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The Long Haul

By August 18, 2016 November 27th, 2018 Tracy's Blog

Last week Sheryl Sandberg — CEO of Facebook, billionaire, best-selling author and, according to Forbes, the 7th most powerful woman in the world — posted an interesting Facebook status. #FirstSevenJobs she wrote, and then listed her work history from babysitter to suburban mall salesclerk to aerobics instructor to receptionist, then on to the World Bank, Children’s Defense Fund and finally to second in command at Facebook. Many thousands of people have liked the post, and it has gotten more than 500 shares, likely because of the truth it tells — no matter how much we want to believe in insta-success, it’s hard work and a long haul that get you where you want to go.

The work we are doing through Charley’s Fund is not easy. It is not sexy. There is no immediate gratification. Success is not one eureka moment that takes us from devastating illness to a miracle cure. It’s lessons learned every day, getting better and better at what we do, and taking the many necessary steps – sometimes seemingly small ones — toward transforming Duchenne from an aggressive killer into a chronic, manageable condition.

We got news of one such advancement last week, when Summit Therapeutics announced positive results from a clinical trial. No, we do not have a cure. We don’t even know yet if the drug stabilizes or strengthens muscles in kids with Duchenne. What we learned is that a new formulation of the drug gets better uptake than the current formulation. This is one of those seemingly small steps, but it is critically important toward ultimate success — sort of like salesclerk to aerobics instructor on the way to self-made billionaire.

The story of Summit, like any real-life success story, is one of determination in the face of setbacks and a commitment to the long haul. We first partnered with the company (then called Vastox) nine years ago to help them screen thousands of molecules in a zebrafish model, in search of compounds that could “upregulate” (i.e., increase) utrophin levels. Utrophin is a protein that can compensate for lack of dystrophin. The company made good progress, and even made a deal with a deep-pocketed pharma partner to conduct the costly human clinical trials that would be needed to determine whether the drug is safe and effective as a treatment for Duchenne.

The first clinical trial in humans didn’t go so well. The drug didn’t have great uptake in healthy adult volunteers. If a drug can’t get where it needs to go, of course it can’t do what it needs to do. So the pharma partner gave up, and Summit faced a turning point.

(Side note here: Sheryl Sandberg notes in her FB post that she was fired from her first babysitting job for letting a pizza delivery guy into the house when no one had ordered a pizza. Clearly, early missteps do not predict ultimate failure.)

Despite these disappointing results from the Phase 1 study, Summit chose to persist, and we stepped in to help clear the hurdle. Charley’s Fund — together with three nonprofit partners — provided the funds to reformulate the compound and try again in healthy adult volunteers. Lo and behold, it worked. The change improved the drug’s bioavailability, and the company proceeded to the next step: testing the drug in boys with Duchenne. Meanwhile, Summit has continued testing out various additional new formulations to get the most uptake with the least amount of drug — hence, this week’s good news.

Clearly, our work does not involve one dramatic success that happens in a single thunder-clap moment. In the fight to end Duchenne, success is made up of these small but important victories that are adding up to change the future for Charley and boys just like him all over the world. Over the past 12 years, it is remarkable how much we have helped transform the Duchenne landscape in profound ways. Multiple clinical trials underway. Hundreds of millions of dollars committed to the development of new medicines. Dozens of congressmen and senators paying attention to the regulatory process. Multiple biotech companies solely dedicated to developing new treatments for Duchenne, and now even large pharma companies the likes of Pfizer and Bristol Myers Squibb building their own Duchenne programs.

I sometimes stress about the fact that the nature of our work does not entail one Major Moment that demonstrates the impact we are having. But, spectacular success doesn’t happen overnight; it happens slowly but surely, through lessons learned from a daily (and nightly) grind that is often not very glamorous. I can’t express how grateful I am that our supporters understand that and stick with us as we slog our way toward achieving what we set out to do. I hope it helps to know that my first job was babysitter, too.