The 2010 NF conference kicked off with a Keynote Presentation from Dr. Susan Lindquist on Heat Shock Factor proteins as candidate signaling targets for NF1 therapeutic intervention, then transitioned to an afternoon of updates on ongoing NF clinical trials chaired by Dr. Kathryn North (University of Sydney) and Dr. Roger Packer (Children’s National Medical Center). Keeping in the 2010 NF Conference theme of ‘Back to the Future’, Dr. Brigitte Widemann (National Cancer Institute) opened with a review of ‘lessons learned’ from NF1 clinical trials and the challenges ahead. Dr. Widemann is widely recognized as a world expert on NF1 tumor trials. Through planning and running a number of NF1 trials to date, she noted that NF1 clinicians now have a solid understanding of how to conduct Phase I (safety) trials and are advancing into therapeutic (Phase II) trials. Front and center in plexiform neurofibroma trials is to monitor tumor volume and ‘burden’; however getting industry to understand this can be challenging, as testing drugs in these tumors will mean long-term trials, something industry may not be familiar with (being more used to short-term cancer trials). Three dimensional monitoring of NF tumors is critical to make trials as rapid and accurate as possible. Other issues addressed by Dr. Widemann included the importance of the clinical community to focus on how to make clinical trials sufficiently safe so as to include the very youngest patients, those under three years old who may benefit significantly from inclusion in trials. Specific ongoing NF1 trials presented included Dr. Dusica Babovic (Mayo Clinic) – Phase II of cediranib; Dr. Michael Fisher (Children’s Hospital of Philadelphia) – Phase II of sirolimus (through the CDMRP consortium); and Dr. Aerang Kim (NCI) – pediatric Phase I of sorafenib – all trials in testing in NF1 plexiform neurofibromas. These are all ongoing with safety data and efficacy being assessed; though as yet no drug looks especially promising the investigators are learning how to do NF1 trials as safely and efficiently as possible. Dr. Maria Acosta (Children’s National Medical Center) presented a promising update on the Phase II trial of Lovastatin for NF1 learning disabilities, a drug that seems to be both safe and at least to some extent effective. Dr. Jay Gibbs (AstraZeneca) reviewed some of the drug pathways that may be of interest in NF therapy including the JAK/STAT pathway which is of increasing interest, and also gave an overview of the drug pipeline and activities of the CTF NF Preclinical Consortium which include currently testing of drugs targeted at PI3K/mTOR, erbB receptors and additional targets in partnership with companies including Novartis, Genentech, Avila Therapeutics. Dr. Scott Plotkin (Harvard Medical School) and Dr. Ambereen Kurwa (NCI) presented data on the use of imaging for three dimensional tumor volume assessment and whole body tumor burden assessment and in Dr. Kurwa’s study even how to use this imaging to sub-classify plexiform neurofibromas. As noted by Dr. Widemann earlier in the afternoon, these technologies are emerging as an invaluable part of NF clinical care and trials. The first patient representative to speak at the 2010 NF Conference, Tracy Galloway shared her family’s experience of her teenage daughter’s diagnosis with NF2, and how she has responded with such determination to accelerate research in any way she can. Communication and sharing between researchers, she noted, is absolutely critical. Dr. Brad Welling (Ohio State University) provided a terrific overview for the group of the recent NF2 meetings in Las Vegas (which we covered extensively in the May blogs). Further NF2 trial reports will come later in the NF Conference, so stay tuned!
2010 NF Conference: Day 1
