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Foundation Funded Research Shows Future Promise as NF1 Malignant Tumor Treatment

By May 11, 2010December 18th, 2023Awareness, Science & Research

‘Tall oaks from little acorns grow’ is an adage that is appropriate for the Children’s Tumor Foundation Drug Discovery Initiative. This program offers modest grants to investigators to test candidate NF drug treatments. To date the program has funded over 30 projects and the first 20 – a cost to CTF of $500,000 – have already garnered $3.5 million in follow-on funds from NIH, DOD and other sources, as well as establishing multiple relationships with the biotech and pharma sector and publishing results of the research.

One such project published this month* describes the development and testing of Schweinfurthin A, a drug originally purified from a natural plant-derived product, as a treatment for NF1-related malignancies glioblastoma and astrocytoma.  CTF has partially funded this research through two DDI Awards to Dr. David Wiemer (University of Iowa) to optimize Schweinfurthin’s chemical function, and the drug was subsequently tested on human NF1 tumor-derived cells models in the laboratory of Dr. Karlyne Reilly at the National Cancer Institute.  The drug halts the growth of human tumor cells but not healthy cells derived from mice. The drug appears to act via blocking signaling target Rho. These findings suggest Schweinfurthin could have a tumor-specific mode of action- an exciting possibility for future development of drugs effective against NF1 malignancies.

* Turbyville et al. (2010. Schweinfurthin A Selectively Inhibits Proliferation and Rho Signaling in Glioma and Neurofibromatosis Type 1 Tumor Cells in a NF1-GRD–Dependent Manner.  Mol Cancer Ther; 9(5); 1234–43.