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

Statement Of Support By The PAP Foundation, Inc. For FDA Approval Of GM-CSF Treatment For aPAP
  • USA - English

The PAP Foundation finds compelling evidence of efficacy of inhaled GM-CSF treatment for aPAP patients through review of recently published and publicly available data in conjunction with learnings from prolonged "off-label" use within the PAP Foundation patient community, which is in dire need of an effective therapy to treat this life-threatening syndrome


News provided by

PAP Foundation

Mar 25, 2020, 10:10 ET

Share this article

Share toX

Share this article

Share toX

LOS ANGELES, March 25, 2020 /PRNewswire-PRWeb/ -- The PAP Foundation, Inc. ("PAPF") is the primary patient advocacy organization for Pulmonary Alveolar Proteinosis ("PAP") in the United States and consists of clinicians, scientists, PAP patients and their family members. With foundational goals of finding a cure and improving the lives of those affected by PAP, we have received numerous testimonials from patients whose lives have significantly improved through the use of inhaled GM-CSF. Outcomes have included improvement in exercise tolerance and quality of life, decreased need for whole lung lavage, and decreased need for oxygen therapy. Moreover, we have witnessed patients who have discontinued GM-CSF therapy after seeing significant improvement only to suffer disease recurrence necessitating the resumption of GM-CSF treatment.

PAPF commends both the PAGE (sponsored by Niigata University Medical & Dental Hospital) (shortened link: https://bit.ly/2WGi07F) and the IMPALA (sponsored by Savara Inc.) (shortened link: https://bit.ly/2y28XDC) investigators for conducting the first two multicenter randomized clinical trials using inhaled GM-CSF in patients with autoimmune Pulmonary Alveolar Proteinosis ("aPAP"). The results of the two studies differed in some regards, yet in aggregate PAPF interprets this data as further evidence inhaled GM-CSF can improve a number of outcomes including quality of life, pulmonary function testing parameters, the oxygenation of the blood as measured by "A-a gradient", and the amount of surfactant seen on images of the lungs.

Based on the above trials and on the experiences of numerous PAPF patients, it is our continued reasoned position that inhaled GM-CSF treatment is not only an effective therapy for many patients, but the best medical option currently available to improve the overall health and quality of life for most aPAP patients. In contrast, the current standard of care for aPAP treatment is Whole Lung Lavage ("WLL"); a highly invasive, time-consuming and dangerous surgical procedure that is often needed throughout a PAP patient's life whenever their symptoms become life-threatening or otherwise too much to bear.

Of particular note to the PAP patient and clinician communities, we believe the most efficacious results for aPAP patients on inhaled GM-CSF treatment take into consideration drug duration and dosage. The experiences shared among our patient and physician community suggest that the full impact of GM-CSF therapy can take multiple months, and treatment that does not allow for this requisite time will likely show suboptimal results. As to dosage, some in our community's experience suggests that GM-CSF treatment is only effective once the dosage is high enough (presumably, to overcome the barrier of elevated anti-GM-CSF autoantibodies in each individual aPAP patient's lungs). Using a number of patients in our community as case examples, 250 ug of GM-CSF daily failed to show improvement, however, when dosage was increased to 500ug daily, these patients no longer required additional whole lung lavages, some for multiple years. This represents a drastic improvement in this subset of patients compared to the year prior to initiating this increased dosage of GM-CSF.

Although the science as to PAP has come a long way in the recent past, since the revitalization of our patient foundation over 3 years ago, we have witnessed a number of patients in our PAP community tragically deteriorate; some requiring lung transplantation and others who are being kept alive on oxygen therapy or life support in a hospital setting. We believe that if these patients were properly diagnosed with PAP earlier and GM-CSF treatment was available to them, their lives and outcomes could have been dramatically improved.

The PAP Foundation remains steadfast in our mission to support the development of efficacious therapies and ultimately a cure for this devastating disease and is committed to its advocacy for FDA approval of inhaled GM-CSF therapy as a primary treatment option for aPAP patients.

In furtherance of the above, the PAP Foundation attended the FDA Rare Disease Day held at the FDA in Maryland on February 24, 2020, where we shared these sentiments during the Open Public Comment period and will be submitting this written statement together with our comments made at the FDA event to Federal Register Docket No. FDA-2019-N-5646.

This statement of support is provided by the leadership of the PAP Foundation, Inc., comprised entirely of doctors, clinicians, and PAP experts and/or patients, as such leadership is listed below:

President
Christopher Hameetman
Los Angeles, CA

Vice President & Clinical Director
Tisha Wang, M.D.
Los Angeles, CA

Second Vice President
Steven Park
Lakewood, CO

Secretary / Treasurer
Brenna Carey, M.S, Ph.D.
Cincinnati, Ohio

Scientific Director
Bruce Trapnell, M.D.
Cincinnati, Ohio

If you would like further information on this topic, please contact Brenna Carey, PAP Foundation Media Liaison, at 513-636-8916 or by email to [email protected].

SOURCE PAP Foundation

Related Links

https://www.papfoundation.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.