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Drug-Based Treatments (Clinical Trials)

Last Updated: 07/17/19


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-486)
  2. Sulforaphane (SFN)
  3. Pembrolizumab (Keytruda™)
  4. Cabozantinib (Cabometyx™)
  5. FRAX597
  6. Ado-Trastuzumab Emtansine
  7. Cetuximab (Erbitux™)
  8. BEZ-235 (NVP-BEZ235)
  9. Vandetanib (Zactima™
    and Caprelsa™)
  10. Somatostatin
  11. 68Ga-NODAGA-E[c(RGDyK)]2
    - Denmark
  12. Endostatin (Endostar™)- US
4. Solid Tumor Pre-Clinical Trials
  1. Brigatinib - USA
  2. RO4929097 - Canada
  3. Cellular Prion Protein (PrPC)
5. Poor Responses in NF2 Drug Trials
  1. Aspirin- USA
  2. Nilotinib (Tasigna™) - Canada
    - Canceled: Trial Death
  3. Dasatinib (Sprycel™)
  4. CD47
  5. Pasireotide - SOM 230
  6. PTC 299
  7. OSU-03012 - AR-12
  8. Hydroxycarbamide (Hydrea™)
Tumor-drug Treatment Definitions

Also See:

NF1 Tumor Drug-Based Treatments
  1. Bevacizumab (Avastin™)
  2. Selumetinib
  3. RAD 001 - Everolimus (Afinitor™)
  4. Esbriet (Pirfenidone)
  5. Sirolimus
  6. Gleevec

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 1: Tumor drug-Treatments for NF2 tumors are capable of attacking only one type of tumor either; 1) schwannoma, 2) meningioma, or 3) ependymoma.
  3. Changes in tumors 2: Medications to manage NF2 tumors is up to, but never over twenty-percent (20%) of tumor sizes.
  4. Delay: While tumor drug-treatments only reduce tumor size, this can help delay damage tumors can do until better treatments are available.
  5. Drug class: NF2 tumor drug-treatments are not chemotherapy, treatments are either molecularly targeted therapy or immunotherapy.
  6. Discuss with doctors: Choice of tumor drug-treatment should be made based on the immediate need for an idividuals most immediate need of tumor management. Currently, no drug treatment can attack all three tumor types.


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

There are limits to tumor-drug treatments. In efforts to find a better treatments research is ongoing in:

  1. oncology medication (molecular targeted tumor therapies and immunotherapy)
  2. over the counter medication
  3. natural food, and supplements (ayurvedic/botanical)

NF2 tumor drug-based 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 not worth continuation of the treatment
  • reduction or stabilization of tumors enough for a better treatment later

Research is needed!!!

The list of 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 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

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.

Important Tumor-drug Treatment Definitions

Definition List

  1. Angiogenesis
  2. Angiogenesis Inhibitor/Antiangiogenesis Agent
  3. Immunotherapy
  4. Molecularly Targeted Therapy
  5. Chemotherapy
  6. Receptor
  7. Cancer/Tumor Condition


  1. Angiogenesis: "the development of new blood vessels. Blood vessel formation. Tumor angiogenesis is the growth of new blood vessels that tumors need to grow. This process is caused by the release of chemicals by the tumor and by host cells near the tumor." [ Cancer.gov "Angiogenesis."]
  2. Angiogenesis Inhibitor/Antiangiogenesis Agent: "A drug or substance that keeps new blood vessels from forming. In cancer treatment, angiogenesis inhibitors may prevent the growth of new blood vessels that tumors need to grow." [ Cancer.gov. "Angiogenesis Inhibitor."]
  3. Immunotherapy: "A type of therapy that uses substances to stimulate or suppress the immune system to help the body fight cancer, infection, and other diseases. Some types of immunotherapy only target certain cells of the immune system. Others affect the immune system in a general way. Types of immunotherapy include cytokines, vaccines, bacillus Calmette-Guerin (BCG), and some monoclonal antibodies." [Cancer.gov. "Immunotherapy."]
  4. Molecularly Targeted Therapy:
    1. "In cancer, a type of treatment that uses drugs or other substances to target specific molecules involved in the growth and spread of cancer cells. Blocking these molecules may kill cancer cells or may keep cancer cells from growing or spreading. Molecularly targeted therapy may cause less harm to normal cells and may have fewer side effects than other types of cancer treatment." [ Cancer.gov. "Molecularly Targeted Therapy"]
    2. "Targeted molecular therapy is a type of personalized medical therapy designed to treat cancer by interrupting unique molecular abnormalities that drive cancer growth. The drugs used in targeted therapy are designed to interfere with a specific biochemical pathway central to the development, growth, and spread of that particular cancer." [ PennMedicine. "Molecularly Targeted Therapy"]
  5. Chemotherapy: "Treatment that uses drugs to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Chemotherapy may be given by mouth, injection, or infusion, or on the skin, depending on the type and stage of the cancer being treated. It may be given alone or with other treatments, such as surgery, radiation therapy, or biologic therapy." [Cancer.gov. "Chemotherapy."]
  6. Receptor: "A molecule inside or on the surface of a cell that binds to a specific substance and causes a specific effect in the cell." [Cancer.gov. "Receptor."]
  7. Cancer/Tumor Condition: assumed as interchangeable terms, until a tumor is proven as not cancer class tumor. Diagnosis of NF2 means tumors seen in scans and otherwise are not cancer, unless a tumor is over a certain size.
    Example: tumor growth from neurofibromatosis type 2 (NF2), can become malignant (cancer), but NF2 is not a form of cancer.


  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.

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