Disc-related back pain may soon have an effective treatment: gene therapy delivered via naturally derived nanocarriers. A recent study demonstrates that this approach can repair damaged spinal discs and reduce pain in mice.

Researchers engineered nanocarriers using fibroblasts, a type of connective-tissue cell, and loaded them with genetic material for a crucial tissue development protein. These nanocarriers were injected into damaged discs in mice simultaneously with the occurrence of a back injury.

Over a 12-week period, imaging, tissue analysis, and mechanical and behavioral tests showed that the gene therapy restored the structural integrity and function of degenerated discs and alleviated back pain in the mice.

“We have a unique strategy that can both regenerate tissue and inhibit pain symptoms,” said co-senior author Devina Purmessur Walter, associate professor of biomedical engineering at The Ohio State University.

Although further research is needed, these findings suggest gene therapy could provide a long-lasting alternative to opioids for managing severe back pain.

“This therapy could be used during surgery to enhance disc healing,” noted co-senior author Natalia Higuita-Castro, associate professor of biomedical engineering and neurological surgery at Ohio State. “Your own cells do the work and return to a healthy state.”

Published recently in Biomaterials, the study builds on previous work in Higuita-Castro’s lab. They had previously reported that nanocarriers loaded with anti-inflammatory agents mitigated tissue injury in damaged mouse lungs.

The new study used FOXF1, a transcription factor crucial for tissue development, as the therapeutic cargo. This protein, important for tissue growth, declines with age. By reintroducing FOXF1, the researchers aimed to revert cells to a healthier, more developmental state.

Mice treated with FOXF1 nanocarriers showed significant improvements compared to controls. Their discs regained stability and function, enhancing spinal flexibility and reducing pain symptoms, although pain response varied by sex.

The researchers highlighted the advantage of using universal adult donor cells to create these therapies, as they are less likely to trigger an immune response. Ideally, the gene therapy would be a one-time treatment, providing lasting benefits.

Future experiments will test other transcription factors involved in disc development and evaluate the therapy in older animals and larger models of back pain. The study’s promising results pave the way for potential clinical trials.

The research, funded by NIH grants, involved multiple collaborators from Ohio State, Icahn School of Medicine at Mount Sinai, and the University of Manchester. Ohio State has filed a patent application for this nonviral gene therapy to treat musculoskeletal disorders.


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