Research has shown that disc-related back pain may one day be treated with gene therapy delivered by naturally derived nanocarriers. A recent study demonstrated that this gene therapy successfully repaired damaged discs in the spines of mice and reduced pain symptoms. The nanocarriers were engineered using mouse fibroblasts and loaded with genetic material for a protein crucial to tissue development. When injected into damaged discs in mice, the gene therapy restored structural integrity and function to degenerated discs, offering a potential long-lasting alternative to opioids for managing debilitating back pain.
Degeneration of intervertebral discs is a common cause of low-back pain, impacting approximately 40% of cases. Current surgical interventions, such as trimming bulging tissue from herniated discs, provide temporary pain relief but do not address the underlying issue of disc degeneration. This new gene therapy approach aims to repair the damaged discs by rejuvenating the cells within the tissue. By delivering genetic material to produce a key protein called FOXF1 important for tissue development, the therapy helps the discs regenerate and maintain their health over time, thus promoting spine function and reducing pain.
Previous work in the researchers’ lab demonstrated that nanocarriers loaded with anti-inflammatory cargo could reduce tissue injury in damaged lungs of mice. By engineering nanocarriers to mimic extracellular vesicles naturally found in humans, the researchers were able to deliver specific proteins and molecules to the damaged discs, promoting tissue repair. The FOXF1 protein delivered to the injured discs helped the tissue regain stability and plumpness, contributing to improvements in range of motion, load bearing, and flexibility in the spine. Behavioral tests also showed a reduction in pain symptoms in the mice treated with gene therapy.
Using universal adult donor cells for creating extracellular vesicle therapies reduces the risk of inducing an immune response, making the gene therapy safer and potentially more effective. The researchers hope that the therapy, which aims to reprogram cells to a healthier state, would function as a one-time treatment. Additional experiments are planned to test the effects of other transcription factors involved in disc development, as well as investigating the therapy’s effects in older animals to model age-related disc degeneration. Eventually, this gene therapy approach may be tested in clinical trials for larger animals with back problems.
The researchers behind this study, including co-senior authors Devina Purmessur Walter and Natalia Higuita-Castro, have received funding from the National Institutes of Health to support their research on gene therapy for back pain. By continuing to explore the potential of gene therapy for musculoskeletal disorders, the team hopes to advance the field of noninvasive treatments for back pain. Collaborating with other experts in the field, the researchers are working towards developing novel therapies that could transform the way back pain is managed, offering patients a more effective and long-lasting solution to this debilitating condition.
The team’s work has resulted in a patent application for nonviral gene therapy for treating painful musculoskeletal disorders, highlighting the potential impact of this research on future clinical applications. By harnessing the power of gene therapy and nanotechnology, the researchers are paving the way for innovative treatments that could revolutionize the field of back pain management. Through further research and collaboration, this promising gene therapy approach may one day provide relief for individuals suffering from disc-related back pain, offering a new hope for improved quality of life and mobility for patients with this condition.
