Home > Treatment Options > Tumor Suppressors >

Drug-Based Treatments (Clinical Trials)

Last Updated: 07/18/22

NF2 Clinical Trials

Vestibular Schwannoma or
other Schwannoma
  1. Bevacizumab (Avastin™)
    and biosimilar (MVASI™)

    - Available in Many Countries
  2. Brigatinib (Alunbrig™)
    - USA (recent 2020)
  3. RAD 001 - Everolimus (Afinitor™)
    - Many Countries
  4. Lapatinib (Tykerb™) - USA
  5. Crizotinib (Xalkori™)
    - USA and Canada
  6. Sorafenib (Nexavar™) - Canada
  7. Erlotinib (Tarceva™)
    - Canada
  8. Imatinib (Gleevec™ Mesylate)
    - USA & Canada
  9. Sunitinib (Sutent™) - USA & Canada
  10. AZD6244 (Selumetinib) - USA
  11. Axitinib (Inlyta™) - USA
  12. rec-2282 (AR-42 OSU-HDAC42) - USA - Ohio
    Status: Unknown
  13. Icotinib - China
  14. Gentimicins - Sweden
  1. AZD2014 (Vistusertib™) - USA
  2. GSK2256098 - USA
  3. rec-2282 (AR-42 OSU-HDAC42)- USA - Ohio
    Status: Unknown
  4. Brigatinib - USA

NF2 Pre-Clinical Trials

Completion of Phase 0 Animal Trials
  1. AAV-P0-ASC
  2. Mifepristone (RU-486)
  3. Sulforaphane (SFN)
  4. Pembrolizumab (Keytruda™)
  5. Cabozantinib (Cabometyx™)
  6. FRAX597
  7. Ado-Trastuzumab Emtansine
  8. Cetuximab (Erbitux™)
  9. BEZ-235 (NVP-BEZ235)
  10. Vandetanib (Zactima™
    and Caprelsa™)
  11. Somatostatin
  12. 68Ga-NODAGA-E[c(RGDyK)]2
    - Denmark
  13. Endostatin (Endostar™)- US
  14. Optune

4. Solid Tumor Pre-Clinical Trials

  1. Brigatinib - USA
  2. RO4929097 - Canada
  3. Cellular Prion Protein (PrPC)

5. Poor Responses in NF2 Trials

  1. Aspirin- USA
  2. Nilotinib (Tasigna™) - Canada
    - Canceled: Trial Death
  3. Dasatinib (Sprycel™)
  4. CD47
  5. Pasireotide - SOM 230
  6. PTC 299
  7. Hydroxycarbamide (Hydrea™)

Schwannomatosis (SWN)

Also See:

NF1 Tumor Drug-Based Trials

Plexiform Neurofibromas, and Optic Nerve Glioma
  1. Koselugo (Selumetinib)
    - FDA approved USA (2020)
    - approved Europe (2021)
  2. Bevacizumab (Avastin™)
  3. NFX-179
  4. Clinical Trial NCT04435665
    "NF: Molecular Pathogenesis and Natural Compounds
    as Potential Treatments"
  5. RAD 001 - Everolimus (Afinitor™)
  6. Esbriet (Pirfenidone)
  7. Sirolimus
  8. Gleevec
  9. Therapeutics' NFX-179 Gel NCT04435665
  10. Trametinib (Mekinist) Phase 1
    Tumor type undetermined trial for
    Glioma and Plexiform Neurofibroma Trial
  11. Device: (electric field therapy)
    Optune Glioblastoma Malignant GBM (DIPG)

Important Facts

  1. Many medications/tumor drug-treatments have been in trials for tumor management for neurofibromatosis type 2 (NF2) tumors.
  2. Changes in tumors 2: Medications to manage NF2 tumors is up to, but rarely reduces tumor size more than twenty-percent (20%).
  3. Delay Advantage: While tumor drug-treatments only reduce tumor size, this can help delay damage tumors can do until better treatments are available and will lower risk of tumor growth changing from non-cancerous to cancerous.


There is no cure to neurofibromatosis type 2 (NF2).

There are limits to tumor-drug treatments for tumors that grow as a result of the NF2 condition.

