MDS Foundation: Significant advances made in understanding, treating MDS, AML

The Myelodysplastic Syndromes (MDS) Foundation today said significant advances are being made in understanding and treating MDS and a related condition, AML (acute myeloid leukemia). Key studies being presented this week include data on new markers and genetic mutations to predict susceptibility to MDS and response to treatment. Studies also look at new dosing and new combinations of existing drugs, and include an early look at new drug formulations showing increased potential and presenting more options for patients. The new findings will be presented at the 52nd annual meeting of the American Society of Hematology (ASH), being held this week in Orlando, Florida.

“These are challenging diseases that affect a growing number of persons each year with the graying of the US population; we are pleased to see the significant progress being made”

MDS is a malignant condition of cells in the bone marrow. Patients require blood transfusions that can lead to iron overload, and the condition can progress to AML, a serious form of leukemia that has a median survival of less than one year. MDS affects the production and function of blood cells—red blood cells, white blood cells or platelets. The incidence of MDS and AML is underestimated, conservatively affecting tens of thousands of persons annually.

"These are challenging diseases that affect a growing number of persons each year with the graying of the US population; we are pleased to see the significant progress being made," said Stephen D. Nimer, M.D., Vice Chair, Faculty Development & Alfred P. Sloan Chair at Memorial Sloan-Kettering Cancer Center in New York and MDS Foundation board member. "While there were very few options for MDS patients in the past, with the FDA approved therapies, patients are able to live a better quality of life, often free from the time-consuming and tiring process of receiving frequent blood transfusions. It is encouraging to see additional confirmation that these new therapies are providing benefits for patients and that additional drugs are in development."

Key MDS and AML studies to be presented at ASH include new ways to measure and determine susceptibility to MDS and predict response to treatment:

• data to validate the MD Anderson Risk Model (MDAS), a new, more accurate way to predict the natural history of a given patient's disease to guide appropriate therapy (abstract #444, 12/6, 11:45am)
• genetic markers to predict susceptibility to MDS (#LBA3, 12/7, 7:30am and #612, 12/6, 4pm)
• and a specific genetic mutation that may help predict response to treatment with VIDAZA^® (azacitidine) (#439, 12/6, 10:30am)

Another key group of studies provide new data on treatment options for patients:

• a reduced dose and longer cycle of VIDAZA compared to the standard of treatment (50mg vs 75mg; 10 days vs 7 days) doubles the rate of hematologic normalization (#601, 12/6, 2:45pm)
• an early look at a new regimen CPX-351, a liposome (encapsuled) formulation of cytarabine and daunorubicin in a specific ratio to maximize synergy, demonstrates higher response rate in newly diagnosed elderly patients with AML and in particular, AML that evolves from MDS (#655, 12/6, 4:30pm)
• an oral version of the drug VIDAZA shows encouraging clinical responses in early testing (#603, 12/6, 3:15pm)

Other studies found positive effects with combinations of drugs, such as REVLIMID^® (lenalidomide) plus intensive chemotherapy in elderly patients with higher-risk MDS and AML (#508, 12/6, 3:30pm).

However, two studies dramatically show the need for a broader range of treatments, documenting a poor outcome for patients when current hypomethylator therapies fail and calling for clinical trials for this group of patients to take priority (#2913, 12/5, 6pm; #443, 12/6, 11:30am).

Alan List, M.D., Physician-in-Chief at the Moffitt Cancer Center in Florida and board member of the MDS Foundation noted: "The growing ability to predict and assess disease incidence and outcome combined with a better understanding of current treatments makes this an important ASH conference for MDS patients and their physicians. Our arsenal is too small and we need more research, but we are encouraged by what we expect to see this year."