Wednesday, May 20, 2015

PHA Brings One-Day Regional Conferences to Patients and Families

When I came to PHA in 1999, one of the first things that struck me was the excitement and anticipation surrounding our International PH Conference. Given the amount of work to produce these very special events, the reason PHA hosts them every two years, instead of annually, also became clear. However, that 24-month gap led to a dream by Board members: someday PHA should produce annual regional conferences, events where people wouldn't have to travel as far to receive the valuable education and networking opportunities that such meetings offer. 

It took almost a decade but in 2009, PHA had grown to the point where we were able to make the dream a reality. The vehicle we created is PHA on the Road, the patient component of PHA’s Medical Education Program, and in this guest blog post from PHA staffers Jodi Holland and Megan Mallory, we share the exciting plans for PHA’s seventh year of PHA on the Road. We hope you will join us in one of our four destination cities.


PHA is hitting the road again this year when PHA on the Road: PH Patients and Families Education Forums visits Atlanta on June 20, Philadelphia on July 25, Phoenix on Oct. 3 and St. Louis on Oct. 10. PHA on the Road recognizes that the pulmonary hypertension patient is the heart of a larger PH community that makes support, connection, information and education available to anyone who needs it. Since the program’s inception in 2009, these free patient-focused educational forums have featured interactive presentations, general sessions, time to meet other patients and caregivers, and exhibits. Forum speakers are leading PH healthcare professionals from the local area.

As Sarah Wisdom, from Yuma, Ariz.,
who has a family member with PH, shares, “[The forum was] very informative and [provided] great information for our whole family.” Patient Rebecca Talob, from San Diego, Calif., agrees, adding that the healthcare professionals who sat on session panels “were great and explained things so they were understandable and useable.”

Many patients also appreciate the opportunity 
PHA on the Road offers for networking. For some, these forums are the only time they and their family meet other patients. “It was very encouraging to know we're not alone,” Sarah notes.

General Session Topics at the Forums May Include:

  • Diagnosis: How is PH Diagnosed?
  • PH Treatments: What are the Choices and Which One is Best for Me? 
  • Long-term Medical Management of PH
  • What is on the Horizon? Clinical Trials and Drug Development

Breakout Session Topics at the Forums May Include:

  • Emergency Situations
  • Exercise
  • Insurance Issues
  • Intimacy Issues
  • Nutrition
  • Pediatric PH
  • PH and Emotional Wellbeing
  • PH and Other Associated Conditions
  • Traveling with PH

To ensure that patients, caregivers and family members get the most out of PHA on the Road, the forums are offered free of charge and include lunch and a kids' room where qualified volunteers and staff keep an eye on children as they play games while their family members receive this invaluable information.

For more information about PHA on the Road or
 to register, visit www.PHAssociation.org/OnTheRoad, contact OnTheRoad@PHAssociation.org or call 301-565- 3004 x763.

PHA on the Road is a program of the PHA Medical Education Fund, made possible 
by unrestricted educational grants from Actelion Pharmaceuticals US (Diamond sponsor), Gilead Sciences (Platinum sponsor), and United Therapeutics and Bayer HealthCare (Gold sponsors).

Wednesday, May 13, 2015

PHA Offers Grants to Support PH Research

This week’s blog is an invitation to researchers to seek PHA funding through our multiple funding programs. It is also a celebration of what a committed rare disease community can do to advance knowledge that will lead to change and a better future. The research that PHA began to fund in 2000 has become building blocks for what came next and what will come tomorrow. Almost all of the money that PHA invests in this effort has come from members of our community who have organized special events and/or given generously themselves.  

A by-product of PHA’s research efforts was highlighted recently by Dr. Karen Fagan, Chair of PHA’s Scientific Leadership Council, when she studied where young researchers go after receiving a PHA research grant. Looking at our sponsorship of Career Development Awards with the National Institutes of Health, Dr. Fagan found that ALL researchers who were awarded this grant from PHA have stayed in academic medicine and importantly in the area of PH-related research and clinical care. This means PHA is not just funding grants. Our donors are making it possible and attractive for the most promising clinicians and researchers to continue in PH research.   

