On April 1, 2022, the Food and Drug Administration approved axicabtagene ciloleucel (Yescarta, Kite Pharma, Inc.) for adult patients with large B-cell lymphoma (LBCL) that is refractory to first-line chemoimmunotherapy or relapses within 12 months of first-line chemoimmunotherapy. It is not indicated for the treatment of patients with primary central nervous system lymphoma. Approval was
Natural killer (NK) cells derived from cord blood that were activated and complexed with a CD30/CD16A bispecific antibody elicited an 89 percent overall response rate in patients with relapsed or refractory CD30+ lymphoma, according to results from a phase I/II clinical trial presented at the AACR Annual Meeting 2022, held April 8-13. “We were favorably surprised by the
A MRNA VACCINE WAS COMBINED WITH CAR T CELLS THAT TARGET TUMOR CELLS EXPRESSING THE CLDN6 ANTIGEN A new chimeric antigen receptor (CAR) T-cell product had an acceptable safety profile and showed early signs of efficacy as a monotherapy and in combination with an mRNA vaccine in patients with solid tumors, according to preliminary data
CD19 CAR T-cell therapy is effective at achieving durable remission for relapsed/refractory ALL across cytogenetic risk groups. CD19 CAR T-cell treatment results for patients with high-risk cytogenetics including Ph+, Ph-like, and KMT2A-rearranged ALL are encouraging. Chimeric antigen receptor (CAR) T-cell therapy can induce durable remissions of relapsed/refractory B-acute lymphoblastic leukemia (ALL). However, case reports suggested differential
Stanford researchers have been working on gene therapies for epidermolysis bullosa, or “butterfly disease,” for over a decade. A new gel helped wounds heal and stay healed in a clinical trial. A gene therapy gel applied to the wounds of nine people — three of whom were children — with the blistering skin disease epidermolysis
A study reporting this new protocol was published in the April 7 print edition of the journal Cell Stem Cell. A Mount Sinai-led team has developed a reproducible and scalable method to advance maturation of human pluripotent stem cell-derived cardiomyocytes (hPSC-CMs)—cells that support heart muscle contraction, generated in the lab from human stem cell lines—which researchers
BACKGROUND Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-associated virus 5 (AAV5)–based gene-therapy vector containing a coagulation factor VIII complementary DNA driven by a liver-selective promoter. The efficacy and safety of the therapy were previously evaluated in men with severe hemophilia A in a phase 1–2 dose-escalation study. METHODS Was conducted an open-label, single-group, multicenter, phase 3
Immunotherapy is increasingly becoming a successful way to treat cancer. Researchers at Uppsala University have now developed armed CAR-T cells that reinforce the immune defense against cancer and that could increase the possibilities to successfully treat solid tumors. The study has been published in the journal Nature Biomedical Engineering. The use of immunotherapy to treat cancer
Researchers at Weill Cornell Medicine have identified a key protein that induces the program to build specialized liver blood vessels. The discovery could lead to engineered replacement hepatic tissue to treat common liver diseases. There are many types of blood vessels in the human body that are functionally different from each other. In the liver,
Findings shine light on underpinnings of COPD, pave new direction for future research on treatments A new type of cell that resides deep within human lungs and may play a key role in human lung diseases has been discovered by researchers at the Perelman School of Medicine at the University of Pennsylvania. The researchers, who
