The first pediatric PH research fund is opening its doors…

In little more than a decade and a half, we have seen incredible growth in the field of pulmonary hypertension.  We have gone from no treatments to nine, to being a disease of little notice in the medical community to one with great and growing educational structures, from hopelessness to hope.

It is now time to bring that same progress to pediatric PH.  Opening the doors for the RobynBarst Pediatric Research and Mentoring Fund is an early and essential step.

Here is an updated version of the note I sent to PHA’s Board and staff about a week ago …

On Saturday evening September 22 at about 7:00pm Pacific, Carl Hicks announced that the Robyn Barst Pediatric PH Research and Mentoring Fund reached its $1,000,000 stage 1 goal.  The doors for the first ever pediatric research fund for PH are now opened!

While the goal was reached at the at the Thirsting for a Cure event in Oregon, it was the culmination of a two year communitywide effort. We owe a great deal of thanks to a great many people…to the Cardiovascular Medical Research and Education Fund (CMREF) for their leadership grant, to Steve White and his family for their generous pledge to get us on our way, to the Linnens, Srinivasans and MacDonalds for their special generosity, to Actelion and United Therapeutics for their high-level sponsiorships for Thirsting for a Cure, to many other donors, to Carl Hicks whose impatience to get us to our goal led to the event that brought us over the top and to Actelion CEO, Shal Jacobovitz who called Carl just before the event and committed to fill any gap to get us to the $1,000,000 target for activation of the Fund.

Most of all, we owe Dr. Robyn Barst for her work in pulmonary hypertension as a clinician, a researcher and builder of the field, especially in the once lonely area of pediatric PH.  Robyn’s generous lead gifts allowed us to establish the Fund and move it from dream to reality in little more than two years.  Her vision in including a mentoring element will help ensure the development of future pediatric PH experts.  Robyn, we are grateful.

Dr. Serpil Erzurum, chair of PHA’s Research Committee recently led a meeting to plan for anticipated implementation of Barst Fund activities.  Based on that meeting, we contacted and rapidly reached agreement with the American Thoracic Society for grant management services. We will now begin to work with them to establish program guidelines based on our existing proposal, set application and review deadlines and make our first grants.

In closing, Saturday’s event was a lot of fun with a lot of wine at the Alexana Winery in Newberg, Oregon.  Mike and Bonnie McGoon, John Hess, Betty Lou Wojciechowski (and family), Mary Jan Hicks, Bruce and  Rita Brundage, Gail and Denis Hayes, Gareth Gwyn and many others joined us, including Charity Sunshine who sang for the gathering.  The Portland Metro Support Group did great work at the registration table and beyond to make the event the success that it was.  Carl got a lot of people driving happily down a long gravel road to get to this event…and more happily back.  Well done. 

The Robyn Barst Pediatric PH Research and Mentoring Fund has gone from dream to reality.  Now it’s time to leverage that reality to growth of the pediatric PH field – for the good of the kids and their families living with this disease…today and tomorrow.

A strange world...

In 2001, Alicia Mundy wrote Dispensing with the Truth.  It is the story of the battle over fen-phen, the drug combination that was ultimately withdrawn from the market because some patients who were taking it developed heart and lung damage, most notably pulmonary hypertension.

Many of PHA's members and some of our board members became PH patients after taking fen-phen.  Indeed, Alicia Mundy's book opens with the story of Mary Linnen, the daughter of former PHA board member Tom Linnen and his wife, Mary Jo.  As the liner notes describe Mary, she was "a healthy young woman...who took the drugs for only twenty-three days to lose weight before her wedding, and then died in the arms of her fiance a few months later".

I've been thinking a lot recently of Mary and Tom and Mary Jo and others like Candi who lost their health or their lives or family members to PH developed through fen-phen (Pondamin and Redux).

On July 13, the New York Times reported under the headline F.D.A. Review of Diet Pill Relieves Investors that the drug Qnexa had received a positive staff review of it's safety and effectiveness.  The maker's shares immediately soared 15% while two competing drugs soon due for FDA review went up 9% and 21 %. 

I understand that weight loss is the holy grail of American life but I - and all of us who are part of the PH community - have seen the collateral damage.  Qnexa contains the amphetamine phentermine -- one half the ingredients of the fen-phen combination.  The makers would say, it's the safe half. 

Maybe.

On June 15, the New York Times carried a second report that the FDA panel of expert advisors reviewing the drug on June 14 had voted 10 to 6 to recommend against approval of the drug based on safety concerns.

Now the FDA has a choice.  We'll see what they do.