20 research outputs found
161 PortaNeb® or CR60®? Inhalation of tobramycin (TOBI®) with the Pari LC Plus® nebuliser: an in vivo pilot study
Proof of principle. Treatment of cystic fibrosis-related diabetes: a possible role for complementary metformin?
Rectal Organoids Enable Personalized Treatment of Cystic Fibrosis
In vitro drug tests using patient-derived stem cell
cultures offer opportunities to individually select
efficacious treatments. Here, we provide a study
that demonstrates that in vitro drug responses in
rectal organoids from individual patients with cystic
fibrosis (CF) correlate with changes in two in vivo
therapeutic endpoints. We measured individual
in vitro efficaciousness using a functional assay
in rectum-derived organoids based on forskolininduced swelling and studied the correlation with
in vivo effects. The in vitro organoid responses correlated with both change in pulmonary response and
change in sweat chloride concentration. Receiver
operating characteristic curves indicated good-toexcellent accuracy of the organoid-based test for
defining clinical responses. This study indicates
that an in vitro assay using stem cell cultures can prospectively select efficacious treatments for patients
and suggests that biobanked stem cell resources
can be used to tailor individual treatments in a
cost-effective and patient-friendly manner