Duchenne Muscular Dystrophy and The Economics of My Son's Life

It's been almost a year since Gus was diagnosed with DMD. On the drive into work this morning, I was thinking about what I've learned about DMD since that November evening in 2009 when the doctor from Dartmouth called.

It comes down to four things:

1. Duchenne Muscular Dystrophy is devastating. In the 149 years since the identification of DMD, that has not changed. We see it again and again in the numerous letters and cards we've received from people who have lost a loved one. Here's a quote from the most recent card: "Our son was born in 1983, we spent much of our lives devoted to giving him all the opportunities we could afford...he did everything he could with what he had, and he died in September, 2007..."
2. Managed care offers a twinkling of hope. By having a neuro-muscular specialist, a cardiologist, a physical therapist, and an occupational therapist working together, boys with DMD have a chance at a slightly better and slightly longer life. It spite of mounting evidence to that effect, however, there are just two clinics in the US that offer such care. One is in Boston, at Mass General, and one is in Cincinnati, at the Cincinnati Children's hospital. A third clinic is planned at UCLA.
   
3. Research is advancing, but DMD often advances faster. Gene and Stem-Cell therapies offer real hope for a cure, but in the year since Gus' diagnosis, we've already seen great hope dashed with PTC-124. We pray that we haven't entered Bill Murray's Goundhog Day of DMD news, hearing about potential treatments and cures that never go anywhere again and again.
4. This is a rare disease. About 38,000 kids were born worldwide with it last year. That means, that since Gus was born, 190,000 families have heard or will hear the same devastating news that we received last November. Seems big, but it's not enough - if you restrict that number to births in the U.S., it's MUCH smaller - just 1182 children each year. That's the number that keeps us up at night. With so few patients in the world who can pay for treatments, will it ever make economic sense for big pharma to treat DMD?

That's where John Crowley, comes in. In 2003, Crowley, whose kids had Pompe disease (an even more rare genetic disorder than DMD - about 1/400,000 births - or 10 cases each year in the US ) created a treatment at Genzyme. He also created an economic model that makes sense to big pharma. The answer to the economics question? Charge A LOT - about $200,000 annually per patient to treat Pompe.

I'm no mathematician, but, based on those numbers, I think we're looking at about $20,000/year to treat Gus when a treatment comes along. When that time comes, we'll happily pay $20,000. At the same time, we can't fund a drug through trial.. How much does it cost to get a drug through all three phases of an FDA trial? I've posed that question to doctors and execs over the months, and the consensus seems to be about $20,000,000. Only Bill Gates or drug companies have $20 mil to spend on trials (are you listening Bill Gates?).

So what's our role?

We fund the research and the treatments. We find the doctors who are figuring out ways to improve kids lives and fund them. We find researchers who have research that's so promising that big pharma can't ignore it and fund them. We stay up later and get up earlier in order to make the single dollar that will break through it all.

In the end, we ignore the economics and we believe that this is the generation of boys who, with our help, will beat DMD. There are 38,000 kids each year who are counting on us. We can't let them down.

Gobaith am Gus - The Hope Goes International!

We were very excited last week when our Welsh friend Kevin Phelps e-mailed that he was inspired to run a 20k race "along the South Wales coast," and the Cardiff Half Marathon in honor of Gus. Kevin is asking for sponsors and plans on giving everything that he raises to The Hope for Gus Foundation. If you're interested in helping Kevin help us, you can donate through our Donate Page - Let us know it's for Kevin in the note.

Thank you Kevin and thank you everyone!

p.s. We did mention to Kevin that the large sign (Hope for Gus in Welsh) might throw off his gait, but he's committed!

Cousin Jake

I wanted to share a poem written by Jake, Gus' oldest cousin. Jake is in 8th grade, and he has shown us his maturity, compassion and love for his cousin by organizing a fundraiser last year at his school that netted $600 (for that, he made it onto our Heros page here).

Recently, Jake approached me with a poem/rap that he wrote in honor of Gus. We hope to get him into a recording studio to record it soon. Gus has a special connection with Jake as well, and this poem shows why:


Hey, hey hey my cousin,
One day I heard,
You have a bit of a problem,
Like something with your muscles isn't working quite right,
But let me the first to say,
There's nothing wrong with you in plain sight,
And I know you're strong,
And I know you're brave,
And nothing in this world,
Can make your smile go away,
Because you're as powerful,
As the sun is bright,
And you won't go down,
Without putting up a tough fight,
We give you hope,
And give you support,
And you have a spot,
In all of our hearts,
We love you as much,
As the sky is blue,
And even on a cloudy day,
We can see the perfection in you.

Most kids wear braces,
On their teeth,
But you wear braces,
On your legs, ankles and feet,
And you take medicine,
To help you along,
But that's not what really,
Make's you strong,
No, it can never compare,
To the care that we give,
And can never measure up,
To your will to live!

The thoughts that run,
Through your mind all day,
Must only be,
Live laugh love and play,
And when I see,
Your face light up,
It shows that you too,
Know we have Hope For Gus,
Yes, we have hope,
Hope for you,
For every amazing thing,
You have done and will do,
Yes our hope is more powerful,
Than any disease,
And what I'm saying here,
Isn't just coming from me,
It's coming from everyone who's been affected,
By DMD,
And we all have Hope For Gus, little buddy!!