Accessibility Statement Skip Navigation
  • Why PRWeb
  • How It Works
  • Who Uses It
  • Pricing
  • Login
  • GDPR
  • Create a Free Account
Return to PRWeb homepage
  • News
  • Resources
  • Contact
When typing in this field, a list of search results will appear and be automatically updated as you type.

Searching for your content...

No results found. Please change your search terms and try again.
  • News in Focus
      • Browse News Releases

      • All News Releases
      • Multimedia Gallery

      • All Multimedia
      • All Photos
      • All Videos
  • Business & Money
      • Auto & Transportation

      • Aerospace, Defense
      • Air Freight
      • Airlines & Aviation
      • Automotive
      • Maritime & Shipbuilding
      • Railroads and Intermodal Transportation
      • Supply Chain/Logistics
      • Transportation, Trucking & Railroad
      • Travel
      • Trucking and Road Transportation
      • View All Auto & Transportation

      • Business Technology

      • Blockchain
      • Broadcast Tech
      • Computer & Electronics
      • Computer Hardware
      • Computer Software
      • Data Analytics
      • Electronic Commerce
      • Electronic Components
      • Electronic Design Automation
      • Financial Technology
      • High Tech Security
      • Internet Technology
      • Nanotechnology
      • Networks
      • Peripherals
      • Semiconductors
      • View All Business Technology

      • Entertain­ment & Media

      • Advertising
      • Art
      • Books
      • Entertainment
      • Film and Motion Picture
      • Magazines
      • Music
      • Publishing & Information Services
      • Radio & Podcast
      • Television
      • View All Entertain­ment & Media

      • Financial Services & Investing

      • Accounting News & Issues
      • Acquisitions, Mergers and Takeovers
      • Banking & Financial Services
      • Bankruptcy
      • Bond & Stock Ratings
      • Conference Call Announcements
      • Contracts
      • Cryptocurrency
      • Dividends
      • Earnings
      • Earnings Forecasts & Projections
      • Financing Agreements
      • Insurance
      • Investments Opinions
      • Joint Ventures
      • Mutual Funds
      • Private Placement
      • Real Estate
      • Restructuring & Recapitalization
      • Sales Reports
      • Shareholder Activism
      • Shareholder Meetings
      • Stock Offering
      • Stock Split
      • Venture Capital
      • View All Financial Services & Investing

      • General Business

      • Awards
      • Commercial Real Estate
      • Corporate Expansion
      • Earnings
      • Environmental, Social and Governance (ESG)
      • Human Resource & Workforce Management
      • Licensing
      • New Products & Services
      • Obituaries
      • Outsourcing Businesses
      • Overseas Real Estate (non-US)
      • Personnel Announcements
      • Real Estate Transactions
      • Residential Real Estate
      • Small Business Services
      • Socially Responsible Investing
      • Surveys, Polls and Research
      • Trade Show News
      • View All General Business

  • Science & Tech
      • Consumer Technology

      • Artificial Intelligence
      • Blockchain
      • Cloud Computing/Internet of Things
      • Computer Electronics
      • Computer Hardware
      • Computer Software
      • Consumer Electronics
      • Cryptocurrency
      • Data Analytics
      • Electronic Commerce
      • Electronic Gaming
      • Financial Technology
      • Mobile Entertainment
      • Multimedia & Internet
      • Peripherals
      • Social Media
      • STEM (Science, Tech, Engineering, Math)
      • Supply Chain/Logistics
      • Wireless Communications
      • View All Consumer Technology

      • Energy & Natural Resources

      • Alternative Energies
      • Chemical
      • Electrical Utilities
      • Gas
      • General Manufacturing
      • Mining
      • Mining & Metals
      • Oil & Energy
      • Oil and Gas Discoveries
      • Utilities
      • Water Utilities
      • View All Energy & Natural Resources

