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Drug-Based Treatments (Clinical Trials)

Last Updated: 09/27/18

1. NF2 Tumor Drug-Based Treatments
  • Genetics and NF2 Treatments
  • Targeted Tumor Therapies
  • How Clinical Trials Work
2. Current NF2 Clinical Trials 3. NF2 Pre-Clinical Trials
Completion of Phase 0 Animal Trials
  1. Mifepristone (RU-482)
  2. Crizotinib (Xalkori™) - USA and Canada
  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. Aspirin- USA
  15. BXCL101 (Bortezomib™)- USA
4. Solid Tumor Pre-Clinical Trials
  1. RO4929097 - Canada
  2. Cellular Prion Protein (PrPC)
4. Determined Unlikely for NF2
  1. Nilotinib (Tasigna™) - Canada
    - Canceled: Trial Death
  2. Dasatinib (Sprycel™)
  3. CD47
  4. Pasireotide - SOM 230
  5. PTC 299
  6. OSU-03012 - AR-12
  7. Hydroxycarbamide (Hydrea™)
5. Also See:

There is no cure to Neurofibromatosis type 2 (NF2). There is no treatment that; stops tumor growth, prevents the growth of new tumors, reduces the size of all tumors, destroys all tumor types, works on everyone with NF2. Research in; 1) oncology medication (molecular targeted tumor therapies/chemotherapy), 2) natural (ayurvedic/botanical), or 3) otherwise, have found one treatment that has proven to help everyone.

NF2 tumor drug-based treatments are experimental and only available through clinical trials.

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

  • tumor size reduction
  • stabilize tumor size
  • no response to tumors at all
  • side effects not worth continuation of the treatment
  • reduction or stabilization of tumors enough for a better treatment later

Research is needed!!!

The list of drug-based tumor treatments here includes a breakdown or treatments noted as a trial option both new, to avoid the possible use of discontinued treatment, over options that are more likely to have better results.

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

Genetics and NF2 Treatments

Why might a treatment work for some people with NF2, but not all people with NF2?

"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]

Molecular Targeted Tumor Therapies

Currently, NF2 drug-based tumor 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

1. Bevacizumab (Avastin™)

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™)

2. Aspirin

Trials should be available before the end of 2019, and while this seems like the preface; simple and inexpensive answer, it is important to read side effects of short-term and long-term use.

WARNING: The first sign of hearing loss is tinnitus, and a known long accepted side effect of aspirin includes tinnitus and blood thinner.

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) https://ghr.nlm.nih.gov/gene/NF2#normalfunction
  2. American Cancer Society, Inc. "Treatments and Side Effects" https://www.cancer.org/treatment/treatments-and-side-effects.html
  3. 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
  4. 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). .
  5. The Government of Canada. "Clinical Trial Search." https://health-products.canada.ca/ctdb-bdec/index-eng.jsp
  6. National Institute of Health and Research. (NHS) "UK Trials The Gateway." https://www.ukctg.nihr.ac.uk/
  7. USA Clinical Trial Database. U.S. National Library of Medicine. "Clinical Trials." https://clinicaltrials.gov/