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).
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).
Nitin Jain, MD, of The University of Texas MD Anderson Cancer Center, discusses findings from two studies showing that the combination of ibrutinib and venetoclax is an effective chemotherapy-free oral regimen for patients with high-risk, previously untreated chronic lymphocytic leukemia (Abstract 34).
The ASCO Post
Mhairi Copland, PhD, MB BChir, of the University of Glasgow, discusses results of a study on the combination of ponatinib and fludarabine, cytarabine, idarubicin, and G-CSF for patients with blast phase chronic myeloid leukemia, a rare complication with a poor outcome (Abstract 497).
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 ).
Jerald P. Radich, MD, of the Fred Hutchinson Cancer Research Center, discusses a gene-expression model that distinguishes patients with chronic myeloid leukemia who achieved a deep molecular response from those with a poor response to treatment. This work could yield new therapeutic targets that could potentially turn a poor responder into a good responder who might even achieve treatment-free remission (Abstract 665).
