National Journal today...

A few weeks ago Donna Pederson, a 24 year old PH patient and nurse, PHA's good friend Congressman Kevin Brady and I agreed to a video interview with the National Journal.  Their video publications are available to all Capitol Hill offices (and heavily trafficked by them), as well as to other subscribers.

I thought they did a pretty good job with the exception of reporting patient survivability at 2.8 years for 50% of patients. That number is true for patients not on medication. With 9 treatments availble - 8 since 2001 - survivability continues to improve.

At any rate, here's the video.  See what you think.

Time to move...again

On February 16, I posted that construction permits went up for PHA's 6th move in 11 years.

Well it's time.

Last weekend the cubicle walls came down and were moved to our new space on the 10th floor.  This week, as you can see from the pictures on our 4th and 5th floor, it's been kind of like a sweatshop here at PHA.  Starting tomorrow and continuing on Saturday, our computers and phones will make the move and our staff should be reunified on a single, functioning floor on Monday.

Wish us luck!

Candid Reflections on Lung Transplantation...

Exactly a week ago, PHA had the opportunity to deliver a Lunch Symposium at the International Society for Heart and Lung Transplantation.

As a high level sponsor of the ISHLT Conference, Gilead Sciences had the right to this block of time but decided a PHA presentation would be more appropriate.  They covered the costs for the lunch event and we accepted and developed the content.

I opened with a slide presentation titled Blending the Patient and Medical Communities: The Pulmonary Hypertension Association Model. Truth be told, it was really more about it's subtitle,,,Heroes.  I told brief stories about individuals like Bonnie Dukart, Diorothy Olson and Barbara Smith, people who by their actions made a difference and built this organization.

Following that, we presented a video PHA developed in 2005, featuring stories of patients experiences pre and post transplant.  I hadn't seen it myself in quite awhile and found it quite moving.  One of the doctors who saw it suggested it would have value as a training tool for medical professionals coming into the field.  I think we'll explore that possibility.

We were fortunate to have Dr. Reda Girgis of Johns Hopkins conclude the program with a presentation on Challenges in Lung Transplantation for PAH.  He educated the audience on a number of transplant issues specific to PH patients.

It was a goos session and a great chance to introduce the work we are doing to the transplant community.

ISHLT..two surprises.

I spent most of last week at the International Society of Heart and Lung Transplant meeting in Chicago.

Their staff did an amazing job navigating around an unexpected problem.  The eruption under the Eyjafjallajokull glacier in Iceland made it impossible for over 500 European registrants to attend...and many of those were speakers.  With 48 hours notice, most of the speakers were able to present their slides remotely and well.  It was an amazing achievement and one I hope we'll never have to match ourselves to at a PHA Conference.

A second surprise was much more pleasant.  In an organization that historically has had little PH education, avery high percentage of content was focused on the disease.  As a matter of fact, the Conference had a full day symposium on PH last Wednesday.

This explosion of PH information is due to the hard work of many of the physicians we work with at PHA.  Two years ago, they formed a new PH Council within ISHLT and drew large attendance to strong PH sessions at last year's Conference in France. 

Christa from our Med Services staff and I attended this year's Council meeting.  The outgoing Chair, Dr. Ray Benza and incoming Chair, Dr. Myung Park led a dynamic discussion of their next steps in keeping PH as a topic of value and importance to the ISHLT membership.

PHA's next step is currently being worked out.  We have offered to support the PH Council's educational process within ISHLT by mailing the next issue of our medical journal to all ISHLT members. Its topic is Transplant.  We'll be sending the ISHLT board a review copy in the next week or so.  Dr. Debbie Levine, Dr. Myung Park, and Dr. Ray Benza, all Council members, have worked on the issue...with Dr. Levine being the primary editor. 

I feel fortunate to have been able to attend the ISHLT meeting.  It was another important element in the fight against PH and another chance to see how the interconnectedness of our community can drive positive change for the good of all. 

...And, while that's a pleasure, I'm pleased to say it is no longer a surprise.

And that's how awareness grows...

PHA hosts over 20 message boards and lists servs.  Many support the work of various committees, while others are active sources of broad communications for various segments of our community.

Lately, there's been a lot of buzz on the support group list servs about two pieces of video involving support group leaders.

Ellen Harris, a support group leader from Northern Virginia, is featured on a PH segment on the PBS show, Healthy Bodies, Healthy Minds (scroll down to episode 1506).  North Carolina support group leader Doug Taylor reported that WTVI in Charlotte, NC also had a recent program on PAH. (Once on the page use the arrow at the bottom to scroll down to the Pulmonary Hypertension show). Host Joey Popp interviewed cardiologist Theodore Frank, MD, and pulmonologist Doug Kelling, MD as well as local PH patients (and twins) Linda Miles and Brenda McCallum.

