Today, we are grateful and encouraged to announce that three of our partner organizations are throwing their weight — and some serious financial firepower — behind one of our most important programs: The Impact Initiative. Launched in 2015, the Impact Initiative speeds and improves all research — not just one particular therapy. Michael’s Cause, Pietro’s Fight, and Ryan’s Quest have come together to commit $442,000 to help us identify field-wide research problems and design and drive innovative solutions.
The Impact Initiative’s main architect, Charley’s Fund President and COO Laura Dalle Pazze, recently sat down with Rob Capolongo of Michael’s Cause, Manni Scarso of Pietro’s Fight, and Dave Schultz of Ryan’s Quest to discuss their major commitment.
Laura Dalle Pazze, Charley’s Fund: As parents of boys with Duchenne and co-founders of your own organizations, you’ve been funding medical research for a long time. What stood out to you about this work? Why did you decide to support it?
Rob Capolongo, Michael’s Cause: We HAVE been doing this a long time, and that’s part of the problem. While we’ve seen progress, it’s not happening fast enough. We clearly need to do something differently. We can’t stop supporting the development of specific therapeutics, but we also need to do more to make sure that the new medicines being developed have the best possible chance of success. We’ve worked with Charley’s Fund for years on various projects, and we know they’re fearless in digging in and solving problems. When they explained to us what they’re doing here, it just made sense.
Laura: On that note, let me back up for a moment to say a bit about what the Impact Initiative is and why it’s important. We’re at a point of tremendous opportunity in Duchenne research. Many potential therapies are at various stages of testing, and more money than ever before is being invested in the development of these new treatments. But think of all these experimental medicines as seeds that have been planted: Some will wither, some will eventually (hopefully) make it. We have to make sure the right tools are in place and the conditions are set to help the strongest seeds survive and thrive — and to weed out the bad ones sooner rather than later.
Charley’s Fund spent a lot of our early years planting and nurturing specific seeds — in other words, funding the development of individual therapies. More recently, we’ve realized we must also focus our attention on tending the soil and cultivating the conditions that help ALL seeds grow. That’s how the Impact Initiative was born. We ask questions like: How can we improve our preclinical models to enable smarter decisions about which drugs to advance to human clinical trials? How can we improve our outcome measures to assess efficacy in clinical trials faster and more sensitively? Then, we start working on solutions.
Dave Schultz, Ryan’s Quest: We have funded a lot of research over the years. We’ve had some victories, but like Laura said, we’ve seen a lot of shots totally miss the goal. (I’m a sports guy so while I dig the seeds metaphor, I tend to think in terms of a ball and a field.) Parents and families with Duchenne don’t want hope — we want drugs that work. And if projects we hope will work don’t turn out that way, we need to come together as a community, buckle down, and figure out how to work smarter. That’s what Charley’s Fund is doing here. With this project, we’re not banking on the outcome of one drug; instead, we’re banking on a smart plan and dogged work ethic to help us develop resources every drug needs. And that is a sure bet. So no matter what, I know my funds will have an impact.
Laura: Dave brings up such a critical point — taking on these problems is a lot of work. And while biopharma companies clearly have a stake in the solutions, advancing the drugs in their pipelines is already more than a full-time job. They know our tools and resources are woefully imperfect, but they have to focus on working with what we’ve got to move their drugs along — otherwise, their investment will dry up.
Solving these problems also requires collaboration and dot-connecting, and Charley’s Fund is in a great position to work with everyone —academics, industry, other organizations — to do it. As long as we lead the charge, companies are happy to participate. They just need someone to put in the organizing effort, critical thinking, and elbow grease to make it happen.
Manni Scarso, Pietro’s Fight: Right. No one drug development company is going to make it a priority to improve the research infrastructure and then let everyone benefit from those improvements, and that makes sense. But these improvements are desperately needed. My son has participated in two clinical trials so far, so I have seen first-hand how difficult, time-consuming, and draining clinical research can be for families, not to mention how expensive. In my opinion, it is ultra-important to have tools in place that give a relatively quick and reliable readout on the treatments being tested. I’m especially enthusiastic about the new at-home, video-based assessment system we’re helping to support with this grant to Charley’s Fund.
Laura: I’m glad Manni brought that up. This tool has real potential as a sensitive, reliable measure of whether a treatment is benefitting patients. Here’s how it works: Caregivers capture video footage of kids, adolescents, adults — anyone with the disease — doing everyday tasks. Young kids are recorded climbing stairs, rising from the floor, and walking or running. Older patients are recorded lifting a fork, rolling over in bed, and sitting up. Trained physical therapists then evaluate the videos using a validated scoring scale. The idea is to identify changes in how patients complete those tasks over time in order to evaluate how quickly the disease is progressing.
Rob: I don’t think we can overstate the need for better outcome measures. My son is participating in a clinical trial right now. Thankfully he is doing really well for a 13-year-old with Duchenne. But is that due to the experimental medication, or other factors such as the supplements he takes, or even luck of the draw? The only way to know for sure is reliable outcome measures that can accurately and quickly determine whether a treatment is providing benefit. That’s why I’m glad our funding will also help support the development of Electrical Impedance Myography, which seems to me like a quick and painless way to get quantifiable information about muscle health. I also like that it can be used on people at any age, whether you’re two years old or 20.
Dave: I know so many families whose kids aren’t eligible for trials because they’re too young or too old. That’s a real problem not only for the families whose kids are left out, but because it slows everything down. When you can only draw participants from a small slice of the population, it takes longer to recruit and fill the trials. Meanwhile, our kids are getting worse every day. We need answers on these treatments, and we need them quickly.
Laura: Rigorous science — undertaken with urgency and focus — is the only way we get to the outcomes we and all Duchenne families want: Drugs that work and work well. Solving these field-wide problems and improving our tools and resources is an absolute must to get results. We wrote about this recently in a blog post addressing an article The New York Times published about Duchenne. And we’re not the only ones pushing forward on these fronts: other big-name (and big-impact) foundations like The Michael J. Fox Foundation for Parkinson’s Research and the Cystic Fibrosis Foundation also prioritize pioneering efforts like this.
I feel tremendously heartened to have Rob, Dave, Manni, and their organizations on board with us in this work. Their support will help us make progress on EIM, the Casimir Duchenne Video Assessments tool, and a third project we have underway to improve the preclinical models and protocols that are used to determine which treatments should advance to clinical testing. We need to be relentless in pushing ourselves and our field to continue to get better and smarter in our quest for new treatments. With their support, we can make huge leaps forward.
Stay tuned for more information about these and other projects advancing under the Impact Initiative. Want to learn more about the work referenced here? Check out the case studies highlighted here as well as Q+As with Seward Rutkove, MD — the Harvard doctor behind Electrical Impedance Myography — and Mindy Leffler — co-founder of Casimir and the Duchenne Video Assessments tool, and mom of Aidan.