
The therapy attempts to restore fetal hemoglobin production.

The FDA has accepted a Biologics License Application as well as granted Priority Review for the gene therapy that treats spinal muscular atrophy (SMA) Type 1.

The anti-BCMA CAR T cell therapy bb21217 demonstrated an objective response rate of 83.3%, with a very good partial response or better rate of 75% in patients with heavily pretreated relapsed/refractory multiple myeloma.

Two-year maintenance therapy with ixazomib led to a 39% improvement in progression-free survival compared with placebo in patients with newly diagnosed multiple myeloma who achieved a partial response to induction treatment with a proteasome inhibitor and/or an immunomodulatory agent following autologous stem cell transplant.

Health resource utilization data gathered from the TRANSCEND-NHL trial have found that longer stays in the intensive care unit have a significant impact on the cost of care due to cytokine release syndrome (CRS) following treatment with chimeric antigen receptor (CAR) T cells.

Checkpoint inhibition showed promise for augmenting or extending response to chimeric antigen receptor T-cell therapy in some patients with relapsed B-cell acute lymphoblastic leukemia.

Receiving a stem cell transplant for the first time following CD19 CAR T-cell therapy induced a reduction in the risk for acute lymphoblastic leukemia (ALL) recurrence, according to a retrospective analysis of the phase I/II PLAT-02 study.

Concurrent treatment with ibrutinib and the CD19-targeted chimeric antigen receptor T-cell therapy JCAR014 was well tolerated and led to an overall response rate of 83% in patients with relapsed or refractory chronic lymphocytic leukemia.

Treatment with the CD19-targeted CAR T-cell therapy tisagenlecleucel demonstrated sustained rates of relapse-free survival and overall survival at 24 and 18 months for pediatric and young adult patients with relapsed or refractory acute lymphoblastic leukemia.

A multicenter retrospective study that evaluated the efficacy and safety of chimeric antigen receptor (CAR) T-cell treatment, axicabtagene ciloleucel (axi-cel; Yescarta), in a real-world setting found a similar response as well as toxicity compared with the ZUMA-1 clinical trial.

Robert J. Kreitman, MD, discusses key attributes of moxetumomab pasudotox and its potential impact in hairy cell leukemia.

Although CAR T-cell therapies have proved successful in certain hematologic malignancies, efforts to employ similar strategies in solid tumors have been challenging. Investigators are working on different forms of adoptive cell therapy in solid tumors and early signs are promising.

The combination of nivolumab (Opdivo) and ipilimumab (Yervoy) did not improve overall survival versus placebo as a maintenance therapy for patients with extensive-stage small cell lung cancer without disease progression following frontline platinum-based chemotherapy.

CD7 gene-edited chimeric antigen receptor (CAR) T cells target CD7-positive acute myeloid leukemia (AML) cells while sparing myeloid and erythroid cells and minimizing toxicity, according to the results of a recent study.

Stephanie Jackson, MSN, RN speaks with Cancer Network about the role oncology nurses play in managing patients with hematologic malignancies who are undergoing CAR T-cell therapy.

The FDA has granted brentuximab vedotin a breakthrough therapy designation for use in combination with chemotherapy for the frontline treatment of patients with CD30-expressing peripheral T-cell lymphoma.

The investigators hope to gain FDA approval on the treatment so they can treat more patients.

Robert Dean, MD, discusses the promise for new therapies in mantle cell lymphoma.

This fall, The American Journal of Managed Care® convened a panel of experts on migraine to discuss calcitonin gene-related peptide (CGRP) inhibitors, an emerging therapy for the condition, which affects 39 million people in the United States.

Therapies designed to treat neurologic conditions have made up 25% of the submissions to the FDA for a Regenerative Medicine Advanced Therapy designation.

There is limited data on when and whether oncologists should change systemic therapy for patients with non–small cell lung cancer, but some studies provide useful guidance.


In light of recent advancements, the current paradigm for choosing first-line therapy for patients with metastatic non–small cell lung cancer who do not harbor an actionable driver oncogene depends upon PD-L1 expression level and histology.

The FDA has granted a fast track designation to selinexor for the treatment of patients with previously treated diffuse large B-cell lymphoma who are ineligible to receive high-dose chemotherapy with stem cell rescue or CAR T-cell therapy.

The high durable response rates seen with CAR T-cell therapies have helped fill a high unmet need for patients with relapsed/refractory diffuse large B-cell lymphoma, with questions remaining on the optimal way to use these agents following the FDA approval of 2 therapies in the past year.

A report from the President’s Cancer Panel has found that use of human papillomavirus (HPV) vaccines remain low, despite improvements; Novartis believes that its new gene therapy to treat spinal muscular atrophy could cost $4 million to $5 million per patient; the chief medical officer at the American Cancer Society (ACS) resigned over concerns regarding controversial fundraising partnerships.

The president of the ANA offered his perspective on these novel genetic therapies, as well as other a few other areas of interest.

Gene-edited glypican-3 (GPC3)-targeted chimeric antigen receptor (CAR) T cells with deficient programmed death–1 have greater cytotoxicity compared with wild-type GPC3-targeted T cells in GPC3-positive hepatocellular carcinoma, based on results from a recent study.

After a single dose of RGX gene therapy, the mean change in BCVA was +8 letters in cohort 3 and the average number of injections over the course of 6 months was 1.3 in a phase 1 cohort study.

Armin Ghobadi, MD, discusses the evolution and expansion of CAR T-cell therapy in oncology and how to maximize its application across liquid and solid tumors.