Drug-Based Treatments (Clinical Trials)

Last Updated: 09/8/17

NF2 Drug-Based Treatments are experimental and only available through clinical trials.

All Clinical Trials for Neurofibromatosis Type 2 (NF2) tumor management that are now or had been in clinical trials for tumor size reduction can be seen in the list included.

Drug-Based trials that ended can also be seen below the list of current trials, for awareness of what treatments have been discontinued over newer options. Links to each discontinued treatment explains the reason the treatment are no longer considered as a possible option.

Links to existing drug-based treatments include known side effects. Awareness of side effects can help allow life adjustments to continue with treatment for a longer period.

1. NF2 Drug-Based Treatments

Targeted Tumor Therapies

The NF2 gene provides instructions for the production of a protein called MERLIN, also known as Schwannomin. This protein is made in the nervous system, particularly in specialized cells called Schwann cells that wrap around and insulate nerves.

Merlin helps regulate several key signaling pathways that are important for controlling cell shape, cell growth, and the attachment of cells to one another (cell adhesion). This protein functions as a tumor suppressor, preventing cells from growing and dividing too fast or in an uncontrolled way. [NIH, Genetics Home Reference]

Currently, NF2 Drug-Based treatments are only Targeted Tumor Therapies, which have yet to do anything other than a percentage of tumor management for one tumor type at best. A few trials have proven to help some people with Schwannoma, but recent trials started in 2017 are showing promise for Meningioma.

How Clinical Trials Work

While there are advantages to considering participation in clinical trials for treatment, it is important to do everything you can to be aware of dangers for treatment before participating in a medical trial. There are different phases of clinical trials, each phase or step in the approval of a drug or treatment is carefully monitored to determine the effectiveness and possible side effects of each treatment.

Learn more about Clinical Trials

2. Current NF2 Clinical Trials

Each Trial is associated with a page here listing side effects and things that can be done to allow people to stay on each trial longer when possible for maximum treatment, just click the trial name. To read further about a trial at www.clinicalrials.gov, click the trial number.

1. Bevacizumab (Avastin™)

  • Trial Availability: Most Countries
  • Tumor Target: Vestibular Schwannoma (Trial Began: 2009) and Ependymoma (Trial Began: 2016_
  • Treatment by: Intravenous or Port
  • Recruiting Trial: NCT01767792
  • Active, Not Recruiting: NCT01125046
  • Completed: NCT01207687

Avastin™ is the longest running NF2 drug-based clinical trial treatment, 2009. When it works, it has the possibility of affecting Vestibular Schwannoma (VS). The rates tumors might change are different for each tumor in the same individual when it works. Study for review of NF2 Meningioma and Ependymoma have been in early stages, started in 2015.

While Avastin™ side effects are minimal, after time, it can become hard to tolerate, and there is a chance of fast tumor regrowth during even three-month breaks for treatment vacations. Read more on side effect issues and other data collected in the Avastin - NF2 Community Informal Study.


2. Vistusertib (AZD2014)

  • Trial Availability: USA
  • Tumor Target: Recurrent Meningioma (Trial Began: 2016)
  • Treatment by: Pill - Oral
  • Recruiting Trial: NCT02831257
  • Not Open: NCT03071874

3. GSK2256098 (Vismodegib)

  • Trial Availability: USA
  • Tumor Target: Recurrent Meningioma (Trial Began: 2017)
  • Treatment by: Pill - Oral
  • Recruiting Trial: NCT02523014

4. Crizotinib (Xalkori™)

  • Trial Availability: USA
  • Tumor Target: Schwannoma (Trial Began: 2016)
  • Treatment by: Pill - Oral
  • Recruiting Trial: NCT02465060

5. BXCL101 (Bortezomib™)

  • Trial Availability: USA
  • Tumor Target: Schwannoma and Recurrent Meningioma (Trial Will Begin: Later 2017)

6. Endostatin

  • Trial Availability: USA
  • Tumor Target: Vestibular Schwannoma
  • Active, Not Recruiting: NCT02104323

7. Axitinib (Inlyta™)

  • Trial Availability: USA
  • Tumor Target: Vestibular Schwannoma (Trial Began: 2015)
  • Treatment by: Pill - Oral
  • Recruiting Trial: NCT02129647

