A challenge to research that inspired hope for patients with fibrous dysplasia

In 2023, the Harvard School of Dental Medicine received a grant from the U.S. Department of Defense to support a four-year investigation of fibrous dysplasia (FD), a serious skeletal condition in which noncancerous tumors lead to bone deformities, fractures, and pain. The grant aimed to explore the cellular and molecular foundations of the condition, which impacts an estimated 1 in 15,000 to 30,000 individuals and presently has no cure. The study held promise not only for treating FD but also for identifying therapies for ailments affecting military personnel, such as blast-induced heterotopic ossification and chronic bone pain.

Initially, the funding was celebrated by patients and advocacy organizations like the FD/MAS Alliance, a nonprofit committed to discovering evidence-based therapies for Fibrous Dysplasia and McCune-Albright syndrome.

“This funding was beyond just a financial grant—it represented a vital investment in comprehending and ultimately treating a debilitating disease,” stated Adrienne McBride, Executive Director of the Alliance. “Advancing research in FD/MAS benefits individuals living with this rare disorder and holds significant potential for wider medical implications.”

The mechanisms explored in FD research could yield relevant insights for numerous other diseases causing bone fragility, pain, and fractures. With federal research funding to Harvard currently frozen, these insights may never come to fruition.

“FD patients and their loved ones had been intently following research advancements, anticipating new, effective treatments. The cancellation of pioneering projects like this diminishes this hope and sends a disheartening message to those affected by an already under-recognized condition,” remarked Yingzi Yang, Professor of Developmental Biology at HSDM and principal investigator on the grant.

Yang and her colleagues at Massachusetts General Hospital (MGH) had been making strides in the years since the grant was awarded. Although some studies continue at MGH, the research conducted in the Yang Lab at HSDM, crucial for enhancing the understanding of the disease mutation, has ceased.

“We had made considerable progress in identifying potential treatment targets for this critical condition based on our increased understanding of the molecular mechanisms,” Yang explained. “Halting our research disrupts the comprehensive understanding of the FD condition and diminishes scientific rigor and impact.”

“The cancellation of this grant marks a significant setback for FD/MAS research and for those, including military personnel, who depend on scientific advancements for hope and assistance,” McBride remarked.

FD/MAS can influence every bone in the body, with the largest subgroup of affected individuals experiencing FD lesions in their craniofacial bones, resulting in severe facial deformities.

HSDM graduate Christopher H. Fox, DMD87, DMSc91, who heads the American Association for Dental, Oral, and Craniofacial Research (AADOCR), also voiced serious concerns over the consequences.

“This reduction in funding for such promising research is a tragedy for the FD/MAS community and indeed for our nation. Through our advocacy initiatives, AADOCR is striving to reverse these misguided decisions,” Fox stated.