An important part of PHA’s mission is to find ways to prevent and cure pulmonary hypertension, and to that end, PHA has committed more than $15 million, over the last 15 years, to researchers seeking to better understand the disease. This year, PHA is making more than $500,000 available to support scientists who are looking for answers that could lead to better outcomes, new therapies and, ultimately, a cure for PH.

Thanks to PHA’s generous community members and historical partnerships, PHA’s Research Program provides support for PH research projects ranging from early, exploratory and developmental work to projects that facilitate mentorship and ensure a future of promising, innovative investigators in the field.

Our donors know that their generosity is leveraged to create even more opportunity for researchers to learn about and change the history of this disease. For example, PHA’s KO8/K23 award annually combines $312,000 in PHA contributions with over $500,000 from the NIH to create an over $800,000-per-year research program. Other PHA research programs are enlarged through similar matches.

In 2015, PHA’s Research Portfolio includes funding for new awards in:

  • The first program focused on pediatric PH research 
  • A grant that encourages investigators to explore novel ideas and avenues that could enhance the understanding of PH 
  • An award that supports faculty-level investigators studying the diagnosis, pathogenesis, treatment or outcomes of PH 
  • A grant that offers support to a recipient of a mentored career development or patient-oriented research grant awarded by the National Heart, Lung, and Blood Institute (NHLBI)

Researchers, be sure to apply for one of these grants. Patients and families, we hope you will share this information with the healthcare professionals in your lives. The 2015 PHA grant opportunities include:


Learn more about PHA’s 2015 funding opportunities, including eligibility criteria and deadlines.

Wednesday, May 6, 2015

World PH Day... and Beyond

Today’s blog was co-written by PHA staffers Megan Mallory and Renee Hockaday. They make the point that awareness doesn’t begin and end on World PH Day. It is an everyday necessity if we are to succeed in our efforts to achieve better lives for PH patients and all those living and working with this disease.

Yesterday, on May 5, pulmonary hypertension patients and their loved ones asked their social media friends around the world to join their fight against a life-threatening disease too often dismissed as asthma. Our PH community did this to mark World Pulmonary Hypertension Day, and we joined in a global effort to educate people about PH and bring awareness to this disease.

The fight for awareness does not end after one day. If we are to succeed, awareness-building must continue today and every day. If you missed out on yesterday’s social media blitz, we ask you to join this awareness movement today.

We have designed a World PH Day celebration social media pin with the message, “PH: A disease of many stripes,” to symbolize the diagnostic challenges and how PH comes in different forms, sometimes without a known cause and in other cases, associated with other diseases. Please share this pin with your social media followers to lead them to PHAssociation.org to learn more about PH, find healthcare resources and/or make an online donation.

Please post this social media pin with the following messages on your website, Facebook, Twitter and other social media pages. Or if you want, create a message of your own. Here are our suggested messages:

Facebook:
Pulmonary hypertension may look like asthma. But on average, the survival rate for those untreated is 2.8 years. For World PH Day, share this message and World PH Day pin with 5 people you love. http://owl.li/LVTdB

Twitter:
World PH Day: the other May 5 celebration. http://owl.li/LVTdB @PHAssociation #PHAWorldPHDay #ILovePHA #PHAssociation

Did you know?
Yesterday also marked the beginning of PHA’s Days of Unity. Days of Unity are events and fundraisers hosted by individuals and organizations to raise money to fight PH. The events range from small walks hosted by PH patients to an endurance bike race fundraising effort led by Team PHenomenal Hope, which raised nearly $117,000 for PHA as participants in the endurance bike event Race Across America in 2014. Our 2015 Days of Unity events will conclude on June 20 after Team PHenomenal Hope completes its 860-mile Race Across the West from Oceanside, Calif., to Durango, Colo. Learn more and get involved

An Unexpected Gift for Support Group Leaders
In honor of World PH Day, Pulmonary Hypertension Association support groups got a special delivery. Gerry Fischer, president of PHA Europe, donated books written by his daughter, PH patient Maleen Fischer, to support group leaders. Maleen's book,“Hope Springs Eternal,” is about her PHight with PH. On behalf of all of PHA support group leaders and members, PHA thanks Gerry and Maleen for this thoughtful donation!

