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NF Bites: Progress in NF2 – Part Two – Developing Drug Therapies

By April 16, 2010December 18th, 2023Awareness, Science & Research

Welcome to the seventh in a series of “NF Bites” – providing snapshots of individual areas of neurofibromatosis research and how the Children’s Tumor Foundation is advancing this. Over the coming days and weeks we will focus on different aspects of neurofibromatosis research. Today: in Part 2 on where are we with research progress in NF2, we focus on advances in preclinical drug testing and clinical trials.

NF2 research activity, especially in the area of testing candidate drug treatments has increased significantly in the past several years. This has been driven in part by our publication in 2009 of consensus documents on NF2 clinical trials and drug pipeline in Clinical Cancer Research, based on an expert workshop convened by the Foundation. We currently fund a significant amount of NF2 research ‘from bench to bedside’ – an indicator of the exciting progress made in NF2 recently in understanding the underlying biology and then applying this to finding effective drug therapies. Importantly CTF is hosting an expert workshop in May 2010 to evaluate progress to date in NF2 clinical trials and how these can be further accelerated.

·         The Foundation was a co-supporter of the groundbreaking pilot Phase II trial of Bevacizumab (Avastin) which caused vestibular tumor shrinkage, restored hearing and was published in New England Journal of Medicine in July 2009.

·         One of our first Clinical Trial Award recipients in 2009 was Jaishri Blakeley (Johns Hopkins University), receiving $125,000 to do a Phase Zero NF2 clinical trial of the drug Lapatinib, currently in progress.

·         Outside of CTF funding, a Phase II NF2 trial of note is underway at Massachusetts General Hospital to assess drug PTC-299 in patients. This trial is co-supported by CDMRP NFRP and PTC Therapeutics.

·         Through the Foundation’s NF Preclinical Consortium, a $4M multi-year initiative to accelerate promising candidate drugs to the clinic, the laboratories of Andrea McClatchey (Harvard/ Massachusetts General Hospital) and Marco Giovannini (House Ear Institute)are testing a pipeline of NF2 drugs, in collaboration with Novartis, Genentech and Avila Therapeutics.

·         In one of our first Drug Discovery Initiative Awards, in 2006, Oliver Hanemann (University of Plymouth) received $11,000 to test the drug Sorafenib on human NF2 schwannoma cells in culture. Positive data emerging from this study has led to planning of a Phase II European clinical trial.

Look for the final instalments of NF Bites next week!