Until recently, the only two hospitals in the U.K. with the expertise to run Duchenne muscular dystrophy (DMD) clinical trials were turning studies away — they didn’t have the resources to test all of the new and potential advancements.
The Hub, a project set up by Duchenne U.K., is building a network of clinical trial sites — and connecting them with companies and patients interested in participating — in hopes of ensuring that every child with DMD has access to experimental therapies.
The group also launched an online clinical trial finder with resources for patients and families searching for information on ongoing or planned DMD studies across the U.K.
Alex Johnson and Emily Crossley, who each have a son diagnosed with DMD, started Duchenne U.K. six years ago. Since then, they’ve raised over £10 million (about $12.8 million), most of which has gone to support more than 50 research projects ranging from preclinical work on gene editing techniques to clinical trials.
Things were going well, Crossley told Muscular Dystrophy News Today, and it’s a “very exciting time for Duchenne’s research.” But she quickly realized that with so many discoveries being made, there weren’t enough hospitals prepared to test them in people, delaying the opportunity for patients to benefit from potential therapies. Crossley said industry is reluctant to run trials at centers lacking experience with DMD studies.
Discovering this was a “huge blow,” Crossley said. “Often the family’s only hope of getting access to drugs and investigative therapy is through a clinical trial.”
They created the DMD Hub in 2016, with the goal of expanding both patient information and site expertise. According to its website, the DMD Hub “provides training, mentoring and resources to sites to help them meet industry requirements for clinical trials …one of the main ways that we will increase clinical trial capacity.”
Since 2016, the group has supported five DMD Hub trial sites: in Alder Hey, Birmingham, Bristol, Glasgow, and Leeds, and plans on supporting four more. It named the Great Ormond Street and Newcastle hospitals — which were previously running all the country’s DMD trials — “DMD Hub Centers of Excellence.” It December, the Hub reported that its support for neuromuscular research at the Osmond Street hospital led to a doubling in the number of children participating in clinical trials there, from 139 in 2016–17 to 351 in 2017–18.
Along with providing initial funding to new DMD Hub sites, Duchenne U.K. also acts as a liaison, connecting pharmaceutical and biomedical companies with these sites, recruiting experienced staff, and offering advice on study protocols and the approval process.
Crossley and her team soon realized that “patients didn’t really know where to go to get information.” So DMD Hub also set up and maintains the clinical trial finder, offering up-to-date information on the studies, along with inclusion criteria, trial goals, contact information, and an easy-to-understand summary.
Patients and caregivers can use filters to search for trials most relevant to them.
“There really is nothing else like it,” Crossley said, “we don’t rely on third parties for information.” The most important thing, she added, is giving patients the “knowledge and information they need to make the right choice for them and their family.