Two children who received a Novartis gene therapy for their neuromuscular disease died following treatment, spotlighting its risks and renewing questions about the safety of genetic medicines like it. The patients developed acute liver failure between five and six weeks after infusion with the gene therapy, called Zolgensma and approved to treat spinal muscular atrophy,
Young people who received doses of tisagenlecleucel, a chimeric antigen receptor T cell (CAR-T) therapy, at the higher end of the FDA-approved dosing range had significantly better survival rates at one year compared with those who received lower doses within this range, according to research published today in Blood Advances. Since its approval as the first
Scientists at St. Jude Children’s Research Hospital developed a simple method to select for more effective cancer-destroying CAR T cells for patients with relapsed T-ALL. Scientists at St. Jude Children’s Research Hospital are improving chimeric antigen receptor (CAR) T-cell therapy. Their new simplified approach selected for an advantageous T-cell type and showed promise in the lab against relapsed T-cell
In rodent models, introduced neuroprotective protein slowed disease progression and increased life span Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease characterized by the progressive loss of motor neurons in the brain and spinal cord responsible for voluntary movements and muscle control. In a new study, published July 11, 2022 in the journal Theranostics, researchers at
A multi-institutional effort led by researchers at the School of Veterinary Medicine is taking steps to develop an effective technique to regenerate photoreceptors cells and restore sight in people with vision disorders. What if, in people with blinding retinal disorders, one could simply introduce into the retina healthy photoreceptor cells derived in a dish from
Interdisciplinary team of researchers from UMass Amherst and UMass Chan Medical School maps the cellular production of protein and discovers how to boost its output In a major new development in the quest to develop better gene therapies with which to treat a host of diseases, researchers at the University of Massachusetts Amherst and UMass
CNS relapse is still a common cause of treatment failure in R/R B-ALL, although chemotherapy, cranial irradiation, and allo-HSCT are all modalities that can be incorporated into the management of CNSL. In the present study, published on Blood by a team of Xuzhou Medical University, was reported the efficacy, toxicity, and clinical feasibility of CD19-specific
Hannover Medical School doctors successfully treat three-year-old girl / Publication in “Nature Cardiovascular Research“ Clinical researchers at Hannover Medical School (MHH) have succeeded for the first time worldwide in stopping the usually fatal course of the disease in severe pulmonary hypertension thanks to a novel therapeutic approach. A three-year-old girl suffering from so-called pulmonary arterial
Given recent progress in cell therapy research, it is clear that the engineering disciplines outlined in this Review will play an increasing role in the creation of new product pipelines with improved safety, efficacy and accessibility for patients. Recent scientific advances have not only demonstrated the potential impact of technologiesdeveloped by each of these fields,
Stanford researchers modified anti-cancer CAR-T cells so they can be controlled with an oral drug. The modified cells are safer, more potent and more active against solid tumors in mice. A cancer treatment that uses a patient’s own genetically modified immune cells to attack cancer cells is safer and more effective when it can be
