Kyle Rohde, PhD, Professor of Biomedical Sciences, University of Central Florida, talks about cystic fibrosis and its impact on the human system.
If you could for our viewers, just explain what cystic fibrosis is and what’s happening in a person’s system?
ROHDE: The patients with cystic fibrosis have a mutation in the CFTR transporter, and it’s in the cells that line the lung airways. This causes a buildup of very thick mucus in the lungs that compromises their immune system and sets them up for recurring bacterial infections of the lung and also impacts other organ systems, too. Most people think of only the lungs, but it has effects on the GI tract and other parts of the body as well.
When a CF patient gets these bacterial infections and these lung infections, I would imagine it’s bad for anyone, but for someone with CF it has added impact?
ROHDE: Right. There’s a lot of these pathogens that don’t normally cause infections in healthy people that have a functioning immune system, but the complications that come along with cystic fibrosis make them susceptible to these opportunistic pathogens like Mycobacterium abscessus.
And I was going to ask, as of right now, no cure for CF, it’s just treatment, right?
RHODE: Yeah, I think the treatments have come a long way and the life expectancy and life quality have improved dramatically in the past years. But it’s still not curable.
You and your colleagues in this lab are looking at one infection. Can you tell me one bacteria in particular, can you tell me a little bit about that?
ROHDE: Right. We’re focused right now on Mycobacterium abscessus, which is kind of a cousin of Mycobacterium tuberculosis, and it is a member of a group of pathogens called non-Tuberculosis Mycobacteria, or NTMs. Those are a big problem in CF patients. They’re extremely hard to treat and is one of the major problems. It’s a very sort of slow, insidious disease process, but very difficult to treat with antibiotics.
And what do they cause? What kind of difficulties?
ROHDE: They cause symptoms very similar to tuberculosis. They will cause granulomas, lesions in the lung and cause just chronic inflammation in the airways as well. So eventually you get destruction of the lung tissue and loss of lung function.
So, tell me a little bit more about your research and what you’re looking at and what you’re hoping to determine.
RHODE: We’re focused on a number of different aspects of Mycobacterium abscessus. Two of the major areas are involved in drug discovery, so trying to find new antibiotics that can kill Mycobacterium abscessus, as well as the mechanisms of drug resistance. Why are these bacteria so hard to kill with the existing antibiotics? A new project that we just started, funded by the Cystic Fibrosis Foundation actually, is to try to implement a cystic fibrosis mouse animal model so that we would have a way to model this infection in a CF patient, in this case a mouse patient. That will open a lot of doors to better study the infection process, to test new developmental antibiotics and treatment options. We’re excited to get that project started thisu summer.
You said you’re studying in a mouse model. Are there more than one antibiotic that you’ll be testing? Are you looking at a whole range of things in an animal model?
RHODE: Once we have an animal model that is up and running, that will open the doors for almost every aspect of our research on that pathogen. We have several ongoing projects where we’re trying to discover new antibiotics that aren’t currently in use for treating this bacteria and oftentimes they work well in the lab. Then something about the bacteria changes during an infection that makes it more difficult to kill. So now we’ll hopefully be able to study that and find the drugs that work in a real-life kind of setting. When you have the mucus and the bacteria in the lungs, it’s very different than killing bacteria in the dish.
How critical is it in terms of treating this disease to find those new antibiotics, something that will work for the patient?
RHODE: I would say it’s extremely important. At the current time, you’re looking at a treatment with four or five antibiotics for up to a year, and that includes injectable antibiotics. You would have to go to the doctor regularly to get your treatment and those have extreme side effects. A lot of people can’t even finish the treatment because the side effects become so bad. I believe the estimate is between 50 and 70 percent failure rate of treatment. You often will see people that are infected with Mycobacterium abscessus for five, six, seven years and they’re trying different treatments to just keep it at bay, and then it comes back again, and so oftentimes it leads to a lung transplant or a resection of a large chunk of your lung.
That was my next question, for our viewers who are not that familiar, how potentially devastating are these infections to people?
RHODE: So infections with Mycobacterium abscessus and other bacteria like Pseudomonas generally the leading cause of death in CF patients, as sort of the CF sets them up for the infection that is ultimately the downfall of the patient. Finding better treatments for these infections that you see in CF patients is critical to extending their life.
Is there anything I did not ask you about your research? I know that you’re trying to set up the animal model. Do you have a timeline or a timeframe for the project?
RHODE: We’re in the process of breeding the mice for the launching that infection experiment. I think during the summer and early fall would be the early pilot experiments .
One other question. Is there anything else I didn’t ask that you would want to make sure people know?
RHODE: It’s hard to say. Not that I know of.
Anything that you would want to make sure that our viewers at home take away from this about your research?
RHODE: I think just an increased awareness of some of these infections that may not be mainstream, or many people may not appreciate the severity of them. Tuberculosis is another infection we work on that people often think is cured. Unless you’re aware of cystic fibrosis patients, you may have never heard of Mycobacterium abscessus. So just, I think, an increased awareness of how the process of research functions. I think one thing people may not be aware of is that most drug discovery for antibiotics is now not being done by the big companies and big pharma is generally leaving that area of work. It’s academic researchers that are picking up the slack, so to speak, to try to discover, you know, the next generation of antibiotics.
And you had mentioned for patients with this particular infection, there might be four or five antibiotics that would work. Is there a concern about resistance? And if you only have a couple of things that work right and you have to take them over a period of time, is that another concern that you guys are looking at?
RHODE: It is as a huge concern because you barely have a front-line regimen that takes multiple drugs. And if it does happen that you have strains that emerge that are resistant to one or two of those you’re not left with many options. That is another part of the problem, is having backup drugs for those circumstances to treat drug resistant strains. And so that’s in the process of discovering drugs, you want to find brand new drugs that work in a completely new way that would be able to kill the drug resistant varieties that are already out there.
I just want to make sure that I heard you correctly, MAB, did you say it’s the leading or one of the leading causes for the need for a lung transplant?
RHODE: I don’t know that it would be accurate to say it’s the leading, but infections like M. abscessus and Pseudomonas that cause sort of irreversible damage is often the outcome when one treatment doesn’t work.
Interview conducted by Ivanhoe Broadcast News.
END OF INTERVIEW
This information is intended for additional research purposes only. It is not to be used as a prescription or advice from Ivanhoe Broadcast News, Inc. or any medical professional interviewed. Ivanhoe Broadcast News, Inc. assumes no responsibility for the depth or accuracy of physician statements. Procedures or medicines apply to different people and medical factors; always consult your physician on medical matters.
If you would like more information, please contact:
CHRISTIN SENIOR
321-900-5624
Sign up for a free weekly e-mail on Medical Breakthroughs called First to Know by clicking here
