Virus-Delivered Gene Therapy Provides Hope for Neuropathic Pain Relief What are the main findings?

Response: A single transforaminal epidural injections of KLS-2031 to target the appropriate dorsal root ganglion (DRG) produced notable long-term analgesic effects in both female and male rats. The three therapeutic genes expressed from KLS-2031 synergistically inhibit the major pathological mechanism of NP. The superior analgesic effect of KLS-2031 is the result of a multiple-target approach, which suppressed neuronal hyperactivity, damage, and neuroinflammation. The therapeutic genes delivered by KLS-2031 not only reverse these neuropathological changes but also normalize the excitability of peripheral nerves, indicating that this treatment controls pain sensation while promoting a favorable environment for nerve function. What should readers take away from your report?

Response: KLS-2031 has shown long-term analgesic effect. It reduced the number of apoptotic cells and activated microglia in the DRG, and restored the sensitivity and excitability of small-sized neurons in the DRG, which are known to contain pain-signaling c-fibers. Our study demonstrated that KLS-2031 not only alleviates NP symptoms but also achieves neurophysiological improvement. These findings demonstrate the potential success of NP treatment by targeting multiple aspects of complex underlying pathophysiology. What recommendations do you have for future research as a result of this work?

Response: So far, we have studied the mechanism of action (MOA) of KLS-2031 in the peripheral nervous system, and we will continue to study the MOA of KLS-2031 in the central nervous system. Furthermore, based on the finding that DRG neuronal excitability was significantly increased in the neuropathic pain model and recovered by KLS-2031 treatment, we are planning to examine the precise mechanism by which neuronal excitability is modulated by KLS-2031. Is there anything else you would like to add?

Response: KLS-2031 has the potential to address unmet medical needs for neuropathic pain. We have demonstrated that a single administration of KLS-2031 has long-term efficacy and has the potential to be a disease-modifying agent. KLS-2031 is currently undergoing a phase1/2a clinical trial for the treatment of lumbosacral radiculopathy in the USA.


Kim, Daewook & Kim, Kyung-Ran & Kwon, Yejin & Kim, Minjung & Kim, Min-Ju & Sim, Yeomoon & Ji, Hyelin & Park, Jang-Joon & Cho, Jong-Ho & Choi, Heonsik & Kim, Sujeong. (2020). AAV-mediated combination gene therapy for neuropathic pain: GAD65, GDNF, and IL-10. Molecular Therapy – Methods & Clinical Development. 18. 10.1016/j.omtm.2020.06.018.

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Last Updated on August 10, 2020 by