In August, top bone researchers from around the world gathered in Sun Valley, Idaho for the 47th Annual International Musculoskeletal Biology Workshop. The Sun Valley workshop is an annual convening of field-leading bone researchers, who use the workshop to discuss their progress and obstacles with other top researchers. This allows researchers to get new, fresh ideas for how to approach a problem.
This year’s meeting was especially exciting for those interested in fibrous dysplasia and McCune-Albright syndrome (FD/MAS). The three-day meeting covered many different bone research subjects, but it spent a half-day exclusively on FD/MAS. Presenting researchers included Drs. Michael Collins and Alison Boyce of the NIH, Dr. Ed Hsiao of University of California, San Francisco, and Dr. David Burr of Indiana University, all of whom volunteer for and advise FDF.
The Fibrous Dysplasia Foundation (FDF) sent executive director Deanna Portero to the meeting to encourage more rapid progress on FD/MAS patient priorities.
“The Sun Valley Workshop draws the most talented minds in bone research, so it was a win-win situation for the FD/MAS community. Some of our most dedicated researchers got feedback and ideas on how to approach stubborn problems that have bedeviled the field. At the same time, the audience of non-FD/MAS researchers learned about the field of FD/MAS research and got a world-class tutorial on some of the most pressing questions. We need more labs to study FD/MAS, so it’s a smart strategy to treat non-FD/MAS researchers as ‘soon-to-be FD/MAS researchers,’” said Portero.
In addition to presentations from established experts, the conference highlighted new researchers with innovative ideas. Dr. Kelly Wentworth of University of California, San Francisco received this year’s Young Investigator Award, and presented her work on McCune-Albright syndrome stem cell models. “When new researchers become interested in FD/MAS, they also bring energy and innovative approaches to the table. Dr. Wentworth’s work leverages CRISPR/cas9 gene editing technology to study FD/MAS. That’s cutting-edge technology. In 2010, that kind of gene editing research was science fiction. It literally didn’t exist.” explained Portero.
The presentations focused on FD/MAS sparked the interest of many researchers and opened conversations on potential future projects. Researchers and representatives of biopharmaceutical companies were interested to hear that the FD/MAS Patient Registry already had 500 registrants, and that 230 of those registrants completed surveys about their medical histories and experiences. “Following the FD/MAS portion of the workshop, two prominent researchers requested the application instructions for tapping into the FD/MAS Patient Registry for new projects,” recounts Portero.
“When a researcher or company chooses to study a rare disease, they are taking a big risk. But when they see patients rallying around the FD/MAS Patient Registry, they see that the patient community is standing by to resolve those challenges with them. Every enrollment, and every survey matters,” said Portero.
Part of the work of the FDF is to foster research connections, and to steer those new connections into meaningful action. Portero left the meeting feeling hopeful and determined. “More than ever, I believe that our progress to a treatment or a cure is at a crucial junction. The pieces of the puzzle–the scientific expertise, animal models, patients, funding sources–are scattered across the world. The faster and more aggressively that we pull them together, the better.”