News

A first-time study of chimeric antigen receptor T-cell therapy in youths with Down syndrome-associated relapsed/refractory (r/r) acute lymphoblastic leukemia produced high remission rates and toxicity results that were similar to those observed in patients with r/r ALL.

Results from a recent study may show why some patients with chronic lymphocytic leukemia are resistant to tisagenlecleucel, while potentially offering a pathway to enhance patient response.

Orchard Therapeutics announced that its gene therapy candidate, OTL-200, has been granted Rare Pediatric Disease designation for the treatment of metachromatic leukodystrophy.

CAR T-cell therapy can induce next generation sequencing negativity in patients with relapsed/refractory acute lymphoblastic leukemia, suggesting a "synergistic" relationship with hematopoietic cell transplant.

Tisagenlecleucel, sold as Kymriah, has gained its second indication following the FDA's approval of the chimeric antigen receptor T-cell therapy for the treatment of adult patients with relapsed or refractory large B-cell lymphoma, the most common form of non-Hodgkin lymphoma.

REGENXBIO Inc. announced that the U.S. FDA has granted Fast Track designation to RGX-131, a novel, one-time investigational treatment for mucopolysaccharidosis type II (MPS II).

The chimeric antigen receptor T-cell therapy tisagenlecleucel has been approved for a second type of blood cancer; the National Institutes of Health has started recruiting individuals for a database that will include data on more than 1 million people; Kansas’ request to impose a 3-year lifetime limit on Medicaid benefits is testing just how open the Trump administration is to allowing states flexibility.

The FDA has approved tisagenlecleucel (Kymriah) for use in adult patients with relapsed/refractory large B-cell lymphoma—including diffuse large B-cell lymphoma (DLBCL), high-grade B-cell lymphoma, and DLBCL arising from follicular lymphoma—after 2 or more lines of systemic therapy.

Alectinib offered significantly improved outcomes over standard chemotherapy in patients with ALK-positive non–small-cell lung cancer who had progressed on crizotinib therapy.

A fully automated artificial intelligence-based system could effectively classify function and potency of cell therapy.

Keith Stewart, MB, ChB, discusses the role of stem cell transplant, minimal residual disease testing, and maintenance therapy in the treatment of patients with multiple myeloma.

Children receiving AVXS-101 have shown such improvement that researchers are calling for an update to the CHOP-INTEND scale used to track their progress.

Maung Myo Htut, MD, discusses the use of CAR T-cell therapy in multiple myeloma and the potential infusion of immunotherapy into treatment.

Drug company bluebird bio, Inc, has announced the publication of positive interim data for its gene therapy to eliminate or reduce chronic blood transfusions in patients with transfusion-dependent β-thalassemia.

Frederick L. Locke, MD, discusses the ongoing ZUMA-2 trial investigating axi-cel for patients with mantle cell lymphoma.

The U.S. FDA has cleared the Investigational New Drug application for DTX401 for the treatment of glycogen storage disease type Ia (GSDIa).

City of Hope is currently the only institution in Southern California to offer both FDA-approved CAR T-cell therapies.

With chimeric antigen receptor (CAR) T-cell therapy being so new, there is going to be a learning curve as providers become more educated about the treatments, the manufacturing process, and the toxicities, Houston Holmes, MD, MBA, FACP, a medical oncologist with Texas Oncology, explained at the Community Oncology Alliance’s (COA) 2018 Community Oncology Conference.

Reimbursement models, novel gene therapy–based treatments, and price transparency: these were some of the topics covered by presenters and panelists at the annual meeting of the Community Oncology Alliance.

C. Ola Landgren, MD, PhD, shares his insight on CAR T-cell therapy, sequencing, and triplet and quadruplet regimens in myeloma.

A new class of injectable monoclonal antibodies directed against calcitonin gene related peptide (CGRP) or its receptor may offer hope to migraineurs who have not found effective prophylactic therapy.

While calcitonin gene-related peptide (CGRP) inhibitors are projected to have a positive impact on the health of patients with chronic or episodic migraines for whom preventive therapy had failed relative to no treatment, it is likely that the inhibitors will exceed commonly-cited willingness-to-pay thresholds, according to a report from the Institute for Clinical and Economic Review (ICER).

The FDA has granted a priority review to a supplemental biologics license application for nivolumab (Opdivo) for the treatment of patients with small cell lung cancer with disease progression following 2 or more lines of therapy.

Treatment with nivolumab reduced the risk of death by 32% compared with investigator’s choice of therapy for patients with metastatic or recurrent squamous cell carcinoma of the head and neck.

An off-the-shelf, dual-targeted chimeric antigen receptor T-cell approach yielded positive results in preclinical specificity, functionality, and efficacy studies.

Pfizer has dosed its first patient in a phase 1 clinical trial of an investigational gene therapy for the treatment of patients with Duchenne muscular dystrophy, a genetic disease that results in the absence of dystrophin, a protein that helps to keep muscle cells intact.

On Thursday, GlaxoSmithKline (GSK) announced that it will be transferring its portfolio of approved and investigational gene therapies to Orchard Therapeutics in exchange for a 19.9% stake in the company.

How are community practices coping with administering chimeric antigen receptor (CAR)-T treatments? At the 2018 Community Oncology Conference hosted by the Community Oncology Alliance, Houston Holmes, MD, MBA, FACP, Texas Oncology, shared his experience with administering CAR T-cells in a community cancer center–based setting.

Pfizer announced that it has begun a Phase 1b clinical trial for PF-06939926, its mini-dystrophin gene therapy candidate, intended for boys with Duchenne muscular dystrophy.

CEO of Krystal Biotech discusses first topical gene therapy for dystrophic epidermolysis bullosa.