A new drug called venetoclax was dubbed "Rookie of the Year" by Dr. Rafael Fonseca at this year's American Society of Hematology meeting in San Diego. It is one of the first therapies that appears to have impact on a specific sub-type of multiple myeloma. Venetoclax is an oral BCL-2 inhibitor that kills myeloma cells, especially for patients with an 11;14 translocation. Patients with this translocation typically have high BCL-2 levels, low BCL-XL levels and a low MCL-1 profile. In a Phase 1 study, relapsed/refractory patients received venetoclax as a single therapy. After a two week period with dose escalations, a final dose was determined. If patients progressed during the venetoclax, they could receive venetoclax plus dexamethasone and continue on the study. In the study, 66 patients were enrolled with a median number of 5 prior therapies. Thirty of the patients had the 11;14 translocation. With a median time on venetoclax of 2.5 months, overall response was 21% and 15% achieved very good partial response. Thirty-nine patients discontinued the study due to disease progression. The most impactful responses were for patients with the 11;14 translocation. In this sub-group, overall response for this group was 40% and 27% achieved a very good response rate. The excitement around venetoclax is the potential to find and utilize more personalized approaches for multiple myeloma patients. We look forward to seeing its use in more combination approaches that are typical for myeloma treatment. To read the full ASH abstract, click here. To find all clinical trials using venetoclax, click here: Venetoclax Clinical Trials
about the author
Myeloma survivor, patient advocate, wife, mom of 6. Believer that patients can help accelerate a cure by weighing in and participating in clinical research. Founder of Myeloma Crowd by HealthTree and the HealthTree Foundation.