News

University of Pennsylvania investigators conducted an interim analysis of a year-long study on intraocular injections of a mutation-specific gene therapy when one patient showed dramatic early improvements.

Updates from the Annual ASH meeting, December 2018.

The agency is anticipating upward of 200 INDs per year by 2020 and between 10 and 20 cell and gene therapy approvals per year by 2025.

The FDA has granted the investigational BTK inhibitor zanubrutinib a breakthrough therapy designation for the treatment of adult patients with mantle cell lymphoma who have previously received at least 1 prior therapy.

Nina Shah, MD, discusses the promise of bb21217 in the complex treatment paradigm of relapsed/refractory multiple myeloma

A phase 2 trial demonstrated that the regimen of rituximab, bortezomib, bendamustine, and dexamethasone is a viable treatment option for older patients with mantle cell lymphoma (MCL), and highlighted the usefulness of using minimal residual disease (MRD) to guide early and late clinical decisions.

Crizanlizumab is being developed to prevent painful and unpredictable vaso-occlusive crises in patients with sickle cell disease.

The study implies that B-cell depletion by rituximab therapy may be therapeutically beneficial in patients with secondary progressive multiple sclerosis.

A recent prospective phase 2 study sought to investigate autologous hematopoietic stem cell transplantation as a therapeutic intervention in multiple sclerosis (MS).

Deepu Madduri, MD, discusses the status of transplant for eligible patients and the developments that are being made with CAR T-cell therapy in multiple myeloma.

Heather Greene, NP, discusses the importance of open communication between patients and providers from the onset of therapy and provided insight on the management of treatment-related adverse events in metastatic renal cell carcinoma.

From chimeric antigen receptor T-cell therapy to value-based contracting, reimbursement issues dominated reader interest.

Gene therapy has generated excitement as a treatment or even a potential cure for inherited diseases. Among them: Duchenne muscular dystrophy.

An ongoing randomized phase III study will compare the safety and efficacy of atezolizumab with placebo as adjuvant therapy after definitive local therapy in patients with high-risk, locally advanced squamous cell carcinoma of the head and neck.

The frontline induction treatment of bendamustine and rituximab has improved event-free surviva compared with R-CHOP therapy in patients with mantle cell lymphoma.

C. Ola Landgren, MD, PhD, discusses the role of minimal residual disease negativity and the emergence of CAR T-cell therapy in multiple myeloma.

The trial results indicate that EBV-specific adoptive T cell therapy is well tolerated and further back this approach in efficacy trials.

Chimeric antigen receptor T cells targeting the tyrosine kinase receptor ROR1 can be transferred into patients safely and the cells expand in vivo.

The use of circulating tumor-cell counts demonstrated strong value for selecting endocrine therapy versus chemotherapy for patients with estrogen receptor–positive, HER2-negative metastatic breast cancer.

A median 19-month follow-up of the JULIET trial—a single-arm, open-label, multicenter, global, pivotal phase 2 trial of the chimeric antigen receptor-T cell therapy tisagenlecleucel directed against CD19-expressing B cells—has found a 40% complete response and a manageable safety profile in adult patients with relapsed/refractory (R/R) diffuse large B-cell lymphoma (DLBCL).

In contrast to the wide array of available therapies in the metastatic setting of renal cell carcinoma, in patients with localized disease, the results of trials investigating adjuvant systemic therapy after nephrectomy have so far been underwhelming.

A multitude of BCMA-targeted CAR T-cell therapies are currently in development, each demonstrating different efficacy and safety profiles and each with different constructs.

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.