Note: apologies for not posting these Day 2 blogs earlier. Meetings were back to back today! An indicator of the huge amount of information exchange.
This afternoon focused on ‘Future Frontiers of NF2 Management’. The first three presentations focused on an NF2 and schwannomatosis genetics update (Ludwine Messiaen), an overview of England’s new NF2 national clinical management system (Sue Huson) and utility of three-dimensional imaging in NF2 management (Gordon Harris). Moving on to candidate therapeutics, I gave a presentation on behalf of CTF offering some thoughts on issues the NF2 community should now be focused on as trials advance, including engaging industry, working with the FDA, and building links between preclinical researchers and clinical trialists. Marco Giovannini then provided a summary of his ongoing NF2 preclinical drug screening in the NF Preclinical Consortium and drug targets of interest including erbB, PI3 kinase and Akt.
Harry Miao of PTC Therapeutics provided an overview of the ongoing Phase II clinical trial of PTC-299 in vestibular schwannoma. This trial is underway and has some positive but very preliminary indications of tumor shrinkage (notably vascular shrinkage). Scott Plotkin reviewed the status of the bevacizumab (Avastin) trial. This has doubled from the original 10 patients published last summer, and results continue to look promising with tumor shrinkage and some regained hearing, though responding tumors do re-grow when drug is stopped. In an exciting development, looking ahead, later in 2010 a second trial is set to commence in to give bevacizumab to children with NF2 who are 12 years and older.
‘NF2: State of the Art’ closed with a terrific sense of promise and a commitment from the organizers to convene again in 2011. This evening we convened a smaller group for ‘NF2: State of the Trial’ to continue through tomorrow. This expert group will provide recommendations for programs to further accelerate NF2 trials. Higlights from this meeting will follow in a blog in a few days!