Except for Bevacizumab (Avastin™), NF2 tumor-drug treatments are experimental and in different phases of clinical trials.

Everyone responds differently to each treatment in some way to each treatment option, but each might result in:

  • no response to tumors at all
  • stabilize tumor size (no growth)
  • tumor size reduction with continued use of treatment
  • side effects complications not worth continuation of the treatment

Research is needed!!!

The list of tumor drug treatments here includes a breakdown of treatments in different phases of trials, and a separate listing of discontinued treatments which have proven ineffective.

Links to existing drug-based treatments include known side effects are noted when after treatment has been in trial long enough for information, therefore not all treatments include a list of possible side effects. Awareness of side effects can help allow life adjustments to continue with treatment for a longer period.

1. NF2 Drug-Based Treatments

Bevacizumab (Avastin™) and biosimilar (MVASI™)

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 of 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.

Learn more about Avastin™ and possible replacement biosimilar treatment Bevacizumab-aww (MVASI™)


The conclusion of the study done for management of indicates was on Aspirin is not helpful enough for management of vestibular schwannoma (VS) tumors.[9, 10]

Given the risk of the side effects, individuals looking for control of tumors should consider other options.

Side effects of Aspirin include; rash, gastrointestinal ulcerations, abdominal pain, upset stomach, heartburn, drowsiness, headache, cramping, nausea, gastritis, and bleeding.[11]

Potential NF2 Clinical Trials

Cruciferous Vegetables: Sulforaphane (SFN)

Sulforaphane (SFN) is an isothiocyanate found in cruciferous vegetables.

SFN is available in both food and vitamin form. Research is necessary to determine if; 1) a specific brand is ideal, 2) if a pharmacy needs to make a better compound, 3) what other than SFN might be necessary for the results seen in early trials, 4) how to determine the correct dose (height, age, weight), and tests necessary for safety.


  1. National Institute of Health. Genetics Home Reference. "NF2 gene" (January, 2017)
  2. American Cancer Society, Inc. "Treatments and Side Effects"
  3. "Types of Cancer (tumor) Treatment" National Cencer Institute (Updated: April 6, 2017 | Last Reviewed: October 2018)
  4. Muhammad Yar, Lubna Shahzadi, Azra Mehmood, et.al. Massachusetts General Hospital. "Deoxy-sugar releasing biodegradable hydrogels promote angiogenesis and stimulate wound healing." Materials Today Communications, (2017); 13: 295 DOI: 10.1016/j.mtcomm.2017.10.015
  5. Massachusetts General Hospital. "Manganese-based MRI contrast agent may be safer alternative to gadolinium-based agents: Imaging compound developed by Mass. General team produces comparable image enhancement to the standard of care with rapid clearance, less likelihood of toxicity." ScienceDaily. ScienceDaily, (15 November 2017).
  6. The Government of Canada. "Clinical Trial Search." https://health-products.canada.ca/ctdb-bdec/index-eng.jsp
  7. National Institute of Health and Research. (NHS) "UK Trials The Gateway."
  8. USA Clinical Trial Database. U.S. National Library of Medicine. "Clinical Trials."
  9. "COX2 expression is associated with proliferation and tumor extension in vestibular schwannoma but is not influenced by acetylsalicylic acid intake." Acta Neuropathologica Communicationsvolume 7, Article number: 105 (2019)
  10. MacKeith S, Wasson J, Baker C et al (2018) "Aspirin does not prevent growth of vestibular schwannomas: a case-control study." Laryngoscope 27(S 02):547.
  11. "Aspirin." WebMd. (Date Reviewed: 2018)
    Source: https://www.webmd.com/drugs/2/drug-1082-3/aspirin-oral/aspirin-oral/details

    Source Importance: Possible Side Effects.

What is NF2? | About Us | Treatment Options | Sitemap

Site Search

Disclaimer: Neurofibromatosis Type 2 - Information and Services, www.nf2is.org, is not run by medical experts, affiliated with any healthcare organization or any other company. No assurance can be made to the accuracy or completeness of the information provided here, the accuracy of other sites to which this site links, or of sites that link to this site. - Read More

Copyright © 2021