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As July concludes, the landscape of hematologic oncology continues to evolve, with notable progress in multiple myeloma, myelofibrosis, and CLL.
As July concludes, the landscape of hematologic oncology continues to evolve, with notable progress in multiple myeloma, myelofibrosis, and CLL.
As July concludes, the landscape of hematologic oncology continues to evolve, with notable progress in multiple myeloma, myelofibrosis, and chronic lymphocytic leukemia (CLL) research. Recent FDA actions, clinical trial data, and real-world insights highlight steps forward for patients.
Developments like U.S. Food and Drug Administration (FDA) approvals and regulatory decisions, as well as clinical trial updates, reflect a continued push toward more personalized and effective treatment for patients with blood cancers.
Read on for more insights from the front lines of hematologic research and care.
The U.S. Food and Drug Administration (FDA) has granted accelerated approval to Lynozyfic(linvoseltamab-gcpt) for adults with relapsed or refractory multiple myeloma who have received at least four prior therapies, including a proteasome inhibitor, immunomodulatory agent, and anti-CD38 monoclonal antibody. This bispecific B-cell maturation antigen-directed CD3 T-cell engager demonstrated a 70% overall response rate in the LINKER-MM1 trial, with durable responses lasting up to 12 months in a majority of patients.
Due to the risk of life-threatening side effects, like including cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome, Lynozyfic carries a boxed warning and is available only through a restricted Risk Evaluation and Mitigation Strategy program. The drug’s step-up intravenous dosing schedule is followed by a maintenance regimen that may be reduced with sustained response.
Lynozyfic also received orphan drug and fast track designations, further supporting its expedited development for a population with significant unmet medical need.
T cells are a natural part of the immune system that help fight infection and cancer, but in blood cancers such as lymphoma, they may fail to eliminate malignant cells. To address this unmet need, researchers have developed CAR T-cell therapy, which involves genetically modifying T cells to express receptors that target tumor cells directly, enhancing their ability to identify and destroy cancer.
“We all have T cells in our blood. These T cells are part of our natural, native immune system; they are meant to protect us against infections and cancers. However, when a patient has a cancer like lymphoma, it means the T cells have not properly done their job of eliminating that malignancy. This new method is a way of weaponizing T cells,” Dr. Matthew Ku said in an interview with CURE.
Ku, a clinical and laboratory hematologist, is a professor and lymphoma stream lead at St Vincent’s Hospital, where he is also the principal investigator on multiple important clinical trials. He is a member of the Australasian Lymphoma Alliance, the Australasian Leukemia & Lymphoma Group and the American Society of Hematology.
Ongoing studies aim to further refine personalized strategies by linking clinical and genetic patterns in diseases like myelofibrosis to future treatment approaches.
Cytopenia at diagnosis was linked to poorer outcomes in patients with myelofibrosis, including a higher risk of progression to acute myeloid leukemia (AML) and reduced overall survival, according to a real-world study published in HemaSphere. Among 1,532 patients studied, those with cytopenia (anemia or low platelet counts) had a median overall survival of 38 months; the median overall survival was not reached in the non-cytopenic group. Cytopenic patients were also older, had more comorbidities, and a five-year AML progression risk of 22% versus 6.9% without cytopenia.
“Understanding risk factors for poor prognoses and how to appropriately tailor Janus kinase inhibitor treatment selection for patients with myelofibrosis who are ineligible for transplant is clinically important,” wrote lead study author, Dr. Lindey Rein, and colleagues in the abstract of the study.
Rein is an associate professor of medicine at Duke University, a member of the Duke Cancer Institute, and a researcher in hematologic malignancies and cellular therapy.
Further research is needed to determine how targeting cytopenia may influence survival, AML progression and transplant eligibility.
In a blog post from Andy Winnegar, who was diagnosed with CLL in 2021, he writes about his experience sitting in a chemo chair at Christus St. Vincent Regional Cancer Center in Santa Fe, New Mexico, preparing for one of four weekly infusions. Winnegar discusses how a long-unexplained case of anemia, initially thought to be due to distance running, was eventually diagnosed as CLL with autoimmune hemolytic anemia (a condition where your immune system attacks red blood cells).
This first-person account details the physical and emotional toll of treatment, from infusion side effects and pacing the floor due to back pain and restlessness, to finding comfort in familiar faces among the oncology team.
During a regulatory meeting held on July 17, the FDA Oncologic Drugs Advisory Committee (ODAC) voted against recommending approval for two proposed Blenrep (belantamab mafodotin) combination regimens in patients with relapsed/refractory multiple myeloma who had received at least one prior therapy. Specifically, the panel voted five to three against the risk/benefit profile of Blenrep with Velcade (bortezomib) and dexamethasone (BVd), and seven to one against Blenrep with pomalidomide (Pomalyst) and dexamethasone (BPd), citing concerns over toxicity.
Although both combinations demonstrated improved progression-free survival in phase 3 trials, the committee raised concerns about high rates of ocular toxicity, unresolved dosing questions, and lack of trial diversity.
The majority of participants were from outside the U.S., with limited representation of older adults and Black patients, leading to doubts about the applicability of results to U.S. clinical practice. Overall, the ODAC ultimately concluded that the safety profile at the proposed dosages did not justify approval at this time.
“This was a challenging decision because the efficacy data were strong, but the toxicity data were also very strong… We've heard impassioned testimonials from key opinion leaders and from many researchers in the myeloma community. All of the building blocks are here to explore this question in the future, from patients to researchers to physicians,” Dr. Neil Vasan, a medical oncologist at NYU Langone, explained on why he voted against both drug combinations.
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