Gene therapy for chronic pain and epilepsy

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Current treatments for chronic pain and epilepsy do not fully satisfy all clinical needs. In the case of chronic pain, epidemiological data shows that one in five adults is affected despite the use of current analgesics. It has a detrimental impact on an individual’s quality of life and results in huge economic costs to society as a whole, much greater than those attributed to heart disease or diabetes. Opioids and NSAIDs are the most commonly prescribed analgesics, but are associated with significant side-effect profiles. Furthermore, these drugs rarely provide adequate pain relief and are particularly ineffective against chronic pain caused by nerve injury (neuropathic pain).

Neuronal hyperexcitability is a key feature underlying neuropathic pain (and epilepsy). CASPR2 (contactin associated protein 2), a member of the neurexin family of proteins, regulates the function of potassium channels. These channels are key determinants of neuronal excitability and loss of their activity is implicated in neuropathic pain and epilepsy. Furthermore, disruption of CASPR2 by autoantibodies is associated with neuropathic pain and epilepsy in patients. Researchers at Oxford have shown for the first time that CASPR2 is directly involved in regulating the excitability of sensory neurons. The overexpression of CASPR2 reduces neuronal excitability and can be used as the basis for a method to treat pain, excessive neuronal activity and epilepsy.

A new approach

Based on these findings, Oxford researchers have developed a potential gene therapy to treat chronic pain and epilepsy. The gene therapy consists of an adeno-associated viral vector delivering a modified version of the CASPR2 gene. When used to target sensory neurons, the gene therapy is envisaged to result in the overexpression of CASPR2 such that the CASPR2 polypeptide reduces the excitability of the targeted neurons by increasing the activity of potassium channels, bringing therapeutic benefit. In experimental models, the resultant overexpression of CASPR2 has so far yielded promising results and further studies are ongoing.

The invention is the subject of a PCT patent application, which is published as WO2018060712(A1), and resultant patent applications in the US and Europe are currently undergoing examination.

Licensing opportunity

Oxford University Innovation is now seeking a commercial partner to develop further and bring to market this potential new treatment for pain and epilepsy under an appropriate licence agreement.

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