FDA approved Gomekli (mirdametinib), a kinase inhibitor from SpringWorks Therapeutics, for adult and pediatric patients two years of age and older with neurofibromatosis type 1 who have symptomatic plexiform neurofibromas not amenable to complete resection.
I was a practicing pediatric hematologist oncologist and researcher for 21 years. As a doctor, it was a tremendous privilege to be invited into the lives of patients and families dealing with blood cancers and be entrusted with their care. As a researcher, it was incredibly rewarding to help bring more effective blood cancer therapies to kids.
Despite steady progress in reducing overall cancer mortality rates, cancer incidence in women is rising, according to the American Cancer Society’s “Cancer Statistics, 2025” report. Incidence rates in women 50-64 years of age have surpassed those in men, and rates in women under 50 are now 82% higher than their male counterparts, up from 51% higher in 2002. In 2021, for the first time, lung cancer incidence was higher in women under 65 than in men.
CureSearch for Children’s Cancer awarded a $225,000 research grant to John Prensner, a physician-scientist at the University of Michigan, to advance research on atypical teratoid/rhabdoid tumors. One of the most aggressive and fatal brain cancers affecting infants and toddlers, ATRT has limited treatment options and devastating outcomes, with more than half of cases proving fatal.
OS Therapies Inc. announced positive data from a phase IIb clinical trial (NCT04974008) of OST-HER2 (OST31-164)—the company’s HER2-targeted immunotherapy candidate in the rare pediatric-designated indication of prevention of recurrent, fully resected, lung metastatic osteosarcoma.
A recent population-based study indicates that among children with cancer, those with obesity at the time of diagnosis may face an elevated risk of dying. The findings are published by Wiley online in CANCER.
FDA granted Rare Pediatric Disease designation for THIO in pediatric-type diffuse high-grade gliomas.
On Saturday, Dec. 21, Congress passed a new spending package, narrowly averting a government shutdown.
FDA approved Ryoncil (remestemcel-L-rknd), an allogeneic bone marrow-derived mesenchymal stromal cell therapy, for steroid-refractory acute graft versus host disease in pediatric patients two months of age and older. Ryoncil is the first FDA-approved MSC therapy.
The Lilabean Foundation for Pediatric Brain Cancer Research raised $900,000 at its 13th Annual Lilabean Foundation Fall Ball, held on Saturday, November 2. Proceeds from the Fall Ball will directly fund groundbreaking research initiatives focused on discovering new treatments and improving survival rates for children fighting pediatric brain cancer.
