A number of cystic fibrosis discoveries, therapy advances and other developments were reported by Cystic Fibrosis News Today during 2017.
Now that the year is over, it’s time to review the articles that appealed most to our readers.
Here are the 10 most-read articles of 2017, with a brief description of what made them interesting and relevant to CF patients, family members, and caregivers.
Bone marrow cells may be a way to treat cystic fibrosis, according to University of Toronto researchers. The team said bone marrow cells can boost the immune system to fight infections and restore levels of functional CFTR protein in airways. The findings suggest that bone marrow transplants could offer a dual benefit to CF treatment. Scientists say CFTR gene mutations that produce adbnormal CFTR protein are the cause of CF.
Caleigh Haber’s story is an example that living with CF can be a struggle. She has done everything she can to prevent the disease from commanding her life. Even though her body has started to reject the double-lung transplant she had in October 2015, she continues to live to the fullest, getting married in October 2017. When doctors told her she was not a suitable candidate for a second double-lung transplant, she and her husband decided “not to waste a day of our lives living with the what ifs. Right now, we’re on a search for a new transplant and living our happily-ever-after in the meantime.”
A harmless bacteria called Streptococcus parasanguinis found on the surface of teeth can inhibit the growth of a dangerous bacteria, a study reports. S. parasanguinis is able to hijack molecular signals that the harmful bacteria Pseudomonas aeruginosa uses to proliferate and form a film on the surface of tissue. The findings could help scientists find ways to prevent P. aeruginosa infections and improve lung function in CF patients.
Since the U.S. Food and Drug Administration approved Kalydeco (ivacaftor) in 2012, it has expanded its approval to cover more cystic fibrosis patients. In May 2017 the agency added patients aged 2 and older with 23 rare CFTR gene mutations. The initial authorization had covered patients with only 10 CFTR mutations. The expansion affects about 900 or 3 percent of American children with CF. Faulty CFTR genes are the cause of the disease.
Novoclem Therapeutics is developing a nitric oxide-releasing therapy that can eliminate nine of the most bacteria most responsible for severe lung infections in CF patients, including treatment-resistant bacteria. This therapy boosts the immune system to fight infections and regulates inflammation. The company said it planned a clinical trial in early 2018 to test the treatment.
For the first time ever, a cystic fibrosis patient has received stem cells to treat their disease. The treatment was the focus of a pioneering Phase 1 clinical trial (NCT02866721) to see if mesenchymal stem cells can regulate CF patients’ inflammation and infection. The Cystic Fibrosis Foundation supported the study, which scientists expect to provide important information to improve treatments for CF patients and develop new therapies for them.
Accumulation of mucus in the lungs is a characteristic feature of CF because of faulty CFTR protein. Researchers at the University of North Carolina said they believe that an important contributor to this is lack of water in airway linings. In fact, some scientists are conducting clinical trials to test the idea of using inhaled salt water to hydrate airways.
An Australian study showed that children with cystic fibrosis sleep less and experience more sleep interruptions than healthy children, even when the disease is well-managed and stable. Children with CF have higher sleep-disturbance and sleep-apnea scores, and lower oxygen levels during sleep, researchers found. The team believes the sleeping disorders affect the children’s mood, behavior, ability to manage their emotions, and quality of life. Finding ways to improve the amount of time that these children sleep, and the quality of their sleep, may not only improve their quality of life but also help them obtain better results from treatment, the researchers said.
Hill-Rom has developed a lightweight vest to help patients with cystic fibrosis and related diseases clear their airways. The company’s Monarch Airway Clearance System comes with a rechargeable battery for better mobility. It can be used as patients go about their day.
Boston University medical researchers have developed a way to transform stem cells into lung cells needed to treat cystic fibrosis, but no other types of cells. They also discovered that airway cells can form tiny balls known as bronchospheres that scientists can used to see how well CFTR genes are functioning. Mutations of that gene cause CF. The two discoveries could pave the way for new personalized medicine strategies for CF, the team said.
Cystic Fibrosis News Today hopes that these developments, and new reports coming your way throughout 2018, will ultimately contribute to educate, inform, and improve the lives of patients living with CF.
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We wish all our readers a happy and inspiring 2018.
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