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NF2 Clinical Trial Overview

By February 28, 2017December 18th, 2023NF2-SWN, Science & Research

By Salvatore La Rosa, PhD, Children’s Tumor Foundation VP, Research and Development

These days there is a lot of excitement and hope for the MEK clinical trial for inoperable NF1 plexiform tumors (click here to read about the MEK trial). Over 70% of patients had a dramatic response and saw their tumors shrink 20% or more. This is unprecedented and really makes us hope that this drug will soon be approved by the FDA as the first ever treatment for NF1. But what about NF2? What is the status in the clinic for this condition? Well, I would like to focus on four drug trials that are ongoing and that promise to bring great news for our patients in the short term.

The first drug I would like to cover is Avastin (Bevacizumab). This is now under investigation in children and young adults with NF2. This drug has already been evaluated for safety and tolerability and the current study will evaluate its effect on hearing.  Click here to read about two patients currently in the trial.

AZD-2014, a molecule belonging to the class of mTOR inhibitors and developed by AstraZeneca, is in a Phase 2 clinical study for growing or symptomatic NF2 meningiomas. In this case, the primary outcome measure is radiographic response as measured by volumetric MRI scans.

Also for meningioma, a drug called Axitinib, which is already approved for the treatment of other types of tumors, is currently being investigated to see if it can shrink vestibular schwannomas, the hallmark of NF2. This drug works by the same mechanism as Bevacizumab but has different characteristics and can be taken by mouth, instead of intravenous administration as needed for Bevacizumab. These differences may prove Axitinib more practical, but still it needs to prove its efficacy.

The last trial I want to cover is a relatively new one, activated last year in Beijing, China.  The drug used is Icotinib and it belongs to the class of EGFR inhibitors. EGFR receptors are responsible for the growth of tumors and are well known in the field of NF2 research. This trial will evaluate tumor volume by MRI and will also assess hearing ability and quality of life in patients affected by NF2 tumors. Though this trial is not accessible to everyone as they are only enrolling locally in the Beijing area, it will shed light on this agent and its utility for NF2.

So, even though Selumetinib and the MEK trial for NF1 is making headlines in NF these days, there is also a lot going on for NF2. The challenge for NF2 lies in its numbers as it is rarer than NF1 (1/25,000 compared to 1/3,000 for NF1) and recruitment of patients and completion of trials are often daunting tasks. This is true for NF as for all rare diseases, which is one reason why the Children’s Tumor Foundation started an online patient registry to help fill clinical trials (visit to find out more).

This is also why the Children’s Tumor Foundation launched Synodos for NF2, multi-year initiative that has assembled a team of researchers from twelve world-class labs and medical centers. This initiative promises to speed up the drug discovery process (click here to read more.)

We will get to the finish line thanks to the determination of our community, which never gives up, and possibly also thanks to one of these current investigational treatments that could prove to be useful to help combat NF2. In two to four years, when these trials conclude, we will have more information and hopefully good news. In addition, what keeps our hopes high is that there is a pipeline of other drugs emerging from new studies showing novel and very promising results for NF2.