We recently launched one of our biggest and most important initiatives: The Duchenne Program at UMass Medical School. This brand-new clinic provides coordinated, comprehensive care to people with Duchenne and also serves as a research site for multiple clinical trials. We caught up with Director Dr. Brenda Wong for a Q and A to capture for you what’s so important about this effort and what’s special and innovative about the model.
Q: What most excites you about The Duchenne Program?
A: I’m pretty much obsessed with Duchenne muscular dystrophy. I think about the disease and my patients all the time, and I’m constantly learning something new from the many research programs that are going on around the world. With so much happening in the world of Duchenne, it’s been a dream of mine to have a clinic and research program that is fully dedicated to the disease and research into treatments and a cure. At The Duchenne Program, 100% of our efforts are focused on DMD (and its close cousin Becker muscular dystrophy). Because we’re “all Duchenne, all the time,” we’re able to provide patients with comprehensive care based on the most recent findings. I’m also very enthusiastic about our “all comers” policy. We are committed to providing seamless, full-spectrum care for everyone from newborns to adults with Duchenne, and even moms who are carriers. Too often, adults with Duchenne are stuck feeling like square pegs in a round hole. It’s not ideal for a 25 year old man to be seen by pediatric providers at a children’s hospital. Nor is it great for young men with Duchenne to experience an abrupt change in care as soon as they turn 18 and are passed on to adult providers. Our model builds in a continuum of care so our growing population of young adults with Duchenne can thrive.
Q: Why did you relocate to the Northeast, and specifically to UMass Medical School?
A: Massachusetts is the world’s epicenter of medical innovation. Our clinic is in a perfect place to engage in collaborations with scientists and biotech companies who are paving the way for new treatments. UMass Medical School and the nonprofit foundations supporting our clinic have a partnership that fosters innovation, collaboration, and problem solving. Because our model is embraced and supported here, my team gets to focus more time and energy on our patients and the research.
Q: The term “comprehensive, coordinated care” seems to get thrown around a lot. Can you explain what that means, and how you deliver it through The Duchenne Program?
A: Duchenne involves every system in the human body. Every day we learn more about what we should be doing to monitor our patients — and what interventions we can employ to ensure the healthiest possible outcomes. Comprehensive, coordinated care means we anticipate and address every single issue our patients may face through a team effort. I am lucky to have stellar physicians and other medical providers participating in our program, from cardiology (adult and pediatric) to pulmonology to nutrition, physical therapy, and more. As we learn more about Duchenne, we bring new team members on board to make sure every base is covered. For example, we now have an ophthalmologist on board to monitor for cataracts, which can result from long-term steroid use.
Q: Are you conducting any clinical trials at The Duchenne Program?
A: Yes, we are currently recruiting for Capricor’s study of allogeneic cardiosphere-derived cells. We are also preparing to launch two additional trials. One trial is testing a gene replacement therapy. The other is testing a medicine that limits myostatin, a protein that keeps the muscles from growing too large. We’re optimistic about the variety of therapeutic approaches that is being explored.
Q: What are your future plans for The Duchenne Program?
A: So many! I am really excited about the research possibilities – not only more clinical trials, but contributing to our understanding of how we can make clinical trials faster, less expensive, less burdensome, and more reliable. We have plans to collaborate with scientists, biotech companies, and nonprofit groups like Charley’s Fund to generate data that will be helpful to the whole community. Also, an important long-term goal of mine is to train the next generation of physicians. We have learned so much about Duchenne; it’s critical to attract talented physicians to this disease and equip them to carry on and expand the care model. As far as short-term plans for the near future are concerned, I’m excited for some physical renovations that will freshen up our clinic space as well as the launch of a new website that will be inviting and engaging for patients and their families.
Q: In a rapidly changing health care environment, how do you ensure that caring for patients and advancing the research remain the program’s top priorities?
A: The Duchenne Program relies on a unique partnership between my team, UMass, and Charley’s Fund. The three of us have aligned on core principles for the program, and we work together closely to ensure that we remain true to those guiding principles. The UMass leadership and physicians completely understand the value of our interdisciplinary model and commitment to research, so they have been extremely accommodating as we set up this novel program. When administrative hurdles arise – and believe me, they do – we collaborate to overcome them. Also critical, Charley’s Fund has led the charge in securing philanthropic commitments to ensure the program is fully financed until it is self-sustaining, including support from other foundations such as JBs Keys to DMD and Hope for Gus.
Q: Have you ever experienced a Nor’easter?
A: I must admit I’ve been nervous about that! But we had our first snowfall a few weeks ago, and it didn’t interfere at all with our program. From what I understand, New Englanders are pretty hardy and don’t let snow get in their way. I’m ready!