8. RAD 001 - Everolimus (Afinitor™)


9. OSU-HDAC42 - AR-42

  • Trial Availability: USA - Ohio only
  • Tumor Targets: Meningioma and Schwannoma (Trial Began: 2013)
  • Treatment by: Pill - Oral
  • Recruiting Trial: NCT02282917

10. Lapatinib (Tykerb™)

  • Trial Availability: USA
  • Tumor Targets: Vestibular Schwannoma (Trial Began: 2011)
  • Treatment by: Pill - Oral
  • Completed: NCT01207687 & NCT00973739
  • Unknown: NCT00863122

11. Nilotinib (Tasigna™)

  • Trial Availability: Canada
  • Tumor Target: Intracranial Schwannoma
  • Treatment by: Pill - Oral
  • Terminated: NCT01201538

12. Sorafenib (Nexavar™)

  • Trial Availability: UK
  • Tumor Target: Intracranial Schwannoma
  • Treatment by: Pill - Oral
  • Recruiting Trial: CPMS10563

13. Erlotinib (Tarceva™)

  • Trial Availability: Canada
  • Tumor Target: Intracranial Schwannoma
  • Treatment by: Pill - Oral
  • Trial Status and Availability, Unknown at this Time

14. Imatinib (Gleevec™)

  • Trial Availability: Canada
  • Tumor Target: Intracranial Schwannoma
  • Treatment by: Pill - Oral
  • Trial Status and Availability, Unknown at this Time

15. Sunitinib (Sutent™)

  • Trial Availability: USA
  • Tumor Target: Intracranial Meningioma
  • Treatment by: Pill - Oral
  • Completed: NCT00589784

The Sunitinib clinical trial # NCT00589784, titled "Phase II Trial of Sunitinib (SU011248) in Patients With Recurrent or Inoperable Meningioma" was a trial for people with; CNS Cancer, Meningioma, Intracranial Hemangiopericytoma, Hemangioblastoma, Neurofibromatosis Type 1 as well as Neurofibromatosis Type 2.


16. AZD6244 (Selumetinib™)

  • Trial Availability: UK
  • Tumor Target: Intracranial Schwannoma
  • Treatment by: Pill - Oral
  • Trial Status and Availability, Unknown at this Time

17. Sulforaphane (SFN)

Sulforaphane (SFN) is an Isothiocyanate found in cruciferous vegetables. While this can be obtained by consumption of the right food or vitamin form, other food and herb forms have been in trials and have proven to have potency enough to control tumor size on their own safely.

Potential NF2 Clinical Trials

2. Aspirin

Trials should be available before the end of 2016 and while this seems like the preface; simple and inexpensive answer.

WARNING: Before the trial starts, we know there are issues people using it to prevent hearing loss will have. The first sign of hearing loss is Tinnitus, and a known long accepted side effect of Aspirin includes Tinnitus. Exact dose by age per individual for many reasons will need to be determined since Aspirin is also a blood thinner and can cause additional issues. A warning statement is in place because even if approved it would likely require a regular blood test and monitoring for whole body health.

6. Cabozantinib (Cabometyx™)

  • Trial Availability: USA
  • Tumor Target: NF1: Plexiform Neurofibroma (Trial Began: ?) and NF2: Vestibular Schwannoma given same time as Avastin™ (Trial Began: 2017)
  • Treatment by: Pill - Oral
  • Inhibitor: VEGF receptor

Cabometyx™ has not been approved as an NF2 tumor treatment trial on its own, but some hospitals are using it in combination with Avastin™ as an enhancer to improve the the potential percentage of tumor reduction. On its own Cabometyx™ is on trial for Neurofibromatosis Type 2 (NF1) - Plexiform Neurofibromas: - NCT02101736.

3. FRAX597

4. Ado-Trastuzumab Emtansine
(Kadcyla™)

5. Cetuximab (Erbitux™)

6. Sirolimus (Fermentek™)

7. BEZ-235 aka NVP-BEZ235

8. Dasatinib (Sprycel™)

9. Vandetanib (Zactima™
and Caprelsa™)

10. Somatostatin

Source

  1. National Institute of Health. Genetics Home Reference. "NF2 gene" (January, 2017) https://ghr.nlm.nih.gov/gene/NF2#normalfunction
  2. American Cancer Society, Inc. https://www.cancer.org/treatment/treatments-and-side-effects.html