Already Approved Drug Shows Promise in Patients with MDS/MPN Overlapping Syndromes

By Sara Bondell - December 08, 2023

Myelodysplastic syndromes (MDS) are conditions that can occur when the blood-forming cells in the bone marrow become abnormal, leading to low numbers of one or more types of blood cells. On the other hand, myeloproliferative neoplasms (MPN) are cancers that cause bone marrow to make too many of one or more types of blood cells.

Some patients have mutations that cause both underproduction and proliferation of blood cells. They are classified as having overlapping MDS/MPN syndromes. While there are separate clinical trials and treatment regimens for MDS patients and MPN patients, those with overlapping syndromes have been historically left out of research.

Dr. Andrew Kuykendall, hematologist
Dr. Andrew Kuykendall, Malignant Hematology Program

“Our approach to treatment for these patients usually comes from trials we just completed on MDS patients or on MPN patients. And we kind of have to extrapolate and say, OK, maybe this will work for you,” said Dr. Andrew Kuykendall, an oncologist in the Malignant Hematology Program at Moffitt Cancer Center.

Hoping to better serve these patients, Kuykendall launched a trial investigating a drug called fedratinib in patients with MDS/MPN overlap syndromes and those with a very rare form of MPN called chronic neutrophilic leukemia. Fedratinib is an immune modulating medication that is FDA approved to treat MPNs including myelofibrosis. The idea behind the trial is that the drug can target certain mutations present in patients with MDS/MPN overlapping syndromes.

“There is a preponderance of data from basic science that show maybe this drug could be uniquely effective in this patient population,” Kuykendall said. “So far, overall, we are seeing very promising efficacy of this drug.”

Kuykendall presented preliminary data from nine patients on the phase 2 trial at this year’s American Society of Hematology meeting. Three of five evaluable patients responded to the treatment at week 24. After 12 weeks of treatment, 80% of patients experienced an improvement in symptom burden. 

Three other cancer centers have begun enrolling patients in the trial. Kuykendall hopes the trial’s data will be able to help patients without a phase 3 trial since fedratinib is already approved and easily accessible.

“If you have this data out there and you have these rare patients that don’t have drugs directly approved for them, you could use this to say this is a reasonable approach to treat,” he said.

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