DMD Trial Results Demonstrate Decrease in Creatine Kinase Levels
July 10th 2018Results of a Phase 1/2a gene therapy clinical trial in children with Duchenne muscular dystrophy (DMD) revealed a significant decrease in levels of serum creatine kinase, an enzyme biomarker associated with muscle damage caused by DMD.
BrainChild-01 Will Evaluate CAR T Cells in Pediatric CNS Tumors
July 5th 2018The trial is designed to test chimeric antigen receptor (CAR) T-cell therapy in children and young adults with relapsed/refractory brain and central nervous system (CNS) tumors, wherein the modified CAR T cells will be directly injected at the site of tumor resection or into the ventricular system of the CNS.
Tisagenlecleucel Nears European Approval for DLBCL, ALL
June 29th 2018The European Medicines Agency’s Committee for Medicinal Products for Human Use has recommended approval of tisagenlecleucel for the treatment of either adult patients with diffuse large B-cell lymphoma that is relapsed or refractory after 2 or more lines of systemic therapy, or patients up to 25 years of age with B-cell acute lymphoblastic leukemia that is refractory, in relapse posttransplant, or in second or later relapse.
AbbVie and Calibr to Collaborate on CAR T-Cell Development
June 29th 2018Drug 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.
Gene Therapy in Patients with DMD Demonstrates Outstanding Preliminary Results
June 22nd 2018Positive 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.
Nivolumab Gains First Indication in China for NSCLC
June 20th 2018Last 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).
CD22 CAR T-cell Therapy Effective After CD19 CAR Therapy Failure in Children With B-ALL
June 16th 2018A 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.
RA Drug May Reduce Toxicity Caused By CAR T Treatment
June 15th 2018Last 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.
FDA Approves Pembrolizumab for Relapsed/Refractory PMBCL
June 13th 2018The 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.
Gilteritinib Proves Promising for the Treatment of FLT3mut + Acute Myeloid Leukemia
June 8th 2018Planned 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).