      • Environ­ment

      • Conservation & Recycling
      • Environmental Issues
      • Environmental Policy
      • Environmental Products & Services
      • Green Technology
      • Natural Disasters
      • View All Environ­ment

      • Heavy Industry & Manufacturing

      • Aerospace & Defense
      • Agriculture
      • Chemical
      • Construction & Building
      • General Manufacturing
      • HVAC (Heating, Ventilation and Air-Conditioning)
      • Machinery
      • Machine Tools, Metalworking and Metallurgy
      • Mining
      • Mining & Metals
      • Paper, Forest Products & Containers
      • Precious Metals
      • Textiles
      • Tobacco
      • View All Heavy Industry & Manufacturing

      • Telecomm­unications

      • Carriers and Services
      • Mobile Entertainment
      • Networks
      • Peripherals
      • Telecommunications Equipment
      • Telecommunications Industry
      • VoIP (Voice over Internet Protocol)
      • Wireless Communications
      • View All Telecomm­unications

  • Lifestyle & Health
      • Consumer Products & Retail

      • Animals & Pets
      • Beers, Wines and Spirits
      • Beverages
      • Bridal Services
      • Cannabis
      • Cosmetics and Personal Care
      • Fashion
      • Food & Beverages
      • Furniture and Furnishings
      • Home Improvement
      • Household, Consumer & Cosmetics
      • Household Products
      • Jewelry
      • Non-Alcoholic Beverages
      • Office Products
      • Organic Food
      • Product Recalls
      • Restaurants
      • Retail
      • Supermarkets
      • Toys
      • View All Consumer Products & Retail

      • Entertain­ment & Media

      • Advertising
      • Art
      • Books
      • Entertainment
      • Film and Motion Picture
      • Magazines
      • Music
      • Publishing & Information Services
      • Radio & Podcast
      • Television
      • View All Entertain­ment & Media

      • Health

      • Biometrics
      • Biotechnology
      • Clinical Trials & Medical Discoveries
      • Dentistry
      • FDA Approval
      • Fitness/Wellness
      • Health Care & Hospitals
      • Health Insurance
      • Infection Control
      • International Medical Approval
      • Medical Equipment
      • Medical Pharmaceuticals
      • Mental Health
      • Pharmaceuticals
      • Supplementary Medicine
      • View All Health

      • Sports

      • General Sports
      • Outdoors, Camping & Hiking
      • Sporting Events
      • Sports Equipment & Accessories
      • View All Sports

      • Travel

      • Amusement Parks and Tourist Attractions
      • Gambling & Casinos
      • Hotels and Resorts
      • Leisure & Tourism
      • Outdoors, Camping & Hiking
      • Passenger Aviation
      • Travel Industry
      • View All Travel

  • Policy & Public Interest
      • Policy & Public Interest

      • Advocacy Group Opinion
      • Animal Welfare
      • Congressional & Presidential Campaigns
      • Corporate Social Responsibility
      • Domestic Policy
      • Economic News, Trends, Analysis
      • Education
      • Environmental
      • European Government
      • FDA Approval
      • Federal and State Legislation
      • Federal Executive Branch & Agency
      • Foreign Policy & International Affairs
      • Homeland Security
      • Labor & Union
      • Legal Issues
      • Natural Disasters
      • Not For Profit
      • Patent Law
      • Public Safety
      • Trade Policy
      • U.S. State Policy
      • View All Policy & Public Interest

  • People & Culture
      • People & Culture

      • Aboriginal, First Nations & Native American
      • African American
      • Asian American
      • Children
      • Diversity, Equity & Inclusion
      • Hispanic
      • Lesbian, Gay & Bisexual
      • Men's Interest
      • People with Disabilities
      • Religion
      • Senior Citizens
      • Veterans
      • Women
      • View All People & Culture

  • Hamburger menu
  • Cision PRWeb provides efficient communication tools to continuously engage with target audiences across multiple online channels
  • Create a Free Account
    • ALL CONTACT INFO
    • Contact Us