Wednesday, April 22, 2015

4 Stars... Building Longer, Better Lives

Shortly after I came to PHA as its first employee in 1999, I was invited to attend an annual meeting that the National Heart, Lung, and Blood Institute of the National Institutes of Health organized for nonprofit organizations in NHLBI’s area of medical interest.

I was pretty excited to attend. It was my first chance to connect with other disease associations. I was hoping to learn something from them … and I did. During the breakout sessions, conversation was dominated by one disease that had four separate organizations. They were all shouting at each other and not accomplishing much. To my knowledge, that’s still the case today.

The lesson of the day for me was that PHA needed to be built as much more than an organization. It needed to be a community, a community that reaches across all constituencies whether they be patient, caregiver or medical. It was important that PHA be managed to its mission by people who could work in solidarity across the many issues that will make life better for PH patients. Fortunately, that lesson was consistent with our founders’ vision.

Does that approach of solidarity in community-building work? Well, there’s a lot that could be written about what PHA has accomplished over the years, but that will be for other blogs. This one is about people looking at this organization from the outside.

According to its Wikipedia profile, Charity Navigator was launched in the spring of 2001, with the mission of helping "donors make informed giving decisions and enabling well-run charities to demonstrate their commitment to proper stewardship" of donor dollars. Initially, Charity Navigator provided financial ratings for 1,100 charities. Charity Navigator currently evaluates more than 5,400 charities in the United States.

Due to their comprehensive review process, Charity Navigator quickly emerged as the most respected of the nonprofit evaluators. PHA became one of those evaluated charities in January of 2004, receiving their highest rating – 4 stars.

In April of this year, PHA received our 12th consecutive 4-star rating. As Ken Berger, Charity Navigator’s CEO, writes, “Less than 1% of the charities we rate have received at least 12 consecutive 4-star ratings, indicating that Pulmonary Hypertension Association outperforms most other charities in America….”

Our score this year, at 98.55 (out of 100), was our third highest ever.

PHA has also been recognized for excellence by many other organizations – including the Better Business Bureau, GuideStar Exchange, Independent Charities of America, Great Nonprofits, National Organization for Rare Disorders (NORD) and the Center for Nonprofit Advancement – based on a variety of standards.

We have accepted these awards as the validation of a system that is essential to success in a small rare disease state. That is, we move forward because of the strength and support of a single and powerful community: people like you, bringing the “power of one” to the table for the good of many.

Wednesday, April 8, 2015

Four Days in March...

Usually, we see things in pieces. Once in a while, we are privileged to see those pieces come together… to understand the greater whole.

The PHPN meeting on Thursday
During the course of four days beginning on Thursday, March 26, I had that opportunity. We began on Thursday morning with a daylong meeting of the PH Professional Network (PHPN) executive committee led by Melisa Wilson, a nurse practitioner from Orlando, Florida. PHPN, founded in 2000, is a structure within PHA to involve nurses, nurse practitioners, physician assistants, respiratory therapists, pharmacists and other non-MD healthcare professionals working in PH. It has become an essential and highly valued body for education and networking in the field. There was much discussion at the meeting about the upcoming PHPN Symposium. This event, which PHPN organizes every two years, has become the largest PH meeting of non-MD healthcare professionals in North America. Among many other topics, PHPN leadership also devoted considerable time to discussing the development of publications and supporting the quality and accuracy of PHA publications.

