Researchers at the University of Waterloo are working on new synthetic drugs which may be the cure for degenerative eye diseases as well as similar issues in other parts of the body.
A drug, developed by Sachdev Sidhu and his associates at Waterloo’s school of pharmacy, aims to treat patients with neovascular age-related macular degeneration (AMD) as well as diabetics who are at risk of developing diabetic macular edema (DME). The drug is currently in trial stages in the U.S.
According to the university, the drug activates a group of proteins that share information into a cell which will improve blood vessel integrity in the eye and also block fluid from building up in the retina.
Sidhu said the drugs he and his team are working on are a new type as they work to regenerate cells rather than look for ways to block something as most recently introduced drugs do.
“If you think of most drugs that have been approved over the last two decades, whether they’re small molecules or the COVID drugs or Herceptin, they turn things off,” the research professor explained.
“So what our drug does is something much more challenging. It takes a pathway that is stopped and turns it back on. So that’s why it’s a fundamental difference.”
He used the analogy of stopping a car from working as a way to explain the difference.
“It’s like building a car rather than blowing it up,” Sidhu said. “It’s very easy to stop stuff. But if you want to fix something, you have to know a lot more. You have to precisely coordinate,” Sidhu said.
“And what’s exciting for us is we have learned how to turn on these fundamental pathways, which nobody has been able to do since the three decades when things were first discovered.”
He said he believes there are many issues that the drug could be applied to as others are already in the works.
For example, he mentions degenerative diseases that prevent cells from growing enough, such as Alzheimer’s, Parkinson’s, inflammatory bowel disease, osteoporosis, liver degeneration, and lung fibrosis.
“What all these have in common, among other things, is that at least some pathways that normally cause these tissues to regenerate are either defective or slow, because tissues don’t just sit around in the body, they have to be actively stimulated to grow.”
With the eyeball drug currently in testing, the researchers have already begun to develop similar medications to treat other parts of the body, including the stomach and lungs.
“Our next one, which we’re already writing up, we’ve patented it. We’re writing up the publication and we’re already looking for a partner in industry to develop it,” Sidhu said.
“Just like this protein stimulates regeneration of eyes, we’ve shown in advanced models of disease that this second one we’re making stimulates regeneration of the gut.”
The scientist said that if the drug ends up being effective, it could be used to help treat irritable bowel disease and Crohn’s disease, which affects tens of millions of people.
Sidhu said the research team is also collaborating with Genentech, where he used to work, on a drug to rebuild damaged lungs.
He said he believes that these two drugs are about two years behind the eye treatment in terms of their developmental process.
“So the cool story that’s emerging is we’ve tapped into a way of turning on pathways and a family of proteins that are responsible for maintaining and regenerating a lot of major tissues,” Sidhu said.
The regenerative drug that Kahn and his colleagues have developed for eye treatment is currently in the first phase of testing in the United States.
“It’s in patients and it seems to be safely tolerated. And within a year or two we should have information on whether it’s working,” he explained.
Given the uncertainty of how medicine is approved, if the eye treatment is effective it will still be a while before it is able to hit the shelves.
“It is already in what’s called ‘Phase 1/2 trials’ in that safety and effectiveness will be measured at that same time,” the scientist said.
The company conducting the testing in the U.S. is currently looking for patients suffering from eye diseases to test the drug, according to Sidhu.
“Once they get enough patients to show it’s both safe and effective, I’m estimating two to three years and then it should be a larger trial after that,” he said. “So maybe, you know, ballpark, as in four years, it would be ready for everybody.”