The Keck School of Medicine at the University of Southern California in Los Angeles was awarded a $2.2 million National Institute of Health (NIH) grant to study “Regional Gene Therapy to Enhance Bone Repair.”
$2.2 Million Grant to Study Bone Injuries
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The five-year grant is earmarked for gene therapy research, which shows promise in aiding challenging bone breaks and fractures. The Keck chair, and a professor of orthopedic surgery, Jay R. Lieberman, M.D. applied to the NIH’s National Institute of Arthritis and Musculoskeletal and Skin Disease funding pool.
The proposal notes, “The ultimate goal of this project is to develop regional gene therapy for the management of difficult bone repair scenarios for which there is no satisfactory solution.” Typical “challenging bone injuries” include fractures with severe bone loss, spinal fusion failures, fractures that don’t heal, and total joint replacement revision surgery. Dr. Lieberman has plans to genetically manipulate cells in human bone marrow to encourage bone morphogenetic protein (BMP). This protein is key in progenitor cells, leading to bone production.
No Bones About It
According to Dr. Lieberman, “There are a number of bone injuries that are very difficult to repair and lack satisfactory solutions. My goal with this grant is to determine whether genetically modifying human bone marrow cells to overproduce BMP will help heal large bone defects in an animal model and, ultimately, provide a better alternative for repairs in humans.”
As part of the study, the safety and efficacy of gene therapy will also be addressed. Dr. Lieberman also aims to pinpoint the best cellular dosage for genetically modified scales, so they can be properly scaled for future use in human subjects.
All funds, totally $2,284,028, are federally sourced. Dr. Lieberman has been awarded five other grants since 2010 to study “The Use of Regional Gene Delivery to Heal Critical Sized Bone Defects.” However, this is by far the largest grant he’s received.
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This is a fascinating development. In my practice we've seen similar outcomes with the revised protocol. The key differentiator seems to be patient selection criteria. Has anyone else noticed the correlation with BMI thresholds?
Great point. I'd push back slightly on the conclusion, the sample size in the cited study is too small to draw population-level inferences. That said, the directional signal is compelling and worth a larger RCT.
We implemented a similar approach last year. Early results are promising but we're still gathering 12-month follow-up data. Happy to share our protocol if anyone is interested.
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