Question: Charley’s Fund completes its tenth year of operation this November. Why is this an important moment?
Answer: Over the course of 10 years, you gain knowledge, experience, resources, and connections. You see surrounding circumstances evolve and new opportunities emerge. But for all we’ve gained, we’re still racing against the clock as we lose our most important resource: time. We are seizing this milestone as an opportunity to reflect on the decade of experience under our belts and refresh our strategy to ensure we make the best possible choices and lay the best possible plans.
Q: What are some of the things you have learned over these ten years?
A: Most importantly, we’ve learned that what we’re doing is working. When Charley was diagnosed, there was not even one clinical trial for a hopeful drug to treat Duchenne. Today, the first treatments ever — two genetic treatments that could benefit about 13% of boys with DMD — are just months away from applying to the FDA for approval. We funded both drug companies early on, and we also played a major role in influencing the FDA’s decision to consider accelerated approval. We created our own biotech company that is conducting clinical testing for an anti- fibrotic, anti-inflammatory treatment that could benefit all boys with Duchenne, and the initial clinical data looks positive. We partnered with a group of scientists at the University of Buffaloto translate their exciting discovery — spider venom might help kids with Duchenne! — into a treatment. We now can hope that boys with Duchenne will have more time, and we can use that time to continue building the cocktail of drugs that will turn Duchenne from an aggressive killer into a manageable chronic condition.
But we’ve also learned that we have a lot more to do, and the role our organization plays is critical. Early success means research moves forward to the more expensive stages of development, which means a lot more money is needed. And more research approaching clinical trials means we desperately need more effective, consistent tools to measure results.
The importance of our role as a creative problem-solver can’t be overstated. Drug companies and the FDA are goliaths that have every incentive to follow established processes and procedures. But established processes and procedures are too slow and too risk-averse for Duchenne. We have to be the ones who challenge the status quo and identify and enact opportunities to do better.
Q: How does Charley’s Fund make a difference when the government and the pharmaceutical industry have such deep pockets for funding research and drug development?
A: As a patient-founded organization, we play a unique role in making research happen better and faster. The amount of capital we can raise and fund into research is small compared to what the government contributes to basic science and what pharma companies invest in later-stage clinical trials. But we step in at a point so critical it is often referred to as “the valley of death.” This is the point in development that usually comes after the government funding stops, but before the research is de-risked enough for a pharmaceutical company to step in. Sadly, many potential therapies languish there and never make it to patients.
Q: What’s next in store for Charley’s Fund?
A: Ten years ago, we decided to plant a large number of seeds in various therapeutic areas. The individual seeds we planted have either flourished (like exon-skipping) or floundered. Additional players have taken up ploughshares and are effectively nurturing certain sectors. This enables us to tighten our focus on the best research opportunities and develop tools that will help all new drugs move faster.
A therapy that promotes healthy muscle regeneration will be an important component of the Duchenne medicinal “cocktail.” We recently partnered with Stanford University to support a stemcell program, and we will be delving further into this excitingarea of muscle regeneration in the next couple of years. We also need better tools to accelerate clinical research. More therapies are approaching the clinical stage, which is hugely encouraging. But we need to ensure that new treatments move through clinical trials as quickly, efficiently, and effectively as possible. There’s little financial upside in developing these tools, so it is a prime opportunity for us to have a major, meaningful impact. The same applies to working with the FDA to identify creative solutions to the regulatory path all therapies will face before they can be approved. We don’t have one company’s interest in mind — we have long, healthy lives for all boys with Duchenne in mind. So we have an opportunity as a neutral party to drive that conversation.
Q: How does starting a biotechnology company figure into this equation?
A: It may sound cliché, but when we say “do whatever it takes” we mean it — including founding a new company and assembling a veteran management team to run it. As parents, we just could not live with the fact that certain assets were languishing undeveloped despite serious potential to help our son and other children with his disease. Akashi Therapeutics picked up three of those assets and has a growing pipeline of promising drugs.
Q: How is Charley doing?
A: Under the circumstances, I would say he’s terrific. Duchenne is a beast and there are certainly times when he gets down. Participating in a clinical trial with the exhausting travel schedule and many doctor visits is a drain on the entire family. But he has an amazing ability to put the grief aside and get back to the business of enjoying life. At the moment his top three “things I love” would probably be the alt-rock band Arctic Monkeys, Key and Peele comedy skits, and coconut Chobani yogurt.