FDA Approves Targeted Therapy for Aggressive Brain Cancer

A recent Food and Drug Administration (FDA) accelerated approval marks a step forward in treating a lethal brain cancer that is predominantly diagnosed in children. The drug, Modeyso (dordaviprone), which is taken as a once-weekly oral capsule, is approved for people age 1 or older who have H3 K27M-mutated diffuse midline glioma (DMG) and whose cancer progressed after other treatments. This marks the first drug approval for this cancer with this mutation, MedPage Today reported. The approval was based on data from five clinical studies that enrolled 50 patients who had DMG with this mutation, which is commonly found in this type of brain cancer. Among the participants, 22% responded to the drug, with the response lasting a median of 10.3 months. Some patients’ responses lasted for over a year. “For the first time, we have an FDA-approved therapy for patients with recurrent H3 K27M-mutant diffuse midline glioma,” Patrick Wen, a study investigator and a neuro-oncologist at Dana-Farber Cancer Institute in Boston, said in a press release. “While outcomes remain challenging for many patients, the objective responses observed with dordaviprone, including durable benefit in some patients, represent a meaningful advancement.”

Colorectal Cancer Screening Rises in People Ages 45 to 49

As U.S. incidence of colorectal cancer continues to climb in people under 50, a series of studies published in JAMA suggests more people ages 45 to 49 are getting screened for the disease—and the cancer is being detected at earlier stages. According to a research letter in the journal, 33.7% of people in this age group were up to date with their colorectal cancer screening in 2023—a jump from 20.8% in 2019. In the study, researchers tracked various screening methods, including colonoscopy and at-home stool-based tests, such as fecal occult blood testing and fecal immunochemical testing. In a separate research letter, researchers also noted a steep increase in detecting early-stage colorectal cancer in this age group between 2019 and 2022. “It’s thrilling to see this,” Rebecca Siegel, an American Cancer Society epidemiologist who was involved in one of the studies, told the Wall Street Journal about the increased screening rates. She also noted that earlier detection means “fewer deaths and higher quality of life.” Still, experts told the Journal more outreach is needed, as most people under 45 are not yet routinely screened, despite rising cancer rates in that group. The American Cancer Society lowered its age recommendation for colorectal cancer screening from 50 to 45 in 2018. The U.S. Preventive Services Task Force made that same recommendation in 2021.

Drug Could Reduce Side Effects Associated With CAR T-cell Therapy

The drug itacitinib, taken twice daily, significantly reduced the risk of serious side effects like cytokine release syndrome (CRS) and neurotoxicity in people receiving CAR T-cell therapy for blood cancer, according to a study published in Blood. CAR T-cell therapy uses a patient’s own modified immune cells to target and destroy cancer, but these responses can cause immune-related side effects. Itacitinib, a JAK1 inhibitor, works by blocking signals that drive a key immune response: inflammation. In the study, which included 111 patients, 17% of participants who received itacitinib developed moderate or worse CRS, compared with 57% of those on a placebo, Medscape reported. These findings suggest itacitinib, which is an investigational drug, may help make CAR T-cell therapy safer and more manageable for patients, study authors noted.