UNIVERSITY OF ILLINOIS (WCIA) — Researchers at the U of I have been working on a cystic fibrosis treatment for more than a decade. It involves a drug that’s already been approved for people — and now they’re finally ready to test it.
One of the people who could benefit is fourth-grader, Sarah Bolton. Her everyday routine isn’t like most of her peers.
“We have this vesting that kind of pounds on my chest, and also we kinda have these different medicines and a mask that I breathe in when I’m doing it,” says Bolton. Bolton has cystic fibrosis. It’s a genetic disease that’s caused by the lack of function of a specific protein. That leads to lung problems, including infections and difficulty breathing. Bolton’s mother is a science professor.
“There are a lot of drugs right now that are in clinical trials and that have come to market that can help fix some forms of that broken protein … These medications work for some patients. None of them right now are working for Sarah and her mutations,” says Rachel Smith-Bolton.
But researchers are working on something that has the potential to change Bolton’s life.
“We are committed to trying to do everything we can to find out as quickly as possible — and as safely as possible — if this can be helpful for people with CF,”says Marty Burke. He’s a chemistry professor and Associate Dean for Research at the Carle Illinois College of Medicine.
Burke was inspired to find a treatment when he was in medical school. He was talking about CF with a woman who had it.
“At some point in the conversation, she stopped me. And said it sounds like you know exactly what the problem is — why can’t you fix it?” says Burke. That moment was life-changing.
“Could we replace her missing protein with a small molecule surrogate — so a drug-like molecule — that would do what the protein that was supposed to be doing … almost like a prosthesis on the molecular scale,” he says.
The possible treatment involves a product that’s already approved by the FDA. It’s called amphotericin. It’s used to help with fungal infections. When they first started looking at it, they knew that the drug formed ion channels — but it was thought that the channel activity caused the drug to be toxic if given through the bloodstream. Years of research proved that wasn’t true. They were able to separate the channel activity from toxicity. They tested this in the lab — partly on donated lung tissue, and sometimes in pigs. Results showed this could help fight lung infections, and in theory, work with everybody who has cystic fibrosis.
Burke and his students have been working on this since 2005. They teamed up with the University of Iowa for the last few years. Patients like Sarah, and her mother, say they’re cautiously optimistic.
“There have been other medications we’ve been excited about that have looked really really good in phase one and two clinical trials. But when it got to that big phase three trial where they looked at a lot of patients — it just wasn’t as we anticipated so we’re really keeping our fingers crossed,” says Smith-Bolton.
Burke knows what’s at stake. He’s hoping for the same outcome as the Bolton’s.
“All the data we have thus far is not showing this can work in people yet — it’s all done in the lab and in pigs — but the data we have thus far gives us a lot of hope and optimism that there may actually be potential here,” says Burke.
They’re still working on figuring out how clinical trials will work.
The project was paid for by the National Institutes of Health and Emily’s Entourage. For more information on Emily and her mission, you can click here.