Treatment designed to work across different genetic mutations At a glance: Efforts to develop a gene therapy for Diamond-Blackfan anemia (DBA) — a rare, life-threatening disorder in which bone marrow cannot make mature, functioning red blood cells — have been hampered by the fact that at least 30 different genetic mutations can cause the disorder.
Trial results supported recent FDA approval for patients with relapsed/refractory disease Patients with relapsed or refractory CD19-positive B-cell acute lymphoblastic leukemia (ALL) who were treated with the novel anti-CD19 chimeric antigen receptor (CAR) T cell therapy, obecabtagene autoleucel (obe-cel), experienced high response rates and most did not need a subsequent stem cell transplant (SCT), according to results from the
Researchers at Case Western Reserve School of Medicine have found that, when compared to seven other anti-diabetic drugs, semaglutide, a popular diabetes and weight-loss drug, may lower the risk of Alzheimer’s disease in people with type 2 diabetes (T2D). Alzheimer’s disease is a brain disorder that slowly destroys memory and thinking skills. According to the Alzheimer’s Association, nearly
Study marks the first time these side effects were reported in children Researchers at Children’s Hospital of Philadelphia (CHOP) revealed for the first time that children, adolescents and young adults may experience very rare neurological issues of paraparesis and quadriparesis following chimeric antigen receptor T-cell (CAR-T) therapy, a type of immunotherapy used to treat B-cell Acute
When the U.S. Food and Drug Administration (FDA) announced in November 2023 that it was investigating a link between receiving CAR T therapy and developing secondary cancers later on, it spurred uncertainty for patients and researchers alike. As many of the more than 30,000 patients who have received the life-saving treatment face new questions about
Study results formed the basis of the therapy’s FDA approval in April 2024 Adults with hemophilia B saw their number of bleeding episodes drop by an average of 71 percent after a single infusion of gene therapy, according to the results of an international Phase III clinical trial published today in the New England Journal of Medicine by researchers from the University of
Scientists from UNIGE and HUG have developed CAR-T cells capable of targeting malignant gliomas while preserving healthy tissues. Glioblastoma is the most common and aggressive primary brain tumor—average survival after diagnosis is less than two years—and current treatments remain ineffective. In recent years, immunotherapies have been giving patients hope, albeit with relatively modest success. A
In an early Stanford Medicine study, CAR-T cell therapy helps some with intractable lymphoma, but those who relapse have few options. Modifying the therapy’s molecular target improved response. CAR-T cell therapy, which targets a specific protein on the surface of cancer cells, causes tumors to shrink or disappear in about half of patients with large
In April, the FDA warned of risk of secondary cancers in people receiving CAR-T cell therapy. A large Stanford Medicine study finds the risk is low and not related to the CAR-T cells. A large study by researchers at Stanford Medicine has found that the risk of secondary blood cancers after CAR-T cell therapy — a
Administering gene therapy in both ears of five children with DFNB9 led to additional benefits compared to previous trial, including sound source localization and hearing in noisy environments. Key Takeaways A novel gene therapy designed to target a form of inherited deafness restored hearing function in five children who were treated in both ears. The
