News

B-cell maturation antigen-specific chimeric antigen receptor T-cell therapy is delivering impressive results in multiple myeloma, demonstrating durable responses and acceptable toxicities.

Despite being labeled as rare diseases, a number of neurologic conditions impact more patients than most would believe. The consultant with expertise in ophthalmology, gene therapy, and rare and orphan diseases, chimed in about how these diseases can often be overlooked.

Chimeric antigen receptor T cells targeting CD30 proved to be safe and active in relapsed/refractory Hodgkin lymphoma.

Every week, The American Journal of Managed Care® recaps the top managed care news of the week, and you can now listen to it on our podcast, Managed Care Cast.

Frederick L. Locke, MD, discusses the optimal management of chimeric antigen receptor T-cell therapy-related adverse events.

Patients with relapsed/refractory chronic lymphocytic leukemia had an improved response rate with chimeric antigen receptor T-cell therapy if they also received ibrutinib.

The FDA has granted a priority review designation to a supplemental biologics license application for pembrolizumab (Keytruda) as a treatment for patients with advanced small cell lung cancer whose disease has progressed following ≥2 prior lines of therapy.

FDA trial approval would make this trial the first ever to test a stem cell-based therapy derived from induced pluripotent stem cells for treating any disease.

The Centers for Medicare & Medicaid Services has proposed to cover chimeric antigen receptor T-cell therapies approved by the FDA, under “Coverage with Evidence Development."

The payment model will gather data that will be used to create a long-term model for patients not enrolled in studies or registries.

Dr. Copelan discusses the use of chimeric antigen receptor (CAR) T-cell therapy in non-Hodgkin lymphoma patients and how these therapies might improve upon the current standard of care.

Edward B. Garon, MD, discusses the landscape of EGFR-targeted therapy in non–small cell lung cancer.

Victor A. Chow, MD, discusses the next steps in understanding how to treat patients with large B-cell lymphomas who progress after chimeric antigen receptor T-cell therapy.

The microdystrophin gene transfer therapy from Solid Biosciences has shown protein expression in the first 3 patients dosed. As a result, the company is expediting the planned dose-escalating activities.

The UK’s National Institute for Health and Care Excellence has authorized the use of the CAR T-cell therapy tisagenlecleucel (Kymriah) for the treatment of adults with relapsed/refractory diffuse large B-cell lymphoma who have received ≥2 lines of chemotherapy.

RNA-based analyses of prospectively-collected tumor specimens from patients with metastatic renal cell carcinoma in the phase III COMPARZ trial revealed 4 molecular subgroups, of which a high angiogenesis expression and a low macrophage infiltrate were indicative of response to frontline TKI therapy.

Federal contractors lost not only wages during the 5-week government shutdown, some of them also lost health insurance; with advances in gene therapy, scientists are beginning to talk about a cure in sickle cell disease; there’s bipartisan interest in attacking the issue of unexpected medical bills, and the effort has been aided by President Donald Trump, who voiced support last week for protecting patients.

Axovant Sciences has announced that, in addition to receiving positive feedback from the FDA on its clinical development, it is expecting data from the first 2 patients dosed to read out in March 2019.

A phase I study examines the use of LCAR-B38M, a CAR T-cell therapy directed against BCMA, in patients with relapsed/refractory multiple myeloma.

An Investigational New Drug application was approved to clinically assess AMT-130 as the first potential drug marketed for the neurodegenerative disease.

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).