Lastest Pulmonary Fibrosis Research and Clinical Trials

I received this very informative and interesting email from The Coalition for Pulmonary Fibrosis. If is full of information regarding the latest research and clinical trial information. This organization has spent over $2M in specific research. 

I write to tell you of the increased hope and enthusiasm as the work of the Coalition for Pulmonary Fibrosis (CPF) continues the fight against Pulmonary Fibrosis (PF).  I want to share some of the good news with you as we prepare for the2012 Pulmonary Fibrosis Awareness Month, this September.

Every day progress is made in PF research and we are very proud that, to date, the CPF has invested more than $2 million towards these aggressive searches for answers.   Recent examples of important research progress include:

-Newer understandings of genetic markers and proteomics in PF - the study of proteins that are linked to PF --- in the biomarkers signaling rate of disease advance, as well as other recent projects looking at stem cell research and PF;

-Better defined disease pathways that are key, most notably chronic injury to the alveolar epithelial cells, cells that covermost of the interior surfaces of the lungs;

-Ever more information about the effect of surfactant protein mutations  - the proteins that reduce surface tension in the alveolus of the lungs where gas exchange is critical, and other cellular challenges that are being linked to PF;

-At the University of Southern California (USC), here in Los Angeles, researchers are looking at the signaling pathways between Wnt/β-catenin, a signaling pathway, and transforming growth factor-β. TGFβ is a protein that regulates proliferation.  The apparent increased expression of the a-smooth muscle actin protein, a fibrosis source, may be treatable with ICG-001;

-At the University of Pittsburgh’s Simmons Center for Interstitial Lung Disease, researchers are looking at biomarkers that could signal the rate of disease progression in patients, alerting some patients to the need of getting listed early for transplant;

-In the pharmaceutical world, Boehringer-Ingelheim is in a Phase III study for a PF
treatment; InterMune continues its Phase III study of Pirfenidone, which is approved
for use in Western Europe; Fibrogen is in a promising Phase IIB study; Celgene
has a Phase II study in CC930; and there are more!

The CPF works to foster an ever-greater expansion of studies. The science around PF is absolutely advancing, with ever-expanding teams of researchers taking on PF!  If you are interested in further in-depth information, we have made multiple research papers available on the CPF website at www.CoalitionForPF.org. 

Add to these achievements the new FDA Safety and Innovation Act, enacted in July, that contains the most groundbreaking measures for rare disease patients and their families since the Orphan Drug Act of 1983. Specific to the rare disease community, the Act provides the following:

• accelerated patient access to new medical treatments
• the development of Humanitarian Use Devices (medical devices for small patient populations)
• accelerated development of "breakthrough therapies" -- those that show early promise
• enhanced FDA consultation with rare disease medical experts
• a rare pediatric disease priority review voucher incentive program
• resolution of conflict-of-interest issues that kept voices of rare disease medical experts from being heard

And, looking at your Coalition for Pulmonary Fibrosis:

- In May the CPF announced the winners of the annual CPF Awards given
in two categories for 2012:  the Marvin I. Schwarz award to Dr. Andrew Tager for his outstanding contributions to PF research (his discoveries in Lycophosphatidic acid
receptor activity in PF now resides with Bristol-Myers Squibb (who purchased
Amira), and the Francis Cabral Humanitarian Award to Tami Rippy for her extraordinary commitment to generating awareness of, advocating for, and raising money for the efforts around PF. The Marvin I. Schwarz award carries a monetary grant to be applied towards further PF research.

-Every month the CPF sees an average of 45,000 unique visits to our website,which tells us that people are hungry for information.  The actual number of monthly “hits” to the website is about 240,000.  Our Daughters of PF Linked-In site and Facebook sites help to further assure that we are sharing the most useful PF information, and we have added a “Know Your Risks” section to the website, which alerts people to the kinds of illnesses, toxins and drugs that may contribute to PF.

