Who gets the award for finding the treatment or cure? I received this question over and over and yesterday again, in an email from a parent. She provided a list of each organization and what projects or companies they have supported. In my head, I wanted to say all of us, some of us, what does it matter as long as the drug is developed, the project funded. Her real question was –if I raise money for you, will you get ‘the cure’ for my son? My first thought was to say – ‘cures’ do not come from one group or organization, it does not work that way. Best guess for the cost of developing a new drug is right around the $800 million dollar range and I doubt that anyone would have that ability and/or want to invest every last dollar in a single program. You already know the numbers, 1 out of 8 compounds ever makes it to approval, so it makes sense that any number of investors come in at different times, sharing risk. I went to bed thinking about it, thinking how, for many of us who support research, the projects we fund feel like they belong to us somehow. The projects and the individual who run them become our children in a strange sort of way.

Let’s imagine you are considering having a child. You think of the options: the impact on your life, the impact on your budget, your job, and who you will need to call upon to help you with the baby.
(*novel project – intense review)

You are pregnant, thrilled of course, and take good care of yourself, trying your best to ensure a healthy baby.
(*early data)

The baby arrives, happy and healthy. Let’s call him Gregory. You find yourself overwhelmed. How in the world could someone so tiny take up so much time, cause so much fatigue. You are dead on your feet and need a break and while you love this little one, there are moments when you feel you are not going to make it. And then, there is that first fever, congestion, irritability. You panic. You are up all night watching him breathe. By morning, the baby is improved (why do they always get sick at midnight?) Aunt Betty/Gram or a dear friend walks in the door, hands you a gift certificate for a massage, followed by dinner, and you nearly run for your car.
(*bridge support)

Time flies. Children grow quickly, too quickly, and soon they are running around, under your feet and before you know it, they start school . Gregory enters kindergarten and after a short time, the teacher suggests Gregory is not interested in school, not interested in learning his letters or his numbers and well, by the sound of her voice, it feels like Gregory is not going to be successful. And he’s only 5! This type of attitude is a game changer and you are prepared for battle. This is Gregory, the crazy little man, talkative, smart and not at all what the teacher implies. You search for tools to help and find a wonderful tutor, who makes learning a game about pirates and dragons. Gregory not only learns to read, but begins to drive his family crazy sounding out every road sign and menu option at Burger King.
(*fellowship – additional staff to focus on the project)

Gregory’s parents relax as he seems to adjust well to the schedule and his teachers. Things come easily for Gregory, until 6th grade math. The thing is Gregory can do the work, but he seems preoccupied with sports, video games, and friends. Grades drop. Test scores drop and you know the rest of the story. Parents are upset and the teacher is not budging. Gregory’s parents try everything they can think of from incentives to discipline to pleading. Summer arrives and Gregory barely passes math.
(*some experiments fail, at least temporarily)

The summer was wonderful for Gregory. Nothing about school, but all about friends, swimming, soccer, and video games. His friend’s dad recommends a free on-line video game - Moonbase Alpha. Playing Moonbase Alpha is not simple. Gregory and his friends step into the role of exploration team members and are immersed in a futuristic 3-D lunar settlement. Their mission is to restore critical systems and oxygen flow after a nearby meteor strike cripples a solar array and life support equipment. Gregory is hooked: inspired and engaged in space exploration, learning about science, technology, engineering, and mathetics (STEM education).
(*Interim support? Tox package?)

Gregory is back! He now sees himself working for NASA and one day, joining the team to land on Mars. High school is uneventful. As graduation gets closer, Gregory starts to worry. His cumulative average is 3.5 and his first SAT score is verbal 560 and math 610, good - but not great in terms of his life goal and unfortunately, not quite good enough to get into MIT, and most certainly not the type of credentials he would need for a scholarship. Gregory is devastated. His dream of admission to the best college program that feeds into NASA’s space program seems pretty impossible. With tears in his eyes, Gregory talks to his Uncle Henry about his dreams, his goals, his apparent failure. Henry believes in Gregory, offers to do whatever it takes to help him improve his SAT and generously offers to pay his college tuition. Day and night Gregory prepares for the test. Gregory’s SAT scores improve: 620 verbal and 680 math. He is admitted to Rice College in Texas, a major feeder school for the NASA program.
(*Venture capital)

On a sunny day in June, 4 years later, Gregory walks down the aisle, lifts his arms to receive his Diploma from Rice and while Gregory felt quite prepared to walk on the moon, he realized he would need additional education and resources to step into an aerospace engineering position. Gregory agrees to an internship at NASA, spends time with a range of experts and finds mentors to guide his journey.
(*Partnering)

All this to say, drug development is not accomplished by one person, one organization, or one company. It is accomplished through hard work, vision, opportunity, investment, and ONE VOICE – all focused on a single goal. Every organization/foundation/parent/patient is reaching for the same goal. We all make investments for different reasons, at different times, with different goals in mind; sometimes to fill gaps, often to provide seed money, frequently at just the right time to keep things moving.

And lest we not forget about the seemingly non-sexy stuff such as promoting early diagnosis, accurate genetic testing, registries, standards of care, workshops to identify gaps in care, clinical trials (ambulatory and non-ambulatory) outcome measures, advocacy (NIH, DoD, FDA, HRSA), post market surveillance, and access to therapies… for all boys, everywhere.

No one can do everything, but everyone can do something.
ONE VOICE

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Comment by Kim Innabi on August 3, 2010 at 8:07pm
I was looking for the "like" button!!! As always Pat, you said it so eloquently (and made it so easy to understand). Thank you.
Comment by cheryl cliff on July 28, 2010 at 12:55pm
I really enjoyed reading this story. Think I'll keep it around and re-read throughout the year to remind myself that we are on a road to recovery. The destination (ending duchenne) is further down and there is still a long way to go. Well put, "no one can do everything, but everyone can do something".

Press on with ONE VOICE. It serves us all, we will do well by it.
Thanks Pat

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