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Drug-Based Treatments (Clinical Trials)

Last Updated: 08/19/18

1. NF2 Drug-Based Treatments

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
    (Kadcyla™)
  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. Dasatinib (Sprycel™)
  2. CD47
  3. Pasireotide - SOM 230
  4. PTC 299
  5. OSU-03012 - AR-12
  6. Hydroxycarbamide (Hydrea™)

5. Also See:

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 tumor treatments 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 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

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™)

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.

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


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.

Potential NF2 Clinical Trials


1. Cruciferous Vegetables: 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.

2. 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

Sources

  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/