Then there's the continuing coverage of the Kilimanjaro Climb which has generated over two million readers and viewers across more than 70 articles and features.  I'm sure you'll enjoy one of the most recent from WQED in Pittsburgh.

While the PH community may not be large compared to other diseases, our drive to get our story out is creating awareness and opportunities beyond our numbers.

I hope you'll take a moment to find ways to get involved through the Awareness pages on the PHA website. It can make all the difference.

Beyond expectations...and yet

On October 9, I posted a blog titled, Keeping our values straight.  It was about the launch of our scholarship efforts for PHA's 2010 International PH Conference and why this event is so important for patients and their families..

Yesterday, we had our monthly Executive Committee call with leaders of the PHA Board. 

Patty, our Finance Director reported that our Scholarship Fund has grown to $219,305.  To understand what this means, you have to think about the number of PH patients in the U.S. in proportion to diabetes (23.6 million), asthma (16.4 million), Bi-polar disorder (5.7 million), Alzheimers (5.3 million) or many others.  Campare these numbers to the 20,000 to 30,000 diagnosed PH patients in the U.S.  Our numbers are small and so our climb to make the changes we need and want on any issue is steep.  It is a climb that involves everyone's best efforts...and, in that, this community has never disappointed.

That our community is able to provide this kind of support for patient scholarships - to what has been described by many who attend as a life-changing event - says a lot about the PH community's values.

We're proud of the scholarship program.  There's no question it's hard work to raise these funds but it's so worthwhile.  My only regret is that, even at this extraordinary level of support, there are still many patients who have to be declined for scholarships.  My dream is that, someday, we will be able to endow this program at a level high enough to guarantee that we can fully meet the need and turn away no patient who wants to come..

Kind words spotlighting PHA chats...

It's always nice to receive kind words  Last week, Chris Akins sent those and also put a spotlight on PHA's chats.  I thought I'd share his note with you...

I wanted to personally thank you for the PHAssociation and the online chat that you provide. My wife of 14+ years Marla was diagnosed with PAH 16 months ago. Last March she was in right hear failure and had a mean pulmonary pressure of 138. At that time she was placed on Flolan and a host of oral meds. If it were not for the online chat and the ability to connect with others with PAH I don't think she could have moved past the initial shock. Today, I am over joyed to report that she returned from her quarterly check up with Dr. James Gossage and her pulmonary pressures and heart size are within the range of normal.

From March 2009 to January 2010 she has successfully been transitioned off of Flolan to Tyvaso and her oral meds have been reduced. The mental stigma of PAH still remains but the PHA chat is a constant source of hope, joy, sadness and renewed belief that together we can overcome. The community that PHA provides is instrumental in not only the physiological well being of PAH patients but their mental well being as well.

From a caregivers perspective and being in the medical field for the past 10 years I have to say PHA is a God send. I had the privilege of attending the PHA on the road in Orlando last year. WOW what refreshing change of paradigm of patient, physician, and pharmaceutical interaction. But most of all it is a great opportunity for me personally to interact with other care givers and PAH patients via the online chat. A year ago I was one who didn't know where the road was going to take us and now I find myself a unique position of knowing where the road could go and sharing it with others.

Again Thank You and PHA for all that you do to make things a little easier.

Anyone in the PH community can connect with a scheduled (or unscheduled) chat through the PHA website.

Blogging for Conference...

Nope, this isn't the shortest blog ever. It's just to let you know I did blog today...but on PHA's new Conference page.

Take a look and see what Bonnie Dukart, one of PHA's early and great leaders, had to say about the event and get a picture of how it evolved.

Keeping government focus on PH research...

PHA's Washington Rep, Gavin Lindberg sent us a report today from the NIH on the investment made in PH research with funds from the economic stimulus bill. PHA joined with the biomedical research community last year in encouraging Congress to provide an additional $10 billon to the NIH as part of the American Recovery and Investment Act (ARRA). NIH has been producing “ARRA Investment Reports” on diseases that benefitted significantly from stimulus funding. Only a handful of rare diseases have been the subject of these investment reports and PH is one of them. Here's the report...


        ARRA Investments in Pulmonary Hypertension (PH)

Public Health Burden

PH is an irreversible, malignant elevation of pulmonary vascular pressures that causes death due to failure of the right side of the heart. Although idiopathic pulmonary hypertension is considered a rare disease, its true epidemiology is being called into question because of an increasing awareness that PH may occur in the setting of more prevalent respiratory diseases such as COPD, that it can develop in systemic diseases such as sickle cell anemia, and that right-heart dysfunction may actually occur earlier in the disease pathogenesis than previously recognized. The annual number of hospitalizations attributable to PH in the United States rose significantly from 1980 to 2000, especially for women, and worldwide estimates of all-cause PH are increasing.

