Ethan Knox

Study shows a drug created by Binghamton researcher used to treat Duchenne Muscular dystrophy avoids suppressing bone- and cartilage-related proteins, biomarker changes recover after boys’ transition from corticosteroid

By Ethan Knox ’20 on SEPTEMBER 02, 2026 @BingUNews

In a new study published in Scientific Reports, an international and multidisciplinary research team including Binghamton University researchers identified a set of blood proteins that may explain why the corticosteroid prednisone, although beneficial in treating Duchenne muscular dystrophy (DMD), can interfere with childhood growth and bone health. 

The findings come from a double-blind clinical trial involving boys with Duchenne muscular dystrophy who were 4-7 years old. During the first 24 weeks, participants received placebo, prednisone at 0.75 mg/kg/day, or vamorolone at a 6 mg/kg/day dose, a drug created with the express purpose of treating DMD. During the second 24-week period, participants who had received placebo or prednisone transitioned to vamorolone, while those receiving vamorolone continued treatment. 

The open access study, “Prednisone, not vamorolone, suppresses novel serum bone and cartilage biomarkers associated with growth failure in children with Duchenne muscular dystrophy,”(opens in a new window) found that prednisone significantly reduced established and newly identified markers of bone formation, bone turnover, and growth-plate activity. These changes were not seen with vamorolone or placebo. When participants receiving prednisone transitioned to vamorolone, the suppressed biomarkers returned toward their pre-treatment levels.

“Prednisone remains an important therapy for Duchenne muscular dystrophy, but its effects on growth and bone health are a major concern for children and families,” said Eric P. Hoffman, senior author of the study and associate dean for research and research development and professor of pharmaceutical sciences at Binghamton University. “Importantly, both prednisone and vamorolone improved motor outcomes in the trial, but only prednisone produced this adverse biomarker pattern.”

Broad protein screen identifies a prednisone-specific signature

The researchers first examined four commonly used clinical laboratory markers of bone formation: alkaline phosphatase, or ALP; osteocalcin; procollagen type I N-terminal propeptide, or P1NP; and collagen type I C-terminal telopeptide, or CTX1.

All four markers declined during prednisone treatment but remained stable in participants receiving vamorolone or placebo. The markers recovered rapidly after prednisone was tapered and participants transitioned to vamorolone.

Further analysis identified 10 additional protein measurements that were reduced by prednisone, including collagen-related molecules and other components of bone and cartilage. Mutations in these proteins can cause inherited disorders affecting bone growth and density, cartilage structure, and skeletal development.

“Following the same participants through the treatment crossover was particularly informative,” said Utkarsh Dang, lead statistician of the study and a professor at Carleton University’s Department of Health Sciences. “The prednisone-associated changes appeared in both standard clinical laboratory tests and the broader analysis, and the markers moved back toward baseline after prednisone was discontinued. That pattern supports a reversible drug effect rather than a change caused only by progression of Duchenne muscular dystrophy.”

A window into the childhood growth plate

Many of the newly identified proteins are associated with osteoblasts, which form bone, or with specialized cells in the growth plate that help drive the lengthening of children’s bones.

Human biomarker data shows that traditional corticosteroids like prednisone can promote the loss of these cells and interfere with osteoblast function, but vamorolone did not produce the same signature, suggesting that it may better preserve growth-plate cells and osteoblasts, consistent with observations showing linear growth during vamorolone treatment.

However, blood biomarkers are not the same as clinical outcomes. The study involved a smaller subset of the overall trial population, focused on young boys with Duchenne muscular dystrophy, and it was not designed to establish whether vamorolone reduces fractures or preserves skeletal strength over many years. This means that larger and longer studies will be needed to determine whether the biomarkers predict subsequent changes later in life and whether they perform similarly in girls and in children with other diseases.

“These proteins could eventually provide a practical way to monitor the biological health of the growth plate during treatment,” Hoffman said. “They may also be useful beyond Duchenne muscular dystrophy in other pediatric conditions in which corticosteroids, chronic disease, or investigational therapies can interfere with normal growth.”