David P. Steensma, MD, on Myeloid Neoplasms: Results From a First-in-Human Trial of a Splicing Modulator
2019 ASH Annual Meeting & Exposition
David P. Steensma, MD, of Dana-Farber Cancer Institute, discusses early study findings on H3B-8800, which decreased the need for red blood cell or platelet transfusion in 14% of patients. This splicing modulator, used in the trial to treat patients with hematologic malignancies, also showed safety, dose-dependent target engagement, and a predictable pharmacokinetic profile (Abstract 673).
C. Ola Landgren, MD, PhD, of Memorial Sloan Kettering Cancer Center, discusses phase II study findings that showed an 83% negative rate of minimal residual disease in newly diagnosed multiple myeloma treated weekly with 8 cycles of the quadruplet regimen of carfilzomib/lenalidomide/dexamethasone/daratumumab, without autologous stem cell transplant (Abstract 862).
Ilaria Iacobucci, PhD, of St. Jude Children’s Research Hospital, discusses her work to more accurately define mutation subtypes in acute myeloid leukemia and myelodysplastic syndromes, as well as the implications for diagnosis, prognosis, and treatment (Abstract LBA-4 ).
Andrew H. Wei, MBBS, PhD, of The Alfred Hospital, Melbourne, discusses phase III findings on oral azacitidine (CC-486), the first treatment used in the maintenance setting shown to improve both overall and disease-free survival in patients with acute myeloid leukemia that is in remission following induction chemotherapy (Abstract LBA-3).
Edward A. Stadtmauer, MD, of the University of Pennsylvania Abramson Cancer Center, discusses phase I results of immune cells, modified with CRISPR/Cas9 technology, and infused in three patients (two with multiple myeloma and one with sarcoma). Researchers observed the cells expand and bind to their tumor targets with no serious side effects (Abstract 49).
Jennifer Crombie, MD, of Dana-Farber Cancer Institute, discusses early study results which showed that duvelisib plus venetoclax showed activity in patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma, with no dose-limiting toxicities observed (Abstract 1763).