      11AM ET Sunday – 8PM ET Friday

  • Send a Release
  • Sign up
  • Log in
  • Resources
  • RSS
  • GDPR
  • News in Focus
    • Browse All News
    • Multimedia Gallery
  • Business & Money
    • Auto & Transportation
    • Business Technology
    • Entertain­ment & Media
    • Financial Services & Investing
    • General Business
  • Science & Tech
    • Consumer Technology
    • Energy & Natural Resources
    • Environ­ment
    • Heavy Industry & Manufacturing
    • Telecomm­unications
  • Lifestyle & Health
    • Consumer Products & Retail
    • Entertain­ment & Media
    • Health
    • Sports
    • Travel
  • Policy & Public Interest
  • People & Culture
    • People & Culture
  • Send a Release
  • Sign up
  • Log in
  • Resources
  • RSS
  • GDPR
  • Send a Release
  • Sign up
  • Log in
  • Resources
  • RSS
  • GDPR
  • Send a Release
  • Sign up
  • Log in
  • Resources
  • RSS
  • GDPR

Children's Tumor Foundation Launches NF2 Accelerator Initiative to Combat Rare Genetic Disorder

Foundation to Invest $2.3 Million to Expand NF2 Drug Pipeline and Accelerate Treatments for Neurofibromatosis Type 2 Patients.


News provided by

Children's Tumor Foundation

May 22, 2019, 09:00 ET

Share this article

Share toX

Share this article

Share toX


NEW YORK, May 22, 2019 /PRNewswire-PRWeb/ -- On the occasion of World NF2 Awareness Day, the Children's Tumor Foundation has announced the establishment of a significant research initiative dedicated to finding effective treatments for NF2, along with a substantial investment of $2.3 million in this bold new effort. Called the 'NF2 Accelerator Initiative,' this three-year undertaking will be dedicated to bringing active NF2 treatments to the clinic (and patients) by expanding the clinical drug pipeline for NF2, improving drug selection through the development of innovative testing models, and the development of gene therapy options that address the underlying genetic causes of NF2.

Neurofibromatosis, also known as NF, is an under-recognized genetic disorder that causes tumors to grow on nerves throughout the body and manifests in three distinct forms: NF1, NF2, and schwannomatosis. NF2 is the second most common type of NF and affects approximately 1 in every 25,000 people of all populations. It is caused by mutations in genes located on chromosome 22, and is a progressive disorder characterized by many different manifestations, including the development of tumors such as vestibular schwannomas, meningiomas and ependymomas. NF2 can lead to a variety of symptoms, including hearing loss, tinnitus, balance problems, and more. While the tumors are benign, their location in the body limits surgical options and can create serious health issues, with no effective non-surgical treatment options currently available.

“By expanding the clinical drug pipeline, improving drug selection, and developing gene therapy options, we’re pushing forward in the development of critical innovations to help solve the challenges of NF2, and most importantly, improve the lives of NF2 patients and their families.”

Post this

As a global leader of NF research, the Children's Tumor Foundation is dedicated to developing cures for all three forms of NF. The Foundation and its partners have bolstered the NF2 research field through a team science approach, called Synodos. Launched in 2014, Synodos for NF2 brought together a multidisciplinary team of scientists from 12 world-class labs at academic and medical centers of excellence who pledged to work together. The Synodos teams shared information, datasets and results in real-time at every step of research development. (All that data is now publicly available at nfdataportal.org). That collaborative effort resulted in the identification of promising new clinical candidates for NF2, and today's announcement of the NF2 Accelerator Initiative leverages that new knowledge into a new and ambitious structure, with the goal of speeding up the drug discovery process.

