Nanoparticles used to create ‘one size fits all’ cystic fibrosis treatment
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US researchers are developing a nanoparticle-based one-size-fits-all treatment for cystic fibrosis (CF) patients which could be inhaled simply at home.
CF is a genetic condition which affects a range of organs, most notably leaving the lungs frequently infected and full of mucus, and renders most men infertile. The condition – which has no known cure – affects 30,000 Americans. Those affected can expect to live an average of just 40 years.
Symptoms and disease progression differ between patients, particularly as there are at least 300 different disease-conferring genetic mutations associated with CF.
Some patients choose treatment to effectively ‘rescue’ the mutant protein produced by the faulty gene, although many patients do not have this option due to their genetic mutations or due to the sheer cost of the treatment and its side effects.
Now, a team of researchers and doctors based at Oregon State University and Oregon Health & Science University have demonstrated the possibility of a far more accessible form of CF treatment, which does not attempt to rescue the mutant protein. Instead, it uses chemically modified RNA messengers for cystic fibrosis transmembrane conductance regulator (CFTR, the faulty gene which causes the disease) into nanoparticles. This results in a medicine that can be inhaled at home.
These nanoparticles, loaded with modified RNA messengers, cause cells to produce the protein that allows cells to regulate chloride and water transport that CF patients lack, severely affecting their respiratory health.
“It’s a platform technology for correcting monogenic disorders and allows the same therapy to be effective for treating all [CF] patients,” said Professor Gaurav Sahay, co-author of the study and assistant professor of pharmaceutical sciences at the Oregon College of Pharmacy. According to Sahay, while other treatments must be tailored to each patient in order to account for differences in genetic mutations, this type of therapy works for any CF patient.
“These systems can be repeatedly administered to a patient and the effects are reversible if someone needs to stop the therapy for any reason.”
Sahay and his colleagues tested the nanoparticle-based treatment in culture using patient-derived bronchial cells, as well as genetically modified mice, and found the treatment successful in these models.
The researchers hope that a future iteration of this treatment could make it far easier to treat all patients – particularly children – with a simple inhalation at home, causing none of the unpleasant side-effects patients with CF often suffer during therapy.
“We made a one-size-fits-all treatment,” said Ema Robinson, first author and a research assistant in the College of Pharmacy. “We’re adding back the gene that makes the protein so the airways can be rehydrated and we can adjust the dosage as the disease gets better.”