As PHPN continued its work in the afternoon, the PH Care Centers (PHCC) leadership began their meeting to discuss the new registry PHA is currently building. The registry is based on an understanding that PHCC accreditations can only achieve full value for the PH community when we look across all centers to create and share data to better understand what works best for patients. One of the compelling reasons for PHA to take the significant financial risk of building this registry was a review of the cystic fibrosis registry results. Over a 24-year period (1986-2010) during which there were no disease-specific treatments, patient survivability increased by over 11 years. While other factors, such as earlier diagnosis contributed to this advance, there is no question that shared knowledge of what works, flowing through the work of the registry, was an essential pillar of this success.

The Registry Committee of the PHCC, led by Dr. Steve Kawut of the University of Pennsylvania, is working to build a similar pillar in PH. It was gratifying to see that leaders of five registries working in PH came to this meeting to discuss creating a consistency of structure that would allow patient data to be used in multiple registries. If successful, this will allow more rapid development of knowledge in the field.

So that was our first day…

We began Friday with an all-day meeting of PHA’s Scientific Leadership Council (SLC). This international and globally-regarded group of medical experts in the field oversees PHA’s entire medical structure. This includes research, our multiple medical education programs, the development of our accreditation and registry structures, and the accuracy and relevance of the medical information we provide to patients and their families.

The SLC meeting had much discussion of the rapid progress we are making on the new PH Care Centers accreditation program. With a target of 60 accreditation reviews by the end of 2015, we have already completed 20, with another 20 submitted and being processed for more information or site-visits, and an additional 17 online applications in process of completion. A little over three months into the year, we are ahead of schedule.

Research updates related to PVDOMICS
A highlight of our research discussion was PHA’s new research partnership with the National Institutes of Health. This relates to the PVDOMICS program, the importance of which was described in a guest blog by Michael Gray.

While the SLC meeting continued, PHA’s Board of Trustees Committees began meeting in the early afternoon. The Development Committee discussed how to fulfill their obligations to help raise the funds for PHA to fulfill its mission. Harry Rozakis, a CTEPH patient, chairs the Development Committee.  

The Governance Committee also met. They are chaired by Laura D'Anna, a former PHA Board chair who lost her sister to PH. This Committee reviews PHA's bylaws - our organizational rules - and works to make sure the Board functions well to oversee all of the many issues in which PHA is involved.

The Strategic Planning Committee, chaired by John Hess, the parent of a child with PH, is charged with overseeing the development of our multi-year plan that tells us where we want to go so we have a direction to steer the organization.

A new committee also met: the Search Committee. They are charged with finding my successor as President/CEO when I retire after PHA's International PH Conference in 2016. Their first task will be to find a search firm that fits well with PHA's goals for the position. PHA's Board chair-elect, Roger Towle, the father of a PH patient, is leading this important effort.

To be clear on those goals, PHA's Board has to have strong focus. To assure that focus, they had an all-day session on Saturday with a consultant from Board Source. Board Source is the most highly regarded nonprofit organization dedicated to nonprofit management and governance issues. PHA is a pretty complex organization these days, much more so than when I started as the first staff person 17 years ago. Does the Board seek someone with a medical background, one in organizational development, corporate or nonprofit management expertise, or the ability to raise funds? Those (and more) are the kinds of questions that will define the next stage of PHA's growth.

The Saturday session was very productive in defining the Board conversation and led us into an abbreviated Board meeting on Sunday where many of PHA's medical and patient-serving programs where presented and discussed. The Board is led by Steve White, an Episcopal priest with a doctorate in health management. Steve lost his daughter to PH.

I should also mention that the Board had the opportunity to meet with over 100 Texas support group leaders and members who had come to the Dallas Omni where PHA will have our upcoming 2016 International PH Conference and Scientific Sessions. It was a great opportunity for the Board to meet our local hosts and share our common excitement about the Conference that will take place in PHA's 25th anniversary year!

In closing, I've always felt the all-volunteer PHA Board is a pretty good reflection of the PH community of patients, caregivers and medical professionals. The members put in a lot of time dealing with the many complex issues a rapidly growing organization must face. Seeing all the meetings I described flow from one to the other during our recent four-day span and understanding the interlocks between those meetings, my view is that PHA is not a series of activities but a single organization that connects those activities for maximum impact for the good of patients. It has always been my privilege to work for PHA.


