Treated mice with benign neurofibromas had significant shrinkage of their tumors after 60 days, compared to untreated mice. In mice implanted with human MPNSTs, the drug diminished cancerous tumor growth and doubled survival time to 52 days, as compared to untreated animals.
Researchers said overall shrinkage of MPNSTs was modest compared to that achieved in benign tumors, possibly because additional molecular pathways are involved in their formation. They said this suggests that MPNSTs may require a combinatory treatment that includes PD0325901 in conjunction with other therapies.
Mutations of the NF1 gene are frequently found in other cancers, such as glioblastoma, lung adenocarcinoma and ovarian cancer. The researchers said this could open the possibility of studying the Ras-MEK-ERK pathway as molecular target for treating these diseases.
Funding for the study came from: the DAMD Program on Neurofibromatosis for the NF1 Microarray Consortium (DODW81XWH- 09-1-0135 and W81XWH-04-1-0273); the Children's Tumor Foundation for support to the NF1 Preclinical Consortium; a National Institutes of Health Bench to Bedside Award for MRI Analyses (NIH-P50-NS05753) and an American Recovery and Reinvestment Act supplement to NIH grant R01-NS28840. The drug was provided by the manufacturer, Pfizer Inc.About Cincinnati Children'sCincinnati Children's Hospital Medical Center ranks third in the nation among all Honor Roll hospitals in U.S. News and World Report's 2012 Best Children's Hospitals ranking. It is ranked #1 for neonatology and in the top 10 for all pediatric specialties. Cincinnati Children's is one of the top two recipients of pediatric research grants from the National Institutes of Health and a research affiliate of the University of Cincinnati College of Medicine. It is internationally recognized for improving child health and transforming delivery of care through fully integrated, globally recognized research, education and innovation. Additional information can be found at www.cincinnatichildrens.org. SOURCE Cincinnati Children's Hospital Medical Center