PARENT PROJECT CONFERENCE 2008 A HUGE SUCCESS

It was the middle of the Friday afternoon session and I was searching for a place to plug in my laptop. You know how no one likes to sit in the front of the room? Well, all the desks up there were empty – except one where scattered notebooks and papers marked out someone’s territory. This was, of course, right beside the one empty power receptacle. I sat down anyway. Hopefully, whoever they were, they wouldn’t mind.

They didn’t. And while Jeff Chamberlain, champion of gene therapy, talked about exons and virus delivery shuttles for dystrophin, the missing gene in duchenne dystrophy, we introduced ourselves. They were Wendy and her husband, Brad, this was their first Parent Project Conference and my fourth, and I felt like I was coming home.

At this Parent Project conference, as in years past, parents of boys with duchenne met with researchers who will someday, hopefully soon, find a treatment for our sons. We network, learn about advocacy with Congress, NIH, and the FDA and how to care for the bodies, minds and souls of our boys. And when our boys become men, like my Pete, we learn to turn that care over to them.

Wendy and Brad were studying what looked like a lab report, similar to the one Pete got a few months ago when a lab rechecked his dystrophin gene deletion at exons 48 to 52.

As for exons, after ‘how old is your son’, the next question duchenne parents ask each other is, ‘what’s his deletion?’ So I asked and Wendy held up the report with a look of embarrassed perplexity. These reports are not a bit parent friendly. Wendy said they didn’t know and had been trying to figure it out.

“May I?” I asked and looked over the report.

Their son, Juan, was five and had been diagnosed just weeks ago. After the doctor told them Juan had duchenne muscular dystrophy, after the stunned silence of having the light in your heart darkened, he asked them to leave - his next appointment was waiting. Questions? He suggested they try the web – it has all the latest information about research and care.

Did I think their son could qualify for the PTC-124 clinical trials? Of all the trials for duchenne dystrophy the PTC trial is the most advanced. When it becomes available, it will help 10 – 15% of the boys with duchenne who have misplaced stop codons – a message in their dystrophin gene that tells the protein making machinery to stop too early, before the protein is made. Juan’s deletion spanned exons 48 – 51- not a stop codon.

One of the big surprises of the conference was the announcement that Genzyme was investing $100,000,000 in PTC and PTC-124. Obviously, they think it’s a winner too.

Besides meeting new friends like Wendy and Brad, I saw some dear old ones. Some of my favorite people, the Gapko’s from Wisconsin and Adele and Jason Abramowitz from Maryland, were there and we got the chance to go out to dinner together. I felt strangely empty-armed but free. No Pete to feed, get into bed, scratch itches, bathroom – I felt exhilarated and at the same time, I felt guilty for feeling so good.

I met a few ‘legends’ in the duchenne community; Wil Verheyen, father to Wilson, Canadian, and retired member of a foundation that supports duchenne research, and Nick Catlin, of actionduchenne fame. Deb Miller from CureDuchenne, and Tracy and Ben Seckler from Charley’s Fund were there. Big Pharma was there in record numbers: AVI, Prosensa, and PTC, who each have candidate therapeutics in clinical trials. Other industry representatives were there as well. And MDA.

MDA Vice President of Advocacy, Anne Kennedy, Head of Research, Sharon Hesterlee, and Chairman of the Board of Directors, Dr. Rodney Howell, were at the conference. I was thrilled to see the two giants of the duchenne community, Parent Project and the Muscular Dystrophy Association, working together. Such a partnership will guarantee the shortest time to a treatment.

Pat Furlong, President and Founder of Parent Project graciously gave me time in her busy schedule to talk about the Foundation and how far the group has come. We both learned a lot since those early days in Tennessee with the failed myoblast transfer therapy we held so much hope for.

Pat spoke about the first conference in Florida, how Parent Project has blossomed over the years and now this conference has grown to include over 400 participants, with 7 staff members running the show. The conference was so packed with information and the food was so good, it was a remarkable bargain. Pat said that subsidizing the cost of the conference so that more parents could attend was Parent Project money well spent.

The highlight of the conference came for me on the last day, when a panel of five young men with muscular dystrophy took to the stage and spoke candidly about their lives and their disease. Jacob Gapko is a PhD student in library science, whose dissertation project includes a bibliography of duchenne literature. Pat Mosechen is a counselor and middle school teacher and all around comedian (Seriously, he could go into the business). Jason Abramowitz is a sports enthusiast who plays soccer and hockey in wheelchair leagues all over the country. Conrad Reynoldsen is a congressional aide in California, who talked about his job and how he made it work. And Josh Wenheld, who’s critically acclaimed book, “Worth the Ride” was a hot item at the conference, regaled us with the business side of writing a book. My son would have fit right in!

Thank you guys. You showed us that you are not duchenne. You are not a wheelchair. You chose making your lives work. You’re blazing a trial.

Parent Project Conference This Weekend, July 17 - 20th

Dear Readers,

Have you seen the agenda for the Parent Project 2008 Conference? They address everything!

How far are we from a treatment for duchenne? How do we face the day to day problems that duchenne brings with it? Genetics, care considerations, psychology, social skills, steroids, cardiac and respiratory care, physical therapy, exons, clinical trials, drug development - they're all there. And that's only the beginning.

One of the most important sessions of the whole conference takes place Friday morning. Lead by Elizabeth McNeil, Medical Officer with the Food and Drug Administration, this session talks about what it takes to bring a drug to clinical trials. In the near future, we will need to approach the FDA and get our 'parent' message across in the best way possible so this is valuable information.

I was particularly pleased to see that Parent Project divides the care sessions by age categories. It makes sense that someone with a newly diagnosed 4 year-old is light-years away from needing all the information that goes with caring for a nineteen year old.


PAT FURLONG

Taking one moment, I would like to acknowledge the debt we parents and our children with duchenne muscular dystrophy owe Pat Furlong. She is responsible for galvanizing the parent activist movement: she started the Parent Project.

Thank you, Pat. Sincerely, thank you.

THE BEGINNING

I met Pat at Dr. Peter Law's facility in Tennessee when Pete, my son, was eight years old (he is now 25). Back then, she was another parent like me, who was there to find out how to help her boys. The next time I would meet her would be at the Parent Project conference in Florida.

That conference was the beginning.

I remember it for many reasons, not the least of which is an oil-pan-ripping tire I ran over in the middle of the night in the middle of nowhere, on our way to the conference (I didn't even know what an oil pan was until then). Somehow we made it.

There was an intimate feeling there. Parents and scientist mingled together, getting to know each other. The man who is my boss today, Eric Hoffman, sat at our table one night for dinner. Terry Partridge and others I met there, now feel like old friends.

I thought they were all gods. I still look up to them.


STAKEHOLDERS

John Porter from the National Institutes of Health and Kate Busby, the head of Treat-NMD, the European Muscular Dystrophy network, will be presenting. Meeting Kate for the first time earlier this year, I knew she was a gem. She is focused, compassionate, and cares about all our boys.

Big and Little Pharma will be at the conference. At the Wellstone Annual Meeting in May, John Porter spoke about Pharma, clinicians, researchers and parent organizations working together to reach our common goal: a treatment for duchenne.

Parent Project, MDA, FED, cureDuchenne and all the others; we are the stakeholders. These organizations don't just represent us, they are us. Are we committed to doing our part to bring a treatment for duchenne to clinical trials?

See you at the conference.