WATCH for Rino's next blog: PHA's 12th straight Charity Navigator 4-Star rating.  

Wednesday, March 25, 2015

Make the Halls of Congress Ring on National PH Call-In Day

This week, PHA’s Advocacy and Awareness Director, Katie Kroner, is our guest blogger. Katie describes an important – and easy – way we can make a real difference in the lives of PH patients (and their families).

PHA staff make calls during
National PH Call-In Day 2014
April 16 is National PH Call-In Day – a day to make the halls of Congress ring with the PH community’s voice. You can take part in less than 10 minutes, without leaving your home.

Why me?
A friend of mine was talking with a staffer in Rep. Chris Van Hollen’s (D-MD) office last week. When she mentioned PH, the staffer jumped in and started to tell the story of a young woman who visited his office almost a year ago and shared her experience of being diagnosed while working in Afghanistan. He told me that he'll never forget the way she described her pain from having pulmonary hypertension: "I could breathe in, but my lungs didn't feel like they were getting any oxygen." He remembered her words a year later and said his boss, Rep. Van Hollen, remembers them too.  He was telling PHer Jessica Armstrong's story.

Everyone whose life has been touched by PH has a story to tell and your story can make a difference. Join PHers across the country on April 16 and make sure that every Member of Congress knows about PH.

How can I help?
PHers Jessica Armstrong (left) and Katie Tobias
advocate to the Food and Drug Administration
On April 16, PHers across the country will call their senators and representatives and ask them to co-sponsor the Patients’ Access to Treatments Act.

The bill aims to limit patients’ out-of-pocket costs for complex, life-saving medications by capping possible co-insurance. For PHers, that means a limit on what your insurance could require you to pay for infused and inhaled PH drugs. The bill is specific to commercial insurance, but its success would likely impact Medicare and Medicaid too.

Learn more and take action.

What Should I Do on April 16?
Follow these steps to add your voice on April 16:

  1. Use PHA’s tools to look up phone numbers for your senators and representatives and view a call script. All you need are your street address and zip code: www.PHAssociation.org/ActionAlert/PHNationalCallInDay 
  2. Make the calls! Ask your senators and representatives to co-sponsor the Patients’ Access to Treatments Act.
  3. Tell PHA … and your friends! Don’t forget to fill out the call feedback form that appears after the talking points. PHA will use this information to help hold your Members of Congress accountable. Then, post the alert to social media or email it to your friends. Anyone can join the “phight” on National PH Call-In Day!
  4. Congratulate yourself! Thank you for adding your voice to this critical issue!

For more information, or to join PHA’s grassroots advocacy network, contact Angelia at 301-565-3004 x753 or Advocacy@PHAssociation.org.

Wednesday, March 11, 2015

PVDOMICS and What It Means for the PH Community

Since 2000, PHA has created five distinct research programs through which we have committed almost $16,000,000. Each year, through those programs, we support about a dozen research projects. Each year, through our partnerships, our donors' research dollars are doubled.  

PHA's most recent research partnership - our second with NIH - will help expand a new program that is generating more excitement among researchers than anything we've seen before. PHA's $500,000 commitment, while adding 2 percent to the overall budget, is allowing the program to expand by 20 percent. We feel that's a great investment!

This week, I'm pleased to introduce PHA's Senior Director of Medical Services, Michael Gray, as our guest blogger. He explains the new PVDOMICS program and what it means to the PH community.  

As I turn this page over to Michael, I should tell you that PH is the first disease that will be studied in this way. NIH has told us we are preceding the diseases that will follow because of the strength and structure of the PH community. As part of this community, you are making this important advance possible.

