Patient Organizations & Doctors Gather to Guide Progress for Better Treatment on a Global Scale
Momentum is building among researchers across the world who study fibrous dysplasia and McCune-Albright syndrome (FD/MAS). From December 1st through December 3rd, a powerful group of clinical researchers met in Lyon, France for the Second International FD/MAS Initiative Meeting. Attendees from the research world included US researchers Michael Collins and Alison Boyce of the National Institutes of Health (NIH), French researcher Roland Charpulat of INSERM, UK researcher Kassim Javaid of Oxford University, and Dutch researchers Neveen Hamdy and Natasha Appelman-Dijkstra of Leiden University Medical Center, among other important FD/MAS researchers.
These researchers met for the first time as a group in the UK in 2015 to discuss the development of an international set of clinical care guidelines, and agreed to meet again the following year to continue work on that project. The clinical care guidelines will be a publicly available, deeply detailed guide of how to treat FD/MAS, that is based on the research and wisdom of the participating researchers. After a successful second meeting in France this December, the clinical researchers have agreed to meet annually for the next few years, to complete the international clinical pathway project, and to potentially work on additional projects in the fight against FD/MAS.
In addition to researchers, representatives from patient organizations from the UK, France, Italy, and the United States also attended the meeting. Each patient group representative gave presentations in an effort to help clinicians understand patient concerns and priorities, and to exchange information about the programs and goals of the different patient groups. From the US, the Fibrous Dysplasia Foundation (FDF) was represented by Lauren Ruotolo, an FDF board member, MAS patient, and content marketing professional.
“It was really exciting to be in France and to connect with so many other people who understand and treat this disease. As a patient that meant a lot to me,” said Ruotolo, “I was also struck by how much patient groups can do when they organize and engage. It was really eye opening to see the work of similar organizations from places like France, The Netherlands, Italy, and the UK.”
During her presentation to clinical researchers, Ruotolo shared preliminary data from the FD/MAS Patient Registry
Ruotolo’s presentation focused on patient priorities and the questions that registered study participants want to see answered most.“People are really affected by pain and they want researchers to find solutions. Patient organizations have the potential to collaborate and address those concerns with the scientific community.” This was the first conversation where the FDF could advocate for the FD/MAS community based off survey data about patient priorities. That’s an important change from the past because researchers tend to pay more attention to information that has been collected in a scientific manner.
Ruotolo also shared information about the Fibrous Dysplasia Foundation’s programs with the other patient organizations, highlighting areas that would benefit from more international collaboration. FDF’s Executive Director Deanna Portero looks forward to increased collaboration between different FD/MAS patient groups, “FD/MAS doesn’t play favorites, it doesn’t care what country you live in. We’re all in this together. Across the world, FD/MAS patient groups have so many shared problems and goals. Working together on more projects will mean more progress for patients in all of our countries. That’s why collaboration is one of FDF’s core values.”
Dr. Boyce, who holds leadership positions on the NIH Natural History Study and the FD/MAS Patient Registry Oversight Committee, felt that the meeting benefitted from formal involvement of patient organizations. “One of the main goals of the meeting was to develop international clinical care guidelines for FD/MAS. This will help patients with FD/MAS all over the world receive the appropriate work-up and treatments for their disease. Partnering with patient advocacy organizations has been invaluable in putting these guidelines together, and will be an important part of sharing them with the international community.” said Dr. Boyce.
Dr. Collins, Chair of the FDF’s Medical Advisory Council, agreed that the meeting was incredibly productive. “We made great progress on the guidelines and forged a real working relationship between the consortium of investigators from all over the world. A standardized treatment protocol will allow clinicians and investigators to better compare their patients and the efficacy of treatment. A nice feature of the gathering was that all of our patient groups were different but complementary. I’m confident this group will coalesce and make tremendous progress in FD/MAS.”
Next year, the same group will meet in The Netherlands. Ruotolo is adamant that a patient advocacy collective should attend as well. “As part of the FDF and also a patient myself, I feel extremely motivated to collaborate with other countries and to set patients everywhere on a course for better care.” said Ruotolo, “There’s a great potential for the international FD/MAS community to learn from each other and for patients to direct progress on a global scale.”