News

We've rounded up the top 5 most read articles of June, including trial results from the 2018 American Society of Clinical Oncology Annual Meeting and a FDA breakthrough therapy designation for a gene therapy.

Drug manufacturer AbbVie, and a nonprofit drug discovery division of Scripps Research, Calibr, announced earlier this week that they are partnering to develop chimeric antigen receptor (CAR-T) cell therapies primarily aimed at treating cancer, particularly, solid tumors.

From making the Oncology Care Model work to the challenges of paying for CAR T-cell therapy, panelists at the National Comprehensive Cancer Network Policy Summit discussed how the cost of innovation affects decisions.

A novel gene therapy has demonstrated impressive early results in a small sample of 3 children with Duchenne muscular dystrophy.

Positive preliminary results from the Sarepta Therapeutics, Inc phase 1/2a gene therapy clinical trial assessing AAVrh74.MHCK7.micro-Dystrophin vector in individuals with Duchenne muscular dystrophy (DMD) indicate outstanding promise.

Across 3 cohorts, the therapy showed improvements in on-time without troublesome dyskinesia, ranging from 2.1 hours to 3.5 hours.

Last week, Bristol-Myers Squibb announced that the China National Drug Administration approved the country’s first immuno-oncology and first PD-1 therapy, nivolumab (Opdivo), for the treatment of locally advanced or metastatic non-small cell lung cancer (NSCLC).

Clinical data demonstrate that CD34+ cell therapy improves exercise capacity, angina frequency and reduces mortality in no-option refractory angina.

Advanced sequencing techniques have revolutionized the understanding of how cancer has developed and enabled treatment selection based on genomic characterization of the tumor.

A CAR T-cell therapy specific for CD22 was safe and provided high response rates for pediatric patients with B-cell acute lymphoblastic leukemia who had failed chemotherapy and/or a CD19-targeted CAR T-cell treatment.

A compound CLL1/CD33-targeted CAR T-cell therapy showed a complete remission with minimal residual disease (MRD) negativity in a single-patient case study from an ongoing phase I study for relapsed/refractory acute myeloid leukemia.

A new approach using gene editing technology could allow chimeric antigen receptor (CAR) T cells to target CD33 in patients with acute myeloid leukemia (AML) but prevent the cells from attacking healthy stem cells, too.

Last year, the FDA expanded the indications of rheumatoid arthritis (RA) drug tocilizumab (Actemra) to include the treatment of cytokine release syndrome (CRS) caused by CAR T-cell therapy. Recently, 2 studies have identified another rheumatoid arthritis drug that could be more effective in the treatment of CRS.

The CD19-directed chimeric antigen receptor T-cell therapy lisocabtagene maraleucel (JCAR017; liso-cel) is showing promising response rates in patients with high-risk diffuse large B-cell lymphoma.

The FDA has approved pembrolizumab for the treatment of refractory primary mediastinal large B-cell lymphoma (PMBCL) or who have relapsed after 2 or more previous lines of therapy.

The FDA has granted an accelerated approval to pembrolizumab for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma, or those who have relapsed after 2 or more prior lines of therapy.

CMS has proposed a different way to handle payment for chimeric antigen receptor T-cell therapy, one that the medical and manufacturing community thinks is unworkable.

CAR T-cell therapy with bb2121 induced deep, long-lasting responses in patients with heavily pretreated MM.

Planned analyses of phase 3 trial of gilteritinib as a maintenance therapy after allogeneic hematopoietic stem cell transplantation in patients with Fms-like tyrosine kinase 3 internal tandem duplication (FLT3-ITD) mutations in acute myeloid leukemia (AML).

The CD19-targeted CAR T-cell product axicabtagene ciloleucel (axi-cel; Yescarta) is currently being investigated in patients with relapsed/refractory MCL in the ongoing ZUMA-2 trial.

Several studies presented at the 2018 ASCO Annual Meeting helped further refine and inform treatment strategies for the budding class of CAR T-cell therapies, with a focus on predicting adverse events and optimizing efficacy.

A study recently published in Cell investigated the deletion of the CD33 protein to enable CAR T-cells to more accurately target and attack cancerous cells in patients with n acute myeloid leukemia (AML).

The FDA has granted a Breakthrough Therapy designation to bluebird bio, Inc’s Lenti-D, a gene therapy for patients with cerebral adrenoleukodystrophy, an X-linked genetic disorder caused by a defect in the gene ABCD1.

In TRANSCEND NHL 001, the CD19-directed 4-1BB CAR T-cell product lisocabtagene maraleucel yielded durable responses in heavily pretreated R/R DLBCL.

At the 2018 American Society of Clinical Oncology Annual Meeting, June 1-5, Chicago, Illinois, Noopur S. Raje, MD, director, Center for Multiple Myeloma, Massachusetts General Hospital Cancer Center, presented results from the phase 1 multicenter study with a second-generation chimeric antigen receptor (CAR) T-cell therapy called bb2121.

Therapy with CAR T cells may benefit patients with highly refractory multiple myeloma, said U Penn’s Adam Cohen at ASCO 2018.

Marijke van den Berg, MD, PhD, Alok Srivastava, MD, and Glenn Pierce, MD, PhD discuss gene therapy in hemophilia.

The FDA has granted crizotinib a breakthrough therapy designation for the treatment of patients with metastatic non–small cell lung cancer with MET exon 14 alterations, and for use in patients with relapsed/refractory ALK+ anaplastic large cell lymphoma.