Study shows treatment developed by international team restored immune function in more than 95% of patients in three clinical trials An experimental form of gene therapy developed by a team of researchers from UCLA and Great Ormond Street Hospital in London has successfully treated 48 of 50 children born with a rare and deadly inherited
In recent years, the interest in adipose tissue mesenchymal cell–derived extracellular vesicles (AT-MSC-EVs) has increasingly grown. Numerous articles support the potential of human AT-MSC-EVs as a new therapeutic option for treatment of diverse diseases in the musculoskeletal and cardiovascular systems, kidney, skin, and immune system, among others. This approach makes use of the molecules transported
Chimeric antigen receptor (CAR) T-cell therapy can induce side-effects such as cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome (ICANS), which often require intensive care unit admission. The aim of the study, published in The Lancet Haematology was to describe management of critically ill CAR T-cell recipients in intensive care. An international, multicentre, observational
PTC Therapeutics has reported results from Part 2 of the pivotal FIREFISH trial of Evrysdi (risdiplam), which demonstrated that infants with type 1 spinal muscular atrophy (SMA) continued to show improvements in survival and key motor milestones after two years of treatment. PTC Therapeutics has reported results from Part 2 of the pivotal FIREFISH trial
Approval of ide-cel for adults who have relapsed after four or more prior lines of therapy follows clinical trials led by Dana-Farber Cancer Institute investigators The Food and Drug Administration (FDA) has granted its first approval of a CAR T-cell therapy for adults with multiple myeloma, bringing welcome hope for patients eligible for the treatment, according to researchers
Key Takeaways A single injection of a gene therapy involving what are called zinc fingers dramatically reduced levels of tau, a protein implicated in Alzheimer’s disease, in mice with the condition. The treatment was long lasting, caused no side effects, and reduced Alzheimer’s-related damage in the brain. The technology worked just the way we had
Gene therapy can be used to restore cell function in monogenic disorders or to endow cells with new capabilities, such as improved killing of cancer cells, expression of suicide genes for controlled elimination of cell populations, or protection against chemotherapy or viral infection. While gene therapies were originally most often used to treat monogenic diseases
Chimeric antigen receptor (CAR)-T cell therapies are on the verge of becoming powerful immunotherapeutic tools forcombating hematological diseases confronted with pressing medical needs. Lately, CAR-NK cell therapies have also come into focus as novel therapeutic options to address hurdles related to CAR-T cell therapies, such as therapy-induced side effects. Currently, more than 500 CAR-T and
A study recently published in the journal Science looked into how CAR T immunotherapy could be used to treat solid tumours in addition to leukaemias. The programming of CAR T cells opens avenues for applying cell therapies to, for example, breast cancer or ovarian cancer in the future. In cancer immunotherapy, cells in the patient’s
Sara Ghorashian, PhD, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK, outlines the challenges associated with CAR T-cell therapy for pediatric B-cell acute lymphoblastic leukemia (ALL). Discovering the best treatment phasing with other immunotherapies, such as blinatumomab and inotuzumab, improving the toxicity profile, and CAR T-cell persistence are some of the remaining
