Those adult patients suffering from rare blood cancers such as acute myeloid lukemia (AML), aggressive systemic mastocytosis (ASM), systemic mastocytosis with a hematologic neoplasm (SM-AHN), and mast cell leukemia now stand a better chance at survival, with the Food and Drug Administration’s (FDA) approval of Midostaurin. When combined with chemotherapy, the risk of death decreases by 23 percent, 25 percent for those who have already had stem cell treatment.
Targeted Oncology has revealed that the drug was approved after passing stages II and III of the RATIFY trial. Stage II was a single-arm study of systemic mastocytosis, while III was focused on treating FLT3-positive acute myeloid leukemia, or AML. The LeukoStrat CDx FLT3 Mutation Assay was also approved in order to test for FLT3 mutations in patients who have been diagnosed with the cancer.
Six doses of midostaurin were administered to phase II participants, with the results being that their CR and ICR rates sit at 38 percent in ASM patients and 16 in SM-AHN, with these rates standing for complete recovery and incomplete recovery. Researchers are not surprised at these findings, as associated hematological neoplasm is far more serious than aggressive systemic mastocytosis, and as such is harder to treat. One individual with mast cell leukemia managed to make a CR with the help of midostaurin.
The novartis drug PKC412, reveals Medscape, is a multi-target protein kinase inhibitor which is given orally and started being looked into as a possible treatment for rare blood cancers in the year 2008. It is said to be ineffective, however, when it comes to curing metastatic melanoma. It is the first new drug centered around curing AML in almost 30 years to be approved by the FDA, who last gave the green light for this cause back in 1990. Midostaurin goes by the pharmaceutical name of Rydapt.
The news that the combination treatment had been finally approved was first broken by the FDA themselves, via their official website. A press announcement was posted on Thursday, April 28, and contained a statement from Dr. Richard Pazdur, who serves as both the acting director of the FDA’s Center for Drug Evaluation and Research’s Office of Hematology and Oncology Products, as well as director of the U.S. Food and Drug Administration’s Oncology Center of Excellence.
Pazdur paid tribute to just how groundbreaking it is that now, doctors can perform a diagnostic test in order to detect mutations in the gene, and as such single out specific patients who would most benefit from receiving the dose. He believes there is potential for thousands of lives to be saved thanks to the approval of midostaurin.
By Lorelai Zelmerlow
Photo Courtesy Novartis