Fighting Infections in the Age of CFTR Modulators

As a mother of two children with cystic fibrosis, I am glad to hear that the Cystic Fibrosis Foundation is launching the Infection Research Initiative to address the chronic infections that dramatically impact the lives of people with CF.

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Dara Riva
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Infections are a huge part of life with cystic fibrosis, and they can cause a lot of anxiety for people with CF and their families, said Tré LaRosa, in a video played during the first plenary today at the North American Cystic Fibrosis Conference in Denver.

Those words resonated with me as a mother of two children with CF. Somewhere in the back of his mind, Tré worries that the next infection could be the one he won't be able to recover from. This fear stems in part from the loss of his sister, Alyssa, to CF in March despite two lung transplants.

“Seeing what my sister went through and knowing that at any point, it's possible that I might not respond to the antibiotics, that is something that does scare me a lot,” Tré said in the video shown to an audience of more than 5,000 clinicians and researchers.

That scenario concerns me, too. That is why I am so excited that the Cystic Fibrosis Foundation is dedicating at least $100 million over five years for the new Infection Research Initiative. Lisa Saiman, M.D., MPH, a professor of pediatrics at Columbia University Medical Center, discussed the objectives of the initiative during her plenary address, Improving Outcomes of Infections in the Age of CFTR Modulators.

Dr. Saiman said the initiative is focused on six key areas of research that have been selected with input from people with CF and their family members:

  1. Improving detection and diagnosis
  2. Understanding CF microorganisms
  3. Developing new treatments
  4. Improving current treatments
  5. Evaluating long-term antibiotic use
  6. Looking at the future of CF infection

I like the initiative because it is truly comprehensive and touches on topics that I care about as a mother and someone who will be working on the initiative at the Cystic Fibrosis Foundation.

There's so much more to infections than just the development of new drugs. For example, are we detecting microorganisms (such as bacteria, viruses, and fungi) before they do much damage? What are the effects of long-term antibiotic use? Will my children's infections change once the triple-combination modulators are available, and they can take them?

Dr. Saiman built her speech around the focus areas of the initiative, including how the initiative will build upon the breadth of research that is already underway. She discussed the challenges of treating infections in people with CF, the results of ongoing work to improve our understanding of CF microorganisms, and strategies to develop new anti-infectives for the future.

The main message of the plenary was that despite the advent of CFTR modulators, people with CF are going to need effective treatments for infections and other complications.

Understand CF Microorganisms Better

People who can take modulators will require treatments for lung damage, and we don't know how they will affect infections, for example, Dr. Saiman said. In fact, we don't know all the implications of current anti-infectives on the CF lung microbiome

 

. Researchers are increasingly focused on the interactions between these bugs, like how killing off one could allow something more dangerous to become dominant. A doctor needs to consider all these factors when deciding whether to treat a positive culture of bacteria.

Although some microorganisms common in people with CF are decreasing, others such as nontuberculous mycobacteria (NTM) are increasing. Dr. Saiman talked about studies such as PREDICT and PATIENCE, which aim to create standard ways to diagnose and treat NTM, and the formation of a consortium of participating study sites that could accelerate future NTM clinical studies.

Optimizing and Developing New Treatments

Dr. Saiman talked about the STOP 2 study, which is looking at the duration of intravenous (IV) antibiotic treatment during pulmonary exacerbations

 

 to determine a standard treatment time. Optimizing current treatments like this is a big win for me as a mom. I'm never sure whether having my children stay on antibiotics longer is better than trying a different antibiotic altogether when they have a stubborn infection.

Another pet peeve: throat swabs! I know they are effective in getting samples to test for bacteria, but I hated the trauma my kids went through as toddlers when they had to get them. I'm looking forward to rapid diagnostic tests under development that could detect bacteria types in a patient's breath. Dr. Saiman said that earlier identification from point-of-service tests like this could help manage exacerbations more quickly.

Dr. Saiman also updated us on the current approaches to fighting infections: borrowing treatments from other infectious diseases and conditions, altering existing anti-infectives to inhaled versions, and developing drugs that fight infections in novel ways. For example, she talked about a drug in a late-stage clinical trial that works on breaking up biofilms

 

. Researchers believe the drug will make bacteria more susceptible to antibiotics.

The Foundation has funded 12 infection-related programs that are in various stages of development, and they are in talks with 17 additional companies about other potential drugs, Dr. Saiman said.

A lot still needs to be done to fight CF infections, but she closed her talk by saying she was optimistic because of the “unbelievable tools” that CF researchers have in their toolbox, including highly motivated people with CF and their families, deeply caring clinicians, international collaborations, and funding.

Tré said that knowing that the Foundation is prioritizing infections gives him hope. It gives me hope, too, for my kids, Penelope and Maximilian, and everyone else with CF out there.

Watch the full plenary below.

In Plenary 2, we learned what the Foundation is doing to treat excessive mucus and inflammation, two other complications that wreak havoc in people with CF.

Join the conversation on Facebook.

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Dara has worked at the Cystic Fibrosis Foundation since 2017. Previously, she was a science officer for the Congressionally Directed Medical Research Programs and Research Associate for the Frederick National Laboratory supporting the National Institutes for Allergy and Infectious Diseases. Dara is committed to helping find a cure for her two children who have CF, Maximilian and Penelope, and actively fundraises for the Maryland Chapter. She is also a member of the Family Advisory Board at the Johns Hopkins CF Care Center where her children receive their care. Dara lives in Frederick, Md., and, in her spare time, she enjoys hiking and being outdoors with her husband, two children, and two rescue dogs, Marshall and Molly.

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