News

Spark Therapeutics CEO Jeff Marrazzo reassured patients and the medical community he has an obligation to ensure access to the novel gene therapy.

New vision loss gene therapy gets a price tag below the expected $1 million mark; new evidence finds that the prevalence of autism spectrum disorders has plateaued; hospitals are ill-equipped to care for dementia patients, but a new effort could change that.

Plinabulin, an antineoplastic agent that activates an immune response, is being investigated as a treatment option for patients with advanced or metastatic non–small cell lung cancer who have progressed after standard-of-care therapy.

The FDA has expanded the label for cabozantinib (Cabometyx) to include first-line treatment of advanced renal cell carcinoma regardless of treatment status. Cabozantinib was initially approved in patients who had previously received anti-angiogenic therapy.

Sagus Sampath, MD, discusses ongoing studies exploring checkpoint inhibitors with radiation therapy and the promise of integrating these 2 treatments in the field of non-small cell lung cancer.

The FDA has granted the PD-L1 inhibitor avelumab a breakthrough therapy designation for use in combination with the VEGF inhibitor axitinib in treatment-naïve patients with advanced renal cell carcinoma.

The combination therapy increased the attraction of immune cells to fight non-small cell lung cancer and also boosted the response to immune checkpoint inhibitors.

Universal gene expression profiling of patients with stage II breast cancer resulted in outpatient savings of $11,000 (inclusive of testing costs) within 6 months of initiation of medical therapy.

CAR T cells targeting CD19 can be effective at treating relapsed/refractory diffuse large B-cell lymphoma or follicular lymphoma, with high rates of durable remission.

The FDA approved Spark Therapeutics Inc.’s voretigene neparvovec-rzyl (Luxturna), the first gene therapy for inherited vision loss caused by faulty gene mutations.

Ghassan K. Abou-Alfa, MD, discusses what he sees coming down the pike in epatocellular carcinoma with regard to novel agents, including CAR T-cell therapy, and the burgeoning questions with optimal sequencing of these treatments.

This is the first gene therapy approved in the US targeting mutations in a specific gene.

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.

Marcela V. Maus, MD, PhD, evaluates the next generation of chimeric antigen receptor (CAR) T cells in both hematologic malignancies and solid tumors

Chimeric antigen receptor (CAR) T cells have hit prime time, with the 2 recent FDA approvals for this class of cell-based therapy.

Renova Therapeutics is planning a 536-patient phase 3 trial, FLOURISH, to begin in early 2018.

Researchers found that 2 naturally occurring molecules can make the process of cell generation faster and more efficient. This could have major implications for multiple sclerosis therapy research.

Maintenance therapy with elotuzumab (Empliciti) and lenalidomide (Revlimid) after autologous stem cell transplant improves the quality of response achieved with induction therapy in patients with multiple myeloma.

MMG49 has been identified as a monoclonal antibody that can be targeted using CAR T-cell therapy for patients with multiple myeloma.

A proof-of-concept study has demonstrated that resistance to treatment in multiple myeloma and mantle cell lymphoma could be linked to a protein called Nrf1, which appears to respond to proteasome insufficiency or pharmacological inhibition.

A much-anticipated session on the second day of the 59th Annual Meeting and Exposition of the American Society of Hematology provided long-term updates on trials evaluating 2 chimeric antigen receptor-T (CAR T) treatments: tisagenlecleucel or CTL019 (Kymriah) for the treatment of adult relapsed/refractory diffuse large B-cell lymphoma (DLBCL) and axicabtagene ciloleucel (Yescarta) evaluated in patients with refractory aggressive non-Hodgkin lymphoma.

Continuous lenalidomide therapy improves outcomes among patients with newly diagnosed myeloma who are not autologous stem cell transplant candidates, according to findings from a large phase III clinical trial.

A single treatment with a second-generation CAR T-cell treatment elicited an overall response rate of 94% in a small study of patients with heavily pretreated multiple myeloma, according to the results of a phase I study presented at the ASH Annual Meeting.

A potentially practice-changing study presented at 2017 ASH shows high rates of sustained complete response in patients with relapsed/refractory diffuse large B-cell lymphoma who were treated with CTL019 CAR T-cell therapy.

The CAR T-cell therapy tisagenlecleucel (Kymriah) achieved an overall response rate of 53.1% in adult patients with relapsed/refractory diffuse large B-cell lymphoma.

Treatment with the BCMA-directed CAR T-cell therapy bb2121 induced complete remissions for 56% of patients with relapsed/refractory multiple myeloma.

Lenalidomide (Revlimid) plus rituximab (Rituxan) is a feasible combination that is also safe and active, as initial and maintenance therapy for patients with mantle cell lymphoma.

Enzalutamide (Xtandi) added to exemestane improved progression-free survival in patients with hormone receptor-positive advanced breast cancer and no prior endocrine therapy who were positive for a gene signature-based biomarker indicating androgen receptor-signaling.

Noelle Frey, MD, discusses the FDA approvals of CAR T-cell therapies, as well as the bright future of the treatment.

The FDA has granted a priority review to a biologics license application for mogamulizumab for the treatment of patients with cutaneous T-cell lymphoma who have received at least 1 prior systemic therapy.