Scientists at Children’s Hospital Los Angeles develop newer CAR T therapy that promises to be more effective and less toxic for children with neuroblastoma. Chimeric Antigen Receptor T-cell therapy—CAR T—has revolutionized leukemia treatment. Unfortunately, the therapy has not been effective for treating solid tumors including childhood cancers such as neuroblastoma. Preclinical studies using certain CAR
A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. Both therapies work by switching on a gene that promotes production of fetal hemoglobin, said Dr. Lewis Hsu, chief medical officer of the Sickle Cell Disease Association
Despite the significant improvement in survival outcomes of multiple myeloma (MM) over the past decade, it remains an incurable disease. Patients with triple-class refractory MM have limited treatment options and a dismal prognosis. Chimeric antigen receptor (CAR) T-cell therapy targeting B-cell maturation antigen has transformed the treatment armamentarium of relapsed/refractory MM (RRMM), with unprecedented overall
Exosomes are nano-sized extracellular vesicles secreted by all cells. Extensive research over recent years has now shown these vesicles to be important players in intercellular signalling and to have a role in tissue regeneration, immunomodulation and other biological functions. A lipid bilayer protects the exosome cargo of nucleic acids and proteins from degradation and permits
Scientists regenerate parts of the skull affected by craniosynostosis, a common birth defect. Using stem cells to regenerate parts of the skull, scientists corrected skull shape and reversed learning and memory deficits in young mice with craniosynostosis, a condition estimated to affect 1 in every 2,500 infants born in the United States, according to the
“Thanks to this technology and to the specific fighting capabilities of these cells, extraordinary results have been obtained: The therapy is a new hope for patients who no longer responded to other treatments. The therapy seems to have a long-lasting effect after a single application Unlike many other therapies, which benefit only a small portion
The CLI study is unlikely to meet its primary endpoint by the time of the final analysis Substantial low number of events in the placebo group in the CLI study decreased the statistical power of the study to meet its primary endpoint PLX-PAD was well tolerated, and no safety concerns were raised Company to terminate the CLI study to focus on different therapeutic areas in its pipeline and expects three clinical readouts within the coming calendar year
Previous studies reported on the safety and applicability of mesenchymal stem/stromal cells (MSCs) to ameliorate pulmonary inflammation in acute respiratory distress syndrome (ARDS). Thus, multiple clinical trials assessing the potential of MSCs for COVID-19 treatment are underway. Yet, as SARS-inducing coronaviruses infect stem/progenitor cells, it is unclear whether MSCs could be infected by SARS-CoV-2 upon
The Janssen Pharmaceutical Companies of Johnson & Johnson unveils longer-term results from the combined Phase 1b/2 CARTITUDE-1 study evaluating ciltacabtagene autoleucel (cilta-cel) for the treatment of patients with relapsed and/or refractory multiple myeloma. Cilta-cel is an experimental BCMA-directed (B cell maturation antigen) chimeric antigen receptor T cell (CAR-T) therapy. Data presented at the ongoing Annual Meeting
Earlier this year, Caron Jacobson, MD, presented the interim analysis of ZUMA-5: A phase 2 study of axicabtagene ciloleucel in patients with relapsed/refractory indolent non-Hodgkin lymphoma. Results from that interim analysis showed that the vast majority of patients with follicular lymphoma or marginal zone lymphoma – two slow-growing forms of non-Hodgkin lymphoma – responded to and
