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Therapies for aggressive, recurrent glioblastomas are sorely needed but frequently fail in trials. A first-in-human trial of CAN-3110, an engineered herpes simplex virus 1 (HSV1), shows that it is safe and seems to extend survival and stimulate immune responses — particularly in people with antibodies to HSV1.
The protein netrin-1 is involved in embryonic development and is upregulated in various cancers, including endometrial cancer. In mouse models and a first-in-human trial, blocking netrin-1 with a humanized monoclonal antibody, NP137, prevents a cellular change called the epithelial–mesenchymal transition and inhibits tumour growth.
Individuals with limited English proficiency were less likely to participate in, and sign consent documents in their primary language for, clinical trials led by academia than those led by industry. This retrospective analysis shows that inadequate funding for translation is a barrier to equitable trial enrolment and appropriate informed consent in academic trials.
Researchers have developed a system to classify genetic mutations that could be addressed by therapeutic interventions that use ‘splice-switching antisense oligonucleotides’. This framework identified multiple eligible mutations among 235 people with the genetic disorder ataxia–telangiectasia. An oligonucleotide that was specific to one of these mutations was advanced to a proof-of-concept individualized trial.
Spinal-cord injury interrupts communication between the brain and spinal cord, leading to paralysis. An implant that decodes the brain signals that control movements and drives electrical stimulation of the spinal cord re-establishes this communication, enabling an individual with spinal-cord injury to walk naturally.
Leukaemias characterized by the rearrangement of the gene KMT2A or mutation of the NPM1 gene depend on the protein menin. In a first-in-human trial, the menin inhibitor revumenib had minimal severe adverse effects and showed promising clinical activity in individuals with these types of leukaemia.
