Over half of the children with high-risk neuroblastoma experience late relapses caused by minimal residual disease. Since chimeric antigen receptor (CAR) T-cell therapy has shown efficacy against minimal residual disease in pediatric patients with hematologic malignancies, several CAR T-cell therapies are being investigated for neuroblastoma.
While most preclinical research on pediatric cancer is carried out using adult mouse models, there are significant biological differences between children and adults, including age-related differences in the immune system, metabolism and growth factor signaling. In the current study, investigators from the University of Western Australia and Telethon Kids Institute developed a new mouse model of pediatric medulloblastoma, which is the most common childhood brain cancer.
Infantile hemangioma (IH) is the most common benign vascular tumor seen in childhood. After an early proliferative phase in the first weeks of life, the majority of hemangiomas complete their growth and start to involute by approximately 12 months.
A new epigenetic editing technique has restored the functionality of neurons in Rett syndrome (RTT), a rare neurodevelopmental disease associated with a mutation of the MECP2 gene on the X chromosome. The origin of this disorder is a heterozygous mutation of the MECP2 (methyl CpG-binding protein 2) gene. Homozygosity is lethal and as it is associated with the X chromosome; Rett mostly affects girls, inhibiting the development of their motor skills and communication.
By combining drug sensitivity with genomic profiling of tumor cells, a study from St. Jude Children's Research Hospital with more than 800 patients has shown a wide diversity in drug sensitivity for pediatric acute lymphoblastic leukemia (ALL) and defined six patterns of response to treatment. “This work provides a framework for ‘functional precision medicine’,” corresponding author Jun Yang, vice chair of the Department of Pharmacy and Pharmaceutical Sciences at St. Jude Children's Research Hospital, told BioWorld.
The FDA has awarded rare pediatric disease designation to Orphagen Pharmaceuticals Inc.'s OR-449 for the treatment of pediatric adrenocortical carcinoma (ACC). OR-449 is a selective, first-in-class, potent and orally bioavailable small-molecule antagonist to steroidogenic factor-1 (SF-1; NR5A1), an orphan nuclear receptor and transcription factor that is essential for the growth and development of the adrenal gland.
As expected, the U.S. FDA gave its go-ahead to lecanemab, an amyloid-beta binder for mild cognitive impairment caused by Alzheimer’s disease (AD) and mild AD from Biogen Inc. and Eisai Co. Ltd, which have assigned to the compound the brand name Leqembi.
As expected, the U.S. FDA gave its go-ahead to lecanemab, an amyloid-beta binder for mild cognitive impairment caused by Alzheimer’s disease (AD) and mild AD – together known as early AD – from Biogen Inc. and Eisai Co. Ltd, which have assigned to the compound the brand name Leqembi.
Rhabdomyosarcoma (RMS) occurs in 3% of all pediatric cancers. Aberrant Hedgehog (Hh) activation can be ligand-dependent or -independent, but current clinical Hh inhibitors targeting SMO are effective only for ligand-independent tumors. Researchers at Vall d’Hebron Institut de Recerca, Barcelona, Spain, developed a targeted therapeutic for Hh ligand-dependent tumors.
Researchers from Astellas Pharma Inc. presented preclinical data for the novel 5-HT5A receptor antagonist, ASP-5736, being developed for the treatment of fragile X syndrome (FXS).
RSS
