News

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.

A life-saving therapy is challenging to administer and poses financial risks for patients and institutions alike. Speakers at the Academy of Managed Care Pharmacy Nexus 2018 address some approaches to paying for it while CMS develops long-term policies.

A survivor of chronic lymphocytic leukemia who participated in the first chimeric antigen receptor T-cell trial in 2010 looks back at his experience and also discusses his views on how patient-reported outcomes (PROs) should be used, if at all, by CMS.

VY-AADC01 demonstrated promising early signs of activity and safety for patients with Parkinson disease.

Frontline pembrolizumab monotherapy showed an improvement in overall survival and duration of response versus standard therapy in patients with PD-L1–positive recurrent or metastatic head and neck squamous cell carcinoma; however, there was not a similar improvement in progression-free survival or overall response rate with the PD-1 inhibitor.

The combination of tepotinib plus gefitinib improved progression-free survival and overall response versus chemotherapy in patients with MET mutated EGFR-positive non–small cell lung cancer resistant to prior EGFR TKI therapy.

Since the inception of the idea more than 3 decades ago and its initial development 20 years later, Sarepta Therapeutics’ micro-dystrophin gene therapy has now made its way to human trials.

The gene therapy is also being assessed for long-term safety data, as well as an additional delivery method.

Switchable chimeric antigen receptor (CAR) T cells with a switch directed towards human epidermal growth factor receptor 2 (HER2) in pancreatic ductal adenocarcinoma (PDAC) has similar efficacy as conventional HER2 CAR T cells while also having a greater control over treatment toxicities.

Although CD19 has proved to be an attractive and effective target for chimeric antigen receptor T-cell therapies in hematologic malignancies, a significant subset of patients treated with this groundbreaking form of immunotherapy eventually relapse.

A novel mesenchymal stem cell therapy produced intriguing phase II data and has moved into a randomized, pivotal phase III clinical trial.

Multiple gene signature assays have been developed that supply prognostic information for decisions on adjuvant chemotherapy and whether therapy should be of extended duration.

Cellular therapies have the potential to make a meaningful difference for patients with neurological disorders, if used correctly.

Tisagenlecleucel's high price is aligned with the benefit the chimeric antigen receptor T-cell therapy provides over a patient's life. Approximately 40% of patients treated are expected to be long-term survivors.

We visited San Francisco, California for a State of the Science Summit on Hematologic Malignancies. The meeting covered novel agents and combinations in lymphoma, updates in acute and chronic leukemias, and modalities in the management of multiple myeloma.

Nina Shah, MD, discusses the present and future of chimeric antigen receptor T-cell therapy in myeloma.

Investigators are evaluating whether eryaspase (Graspa), an L-asparaginase-based therapy that triggers tumor cell death, can extend survival for patients with pancreatic cancer.

Brian Till, MD, discusses the rapidly evolving field of chimeric antigen receptor (CAR) T cells.