Treatment

Within the past few years, drugs to dilate the vasculature have become available for use in PH. Current therapies modestly extend survival but do not lead to regression or cure, and no clear indicators of therapeutic efficacy, such as surrogate markers, have been identified. The sole alternative when medical therapy fails is lung transplantation, and it extends survival only minimally. ARRA funds are supporting projects to:

• Identify new ways to direct current vasodilatory therapeutics to the pulmonary vasculature using stem cell-based drug delivery.1

• Use stem cell-based drug delivery methods to test newly identified interventional targets.2

• Design biologic delivery vectors to provide stable and selective production of therapeutic gene products in the pulmonary vasculature.3

• Use cell-specific diagnostic imaging to deliver disease-modifying drugs directly to relevant pulmonary vascular lesions.4

• Identify biomarkers of treatment response that can guide therapeutic decision-making in pediatric patients suffering from idiopathic PH. 5

• Test new devices for acute right-ventricular support during periods of critically elevated pulmonary vascular pressures that cause right-ventricular failure.6

PH Disease Pathogenesis

As understanding of PH has evolved, new hypotheses have been proposed that better describe its development. ARRA-funded projects include research to:

• Determine the relationship between innate or adaptive immune responses and the development of pulmonary vascular disease.7 8

• Delineate the pathophysiologic and genetic factors specific to the heart that may contribute to the development of integrated pulmonary vascular–right-heart disease.9

• Study the ultrastructural and intracellular components of phenotypes that are abnormal or become impaired in function during the development of PH.10 11

• Probe the biologic reasons why more women than men develop PH 12

Pulmonary Vascular Biology

While PH is well-known as originating within the pulmonary vascular–right-heart axis, it has become increasingly apparent from PH studies that our fundamental knowledge of the pulmonary vasculature is incomplete. ARRA funding is supporting important basic and translational investigations in pulmonary vascular biology to:

• Examine the critical roles of oxygen and oxygen deficiency in pulmonary vascular smooth muscle function.13

• Explore the mechanisms by which other oxygen-based compounds, such as carbon monoxide and nitric oxide, regulate lung cell function during normal physiology, injury, and repair of vascular damage.14 15

• Define the unique characteristics of the cells lining the vascular surface in relation to function and location along the pulmonary vascular bed 16 and uncover critical signals needed for lung endothelial cells to form healthy physiological barriers.17

________________________________________


1. 1RC1 HL100807-01 Prostacyclin-secreting Cells as Therapy for Pulmonary Artery Hypertension – Dixon, Richard (TX)

2. 1RC1 HL099980-01 ACE2 as a Target for Pulmonary Hypertension Therapeutics – Raizada, Mohan and Grant, Maria (FL)

3. 1R01 HL092941-01A1 Development of Double-targeted Vectors for Long-term Vascular Expression in vivo – Curiel, David (AL)

4. 1RC1 HL100849-01 Zipcode Based Nano-imaging of Hypertensive Pulmonary Arteries – Tuder, Rubin (CO)

5. 1RC1 HL099786-01 Quantifiable Biomarkers of Vasodilator Therapeutic Response in Children with IPAH – Everett, Allen (MD)

6. 1 RC1 HL100585-01 Minimally Invasive VAD for Treatment of Acute Right Heart Failure – Corbett, Scott (MA)

7. 3 R01 HL082662-03S1 An Autoimmune Basis for Pulmonary Hypertension – Nicholls, Mark (CA)

8. 1 R21 HL092370-01A2 Muscularization of Pulmonary Arteries Induced by an Adaptive Immune Response – Grunig, Gabriele (NY)

9. 3 P50 HL084946-03S1 Molecular Determinants of Pulmonary Arterial Hypertension – Hassoun, Paul (MD)

10. 3 R01 HL086939-03S1 Vascular Collagen Accumulation & Mechanical Mechanisms in Pulmonary Hypertension – Chesler, Naomi (WI)

11. 1 RC1 HL099462-01 Impaired Mitochondrial Fusion in Pulmonary Arterial Hypertension – Archer, Stephen (IL)

12. 1 R21 HL093181-01A1 Role of 15-lipoxygenase in Enhanced Pulmonary Vasoconstriction in Females – Pfister, Sandra (WI)

13. 2 R01 HL067191-05A2 Chronic Hypoxia and Pulmonary Vascular Smooth Muscle – Shimoda, Larissa (MD)

14. 2 R01 HL060234-10A1 Heme oxygenase1/Carbon Monoxide in Lung Vascular Injury – Choi, Augustine (MA)

15. 3 R01 HL060917-10S1 Nitric Oxide Production and Reactions in the Lung – Erzurum, Serpil (OH)

16. 3 P01 HL066299-08S1 Lung Endothelial Cell Phenotypes – Stevens, Troy (AL)

17. 3 R01 HL088328-03S1 Natriuretic Peptides in Pulmonary Endothelial Cell Barrier Function – Klinger, James (RI)

The quiet side of the fight...

