Pharmaceuticals

Last Updated: 09/25/14

All treatments that are and have been considered for tumor suppression for NF2 are and will be included here. Previously considered treatments are included to note the types of treatments considered, to help learn what advantages they had to help determine criteria of new and better treatments, since none of the tumor suppressors has found to be effective enough alone to date.

For Tumor Suppression, there are and have been several medications in trials for NF2, no medication has made it further than trials for NF2, to date. So far none has been declared as 100% effective in every individual in reducing the size of tumors in individuals with NF2.

About Clinical Trials

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 a 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

Clinical Trials for NF2

1. Bevacizumab (Avastin™)

  • Expectation: Change in Meningioma and Schwannoma
  • Treatment by: Intravenous or Port
  • Inhibitor: VEGF-A
  • Pharmaceutical Co.: Genentech / Roche
  • Trial Availability: Most Countries

Avastin is the longest running tumor suppressor medication. When it works, it has the possibility of affecting all tumor types in the brain and spine. The rates tumors might change are different for each, when it works.

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


2. OSU-HDAC42 - AR-42

  • Expectation: Change in Meningioma and Schwannoma
  • Treatment by: Pill - Oral
  • Trial Availability: USA - Ohio only

AR42 is a new drug, no long term data to know what side effects to expect, but NF2 mice trials indicate chances of effectiveness of both Meningioma and Schwannoma.


3. RAD 001 - Everolimus (Afinitor™)

  • Expectation: Change in Intracranial Schwannoma
  • Treatment by: Pill - Oral
  • Trial Availability: USA

This trial only recently opened a few months ago, therefore no long term data is available to say what rate it is effecting tumors or percentage chances on side effects. But this medication has been available for some time for other uses and side effects would likely be similar.


4. Lapatinib (Tykerb™)

  • Expectation: Change in Intracranial Schwannoma
  • Treatment by: Pill - Oral
  • Trial Availability: USA

Trial for this ended with the results that only Schwannoma were affected, and tumors did not shrink as well as they do with Avastin. However it has minimal easily managed side effects, is in easy to take pill form and might be a good alternative for individuals who have issues with Avastin till RAD-001 or something better is more readily available.


5. Nilotinib (Tasigna™)

  • Expectation: Change in Intracranial Schwannoma
  • Treatment by: Pill - Oral
  • Trial Availability: Canada

6. Sorafenib (Nexavar™)

  • Expectation: Change in Intracranial Schwannoma
  • Treatment by: Pill - Oral
  • Trial Availability: Canada

7. Erlotinib (Tarceva™)

  • Expectation: Change in Intracranial Schwannoma
  • Treatment by: Pill - Oral
  • Trial Availability: Canada

8. Imatinib (Gleevec™)

  • Expectation: Change in Intracranial Schwannoma
  • Treatment by: Pill - Oral
  • Trial Availability: Most Countries

9. Sunitinib (Sutent™)

  • Expectation: Change in Intracranial Meningioma
  • Treatment by: Pill - Oral
  • Trial Availability: USA

This is a new NF2 treatment and facts collected are based on use of Sunitinib for other treatments. Side effects have been found to be minimal and any severe issues disappear with dose reduction, break in treatment or other treatment.

Sunitinib has been found more effective in situations where Sorafenib (Nexavar), Temsirolimus (Torisel), Interleukin-2 (Proleukin), Everolimus (Afinitor), Bevacizumab (Avastin) and Aldesleukin helped, but they only had minimal effect.

10. Selumetinib - AZD6244

  • Expectation: Change in Intracranial Schwannoma
  • Treatment by: Pill - Oral
  • Trial Availability: Europe
  • More Information: http://www.nf2is.org/selumetinib.php

This trial recently started in the last few months. No data has been collected on this specifically for NF2.


11. Somatostatin

  • Expectation: Change in Meningioma?
  • Possible Trial: France
  • More Information: Wikipedia

Medications Determined Ineffective for NF2

1. Pasireotide - SOM 230

  • Expectation: Change in Intracranial Meningioma
  • Treatment by: Intravenous
  • Trial Location: USA

Pasireotide was not made available under an NF2 specific trial. It was in trial for people with recurrent or progressive Meningioma. It was found as not effective enough.


2. PTC 299

  • Expectation: Change in Intracranial Schwannoma
  • Treatment by: Pill - Oral
  • Trial Availability: USA - No Longer Available

PTC 299 was found as effective for ability to change NF2 tumors. However, after some time, individuals built up a tolerance to the effectiveness of the treatment and tumors would start to grow again.


3. OSU-03012 - AR-12

  • Expectation: Change in Meningioma and Schwannoma
  • Trial Location: USA

It was determined that AR-42 (OSU-HDAC42 - AR-42) would be the more effective treatment than AR-12 (OSU-03012 - AR-12


4. Hydroxycarbamide (Hydrea™)

  • Expectation: Change in Intracranial Schwannoma
  • Treatment by: Pill - Oral
  • Trial Location: Several Countries before discontinued

Hydroxycarbamide, more commonly known as Hydroxyurea, resulted in complications resulting in the discontinuation of distribution and manufacturing of this treatment in favor of other treatment choices for all uses of this medication.



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