Testing CFTR Modulation drugs like Kalydeco, in vitro, on rare mutations.


New member
A lab in the Netherlands have found a new, reliable way to test drugs like Kalydeco in vitro, by growing an organoid of a patient´s intestine. If the gut reacts positively on the drug this could be a way to help patients with rare mutations (with residual function) to access drugs like Kalydeco even though these mutations haven´t been tested in clinical trials (and hence haven´t been included in the approval by the FDA).
The primary aim seems to be to improve in vitro studies, making them more reliable and applicable on rarer mutations and I don´t know if it will be used as means for physicians to prescribe experimental drugs to patients who would otherwise be left out. I just hope cf-physisians will see the potential for clinical practice here!