Today was about one of those quiet pieces of work that makes a big difference for PH patients...

Several years ago, PHA noticed that many patients who applied for Social Security Disability were being rejected.  While most negative opinions were reversed on appeal, a lot of time and energy was lost.

That led us to a series of meetings with the Social Security Administration through which it became clear we had two major issues to resolve. 

The first - and quicker to solve - was to provide better training related to PH cases for the Social Security adjudicators.  About a year ago, with the support and cooperation of the Social Security Administration, we were able to script an official SSA training film, through the volunteer efforts of Dr. Adaani Frost.  That film is now available for the adjuducators and you can see it too.

The second issue took a major leap forward today. 

The standards on which the SSA makes disability judgments for PH patients were written at a time when there was much less understanding of the disease. Consequaently, they need to be updated to allow for a fair judgment. For the past two months, a team of PHA volunteer physiciansand lay experts, led by Drs. Ron Oudiz and Robyn Barst entered into discussion with SSA physicians and administrators.  This resulted in the development of a targeted presentation by Dr. Oudiz that was made today.  A very rich and extended discussion led by Drs. Oudiz, Barst and Hap Farber followed.  The education they provided and followups they were invited to provide will play an important role in helping SSA's Working Group complete the task of creating practical, meaningful and fair criteria for PH patients applying for Social Security disability.

A special thanks to Hap Farber, M.D., Alan Harder, Gavin Lindberg, Jennifer Jaff, Katie Kroner and Carol Vreim who worked extensively with Drs. Oudiz and Barst on this project, to Drs.Val McLaughlin, Greg Elliot, Adaani Frost, Mike McGoon, Nick Hill, Erika Berman Rosenzweig and Charlie Burger who provided medical review and to Margaret Beardsworth who staffed the project.

Issue by issue, it's always gratifying to see how ready members of the PH community are willing to jump in when needed to move our common cause forward.

You can see the slides presentation from today's meeting by going here and then clicking on other meeting resources in the right column.

Fitting the pieces...

About 18 months ago, PHA was funded by the Medtronic Foundation to develop a program we had conceived long ago...and have recently grown to make practical.  The Defined Populations Project will let us reach out and create programming in five new areas: newly diagnosed patients, young adults with PH, PH patients with associated illnesses, parents of children with PH and caregivers. Surveys and focus groups have led to advisory groups and now new programming for each of these groups.

Recently, I had the opportunity to begin to explore how we can work with PH pediatricians to integrate into and support the family aspects of these growing segments of the PH community.

Following several calls between leaders of PHA's Scientific Leadership Council and the recently organized PPHnet group of pediatricians, I was invited to meet with them in Banff in the Canadadian Rockies.  They had gathered there for the 3rd International Neonatal & Childhood Pulmonary Vascular Disease Conference and for their own planning session.  The meeting organizers picked a beautiful site. Banff is truly one of the world's special places.

While there are strong connections between the PH pediatricians from this group and PHA - Drs. Ivy and Berman Rosenzweig sit on PHA's SLC and Dr. Barst who is on their advisory group is a past SLC Chair -I appreciated the opportunity to present background on PHA for those who are not as familiar with us.

The conversations were produictive and are continuing.  We look forward to developing this relationship in ways most productive for our patients and the entire PH community.

From 200 to 2,000 in 2 years...

Jack and Marcia Stibbs, Matacha Saul and the great and loyal volunteers of The Woodlands, Texas are rainmakers in support of PH research!

Jack's first event for PH research was a golf tournament in 1999.  It grew and grew, until three years ago, Jack said, "I think it's time for a changeup."

And changeup he did...from golf to bugs. Well, allright, crawfish. 
That first year I went to the Bug Boil. It was a great and sunny day, attracting a crowd of over 200people.  We ate a lot of crawfish, alligator and potatoes, drank a lot of beer and enjoyed a great zydeco band.  It was a great and fun day.

This year, two years later, 2,000 people consumed 6,000 lbs of crawfish, 160 lbs of alligator and 2,000 pieces of chicken.  Oh, and there was a refined name change to Crawfish Festival....with even more fun.

Last Saturday's event had to turn people away - and what I hear from Jack - is already planning on growing from A Houston-area event to a regional event in 2011. 

Over the years, the Woodlands team has raised well over a million and a half dollars for research through their great efforts and built tremendous awareness of pulmonary hypertension in the Houston area.  Despite their strange food tastes (sorry Jack, I couldn't resist!), all of us at PHA are deeply grateful for what they have done and continue to do to fight this disease.  THANK YOU TEXAS!

Enjoy a great slideshow of this event