The NF2 Accelerator Initiative will concentrate on opportunities in three areas:

1. Expand the Clinical Drug Pipeline for NF2
This effort will focus on discovering novel therapeutic targets and the development of preliminary biomarkers to help validate existing therapeutic targets for NF2. In order to expand the pipeline, CTF is planning to:
     •    Participate in and co-fund a phase 2 clinical trial using Brigatinib, a promising drug that came out of the Synodos for NF2 effort. Takeda Pharmaceutical Company has agreed to co-invest in the trial. Study protocol and clinical trial site selection is being developed. The trial is aimed to be open for recruitment soon.
     •    Offer Drug Discovery Awards to researchers and institutions focused on the next phase of NF2 clinical candidates. An RFA (Request for Application) is being launched today to support exploratory and development drug discovery projects. Information can be found at ctf.org.
     •    Offer Young Investigator Awards to early-stage researchers as an investment in basic research and the expansion of understanding of the processes that drive NF2. These awards will be available in 2020.

2. Improve Drug Selection for NF2
This focus is on the development of animal models to improve drug testing and efficacy within NF2.
     •    Based on the success of the Foundation's Synodos for NF1 pig models, the National Institute of Neurological Disease and Stroke (NINDS) recently developed similar animal models for NF2. This phase of the NF2 Accelerator project will expand research on these models and provide better understanding of how different manifestations of NF2 develop over time.
     •    Development of image-based animal models for vestibular schwannomas to combat the limited availability of preclinical resources that allow for efficient drug testing.

3. Gene Therapy for NF2
Much of NF2 drug discovery and development is primarily focused on inhibiting the processes that are activated as a consequence of the loss of the NF2 gene. CTF has invested in gene therapies in the past but has now received a very exciting gene therapy proposal that has the ability to be a real treatment option for patients with NF2.

"Today on NF2 Awareness Day, we're proud to announce the next chapter of NF2 research aimed at bringing treatment options to those who are living with neurofibromatosis type 2 faster," said Annette Bakker, PhD, President of the Children's Tumor Foundation. "By expanding the clinical drug pipeline, improving drug selection, and developing gene therapy options, we're pushing forward in the development of critical innovations to help solve the challenges of NF2, and most importantly, improve the lives of NF2 patients and their families."

Lead funding for the NF2 Accelerator Initiative has been provided by Jim and Kathy Thoms; Nicole and Roland Thoms; Carol Harrison Kalagher; and Beth and John Morris. To support the NF2 Accelerator Initiative, please visit ctf.org/endnf2.

ABOUT THE CHILDREN'S TUMOR FOUNDATION:
The Children's Tumor Foundation is a 501(c)(3) not-for-profit organization dedicated to funding and driving innovative research that will result in effective treatments for the millions of people worldwide living with neurofibromatosis (NF), a term for three distinct disorders: NF1 (1 in 3,000 people); NF2 (1 in 25,000), and schwannomatosis (1 in 40,000). NF causes tumors to grow on nerves throughout the body and may lead to blindness, deafness, bone abnormalities, disfigurement, learning disabilities, disabling pain, and cancer. It affects all populations equally. There is no cure yet – but the Children's Tumor Foundation mission of driving research, expanding knowledge, and advancing care for the NF community fosters our vision of one day ending NF. For more information, please visit http://www.ctf.org.

SOURCE Children's Tumor Foundation

Related Links

www.ctf.org

Modal title

Contact PRWeb

  • 11AM ET Sunday – 8PM ET Friday
  • Contact Us

About PRWeb

  • About PRWeb
  • Partners
  • Partnership Programs
  • Editorial Guidelines
  • Resources

Why PRWeb

  • Why PRWeb
  • How It Works
  • Who Uses It
  • Pricing

Accounts

  • Create a Free Account
  • Log in
  • Contact Us

Do not sell or share my personal information:

  • Submit via [email protected] 
  • Call Privacy toll-free: 877-297-8921

Contact Cision

Products

About

My Services
  • All News Releases
  • Online Member Center
  • ProfNet
Cision Distribution Helpline
888-776-0942
  • Legal
  • Site Map
  • RSS
  • Cookie Settings
Copyright © 2025 Cision US Inc.