Cystic Fibrosis News Today brought you daily coverage of important discoveries, treatment developments, clinical trials, and other important events related to cystic fibrosis (CF) throughout 2019.
As we look forward to bringing you more CF news this year, we present our 10 most-read stories of 2019.
A study published in the journal Frontiers in Microbiology found that Manuka honey, a type of honey produced from the nectar of a flowering plant native to Australia and New Zealand, has strong activity against Pseudomonas aeruginosa, a bacteria highly resistant to antibiotics that often causes lung infections in CF patients. The honey’s strong antibacterial activity is due to the presence of a compound called methylglyoxal in its composition. In the study, researchers put the antibacterial activity of Manuka honey to the test against 28 isolates of P. aeruginosa. They found that in two ex vivo porcine lung models, a solution containing 64% of Manuka honey was able to destroy many bacteria isolates, whereas high concentrations of antibiotics could not. Moreover, they showed that when combined with antibiotics, Manuka honey eliminated 90% of the bacteria.
In a study published in the journal Chemical Science, researchers described a new method to treat CF that involves delivering artificial molecule transporters, called anionophores, to replace the faulty cystic fibrosis transmembrane conductance regulator (CFTR) protein, and restore the transport of water and salt in and out of lung cells. Scientists identified four promising candidates that were found to improve the transport of molecules in and out of cells containing the F508 deletion, the most common CFTR mutation associated with CF, when used in combination with lumacaftor and ivacaftor, the active ingredients of two of Vertex Pharmaceuticals’ approved CF therapies, Orkambi and Kalydeco.
In a study published in the journal Nature Communications, researchers from the University of Iowa Carver College of Medicine described a new technique to genetically prevent disease-related mutations from occurring. This new method uses man-made RNA molecules, called anticodon engineered transfer RNAs or ACE-tRNA, that prevent cell machinery components from recognizing abnormal stop signals introduced by mutations in the DNA sequence of a gene, resulting in the production of functional, normal-length proteins. According to scientists, this new method can potentially be used as a form of gene therapy to correct defects that are the underlying cause of many genetic disorders, including CF.
Cystic Fibrosis News Today interviewed Preston W. Campbell, MD, president and CEO of the Cystic Fibrosis Foundation (CFF), to discuss treatment developments for CF. Campbell mentioned that triple combination therapies of CFTR modulators — a group of CF therapies that attempt to correct the defects in the CFTR protein that lead to the onset of disease — seem promising. When asked about lung transplant, Campbell said that deciding to undergo a transplant is a “personal decision,” and noted that CFF established a multi-year Lung Transplant Initiative to support those who decide to move forward on that path.
In October, Cystic Fibrosis News Today interviewed Jeffrey Leiden, MD, PhD, chairman, president, and CEO of Vertex Pharmaceuticals, to discuss the approval of Trikafta, the company’s first triple combination therapy — elexacaftor (VX-445), plus tezacaftor, and ivacaftor (Kalydeco) — for those with CF, 12 and older, carrying one F508del and one minimal function mutation, or two F508del mutations. Trikafta is meant for those who failed to respond, or responded poorly, to other approved Vertex CF therapies, including Kalydeco, Orkambi, and Symdeko (tezacaftor/ivacaftor, ivacaftor — known as Symkevi in the European Union). Leiden was especially thankful to the CF community for its commitment and effort to make this approval a reality.
Cystic Fibrosis News Today covered the FDA approval in October of Vertex Pharmaceuticals’ Trikafta, the first triple combination therapy of CFTR modulators that holds the potential to treat 90% of patients with CF. The approval was based on data from two Phase 3 trials, AURORA F/MF (NCT03525444) and AURORA F/F (NCT03525548), which were both part of the company’s AURORA clinical trial program. AURORA F/MF enrolled 403 CF patients who carried one F508del mutation, and AURORA F/F enrolled 107 CF patients who carried two copies of the F508del mutation. Pooled data from both studies showed that treatment with Trikafta led to significant improvements in lung function, CFTR activity, and quality of life, with minimal side effects.
In a study published in the journal Nature Communications, researchers from Trento University in collaboration with investigators from the Katholieke Universiteit Leuven, described how they used the CRISPR gene editing tool to permanently correct two mutations in the CFTR gene that prevented the production of a normal and functional CFTR protein. With this strategy, they managed to correct CFTR mutations in lung and intestine organoids made up of cells from CF patients. The team said this strategy can potentially be used to correct other mutations that cause CF, and even be applied to other genetic diseases.
In August, Cystic Fibrosis News Today reported on research currently underway in the lab of Laura Niklason, MD, PhD, at Yale University. Yale scientists are seeking to address the shortage of donor lungs by finding a way to create these organs artificially in the lab. They are attempting to create these lab-made lungs by using a harvested lung that’s devoid of cells as a scaffold. They will then place hundreds of millions of cells on these scaffolds which will grow and form new airways and blood vessels. This technique has already been used successfully to create lungs from adult rats. It now remains to be seen if the same approach can be used in humans.
In March, Cystic Fibrosis News Today interviewed Somer Love, a patient activist from Salt Lake City, Rima Manomaiitis, a CF patient from Denver, and Gunnar Esiason, who runs the New York-based Boomer Esiason Foundation, to discuss the controversy that the movie “Five Feet Apart” has created within the CF community. “Five Feet Apart” tells the story of two teenagers with CF who meet and fall for each other while being treated in a hospital. Although aware that they should stay six feet apart to avoid cross-infections, they decide to get closer and remain within five feet of each other, hence the name of the movie. While many people, including Love, felt the movie did a good job depicting the life of those with CF, and could be a great way to raise public awareness about the disease, others argued that the movie overly romanticized the disease. Some, including Esiason, also criticized the movie because they believed it failed to represent the full spectrum of people living with CF, since its main focus was solely on people who were already very sick. Some also said the movie may send a wrong and potentially dangerous message that it’s OK to break the six-feet-apart rule. Manomaiitis, who liked the movie, told Cystic Fibrosis News Today that she hopes this movie encourages the general public to make more donations to help in the fight against CF, and get involved in other ways.
In a study published in the Journal of Cystic Fibrosis, investigators described how a device that helps those with diabetes control their blood sugar levels could also be used by patients with cystic fibrosis-related diabetes (CFRD) — the most common non-pulmonary manifestation of CF — to manage their disease. The device, called the bionic pancreas (BP), uses mathematical algorithms to calculate exactly when and how much insulin and/or glucagon needs to be administered through its automatic pump to normalize an individual’s blood sugar levels. Researchers tested the BP device on three women with CFRD. Their findings showed that the device could be used to keep blood sugar levels near the upper normal range of 100 mg/dl in all participants. The researchers said that larger studies with more patients will be needed to evaluate the safety and efficacy of the BP device among those with CFRD.
Cystic Fibrosis News Today hopes these stories and our reporting throughout 2020 contribute to informing and improving the lives of everyone affected by CF and their loved ones.
We wish all our readers a happy 2020.