Gene therapy offers hope for patients with muscle spasticity

Spinal cord injury is often accompanied by a condition in which muscles become stiff, tense, or contract involuntarily and uncontrollably. This condition is known as muscle spasticity. Its severity varies considerably from patient to patient, ranging from mild muscle stiffness to severe spasms that can significantly interfere with everyday activities. Current treatment of muscle spasticity following spinal cord injury requires the long-term administration of medication. An international team of researchers has now developed a gene therapy that, according to the results of a preclinical study, could reduce the symptoms of the condition after a single administration.

Our colleague, Hana Studenovská, is a member of the international research team that has been working for many years on the development of gene therapy to reduce muscle spasticity. The team, led by Martin Marsala at the University of California San Diego, recently published its findings in the prestigious journal Molecular Therapy. The study demonstrates that a single administration of segment-targeted gene therapy to the spinal cord could provide a long-lasting therapeutic strategy not only for reducing muscle spasticity following spinal cord injury, but potentially also for treating other neurological disorders associated with abnormal muscle tone or increased neuronal activity.

As part of this long-term international collaboration, Hana Studenovská's research group developed biodegradable polymer microparticles for the controlled release of the immunosuppressive drug tacrolimus. Immunosuppression is the targeted suppression of the immune system using drugs such as tacrolimus. As Hana Studenovská explains: "The technology developed by my team enabled the safe testing and validation of the entire therapeutic strategy. This represents one of the key prerequisites for the potential translation of the technology into clinical practice." The polymer microparticles gradually release tacrolimus over several days, providing long-term immunosuppression following a single subcutaneous injection. As she further adds: "This allowed us to establish the conditions necessary for the safe evaluation of the gene therapy in large animal models."

The validation of the therapeutic strategy in a porcine model was carried out in collaboration with researchers from the Institute of Animal Physiology and Genetics of the Czech Academy of Sciences, who also issued a press release describing the study: https://www.iapg.cas.cz/cs/Verejnost-a-media/aktuality/Nova-genova-terapie-snizuje-svalovou-spasticitu-po-poraneni-michy.

An overview of the study is also available in a news article published by the University of California San Diego: https://today.ucsd.edu/story/gene-therapy-approach-reduces-muscle-spasticity-after-chronic-spinal-cord-injury.

We warmly congratulate the entire research team and wish them continued success in advancing this promising therapeutic approach.