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Clinical Trial Advances and New Mouse Models: NF Conference Concludes

By June 14, 2011December 5th, 2023Awareness, NF Conference

The 2011 NF Conference concluded today with a packed agenda of exciting talks, kicking off with some clinical trial updates.Kent Robertson (Indiana University) reported on a trial of Gleevec (Imatinib) in NF1 patients with clinically significant plexiform neurofibromas, age 3-65.  Individuals received drug for 6 months, and if they received benefit they were given the opportunity to stay on the drug.  60% of patients showed a response in one or more of their plexiforms, and 40% of those patients showed an improvement in ther symptoms such as issues with bladder control and airway function.  Dr. Dusica Babovic-Vuskanovic (Mayo Clinic)reported on the concluding Phase II trial assessing the response of NF1 plexiform neurofibromas to Cediranib (AZD2171), a VEGF-targeted agent. 26 patients, 18 years and older participated in the trial and received drug for periods of 18-41 months. 4 patients showed continuous and sustained tumor shrinkage. In addition the trial assessed changes in pain via a quality of life survey, and found that pain levels decreased over the period of drug treatment.  Kimberly Jett (University of Alberta) reported on her study showing that NF1 patients can have reduced levels of Vitamin D in the serum, and that treatment with Vitamin D can increase bone mineral density in these individuals, potentially helping to allay future bone problems.

Sarah Burns (Ohio State University)described research using the PI3K/AKT targeted drugs AR42 and AR12. These drugs are in development at Arno Therapeutics and in clinical trials for a number of cancers. Previous CTF-funded preclinical research by this group has shown that these drugs are candidate therapies for NF2 vestibular schwannomas.   This presentation reported potential efficacy of these drugs in halting or slowing the growth of benign meningiomas.  David Ingram (Indiana University) is focused on the area of vascular disease in NF1. Persons with NF1 are at increased risk of vascular disease. Dr. Ingram has developed mouse models that develop the features of vascular disease, such as infiltration of macrophages. These mice will be valuable study models for understanding the biology and testing drug interventions. Looking ahead, the goal is to be able to identify early subclinical vascular disease so it can be treated before it progresses.

112 posters were presented at this year’s NF Conference, and these were judged by small committees of researchers and clinicians to determine Best Poster in Basic Research and Clinical Research categories.  Consideration was given to merit, originality and depth of data. The winners were: Best Poster – Basic Research: Juliana Ferreira de Souza(Federal University of Mina Gerais, Brazil) – “Aerobic Capacity Is Reduced In Patients With NF1: A Preliminary Report”; and Best Poster-Clinical Research:  Melissa Hinman(Case Western Reserve University) – “The Biological Role of the Regulation of NF1 Exon 23a alternative splicing”.