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NF Clinical Trials: Emerging Options

The 2009 NF Conference got off to an exciting start Saturday with a full half day of presentations on ongoing or planned NF clinical trials. Roger Packer opened with an update on the Phase II NF Clinical Trials Consortium. The group has already fulfilled enrolment on the first NF1 trial - of rapamycin in plexiform tumors - and recently opened a trial to assess Lovastatin in NF1 learning disabilities. 

Next Consortium trials may include RAD001 (mTOR inhibitor) for optic pathway glioma as well as a bone trial. Scott Plotkin presented data from a new pilot trial to assess the blood vessel-targeted drug ranibizumab in NF1 dermal neurofibromas. Drug is injected directly into individual tumors and tumor size will be monitored. This trial is still in its earliest stages. Dr. Brigitte Widemann reported that by the end of 2009 another new NF1 trial will commence that combines two drugs - RAD001 and bevacizumab - for treatment of malignant peripheral nerve sheath tumors that have been resistant to other treatment. This combined therapy approach is, as I reported from ASCO, now being used in many tumor therapies.  

 In NF2 trials, Jaishri Blakeley reported on the Children's Tumor Foundation-funded Phase Zero trial to test lapatinib (kinase inhibitor) in vestibular schwannomas. This trial will be ready to enroll the first patient in July. Harry Miao reported on the new trial of PTC-299 (targeting blood vessel growth) in vestibular schwannomas also to commence soon.  

It is tremendous to see these clinical trial options opening up. Looking ahead, the Children's Tumor Foundation will endeavor to provide comprehensive information on all ongoing trials via our website to keep the community abreast of opportunities to participate.  

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