Kiavasch Mohammad Nejad Farid, MD, of Heidelberg University Hospital, discusses the compassionate use of a novel BCMA-directed CAR T-cell therapy, MDC-CAR-BCMA001, for patients with relapsed/refractory multiple myeloma and systemic light chain (AL) amyloidosis. He explains that although BCMA is a validated target in myeloma, CAR T-cell therapies are not currently approved for AL amyloidosis, prompting the need for alternative strategies. In a small cohort of six heavily pretreated patients, five achieved hematologic responses, including four complete responses and one very good partial response. Additional benefits included minimal residual disease negativity and organ improvements, with a rapid median time to response of just 14 days.
Sara M. Tolaney, MD, MPH, of Dana-Farber Cancer Institute, discusses the potential of the SERENA-6 trial (NCT04964934) to change how ESR1 mutations are monitored in hormone receptor–positive breast cancer. Traditionally, ESR1 testing is performed at the time of disease progression, but emerging data suggest that earlier detection through serial circulating tumor DNA monitoring could allow for more timely treatment changes. The SERENA-6 trial demonstrated that switching to camizestrant upon early ESR1 mutation detection improved progression-free survival compared with continuing aromatase inhibitor therapy. These findings support a new approach in clinical practice, where proactive ESR1 monitoring may guide earlier therapeutic intervention and improve patient outcomes.
Adrián Mosquera Orgueira, MD, PhD, of the Health Research Institute of Santiago de Compostela, discusses the use of a machine learning–based model to improve risk stratification in patients with myelofibrosis undergoing allogeneic stem cell transplant. The model, built using random survival forest techniques, outperformed traditional tools like the CIBMTR and Cox-derived scores in identifying high-risk patients. By determining an optimal threshold—the top 25% of predicted risk—the model more than doubled the number of high-risk patients identified compared with conventional methods, better aligning with real-world clinical observations. This approach could enhance clinical decision making by more accurately flagging patients who may require intensified care or alternative treatment strategies.
R. Lor Randall, MD, FACS, of University of California (UC) Davis Health, discusses the untapped potential of collaboration between oncologists and biomedical engineers to advance cancer research, particularly in sarcoma. He emphasizes that although oncologists typically work with molecular biologists and epidemiologists, engineers offer unique skills in designing physical and computational models that better simulate tumor biology. At UC Davis, the strong engineering faculty presents opportunities to co-develop innovative preclinical platforms, such as 3D tissue constructs and organ-on-chip systems, to explore therapy response and resistance. Randall encourages academic oncologists to partner with engineering departments to build more precise, patient-relevant models that could drive progress in treatment development for rare and understudied cancers.