDS—we hope to improve how well patients respond to therapy and slow disease progression,” said Dr. Guru Murthy. “This study follows a 3+3 dose-escalation design, where we start at a low dose and gradually increase it in small groups of patients to find the safest and most effective level.”
MDS is a disorder where the bone marrow does not produce enough healthy blood cells, leading to anemia, increased infection risk, and in some cases, progression to AML, an aggressive form of blood cancer. Standard treatments like azacitidine can help improve blood cell production, but not all patients respond well, and relapses are common. If successful, this new combination treatment could help reduce complications, and improve patients’ quality of life.
The MCW Cancer Center is leading multiple efforts to improve MDS treatment and outcomes. Researchers are studying protein patterns in blood samples to better predict which patients may relapse after treatment, which could help doctors personalize care. MCW scientists also played a key role in expanding Medicare coverage for stem cell transplants, ensuring more older MDS patients have access to this potentially lifesaving option.
“These efforts align with our mission to bring innovative therapies to our patients,” added Dr. Murthy. “By leading early-phase trials like this one, we’re creating opportunities for patients to receive cutting-edge treatments that could redefine the standard of care.”
The Cancer Center research team aims to enroll 12 participants to help determine the best dose for future studies.