News

AAV2/5-OPTIRPE65 showed improvements in vision-guided mobility, retinal sensitivity, and foveal-driven visual function for patients with RPE65-associated retinal dystrophy.

Data from a phase 1/2a study show RGX-314, a gene therapy from RegenX, was well tolerated and improved visual acuity in patients.

Results of the phase 1 OPTIC trial reveal ADVM-022 was effective at maintaining visual acuity in patients with wet AMD with a favorable safety profile.

Speakers at AAO 2019 event describe journey leading to approval of the first gene therapy for a genetic disease.

The neoantigens found in the patients’ tumors (non-small-cell lung cancer) which were highly dissimilar were enriched for hydrophobic sequences, and correlated with survival rates after the PD-1 checkpoint therapy.

The trial, which received a $4.1 million in grants from the National Institutes of Health and Gateway for Cancer Research, combines City of Hope’s unique CAR T cell therapy with immune checkpoint inhibitors.

Eduardo Sotomayor, MD, discusses the evolution of CAR T-cell therapy, adverse events that require careful monitoring, and novel strategies under development that may mitigate toxicity and improve T-cell persistence.

CAR T cells are a more effective therapy if manufactured for patients with multiple myeloma prior to the onset of relapsed or refractory disease.

Durvalumab added to etoposide and platinum-based chemotherapy as a first-line treatment for patients with extensive-stage small cell lung cancer delays development of new lesions and improves patient-reported outcomes compared with etoposide and platinum-based therapy alone.

Phase III studies of a gene therapy for Leber Hereditary Optic Neuropathy have generated some unexpected positive findings. There is biologic plausibility to explain the data.

Researchers said Wednesday they created a second-generation chimeric antigen receptor (CAR) T-cell therapy that prevented relapse of lymphoma and leukemia and led to 100% long-term survival in early laboratory studies.

Andrew D. Zelenetz, MD, PhD, discusses the use of emerging targeted therapies in mantle cell lymphoma.

Wayne A. Marasco, MD, PhD, discusses the intricacy of engineering CAR T cells and the early data he has observed with CAR T-cell therapy in renal cell carcinoma.

Andre Goy, MD, MS, discusses cutting-edge CAR T-cell therapy and other groundbreaking investigations, as well as his thoughts on general developments in oncology and hematology.

Joshua P. Sasine, MD, PhD, spotlights some of the strategies under investigation to improve the safety and efficacy of CAR T-cell therapy in hematologic malignancies.

Despite an embrace of greater patient populations by the FDA, cardiovascular research into stem cell therapy has been slow and burdened.

As safety and efficacy programs advance, clinicians consider the investigative therapy's potential in cardiology.

Novel strategies are needed to enhance the efficacy of CAR T-cell therapies in patients with acute lymphoblastic leukemia, including new constructs that target more than 1 antigen.

The FDA has granted a breakthrough therapy designation to the MET inhibitor tepotinib as a treatment for certain patients with metastatic non–small cell lung cancer with MET exon14-skipping alterations.

New data from clinical trials of ocrelizumab showed that the anti-CD20+ B cell therapy lowered serum NfL levels, and that the NfL levels offered prognostic value for disease progression in MS.

In this phase III trial, investigators assessed the clinical efficacy and safety of durvalumab with or without tremelimumab with etoposide and carboplatin or cisplatin chemotherapy followed by durvalumab with or without tremelimumab maintenance therapy compared with EP alone as first-line treatment in extensive-stage small-cell lung cancer.

Combination immunotherapy with nivolumab plus ipilimumab was examined as a first-line therapy for patients with advanced non–small-cell lung cancer. Results were presented at the International Associate for the Study of Lung Cancer 2019 World Conference on Lung Cancer.

The FDA has granted a fast track designation to AMG 510 for the treatment of patients with KRAS G12C–mutated metastatic non–small cell lung cancer who received prior therapy.

The FDA has granted a breakthrough therapy designation to capmatinib (INC280) as a first-line treatment for patients with MET exon14 skipping—mutated non–small cell lung cancer.

While the results are early, if further research proves the approach effective, it could help boost the impact of treatments like chimeric antigen receptor (CAR) T-cell therapy, which to date hasn’t had much luck in solid tumors.

Delays in CAR T-cell therapy may significantly decrease gains in survival and productivity for patients with diffuse large B-cell lymphoma and pediatric acute lymphoblastic leukemia.

ONCOLOGY discussed therapy options, including chimeric antigen receptor (CAR)-T-cell therapies for pediatric acute lymphoblastic leukemia (ALL), with Susan R. Rheingold, MD, Medical Director of the Oncology Outpatient Clinic and attending physician with the Cancer Center at Children’s Hospital of Philadelphia. 

The FDA has granted a priority review designation to a new drug application for zanubrutinib for the treatment of patients with mantle cell lymphoma who have received ≥1 prior therapy.

The emergence of monoclonal antibodies, immunomodulatory agents, immunotoxins, bispecific T-cell engagers, and CAR T-cell therapies will redefine multiple myeloma treatment. However, these new approaches, by themselves, are not enough to achieve cure; they must be used in combination.

Saul Priceman, PhD, City of Hope assistant professor in the Department of Hematology & Hematopoietic Cell Transplantation, and his research team have received a $9.28 million award from the California Institute for Regenerative Medicine to support a chimeric antigen receptor T cell phase 1 clinical trial for the treatment of women with HER2-positive breast cancer that has spread to the brain.