-  Every  day the CPF takes calls from patients and caregivers throughout America who need answers and help now. These include everything from medical referrals, to questions about where to get personal support and information, to simply wanting to understand better what is known about the disease.  We arm callers with
information and resources, so that they can better empower themselves as patients or caregivers.

-  Every day, just as importantly, we actively encourage  people to tell others about PF – we are spreading the word, reaching out everywhere, from the NBC Nightly
News, to daily newspapers, to actress Rose McGowan’s vibrant fan club, and
countless other national and local media resources.

The September PF Awareness month and Capitol Hill efforts on the
Pulmonary Fibrosis Research Enhancement act will help us recruit an expanded chorus of voices demanding change.  

Fifty-two weeks a year the CPF staff is working to make certain the nation understands the impact of PF and to energize the American public to support our urgent work.   With your collaboration and support, positive change continues, and more is on its way!

Sincerely,

Mishka Michon, CEO

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CALLING ALL PATIENTS! CLINICAL TRIAL RECRUITMENT

Treatments for PF cannot be found without patient participation.

Please consider joining a trial to help  find treatments.  Go to the CPF Websitewww.coalitionforpf.org or ww.ClinicalTrials.gov for more information.

This is a partial list:    

- Phase III –InterMune – Pirfenidone-Visit AscendTrial.com

- Phase II:  Fibrogen - FG 3019

- Phase II:  Novartis – QA576

-Phase II: Stromedix – STX100 

-Phase I:  Gilead – GS6624

-Phase I: Promedior – PRM 151

If you’re interested in joining a clinical trial, discuss the option with your doctor and contact the company holding the trial in which you’re interested.

To our Donors:

It’s difficult to compress in a short letter everything we are able to do because of your support and how grateful we are for that support.   The over $2 million invested in research grants, the aggressive and productive advocacy work in Washington D.C. to protect our patients’ rights and to advance federal attention to PF,  the ongoing effort to engage the press in helping tell the world about the threat of PF, the partnerships with key organizations that can make a difference in a rare disease such as the American Thoracic Society, the National Institutes of Health and key PF centers around the country, and the building of programs like the Daughters of PF to help generate unified efforts to fight this disease, are all possible because of our donors.  Nothing we do is possible without those who care enough to make this battle possible.  

To our Volunteers and all other Activists: 

We offer our humble gratitude for your contribution to this fight, whether you are
an advocate for change, a caregiver, a volunteer, or in any other way help to
advance this work.  The wonderful events around the country that build awareness and funding are all due to those who take the time to help make a difference.  There is no progress, no hope, and no real investment in the future without all of you who add to the impact of the work we do!

There is still a lot of misunderstanding about who gets Pulmonary Fibrosis.  There are
millions of people who don’t know what the words mean, and many who think there
must be “some reason” that you get PF, having to do with bad health habits, such as smoking.  Though some toxins can generate PF, the vast majority of people have yet to fully grasp that PF can strike completely randomly, and that there is actually no known cause. 

Just ask Heather S., who was blindsided by the diagnosis in her 30’s;

Or ask Elliott W., who was utterly shocked by his diagnosis in his 60’s, after having lived a very healthy lifestyle;

Or ask Heather D., who has struggled with PF since her fifties, and had a difficult and long wait for Medicare even though she was unable to work;

Or ask Olene W., who has always had extraordinary stamina and been very active, but whose life was suddenly changed when she was diagnosed with PF;

Or just ask Kitty L., a healthy and phenomenally active woman who has been a
lifelong tennis player and was struck with PF last year.

We know, very clearly, why this work is so important.  It is because every patient with this diagnosis is facing a terrible truth, and they deserve the best support possible along with a truly passionate effort to build a crescendo of voices demanding progress in research.    

DON’T FORGET – PF AWARENESS month is coming!  We’ll be asking you to contact your legislators to support our Congressional bill.

If you can contribute to support the fight for research, please do so.  Every dollar counts!

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P.S.
Your gift at this time will support our September PF month

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