“This has the potential to be a blockbuster,” said Dr. Stephen Waxman, a professor of neurology, neuroscience and pharmacology at Yale. Dr. Waxman was not associated with the study but was paid a speaking honorarium by the company. He predicted that the Vertex drug would be only the first foray into this new area.
“I like to think it’s the beginning of nonaddictive medicines for pain,” he said.
For now, most people needing relief from moderate to severe pain have two options: drugs like ibuprofen and COX-2 inhibitors, or opioids. The drugs like ibuprofen are not very effective, and the opioids, as is well known, can be addictive because of the way they work. There is no way to separate the effects of opioids — pain relief — from the side effects: changes in thinking, cognition, energy and emotions.
The opioid crisis, one of the gravest public health concerns in the United States, began more than two decades ago and included people who started out taking the drugs for pain but became addicted. As states tightened regulation of prescription opioids, many turned to illegal street drugs like heroin and fentanyl. Though doctors are more cautious about prescribing opioids now, many still do so because there are few alternatives.
Efforts to develop a new class of pain-treating drugs began in earnest in the 1990s. Researchers asked if there were sodium channels that were specific for peripheral nerves. These are portals that open to send pain signals from the nerves to the brain and then close to stop transmitting. If there were portals that only controlled signals from peripheral nerves, that suggested the possibility of drugs to block them and control pain without affecting the brain, and without causing addiction. Pain might be stopped at its source.
So researchers began scouring the globe for people who had genetic mutations that prevent peripheral nerves from transmitting pain signals, or that made peripheral nerves signal pain nearly constantly. If they found those mutations, the genes involved could be targeted with drugs.