Melflufen Could be a New Treatment Option for Multiple Myeloma

According to a story from Hematology Times, a medicine called melflufen, of the alkylating peptide class, appears to be effective in treatment resistant multiple myeloma. In a Phase 2 trial, the drug was used in combination with dexamethasone. This treatment approach was able to yield a response rate of 32.1 percent in multiple myeloma patients that had already tried a number of different treatments, such as proteasome inhibitors, immunomodulators, pomalidomide, and daratumumab.

About Multiple Myeloma

Multiple myeloma is a type of rare blood cancer that affects plasma cells. Plasma cells are a type of white blood cell that is responsible for the production of antibodies. When affected by this cancer, they release abnormal antibodies that can cause a number of complications, such as liver dysfunction, thickened blood, and amyloidosis. The direct cause of multiple myeloma is not well understood, but there are several risk factors, including alcohol consumption, monoclonal gammopathy of undetermined significance, obesity, exposure to radiation, certain chemicals, and family history. Symptoms include neurological problems, kidney failure, anemia, bone pain, and infections. Following treatment, multiple myeloma often relapses; it may not produce symptoms in the early stages. Five year survival rate is 49 percent in the US. To learn more about multiple myeloma, click here.

About The Study

The downside of the study was that almost every patient in the trial experienced an adverse side effect due to the treatment. Despite this disadvantage, the need for new treatments is urgent for multiple myeloma, especially since it is common for the cancer to ultimately relapse. Compared to other treatments, melflufen offers a unique mechanism of action. As a result of this need, the issues with side effects were overall considered manageable, and worthwhile considering the drug’s effectiveness.

It is worth noting that, overall, the drug did not display benefits for all patients; about 47 percent discontinued use when the disease continued to progress, and 15 percent had to discontinue because of adverse events. Adverse events included neutropenia, thrombocytopenia, and anemia. In the study, overall response rate was defined as partial response or better.

Although it melflufen does not appear to work for everyone and can have significant side effects, it still could be a viable option for patients whose disease have developed resistance to currently available treatments.


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