NACFC 2023: “Micro-Management”: The Changing Face of Infections in CF

Steven Rowe, MD, executive vice president and chief scientific officer of the CF Foundation, awards the Dorothy Andersen and Paul di Sant’Agnese Distinguished Scientific Achievement Award to Robert J. Bridges, PhD, professor emeritus and director of cystic fibrosis research at Rosalind Franklin University of Medicine and Science.

Introduction to the plenary and recap of the Infection Research Initiative

What are the bacteria that infect people with CF?

How has the prevalence of CF respiratory pathogens changed over the years?

How do we detect CF respiratory infections?

What are the problems with diagnosing infections?

What are some of the techniques being developed to address the problems with diagnosing infections?

What have we learned about CF infections?

What are the upsides and downsides of using antibiotics in CF?

What are the unintended effects of antibiotics on the bacteria living in people with CF?

People with CF are producing less sputum — another problem in diagnosing and treating infections

What do ongoing research studies tell us about the effects of CFTR modulators on respiratory microbiology?

What can we learn from microbiology in other countries? What are the lessons from Europe?

Nick Kelly, an adult with CF, explains why it is important to find new and better treatments for infections

How will we treat CF infections in those on and off Trikafta®?

What have we learned and where are we going — pulmonary exacerbation trials

How many antibiotics should we use to treat infections? The STOP360 trial.

What did the STOP-2 pulmonary exacerbation trial tell us?

What is the STOP-PEDS trial?

Why do women with CF have worse outcomes than men?

Will infections have the same implications in the era of Trikafta?

What anti-infectives are in development with the support of the CF Foundation?

What is bacteriophage therapy?

What were the results of the BiomX bacteriophage trial?

Hope for All: Addressing the Needs of Those With Untreated CF Mutations

CF Foundation President and CEO Michael Boyle, MD, welcomes viewers to Plenary 1 and gives a brief update on the Path to a Cure.

Cam McLoud, immediate past chair of the CF Foundation’s Board of Trustees, thanks the cystic fibrosis community for their commitment to find a cure for CF and introduces the Foundation’s current board chair, KC White.

KC White, chair of the CF Foundation Board of Trustees, shares her personal experience as an adult with CF.

Michael Boyle introduces the topic and speakers for Plenary 1.

Steven Rowe, MD, chief scientific officer of the CF Foundation, shares his experience as a physician and researcher in CF.

Improvements for people with CF on modulators and turning our focus to people who have not been able to access these treatments

Modulators are not a cure for cystic fibrosis; data show lung disease still progressing.

Path to a Cure: programs in development

The McClains discuss their experience raising their daughter, Abigail, who is not able to take modulators to treat her type of cystic fibrosis.

How nonsense mutations prevent the successful production of the CFTR protein and potential treatments

Lab data from three specific approaches to restoring CFTR expression and function

Closing remarks and introduction of Michelle Hastings, PhD, professor at the Chicago Medical School at Rosalind Franklin University of Science and Medicine

Dr. Hastings introduces antisense oligonucleotides (ASOs).

How RNA splicing removes additional genetic sequences, called introns, so that proper gene expression can occur

ASOs may be a potential treatment to correct splicing mutations

How ASOs may be used to treat frameshift and nonsense mutations when paired with a CFTR modulator

Closing remarks and introduction of Rebecca Darrah, PhD, MS, associate professor and genetic counselor at Case Western University

Dr. Darrah reflects on the power of the CF community and prioritizes the need for treatments for people of color with cystic fibrosis, who disproportionately face more challenging disease courses.

How mRNA-based therapies could potentially treat people with cystic fibrosis regardless of their type of mutations

Data from two companies pursuing mRNA therapy for CF, ReCode and Arcturus

Conversations about CF genetics can be confusing

A helpful technique that can help people more easily understand basic CF genetics and mRNA therapies

Closing remarks

NACFC highlights

Projected growth in the CF population over time

Higher lung function expected over time

Lung function and symptoms after Trikafta

Effect of withdrawal of medications after Trikafta (Simplify study)

Detecting infections post-Trikafta

NTM infections in CF

Some results from survey on well-being post-Trikafta

Liver disease in CF: new guidelines coming

Impact of Trikafta on the pancreas

Report on baby born to mother with CF on Trikafta

Use of Trikafta in people with CF post-lung transplant

Results of a study of Trikafta in children with CF ages 2-5

Lab test results of a potential new modulator

Discussion of genetic therapies in development

Sionna study: Is this a different formulation of Vertex’s 121 modulator study?

Do we know how close mRNA therapies are to phase 1,2,3 clinical trials?

Are people with CF able to take Paxlovid if they get COVID and they are also taking Trikafta?

How are we studying the effects of withdrawing medications while on Trikafta following the Simplify study?

Why do we use viruses to deliver genetic therapies?

Is CRISPR gene editing something that can be used in treating CF some day?

If a modulator study shows a decrease in sweat chloride, will that translate into a better clinical effect in patients?

Does Trikafta affect hormones in people with CF?

What research is happening to help people with nonsense mutations?

How do you try to balance the two patient populations — those with a modulator and those without — which are starting to have very different needs?