Margaret Zacharin, an associate professor in the department of endocrinology and diabetes at Royal Children's Hospital in Parkville, Victoria, Australia, has been awarded an MDA research grant totaling $268,021 over two years. The award will help support a clinical trial of the drug zoledronic acid in children and adolescents with Duchenne muscular dystrophy (DMD).
Zoledronic acid is part of a class of medications known as bisphosphonates, which are used to prevent bone loss in osteoporosis and other bone conditions.
Children with DMD lose bone in part because of their underlying disease and in part because of the effects of corticosteroid medications. These are commonly used to prolong muscle function in DMD but have adverse effects on bone density.
"Bisphosphonates alter the course of corticosteroid-induced bone loss and largely prevent this complication in the adult population," Zacharin said. "It's more difficult to provide such evidence in a pediatric population." However, she noted, with newer techniques of calculation available, more accurate data can now be obtained.
"We aim to determine whether intravenous zoledronic acid plus vitamin D and calcium is better than vitamin D and calcium alone in increasing spinal bone density, reducing bone turnover and/or reducing fracture rate over two years in boys with Duchenne muscular dystrophy."
Zacharin will work closely with Craig Munns, an associate professor in the Department of Endocrinology at Children’s Hospital in Westmead, New South Wales, Australia, on this project.
She said results of the zoledronic-acid study may be used to develop a larger trial to assess fracture reduction in the DMD population and might ultimately be extended to other diseases, including other neuromuscular disorders.
MDA support, she said, "will allow us to undertake and complete this important study in a timely fashion."
Funding for this MDA grant began August 1, 2011.