"The only simple truth is that there is nothing simple in this complex universe. Everything relates. Everything connects"
-Johnny Rich, The Human Script

For centuries, scientists have been fascinated with how traits are passed from one generation to another. Significant progress has been made in this understanding, from Gregor Mendel’s discovery of the laws of inheritance with pea plants in the mid-nineteenth century to Dr. Marshall Nirenberg decoding the language of DNA at the National Institutes of Health (NIH) in the mid-twentieth century. Despite our increased understanding, we still have not solved how genes and changes to your genetic code called “mutations” affect most diseases, including pulmonary hypertension. A new initiative through the NIH, the “Redefining Pulmonary Hypertension through Pulmonary Vascular Disease Phenomics” (PVDOMICS) program, seeks to change that for this particularly devastating disease state.

Mutations, or accidental changes in the DNA code, can occur frequently as cells duplicate, and can even be passed onto the next generation when this mutation occurs in a sperm or egg cell. Mutations sometimes will cause no changes to the individual, sometimes will cause harm to the individual, and sometimes can even provide a benefit. Genetic changes like this have been directly implicated in a number of diseases, including cystic fibrosis, sickle cell anemia, Tay-Sachs disease, and phenylketonuria. Genetic changes have also been implicated in PH, most famously with the discovery of the “bone morphogenetic protein receptor, type II” (BMPR2) gene. However, we don’t know exactly how changes in the BMPR2 “code” lead to PH. We know that BMPR2 genetic mutations are present in more than 70 percent of people with hereditary PAH and 25 percent of people with idiopathic PAH; but having the genetic mutation does not mean you eventually have PAH.

Think of DNA like letters in a book. Individually, these letters have no meaning; together, they tell the story that makes you, you. This story can be changed in a number of ways. Certainly letters and even entire words can be added, removed or changed, which might or might not change the meaning of the story. But direct changes to the letters on the page is only one modification that can have an impact on how you read the story. Maybe your book has a sentence that has been highlighted, or bolded; the words and the meaning of this sentence have not actually changed with this modification, but it has been emphasized in a way that has consequences in context. Maybe your book has a stain on one of the pages masking several words; the words are still there, but hiding these words could change the entire meaning. These different types of changes to how the “story” of your DNA is told make up a web of interactions that can have consequences nearly as drastic as finding one disease causing “gene.” This web includes the molecules that take chapters of your DNA “story” from the nucleus to your entire cell (called RNA), the proteins that are made by this RNA and used by your entire body, and how your cell uses energy to name just three strands of this web.

The NIH in Bethesda, Md., is the world’s largest biomedical research institute, with more than 5,000 scientists working on a number of scientific advancements with basic scientists, physician scientists, patient advocacy organizations, pharmaceutical companies and, of course, patients and family members themselves. The NIH has a long history working in pulmonary hypertension, beginning more than 35 years ago with an early, important registry characterizing the devastating nature of PAH. Since then—with significant advances in the field including 12 PAH-targeted therapies, a successful surgical therapy for CTEPH, and a medical therapy for a specific form of CTEPH—the NIH continues to invest in successful PH research. The most recent demonstration of this is the PVDOMICS program.

PVDOMICS seeks to ask research questions about PH in a way that will lead to a better, more complete understanding of the disease and to developing therapeutics that are more precise by building a clinical research network of seven centers across the United States. They will be performing a comprehensive analysis on around 1,500 patients with all types of PH (WHO Groups 1-5), collecting a range of clinical information; blood for analysis of genetics and the molecules that turn on and off expression of these genes; and imaging studies normally used in the clinic to better understand the difference between normal and diseased lung vascular tissue. There are many benefits from analyzing these very different types of information together, according to the NIH. If the investigators are able to develop this robust, more complete understanding of why some people develop PH and others with similar risk factors do not, more sensitive measures of diagnosis, more uniform responses to medications, and new biomarkers of disease worsening could be discovered. It is anticipated that most WHO Group 1-5 pulmonary hypertension patients who want to participate in this research study may be able to enroll.

The Pulmonary Hypertension Association is a proud partner with the NIH in this important study, and in 2014 awarded a five-year grant of $500,000 to the program to support one of the clinical centers in collecting data from 250-300 patients. For more information on the PVDOMICS program, please see the Pathlight article on PVDOMICS from the winter 2015 issue.