Throughout the past year, Cystic Fibrosis News Today has brought you daily coverage of important discoveries, treatment developments, clinical trials, and other events related to cystic fibrosis (CF).
As we look forward to bringing you more news in 2019, we would like to present you with the 10 most-read stories of 2018.
No. 10 — “Orkambi’s Components Trigger Liver Enzyme That Decreases Its Ability to Fight CF, Study Finds”
Orkambi, developed by Vertex Pharmaceuticals, is a combination therapy of ivacaftor and lumacaftor, but the therapy’s efficacy may be compromised, according to this study. Researchers reported that the two components of Orkambi act separately and trigger a liver enzyme that decreases the therapy’s ability to fight CF.
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Highlighting the need for continuing to develop more effective therapies for CF, the U.S. Food and Drug Administration granted breakthrough therapy designation to Proteostasis Therapeutics’ CF therapy PTI-428. PTI-428 is a therapy candidate that works by increasing the amount of an immature form of CFTR protein, which is then targeted by other CFTR-based therapies. Data from a Phase 2 trial (NCT02718495) showed that CF patients treated with PTI-428 along with Orkambi had improved lung function compared with those who received a placebo, plus Orkambi.
Vertex Pharmaceuticals completed enrollment in two Phase 3 studies that will test a triple combination therapy — Vertex’s new CFTR corrector VX-659 together with tezacaftor (VX-661) and Kalydeco (ivacaftor). One of the trials (NCT03447249), which enrolled 385 CF patients, ages 12 and older, with an F508del mutation and one minimal function mutation, will test the efficacy of the triple therapy in improving lung function after 24 weeks of treatment. The second Phase 3 trial (NCT03460990) will test the triple combination after treatment with tezacaftor plus ivacaftor for four weeks in 111 CF patients, ages 12 and older, with two F508del mutations in the CFTR gene. The main goal of the trial is to improve lung function post-treatment.
A special formulation of an antioxidant-enriched multivitamin helped decrease the frequency of pulmonary exacerbations in patients with CF, a Phase 2 trial (NCT01859390) performed at the University of Colorado showed. Researchers found that a “cocktail” of multiple antioxidants — compounds that halt damage to cells by harmful substances called free radicals — called AquADEKs-2, temporarily reduced inflammation. Moreover, the antioxidant treatment prolonged the time to the first pulmonary exacerbation requiring antibiotics, and reduced the overall frequency of pulmonary exacerbations.
No. 6 — “Translate Bio Readies 1st Clinical Trial of MRT5005, Potential Treatment for All CF Patients”
Translate Bio’s lead candidate, called MRT5005, to treat the underlying cause of CF received FDA approval to be tested in a Phase 1/2 clinical trial. The trial (NCT03375047), currently recruiting participants, will test the safety and tolerability of single and multiple escalating doses of nebulized MRT5005, compared with each other and a placebo. MRT5005 is a messenger RNA (mRNA) molecule that contains the blueprint, or instructions, for the proper production of a working CFTR protein. The therapy is delivered directly to the lungs in nebulized form, and is the first potential mRNA treatment specifically targeting the lungs.
Two new studies reported a discovery that may help design more effective and targeted therapies for CF. Researchers found that complex types of sugar called glycans, found in the mucus that coats organs such as the lungs, throat, and gastrointestinal tract, interact in unique ways with each type of ion (electric-charged molecule). Since ion-gylcan interaction can affect mucus’ structure and fluidity, the findings carry important implications for therapy delivery in CF.
The anti-inflammatory ibuprofen has a moderate antimicrobial activity, and may help fight two key harmful bacteria, Pseudomonas aeruginosa and Burkholderia, that colonize in CF patient’s lungs, according to a study. Moreover, since ibuprofen delivered orally is linked with cases of kidney toxicity and hemorrhage in the gastrointestinal tract, researchers developed an aerosolizable formulation of ibuprofen. The new formulation was effective at inhibiting P. aeruginosa growth in a laboratory setting.
No. 3 — “Cinnamon Oil Compound Might Block Bacteria Like P. aeruginosa from Forming Biofilms, Researchers Say”
A natural component found in cinnamon oil, called cinnamaldehyde or CAD, may help halt the proliferation of Pseudomonas aeruginosa bacteria, preventing it from spreading and inhibiting its ability to form antibiotic-resistant biofilms. These findings support further studies into antimicrobial medications that can help control treatment-resistant bacteria, which represent a serious healthcare problem for people with CF and other diseases.
An inhaled formulation of the salt bicarbonate may carry therapeutic potential for CF after a study showed that the salt impaired bacterial growth when added to the culture medium of Pseudomonas aeruginosa and Staphylococcus aureus — two of the most common bacteria underlying lung infections in people with CF.
No. 1 — “FDA Approves Symdeko, Vertex’s Combo Therapy for Patients with Certain Mutations in CFTR Gene”
In February 2018, Vertex Pharmaceuticals’ Symdeko (tezacaftor/ivacaftor and ivacaftor) was approved by the FDA as a therapy for CF patients, ages 12 and older, carrying two copies of the F508del mutation in the CFTR gene or with one mutation that responds to tezacaftor/ivacaftor. The combination therapy was also approved last year in Canada, and in Europe, where it is sold under the brand name Symkevi.
Cystic Fibrosis News Today hopes that throughout 2019, we will contribute to educate, inform, and improve the lives of patients living with CF, as well as their loved ones.
We wish all our readers a happy 2019.
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