This week an article was published on consensus recommendations on planning and conducting NF2 clinical trials; the result of a Children’s Tumor Foundation-driven initiative to accelerate progress in the identification of effective drug therapies for NF2. The genesis of this paper was a lunchtime brainstorming session at the 2007 NF Conference followed by a Foundation-convened two-day landmark NF2 Workshop held in New York in October 2007. The paper was published in the August 15th issue of Clinical Cancer Research (http://bit.ly/Cwox9) and is authored by an array of leading international NF2 experts headed by Dr. Marco Giovannini (House Ear Institute) and Dr. Gareth Evans (University of Manchester).
Some remarkable progress has already resulted from this initiative with three of the candidate NF2 drugs prioritized in the paper now in clinical testing. Bevacizumab NF2 Phase II pilot trial results were reported by Scott Plotkin et al (MGH) in the New England Journal of Medicine this summer. Lapatinib is being tested in a Children’s Tumor Foundation-funded Phase Zero clinical trial (headed by Jaishri Blakeley (Johns Hopkins) and Matthias Karajannis (NYU)). Finally Phase II NF2 trials are now underway, also under direction of Dr. Plotkin at MGH, to test the PTC Therapeutics drug PTC299.