A UCSF team engineered immune cells to activate exclusively in the brain and attack diffuse midline glioma. It worked in mice and it’s poised for a clinical trial at UCSF. Scientists at UC San Francisco have engineered CAR-T cells that stay dormant until they reach the brain, where they switch on and destroy tumor cells.
Researchers at the Johns Hopkins Sidney Kimmel Comprehensive Cancer Center and Johns Hopkins Children’s Center report that bone marrow transplant using half-matched family donors achieved a 96% survival rate among children, adolescent, and young adult patients with severe aplastic anemia, a rare, life-threatening disorder in which the bone marrow fails to produce enough healthy blood
Experts from EBMT, ISCT and JACIE, with representatives from EULAR and ECTRIMS, have published updated recommendations supporting careful patient selection, multidisciplinary care and long-term follow-up as CAR T-cell therapy expands beyond cancer. An international multidisciplinary group of 31 experts has updated consensus recommendations for using chimeric antigen receptor (CAR) T-cell therapy in people with severe,
Five years after a landmark result in a single patient, new findings from the first treated cohort show that optogenetic vision restoration can work beyond an individual case. Scientists have taken another important step toward restoring vision in people with certain inherited forms of blindness. In a study published in the New England Journal of
Persistent CAR-T cells in the intestine and widespread immune disruption seen as possible drivers of a rare but serious complication Researchers at the Icahn School of Medicine at Mount Sinai have identified biological mechanisms that may contribute to CAR-T cell-associated enterocolitis, a serious inflammatory condition of the intestine that can occur after a type of
The publication consolidates more than two decades of research and clinical development and confirms the long-term clinical benefit of an autologous gene therapy for Wiskott-Aldrich syndrome, a rare genetic disorder affecting the immune system and platelets. A single infusion of genetically corrected autologous hematopoietic stem cells can provide durable clinical benefit for patients with Wiskott-Aldrich
Innovative gene therapy offers a new option for children with a rare, progressive neurodevelopmental disease The U.S. Food and Drug Administration approved Fayuvi (rebisufligene etisparvovec-hopf), the first treatment for pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A., MPS IIIA is a rare inherited disease that progressively damages the
A review led by University of Miami researchers examines how delivering stem cells directly into brain-supplying arteries could support recovery after ischemic stroke and identifies the evidence still needed before the approach becomes part of standard care. Key Takeaways Researchers from the University of Miami Miller School of Medicine and collaborating institutions have published a
The interim Phase I trial data was only from 35 people, but results look good so far. An experimental gene-editing therapy that aims to lower bad cholesterol for the long-term after a single infusion is off to a positive start in an early clinical trial. Researchers running a Phase I safety trial for the drug,
Researchers are testing CAR T cell therapy as a way to reset the immune system in lupus, Graves’ disease and other conditions where the body’s defenses go rogue At age 49, Jan Janisch-Hanzlik’s multiple sclerosis was destroying her freedom to live the life she wanted. She gave up her active nursing job for a desk
