New $2.3M Research Could Transform Treatment for a Rare Childhood Hip Disease

Researchers at Scottish Rite for Children have received a $2.3 million grant from the National Institutes of Health (NIH) to develop groundbreaking treatments for Perthes disease, a rare childhood hip condition that currently has no biological therapy options. The five-year study aims to understand how the body repairs bone and restores blood flow in this painful condition that primarily affects children ages 4 to 8.

What Is Perthes Disease and Why Is It So Challenging?

Perthes disease occurs when blood supply to the ball of the hip joint is disrupted, causing bone tissue to die in a process called osteonecrosis. As the weakened bone breaks apart and loses its round shape, children experience pain, stiffness, and limited hip movement that makes walking and playing difficult. The condition shows a marked male predominance, affecting approximately four boys for every one girl.

Currently, treatment options are limited to managing symptoms rather than addressing the root cause. Children often need to use wheelchairs or crutches for mobility while doctors rely on surgery, braces, or physical therapy to keep the hip ball properly positioned. "Children with Perthes disease can face long, difficult recoveries with few effective treatment options," said Yinshi Ren, Ph.D., the study's principal investigator and scientist at Scottish Rite for Children.

How Will This Research Change Treatment?

The research team will use advanced genetic imaging and single-cell RNA sequencing to examine how bone cells respond to the disease at the molecular level. This cutting-edge approach will reveal gene activity within individual cells, providing unprecedented insight into bone repair mechanisms and potentially identifying new cell populations involved in regeneration.

The NIH funding will support several critical research components:

• Equipment and Models: State-of-the-art laboratory equipment and disease models to study bone regeneration processes
• Expert Collaboration: Partnerships with specialists at UT Southwestern Medical Center and Duke University to accelerate discoveries
• Research Team Expansion: Recruitment of new researchers and trainees to build expertise in this specialized field
• Knowledge Sharing: Dissemination of findings through conferences and publications to physicians and researchers worldwide

"Our goal is to move beyond managing symptoms and toward true healing," Ren explained. "Understanding how bone and blood vessels regenerate in patients with osteonecrosis or Perthes disease could lead to new therapies that stimulate tissue repair."

What Could This Mean for Affected Families?

The research collaboration includes world-renowned Perthes expert Harry Kim, M.D., M.S., Director of the Center of Excellence in Hip at Scottish Rite for Children, and Matthew J. Hilton, Ph.D., from Duke University School of Medicine. Together, they aim to develop the first targeted biological treatment for this condition, which could transform outcomes for thousands of children.

"Many live with pain or long-term joint problems that can lead to early arthritis or disability. Our research will not only fill critical gaps in our understanding of how bone heals after osteonecrosis but also lay the groundwork for developing a targeted biological treatment, which does not exist currently," Ren noted.

This breakthrough research could offer patients faster recovery times, improved joint function, and better long-term outcomes, potentially preventing the chronic pain and disability that many children with Perthes disease currently face. The work represents a significant step toward true regenerative medicine for bone conditions that have long relied only on symptom management.