A drug discovery startup called Pantherics Incorporated is developing a pill-based alternative to current asthma treatments based on research conducted at UW-Milwaukee.
Doug Stafford, the company’s president and CEO, says his business is the first to develop drugs that affect a well-known target outside of the brain and central nervous system. He presented for WARF Innovation Day, held this week in Madison.
“We’ve shown extensively in animal models… that our drug can do two things we want it to: reduce inflammation and reduce bronchospasms,” Stafford said. “The drug opens the airway that’s closed due to inflammation-induced muscle constriction.”
Pantherics is broadly developing treatments for inflammatory diseases of smooth muscle, starting with asthma. This disease affects around 14 percent of the world’s population, but Stafford explains many other conditions have a similar mechanism. That includes diseases of the gastrointestinal tract and bladder, the heart and other parts of the body.
“In all of these conditions, underlying inflammation leads to unwanted contraction of muscle,” he said.
Patients with inflammatory bowel disease or irritable bowel syndrome experience unwanted muscle contractions that cause cramps and diarrhea. And for those with overactive bladders, inflammation causes the smooth muscles of the bladder to contract.
Stafford said treatments for diseases like these are somewhat limited, claiming the combination of inflammatory drugs and muscle relaxants are “not very effective” for many conditions. In asthma, the disease becomes resistant to the drugs, and other adverse effects are associated with steroid-based treatments.
By using a “novel mechanism of drug action,” Stafford says a single pill-based treatment can reduce underlying inflammation and relax smooth muscle contractions.
He explained that GABA receptors are well-understood biological targets for drugs that work within the central nervous system in the brain. Drugs commonly used to treat seizures or anxiety interact with this receptor, and Stafford said some of these therapies have been on the market for 50 years.
His research team was among a number of groups that discovered that GABA receptors are also found in tissues outside of the brain, which he said “creates really interesting opportunities.”
“In a broad sense, our drug discovery platform repurposes decades worth of knowledge on modulating GABA-A receptors for drugs that work within the central nervous system now for conditions outside,” he said.
Because the GABA receptor is a validated drug target, he said scientists understand “quite clearly” how it works, including the effects of other drugs that influence the receptor.
“So there are a number of aspects of this technology where we’re not starting from a dead stop,” he said. “We’re taking advantage of many decades of drug knowledge that we’re now applying to a new problem.”
Stafford said more than 334 million people have asthma worldwide, and the economic burden of the condition in the United States was around $81 billion in 2013. He noted associated prescription drug costs are “also rather staggering,” with 14 million individuals treated annually each paying around $1,800.
“That translates into a $25 billion plus therapeutic market for asthma treatment,” Stafford said.
Aside from his leadership position at Pantherics, Stafford is also the director for the Milwaukee Institute for Drug Discovery.
See more on the asthma research being done at the MIDD: http://uwm.edu/drug-discovery/research-2/asthma-2/