Combination of demineralized bone and gene therapy shows promise for treating pseudarthrosis

A team headed by Professor Albert Rizvanov, director of the Gene and Cell Technologies Open Lab, created a gene therapy drug that encodes growth factors for the stimulation of blood vessel and bone formation. The combination was highly effective in a patient admitted to the Republican Clinical Hospital in Kazan, Russia. The treatment was approved by the ethical committee, supported by the Ministry of Healthcare of Tatarstan and published in BioNanoScience.

Professor Rizvanov explains: We combined a demineralized bone transplant with recombinant genetic material, which carries genes for vascular endothelial growth factor, to stimulate new blood vessel growth (angiogenesis), and bone morphogenetic protein to stimulate bone growth (osteogenesis). Thus survival of transplant and bone tissue formation was achieved at the desired location. We were able to translate our basic and pre-clinical research and are the first to document the efficacy of such therapy in a real clinical case of pseudarthrosis.

As the team leader noted, for a long time clinicians have tried standard methods of bone regeneration - osteosynthesis and osteoplasty - when an absent bone part is replaced with allogenic, cadaveric or demineralized bone matrix. In the latter case cells and minerals are removed from an animal bone tissue, and only the matrix is left - a "bone-like sponge" that can be used to fill in defects in the bone to stimulate formation of a new bone. However, current procedures often result in complications, such as resorption of the transplant. Parts of the problem are poor blood supply and low levels of pro-osteogenic growth factors.

Our therapy, a combination of demineralized bone with gene therapy, is a promising solution for the currently existing complication problems in pseudarthrosis and other bone defects and fracture treatments. We now plan to offer such innovative treatments at the Kazan University Clinic as a part of a new clinical trial program at strategic academic unit Translational 7P Medicine for biomedical and translational research.