Children with solid tumors who relapse are being treated with the same chemotherapy they would have been given 40 years ago, as “there have been no major approvals for pediatric solid tumors,” Catherine Bollard, senior vice president and chief research officer at Children’s National Hospital, said at a June 5 FDA roundtable on cell and gene therapies (CGTs). The problem isn’t the science. Bollard said many groups are working on curative CGTs “for these children who have lost all other hope for survival.” The real gap is that “big pharma doesn’t see the business model because it’s a rare disease,” she added.
Researchers from the University of Arizona have unveiled that coordinated Y chromosome loss in both cancer cells and immune cells may explain the worse prognosis in people with this alteration. The loss of the Y chromosome is one of the most frequent somatic mutations in men, particularly with advancing age.
Kumquat Biosciences Inc. has described compounds acting as GTPase KRAS wild-type and mutant inhibitors reported to be useful for the treatment of cancer.
Beijing Tide Pharmaceutical Co. Ltd. has identified protein arginine N-methyltransferase 5 (PRMT5) inhibitors reported to be useful for the treatment of cancer and myelofibrosis.
Researchers from the University of Arizona have unveiled that coordinated Y chromosome loss in both cancer cells and immune cells may explain the worse prognosis in people with this alteration. The loss of the Y chromosome (LOY) is one of the most frequent somatic mutations in men, particularly with advancing age. Investigating the mechanisms and effects of LOY in PBMCs and its association with immune and tumor cells, they “found a relationship between the Y chromosome loss in normal cells inside the tumor and the Y chromosome loss inside the cancer cell,” Dan Theodorescu, senior author of the study, told BioWorld.
Dizal (Jiangsu) Pharmaceutical Co. Ltd.’s LYN/BTK dual inhibitor, DZD-8586, saw tumor shrinkage in 94.1% of patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma following Bruton's tyrosine kinase inhibitors, the company reported during an oral presentation at the American Society of Clinical Oncology (ASCO) 2025 conference.