Category REGENERATIVE MEDICINE

PIER MARIA FORNASARI
2021-04-22

Outcomes in patients treated with chimeric antigen receptor T-cell therapy who were admitted to intensive care (CARTTAS): an international, multicentre, observational cohort study published on The Lancet Haematology

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

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PIER MARIA FORNASARI
2021-04-15

Risdiplam improves motor function and survival in infants with SMA

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

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PIER MARIA FORNASARI
2021-04-04

First FDA approval of CAR T-cell therapy for multiple myeloma marks milestone for patients

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

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PIER MARIA FORNASARI
2021-03-23

Gene therapy using “zinc fingers” may help treat Alzheimer’s disease, animal study shows

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

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PIER MARIA FORNASARI
2021-03-23

Retroviral gene therapy with a view on previous experience and future perspectives

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

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PIER MARIA FORNASARI
2021-03-23

Current status and perspective of CAR-T and CAR-NK cell therapy trials in Germany

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

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PIER MARIA FORNASARI
2021-03-19

A leap for­ward in research on CAR T cell ther­apy – Cel­lu­lar en­gin­eer­ing makes it pos­sible to tar­get the ther­apy also against solid tu­mours

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

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PIER MARIA FORNASARI
2021-03-08

The challenges of CAR T-cell therapy for pediatric B-ALL. RESTORE launched Fragments of Life: A success in the frontier of medicine becomes a video game.

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

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PIER MARIA FORNASARI
2021-02-24

Chimeric antigen receptor T-cell therapy in multiple myeloma: a systematic review and meta-analysis of 950 patients

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

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PIER MARIA FORNASARI
2021-02-24

European Society Blood and Marrow Transplantation Annual Report shows the rise of CAR-T Cells in Europe during year 2019 and changes in medical practices in the field of hematopoietic cell transplantation over 30 years.

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

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