Targeting Chronic Pain
Chronic pain, including that experienced by people with arthritis, affects more than 50 million Americans. However, currently used painkillers, like opiates such as morphine, are typically administered systemically and often come with unwanted side effects.
Benjamin Storek and colleagues at Mount Sinai School of Medicine in New York have developed a targeted gene therapy technique that simulates the pain-killing effect of opiate drugs but avoids the side effects associated with oral or intravenous treatment. The researchers designed a viral vector to carry the prepro-β-endorphin gene into primary sensory neurons and selectively activate opiate receptors. They delivered the agents directly to the spinal fluid of rats via lumbar puncture, or spinal tap. With a single injection, the rats remained symptom-free for over three months. The researchers found that the prepro-β-endorphin gene was targeted selectively to nerve cells at the “pain gate” and that its activity remained long-lived. Another therapeutic gene, interleukin-10, was also effective when similarly administered in small doses directly at the spine. Together, the improvement in symptoms and the narrow range of targeting suggest that the therapy could be an effective, and specific, way to treat chronic pain.
This article was adapted from a press release issued by the National Academy of Sciences.
Storek B, Reinhardt M, Wang C, et al. Sensory neuron targeting by self-complementary AAV8 via lumbar puncture for chronic pain. Proceeding of the National Academy of Sciences