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
Despite dramatic improvements in survival, multiple myeloma (MM) remains largely incurable, and most patients develop disease that is refractory to available treatment options. A paper just published on Blood Advances by researchers of Department of Hematological Malignancies and Cellular Therapeutics, Kansas University Medical Center, Westwood, Division of Gastroenterology, University of Toledo, Division of Hematology and
Hematopoietic cell transplantation (HCT) is an established procedure for many acquired or inherited disorders of the hematopoietic system, benign or neoplastic, including those of the immune system, and as enzyme replacement in metabolic disorders. The activity survey of the EBMT, describing the status of HCT, has become an instrument with which to observe trends and
In drug development, scientific progression was never a solo effort. Rather, to advance new therapies, particularly cell and gene therapies, drug development is a team sport. Why is the team so important? Oftentimes when one tests novel cell and gene therapies in the clinic for the first time, one is treading completely new ground. The
Breyanzi demonstrated a 73% overall response rate and 54% complete response (CR) rate in the largest pivotal trial in 3L+ LBCL, TRANSCEND NHL 001 trial Breyanzi demonstrated sustained responses in patients who achieved a CR with median duration of response not reached Grade ≥3 cytokine release syndrome and Grade ≥3 neurologic toxicities following Breyanzi treatment occurred in 4% and
The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) will perform an accelerated assessment of the Marketing Authorisation Application (MAA) for the B-cell maturation antigen (BCMA)-targeted chimeric antigen receptor T-cell (CAR-T) therapy ciltacabtagene autoleucel (cilta-cel). Accelerated assessment is
Chimeric antigen receptor T-cell therapy, or CAR T, has been a breakthrough in the treatment of blood cancers such as acute lymphoblastic leukemia and diffuse large B-cell lymphoma. Clinical studies have shown overall response rates of more than 80% with an ongoing response of nearly 40% more than two years after therapy. However, the cellular
